On November 5, 2020 EyePoint Pharmaceuticals, Inc. (NASDAQ: EYPT), a pharmaceutical company committed to developing and commercializing innovative ophthalmic products, reported financial results for the third quarter ended September 30, 2020 and highlighted recent corporate developments (Press release, pSivida, NOV 5, 2020, View Source [SID1234570148]).

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"With the continued increase in patient office visits and resumption of operations at many healthcare facilities during the quarter, we were encouraged to see customer demand for both YUTIQ and DEXYCU near pre-COVID-19 pandemic levels," said Nancy Lurker, President and Chief Executive Officer of EyePoint Pharmaceuticals. "We enter the remaining months of 2020 with the EyePoint and ImprimisRx combined sales teams mobilized to promote DEXYCU to their established account base and our YUTIQ team continuing to call on uveitis and retinal physician offices, subject to evolving COVID-19-related restrictions."

Ms. Lurker continued, "With the recent completion of our good laboratory practice (GLP) toxicology study of EYP-1901, a potential six-month sustained delivery intravitreal anti-VEGF treatment for wet age-related macular degeneration (wet AMD), we remain on track to submit an Investigational New Drug (IND) application before the end of the year. Upon acceptance of the IND by the U.S. Food and Drug Administration (FDA), we look forward to initiating a Phase 1 trial of EYP-1901 in wet AMD, a sight-threatening eye disease that is in significant need of longer-lasting treatment options that may slow its progression. In addition, we continue to diligently manage our cash and burn rate and ended October 31, 2020 with $30.5 million in cash."

Commercial Performance in Third Quarter 2020

Customer demand for YUTIQ, represented as units purchased by physicians from the Company’s distributors, was approximately 450 units in Q3 2020 as compared to approximately 430 units in Q2 2020.
Customer demand for DEXYCU, represented as units purchased by ambulatory surgery centers (ASCs) from the Company’s distributors, was approximately 4,700 units in Q3 2020 as compared to approximately 2,100 units in Q2 2020.
Separate purchasing and marketing agreements for expanded access to DEXYCU across the U.S. were recently executed with Vantage Outsourcing and another undisclosed healthcare network. The Company is actively negotiating agreements with additional group purchasing organizations and networks.
In August, the Company and ImprimisRx signed a commercial alliance for the joint promotion of DEXYCU in the U.S. which more than doubles the size of our team engaging directly with physicians and ASCs. During September, ImprimisRx’s sales specialists and inside sales team completed product training and are positioned to call on their ophthalmologist, hospital and ASC accounts.
The Company continues to actively monitor the COVID-19 pandemic and associated public health recommendations to ensure the safety of our patients, physicians and employees.
Operations Update

In October, based in part on the return in customer demand for both products following COVID-19-related closures, the Company secured an amendment to its existing debt facility with CRG Servicing LLC (CRG) in which CRG waived the covenant associated with the Company’s net product revenue for DEXYCU and YUTIQ for the twelve-month period ending on December 31, 2020. CRG also agreed to a reduction of the calendar year 2021 net product revenue covenant to $45 million from $80 million. There were no other material changes made to the term loan agreement and the Company incurred no incremental charges in connection with the amendment.
In August, the Company and Ocumension Therapeutics announced an expansion of their exclusive license agreements for YUTIQ and DEXYCU in certain markets in Asia. Under the expanded agreements, Ocumension made a one-time $9.5 million payment to EyePoint for rights to commercialize both products under its own brand names in South Korea and other jurisdictions across Southeast Asia and as the full and final payment of all remaining development, regulatory, and commercial sale milestone payments under the original license agreements. Royalties for future product sales remain payable to the Company pursuant to the license agreements.
R&D Highlights

In October, the Company completed a GLP toxicology study for EYP-1901, a potential six-month sustained delivery intravitreal anti-VEGF treatment using its bioerodible Durasert technology for wet AMD. The Company expects to file an IND application with the FDA in the fourth quarter of 2020 with a Phase 1 human clinical trial to commence shortly after IND acceptance by the FDA.
Review of Results for Third Quarter Ended September 30, 2020
For the three months ended September 30, 2020, total net revenue was $15.7 million compared to $2.5 million for the three months ended September 30, 2019. Net product revenue for the three months ended September 30, 2020 was $5.8 million, with $3.5 million for YUTIQ and $2.3 million for DEXYCU, compared to net product revenue for three months ended September 30, 2019 of $1.0 million generated primarily by DEXYCU. Net product revenue represents product purchased by EyePoint’s distributors whereas customer demand represents purchases of product by physician practices and ASCs from EyePoint’s distributors.

Net revenue from licenses, royalties and collaborations for the three months ended September 30, 2020 totaled $9.9 million compared to $1.5 million in the corresponding quarter in 2019.

Operating expenses for the three months ended September 30, 2020 totaled $17.7 million compared to $16.6 million in the prior year period. This increase was driven by a $1.6 million increase in cost of sales, a $1.4 million increase in G&A expense and $0.6 million increase in R&D expense being offset by a $2.5 million reduction in sales and marketing expense. Non-operating expense, net, for the three months ended September 30, 2020 totaled $1.8 million of net interest expense. Net loss for the three months ended September 30, 2020 was $3.8 million, or $0.03 per share, compared to a net loss of $15.6 million, or $0.15 per share, for the prior year quarter.

Review of Nine Months Results Ended September 30, 2020
For the nine months ended September 30, 2020, total net revenue was $27.3 million compared to $11.7 million for the nine months ended September 30, 2019. Net product revenue for the nine months ended September 30, 2020 was $14.2 million, compared to net product revenues for the nine months ended September 30, 2019 of $8.9 million.

Net revenue from royalties and collaborations for the nine months ended September 30, 2020 totaled $13.2 million compared to $2.8 million in the corresponding period in 2019.

Operating expenses for the nine months ended September 30, 2020 totaled $51.9 million from $50.6 million in the prior year period. This increase was primarily due to a $2.0 million increase in cost of sales, a $1.0 million increase in R&D expense and a $1.2 million increase in G&A expense offset by a $2.9 million decrease in sales and marketing expense. Non-operating expense, net, for the nine months ended September 30, 2020 totaled $5.4 million. Net loss for the nine months ended September 30, 2020 was $29.9 million, or $0.24 per share, compared to a net loss of $46.4 million, or $0.45 per share, for the prior year period.

Cash and cash equivalents at October 31, 2020 totaled $30.5 million compared to $28.7 million at September 30, 2020 and $22.2 million at December 31, 2019.

Financial Outlook
We expect that the Company’s cash and cash equivalents combined with projected cash inflows from anticipated YUTIQ and DEXYCU product sales and other expected financing activities can fund the Company’s operating plan into 2021 assuming no significant increase in COVID-19-related closures that would materially affect the frequency of ophthalmology office visits or the number of cataract surgical procedures performed across the U.S.

The Company continues to assess additional cash conservation and generation measures to support its operations through the COVID-19 pandemic.

Conference Call Information
EyePoint will host a conference call today, Thursday, November 5, 2020, at 8:30 AM ET to discuss the results for the third quarter ended September 30, 2020 and recent operational developments. To access the conference call, please dial (877) 312-7507 from the U.S. and Canada or (631) 813-4828 (international) at least 10 minutes prior to the start time and refer to conference ID 1452724. A live webcast will be available on the Investor Relations section of the corporate website at View Source A replay of the webcast will also be available on the corporate website.

On November 5, 2020 Teva Pharmaceutical Industries Ltd. (NYSE: TEVA, TASE: TEVA) reported results for the quarter ended September 30, 2020 (Press release, Teva, NOV 5, 2020, View Source [SID1234570147]).

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Mr. Kåre Schultz, Teva’s President and CEO, said, "Teva’s business and operations have shown resilience as the COVID-19 pandemic continues to impact the world. The quarter saw continued strong performance from our key growth drivers, led by AUSTEDO and the biosimilar TRUXIMA, while the market share of AJOVY continued to grow in the U.S. and Europe. During this quarter we also launched our digital inhalers AirDuo Digihaler and ArmonAir Digihaler in the U.S. The DigiHaler portfolio is now the first and only family of digital inhalers with built-in sensors available to patients."

Mr. Schultz continued, "Over the past three years we have reduced our net debt by more than $10 billion to $23.8 billion. This debt reduction, and the continued improvement of our profitability, keeps us on track to achieve our long-term financial targets by the end of 2023."

Third Quarter 2020 Consolidated Results

Revenues in the third quarter of 2020 were $3,978 million, a decrease of 3% in both U.S. dollar and local currency terms, compared to the third quarter of 2019. This decrease was mainly due to lower revenues from generics, OTC and COPAXONE in all regions and lower revenues from QVAR and BENDEKA/TREANDA in our North America segment, as well as reduced demand for certain products resulting from the impact of the COVID-19 pandemic, partially offset by higher revenues from AUSTEDO and AJOVY.

Exchange rate differences between the third quarter of 2020 and the third quarter of 2019, including hedging effects, positively impacted our revenues by $14 million and negatively impacted our GAAP and non-GAAP operating income by $18 million and $13 million, respectively.

GAAP gross profit was $1,852 million in the third quarter of 2020, an increase of 1% compared to the third quarter of 2019. GAAP gross profit margin was 46.6% in the third quarter of 2020, compared to 44.7% in the third quarter of 2019. The increase in gross profit margin was mainly due to higher profitability in North America resulting from the change in mix of products as well as operational cost efficiencies and network optimization, partially offset by lower sales of COPAXONE and other specialty products in all segments. Non-GAAP gross profit was $2,084 million in the third quarter of 2020, a decrease of 1% compared to the third quarter of 2019. Non-GAAP gross profit margin was 52.4% in the third quarter of 2020, compared to 51.4% in the third quarter of 2019.

GAAP Research and Development (R&D) expenses in the third quarter of 2020 were $258 million, an increase of 7% compared to the third quarter of 2019. Non-GAAP R&D expenses were $233 million, or 5.8% of quarterly revenues, in the third quarter of 2020, compared to $242 million, or 5.9%, in the third quarter of 2019. In the third quarter of 2020, our R&D expenses related primarily to specialty product candidates in the pain, respiratory and neuropsychiatry therapeutic areas, with additional activities in selected other areas and generic products including biosimilars. Our higher R&D expenses in the third quarter of 2020, compared to the third quarter of 2019, were mainly due to an upfront payment made in the third quarter of 2020.

GAAP Selling and Marketing (S&M) expenses in the third quarter of 2020 were $605 million, an increase of 2% compared to the third quarter of 2019. Non-GAAP S&M expenses were $566 million, or 14.2% of quarterly revenues, in the third quarter of 2020, compared to $551 million, or 13.5%, in the third quarter of 2019.

GAAP General and Administrative (G&A) expenses in the third quarter of 2020 were $279 million, a decrease of 2% compared to the third quarter of 2019. Non-GAAP G&A expenses were $269 million, or 6.8% of quarterly revenues, in the third quarter of 2020, compared to $270 million, or 6.6%, in the third quarter of 2019.

We recorded a goodwill impairment charge of $4,628 million related to our North America reporting unit in the third quarter of 2020, in connection with current market capitalization influenced by uncertainty regarding the timeframe for resolution of certain litigations.

Other income in the third quarter of 2020 was $8 million, compared to $14 million in the third quarter of 2019.

GAAP operating loss in the third quarter of 2020 was $4,342 million, compared to GAAP operating loss of $81 million in the third quarter of 2019. This decrease was mainly due to the goodwill impairment charge and higher intangible asset impairment in the third quarter of 2020, partially offset by lower legal settlements and loss contingencies charges in the third quarter of 2020.

Non-GAAP operating income in the third quarter of 2020 was $1,025 million, a decrease of 3%, compared to $1,051 million in the third quarter of 2019.

EBITDA (non-GAAP operating income, which excludes amortization and certain other items, as well as depreciation expenses) was $1,153 million in the third quarter of 2020, a decrease of 3% compared to $1,183 million in the third quarter of 2019.

GAAP financial expenses were $117 million in the third quarter of 2020, compared to $211 million in the third quarter of 2019. Non-GAAP financial expenses were $241 million in the third quarter of 2020, compared to $208 million in the third quarter of 2019.

In the third quarter of 2020, we recognized a GAAP tax expense of $16 million, on pre-tax loss of $4,459 million. In the third quarter of 2019, we recognized a tax expense of $11 million, on pre-tax loss of $292 million. Our tax rate for the third quarter of 2020 was mainly affected by the goodwill impairment charge that does not have a corresponding tax effect and other changes to tax positions and deductions. Non-GAAP income taxes for the third quarter of 2020 were $133 million, or 17%, on pre-tax non-GAAP income of $784 million. Non-GAAP income taxes in the third quarter of 2019 were $183 million, or 22%, on pre-tax non-GAAP income of $843 million.

We expect our annual non-GAAP tax rate for 2020 to be 17-18%, unchanged from our outlook provided in February 2020.

GAAP net loss attributable to Teva and GAAP diluted loss per share were $4,349 million and $3.97 respectively, in the third quarter of 2020, compared to GAAP net loss and GAAP diluted loss per share of $314 million and $0.29 in the third quarter of 2019. This decrease was mainly due to the goodwill impairment charge and higher intangible asset impairment charges in the third quarter of 2020, partially offset by lower legal settlements and loss contingencies in the third quarter of 2020. Non-GAAP net income attributable to Teva and non-GAAP diluted EPS in the third quarter of 2020 were $637 million and $0.58, respectively, compared to $637 million and $0.58 in the third quarter of 2019.

The weighted average diluted shares outstanding used for the fully diluted share calculation for the three months ended September 30, 2020 and 2019 was 1,096 million and 1,092 million shares, respectively. The weighted average diluted shares outstanding used for the fully diluted share calculation on a non-GAAP basis for the three months ended September 30, 2020 and 2019 was 1,100 million and 1,093 million shares, respectively.

As of September 30, 2020 and 2019, the fully diluted share count for purposes of calculating our market capitalization was approximately 1,118 million and 1,107 million, respectively.

Non-GAAP information: Net non-GAAP adjustments in the third quarter of 2020 were $4,986 million. Non-GAAP net income and non-GAAP EPS for the third quarter of 2020 were adjusted to exclude the following items:

A goodwill impairment charge of $4,628 million related to our North America reporting unit in the third quarter of 2020;
$565 million impairment of long-lived assets comprised mainly of impairments of identifiable intangible assets totaling $509 million ($360 million of IPR&D assets and $149 million of identifiable product rights);
Amortization of purchased intangible assets of $251 million, of which $221 million is included in cost of sales and the remaining $31 million in S&M expenses;
Contingent consideration income of $179 million, mainly related to a decrease in future royalties;
Gain from equity investment of $134 million, reflecting the difference between the book value of our investment in American Well Corporation and its fair value as of the date it completed its initial public offering in September 2020;
Finance income of $124 million, mainly related to the American Well equity holding;
Purchase of in-process R&D of $21 million;
Legal settlements and loss contingencies of $21 million;
Equity compensation expenses of $30 million;
Other items of $24 million; and
Income tax of $117 million.
Teva believes that excluding such items facilitates investors’ understanding of its business. For further information, see the tables below for a reconciliation of the U.S. GAAP results to the adjusted non-GAAP figures and the information under "Non-GAAP Financial Measures." Investors should consider non-GAAP financial measures in addition to, and not as replacement for, or superior to, measures of financial performance prepared in accordance with GAAP.

Cash flow generated from operating activities during the third quarter of 2020 was $307 million, compared to $325 million in the third quarter of 2019. The decrease in the third quarter of 2020 was mainly due to an increase in inventory during the third quarter of 2020 compared to a decrease in inventory in the third quarter of 2019.

Free cash flow (cash flow generated from operating activities, net of cash received for capital investments and beneficial interest collected in exchange for securitized trade receivables) was $506 million in the third quarter of 2020, compared to $551 million in the third quarter of 2019. The decrease in the third quarter of 2020 resulted mainly from lower cash flow generated from operating activities.

As of September 30, 2020, our debt was $25,621 million, compared to $26,266 million as of June 30, 2020. This decrease was mainly due the repayment at maturity of our €1,010 million 0.375% senior notes in July 2020, partially offset by exchange rate fluctuations. The portion of total debt classified as short-term as of September 30, 2020 was 8%, compared to 6% June 30, 2020. Our average debt maturity was approximately 6 years as of September 30, 2020, compared to 6.1 years as of June 30, 2020. Our financial leverage was 71% as of September 30, 2020, compared to 64% as of June 30, 2020.

Segment Results for the Third Quarter of 2020

North America Segment

Our North America segment includes the United States and Canada.

Revenues from our North America segment in the third quarter of 2020 were $2,017 million, a decrease of $34 million, or 2%, compared to the third quarter of 2019, mainly due to a decrease in revenues of COPAXONE and BENDEKA/TREANDA, partially offset by higher revenues from AUSTEDO, generic products and AJOVY. Our North America segment has experienced some reductions in volume due to less physician and hospital activity during the COVID-19 pandemic, but has also experienced increase in demand for certain products related to the treatment of COVID-19 and its symptoms. In addition, the ability to promote our new specialty products, primarily AJOVY and AUSTEDO, has been impacted by less physician visits by patients and less physician interactions by our sales personnel.

Revenues in the United States, our largest market, were $1,887 million in the third quarter of 2020, a decrease of $20 million, or 1%, compared to the third quarter of 2019.

Generic products revenues in our North America segment (including biosimilars) in the third quarter of 2020 were $928 million, an increase of 2% compared to the third quarter of 2019. This increase was mainly due to higher revenues from TRUXIMA and from our ProAir authorized generic, partially offset by lower volume of other generic products.

On September 30, 2020, we launched emtricitabine and tenofovir disoproxil fumarate tablets, 200mg/300mg (the generic equivalent of Truvada), and efavirenz, emtricitabine and tenofovir disoproxil fumarate tablets (the generic equivalent for Atripla) for the prevention and treatment of HIV, respectively. We did not recognize revenues for these products in the third quarter of 2020.

In the third quarter of 2020, we led the U.S. generics market in total prescriptions and new prescriptions, with approximately 365 million total prescriptions (based on trailing twelve months), representing 10.0% of total U.S. generic prescriptions according to IQVIA data.

AJOVY revenues in our North America segment in the third quarter of 2020 were $35 million, an increase of $10 million, or 42% compared to the third quarter of 2019, mainly due to growth in volume in the third quarter of 2020.

AUSTEDO revenues in our North America segment in the third quarter of 2020 increased by 60% to $168 million, compared to $105 million in the third quarter of 2019. This increase was mainly due to growth in volume in the third quarter of 2020.

BENDEKA and TREANDA combined revenues in our North America segment in the third quarter of 2020 decreased by 15% to $105 million, compared to the third quarter of 2019, mainly due to the emergence of alternative novel therapies and continued competition from Belrapzo (a ready-to-dilute bendamustine hydrochloride product from Eagle Pharmaceuticals, Inc.).

COPAXONE revenues in our North America segment in the third quarter of 2020 decreased by 13% to $236 million, compared to the third quarter of 2019, mainly due to generic competition in the United States.

ProAir revenues in our North America segment in the third quarter of 2020 decreased by 30% to $50 million, compared to the third quarter of 2019. In January 2019, we launched our own ProAir authorized generic in the United States following the launch of a generic version of Ventolin HFA, another albuterol inhaler. Revenues from our ProAir HFA authorized generic are included in "generic products" above. In July 2020, we announced the launch of ProAir Digihaler (albuterol sulfate 117 mcg) inhalation powder, which is the first and only digital rescue inhaler with built-in sensors which connects to a companion mobile application and provides inhaler use information to people with asthma and COPD. In September 2020, we announced the launch of AirDuo Digihaler (fluticasone propionate and salmeterol) inhalation powder and the launch of ArmonAir Digihaler (fluticasone propionate) inhalation powder, two digital maintenance inhalers for adolescent and adult patients with asthma.

QVAR revenues in our North America segment in the third quarter of 2020 decreased by 29% to $42 million, compared to the third quarter of 2019, mainly due to increased price competition and lower volumes.

Anda revenues in our North America segment in the third quarter of 2020 decreased by 3% to $341 million, compared to $351 million in the third quarter of 2019, mainly due to slightly lower demand.

North America Gross Profit

Gross profit from our North America segment in the third quarter of 2020 was $1,056 million, an increase of 1%, compared to $1,048 million in the third quarter of 2019.

Gross profit margin for our North America segment in the third quarter of 2020 increased to 52.4%, compared to 51.1% in the third quarter of 2019. This increase was mainly due to the change in mix of products.

North America Profit

Profit from our North America segment consists of gross profit less R&D expenses, S&M expenses, G&A expenses and any other income related to this segment. Segment profit does not include amortization and certain other items.

Profit from our North America segment in the third quarter of 2020 was $560 million, flat compared to $565 million in the third quarter of 2019, mainly due to lower revenues, offset by a change in mix of products.

Europe Segment

Our Europe segment includes the European Union and certain other European countries.

Revenues from our Europe segment in the third quarter of 2020 were $1,116 million, a decrease of 4%, or $47 million, compared to the third quarter of 2019. In local currency terms, revenues decreased by 7%, mainly due to reduced demand for certain products resulting from the COVID-19 pandemic. The COVID-19 pandemic has led to a decline in doctor and hospital visits by patients resulting in fewer prescriptions during the third quarter of 2020. This decrease is also attributed to price declines for oncology products as a result of generic competition and a decline in COPAXONE revenues due to competing glatiramer acetate products.

Generic products revenues in our Europe segment in the third quarter of 2020, including OTC products, decreased by 1% to $824 million, compared to the third quarter of 2019. In local currency terms, revenues decreased by 6% compared to the third quarter of 2019, mainly due to a reduced demand for certain products resulting from the COVID-19 pandemic. The COVID-19 pandemic has led to a decline in doctor and hospital visits by patients resulting in fewer prescriptions during the third quarter of 2020.

COPAXONE revenues in our Europe segment in the third quarter of 2020 decreased by 5% to $101 million, compared to the third quarter of 2019. In local currency terms, revenues decreased by 9%, due to price reductions and a decline in volume resulting from competing glatiramer acetate products.

Respiratory products revenues in our Europe segment in the third quarter of 2020 decreased by 12% to $77 million, compared to the third quarter of 2019. In local currency terms, revenues decreased by 15%, mainly due to reduced demand resulting from the impact the COVID-19 pandemic had on purchasing patterns. The COVID-19 pandemic led to increased demand in the first quarter and a correlating decrease in the following quarters.

AJOVY revenues in our Europe segment in the third quarter of 2020 were $8 million, compared to $1 million in the third quarter of 2019.

Europe Gross Profit

Gross profit from our Europe segment in the third quarter of 2020 was $637 million, a decrease of 4% compared to $662 million in the third quarter of 2019. This decrease was mainly due to lower revenues, as discussed above.

Gross profit margin for our Europe segment in the third quarter of 2020 increased to 57.1%, compared to 56.9% in the third quarter of 2019.

Europe Profit

Profit from our Europe segment consists of gross profit less R&D expenses, S&M expenses, G&A expenses and any other income related to this segment. Segment profit does not include amortization and certain other items.

Profit from our Europe segment in the third quarter of 2020 was $312 million, a decrease of 8%, compared to $341 million in the third quarter of 2019. This decrease was mainly due to lower revenues, as discussed above.

International Markets Segment

Our International Markets segment includes all countries in which we operate other than those in our North America and Europe segments. The key markets in this segment are Japan, Russia and Israel.

On July 30, 2020, we entered into an agreement to sell the majority of the generic and operational assets of our business venture in Japan. We expect this transaction to close by early 2021. The closing of the transaction is subject to customary closing conditions.

**The data presented for prior periods have been revised to reflect a revision in the presentation of net revenues and cost of sales in the consolidated financial statements. See note 1c to our consolidated financial statements for additional information.

Revenues from our International Markets segment in the third quarter of 2020 were $529 million, a decrease of $35 million, or 6%, compared to the third quarter of 2019. In local currency terms, revenues decreased by 1% compared to the third quarter of 2019, mainly due to lower sales in Japan resulting from regulatory price reductions and generic competition to off-patented products, as well as loss of revenues from the sale of certain assets in the Israeli market, partially offset by higher revenues in other markets. Revenues in the third quarter of 2020 were also impacted by reduced demand for certain products resulting from the impact of the COVID-19 pandemic. The COVID-19 pandemic has led to a decline in doctor and hospital visits by patients resulting in fewer prescriptions during the third quarter of 2020.

*The data presented for prior periods have been revised to reflect a revision in the presentation of net revenues and cost of sales in the consolidated financial statements. See note 1c to our consolidated financial statements for additional information.

Generic products revenues in our International Markets segment in the third quarter of 2020, which include OTC products, decreased by 10% to $429 million, compared to the third quarter of 2019. In local currency terms, revenues decreased by 4%, mainly due to lower sales in Japan resulting from regulatory price reductions and generic competition to off-patented products. Revenues in the third quarter of 2020 were also impacted by reduced demand for certain products resulting from the impact of the COVID-19 pandemic. The COVID-19 pandemic has led to a decline in doctor and hospital visits by patients resulting in fewer prescriptions during the third quarter of 2020.

COPAXONE revenues in our International Markets segment in the third quarter of 2020 decreased by 27% to $14 million, compared to $20 million in the third quarter of 2019. In local currency terms, revenues decreased by 14%.

AJOVY On May 12, 2017, we entered into a license and collaboration agreement with Otsuka Pharmaceutical Co., Ltd. ("Otsuka") providing Otsuka with an exclusive license to conduct phase 2 and 3 clinical trials for AJOVY in Japan and, once approved, to commercialize the product in Japan. On July 29, 2020, Otsuka submitted an application to obtain manufacturing and marketing approval for AJOVY in Japan. As a result, Otsuka paid Teva a milestone payment of $15 million in the third quarter of 2020, which was recorded as revenue under "Other" in the table above.

International Markets Gross Profit

Gross profit from our International Markets segment in the third quarter of 2020 was $275 million, a decrease of 7% compared to $295 million in the third quarter of 2019.

Gross profit margin for our International Markets segment in the third quarter of 2020 decreased to 52.0%, compared to 52.2% in the third quarter of 2019. This decrease was mainly due to a different portfolio mix.

International Markets Profit

Profit from our International Markets segment consists of gross profit less R&D expenses, S&M expenses, G&A expenses and any other income related to this segment. Segment profit does not include amortization and certain other items.

Profit from our International Markets segment in the third quarter of 2020 was $125 million, a decrease of 4%, compared to $130 million in the third quarter of 2019. This decrease was mainly due to lower sales in Japan, partially offset by lower S&M expenses.

Other Activities

We have other sources of revenues, primarily the sale of APIs to third parties, certain contract manufacturing services and an out-licensing platform offering a portfolio of products to other pharmaceutical companies through our affiliate Medis. Our other activities are not included in our North America, Europe or International Markets segments described above.

Our revenues from other activities in the third quarter of 2020 were $316 million, an increase of 1% compared to the third quarter of 2019. In local currency terms, revenues decreased by 1%.

API sales to third parties in the third quarter of 2020 were $175 million, flat in both U.S. dollar and local currency terms, compared to the third quarter of 2019.

Conference Call

Teva will host a conference call and live webcast including a slide presentation on November 5, 2020 at 8:00 a.m. ET to discuss its third quarter of 2020 results and overall business environment. A question & answer session will follow.

A live webcast of the call will be available on Teva’s website at: ir.tevapharm.com. Please log in at least 10 minutes prior to the conference call in order to download the required software.

Following the conclusion of the call, a replay of the webcast will be available within 24 hours on the Company’s website or by calling United States 1-866-331-1332; International +44 (0) 3333 009785; passcode: 7275239.

On November 5, 2020 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, reported third quarter 2020 financial results and provided a corporate update (Press release, Kura Oncology, NOV 5, 2020, View Source [SID1234570120]).

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"Our team is focused on developing novel therapies for patients with cancer," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "With our menin inhibitor, KO-539, we are encouraged by the early evidence of activity in our KOMET-001 Phase 1/2 clinical trial in patients with acute myeloid leukemia (AML), and look forward to presenting updated data from the trial in an oral presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting next month. With tipifarnib, in addition to conducting our ongoing AIM-HN registration-directed trial of tipifarnib in recurrent or metastatic HRAS mutant head and neck squamous cell carcinoma (HNSCC), we recently presented preclinical data that underscore the potential to combine tipifarnib with a PI3Kα inhibitor to treat between 25% and 50% of HNSCC patients, and look forward to initiating a combination trial of these two targeted therapies in mid-2021."

Corporate Update

Preliminary data for KO-539 accepted for oral presentation at ASH (Free ASH Whitepaper) – An abstract reporting preliminary data from KOMET-001, a first-in-human study of the Company’s oral, potent and selective menin inhibitor, KO-539, has been accepted for oral presentation at ASH (Free ASH Whitepaper). The abstract, posted on the ASH (Free ASH Whitepaper) website on November 4, 2020, highlighted encouraging safety and tolerability, as well as evidence of anti-leukemic activity as of the data cutoff of August 10, 2020. Kura plans to present a more mature dataset, including data from approximately 10 patients, in the oral presentation at ASH (Free ASH Whitepaper) on December 5, 2020, followed by a virtual investor event featuring two of the trial’s investigators.

KO-539 approaching recommended Phase 2 dose, expansion cohorts – Kura remains focused on its goal of reaching a recommended Phase 2 dose for KO-539 as KOMET-001 continues in dose escalation. The Company continues to add clinical sites in anticipation of moving into the expansion cohorts, pending additional clinical data. The planned expansion cohorts include NPM1-mutant AML and KMT2A(MLL)-rearranged AML, selected patient populations where KO‑539 has the potential to demonstrate increased clinical benefit. Kura believes KO-539 represents a differentiated approach to target genetic subsets representing potentially 35% or more of the total adult AML population. In addition, Kura continues to explore options to potentially broaden the opportunity in the treatment of acute leukemias.

Preclinical data support expansion opportunity for tipifarnib in HNSCC – Last month, Kura reported new preclinical data showing compelling activity of its late-stage drug candidate, tipifarnib, when combined with a PI3Kα inhibitor in models of HRAS-dependent and/or PI3K dependent HNSCC. The data, presented at the EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium on Molecular Targets and Cancer Therapeutics, support the Company’s upcoming Phase 1/2 trial of tipifarnib in combination with a PI3Kα inhibitor in advanced or unresectable relapsed/refractory HNSCC harboring PIK3CA mutations or amplifications and/or HRAS overexpression. Kura believes that the total addressable population for tipifarnib may be between 25-50% of HNSCC.

Leadership team enhanced with addition of Dr. Stephen Dale – In August, Kura appointed Stephen Dale, M.D., as its new Chief Medical Officer. Dr. Dale joined the Company most recently from Kyowa Kirin, where he served as Senior Vice President and Global Head of Medical Science with a primary focus in oncology. Previously, he was Global Clinical Vice President and Clinical Head of Oncology at AstraZeneca, where he oversaw the development of Tagrisso (osimertinib) for metastatic EGFR-T790M mutation-positive non-small cell lung cancer.
Financial Results

Research and development expenses for the third quarter of 2020 were $16.6 million, compared to $12.5 million for the third quarter of 2019.

General and administrative expenses for the third quarter of 2020 were $7.6 million, compared to $5.1 million for the third quarter of 2019.

Net loss for the third quarter of 2020 was $23.8 million, compared to a net loss of $16.4 million for the third quarter of 2019.

Cash, cash equivalents and short-term investments totaled $325.4 million as of September 30, 2020, compared with $236.9 million as of December 31, 2019.

Management expects that current cash, cash equivalents and short-term investments will be sufficient to fund current operations into 2023.
Conference Call and Webcast

Kura’s management will host a webcast and conference call today at 8:00 a.m. ET / 5:00 a.m. PT today, November 5, 2020, to discuss the financial results for the third quarter 2020 and provide a corporate update. The live call may be accessed by dialing (877) 516-3514 for domestic callers and +1 (281) 973-6129 for international callers and entering the conference code: 7456326. A live webcast of the call will be available from the Investors and Media section of the Company’s website at www.kuraoncology.com, and will be archived there for 30 days.

On November 5, 2020 bluebird bio, Inc. (Nasdaq: BLUE) reported that data from its gene and cell therapy programs for sickle cell disease (SCD), transfusion-dependent beta-thalassemia (TDT) and multiple myeloma (MM) will be presented, including seven oral presentations, at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, taking place virtually from December 5-8, 2020 (Press release, bluebird bio, NOV 5, 2020, View Source [SID1234570119]).

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Updated results from patients in Group C of the company’s Phase 1/2 HGB-206 study of LentiGlobin for SCD gene therapy (bb1111) will be presented.

bluebird bio will also present updated long-term efficacy and safety results from the LTF-303 follow-up study; outcomes across genotypes; and outcomes in pediatric patients from Phase 3 studies HGB-207 and HGB-212 of betibeglogene autotemcel (beti-cel; formerly LentiGlobin for β-thalassemia) in TDT.

Data from across the company’s multiple myeloma program will be presented. Presentations will include updated safety and efficacy results from the Phase 1 CRB-401 clinical study of idecabtagene vicleucel (ide-cel, bb2121) and preliminary data from the ongoing Phase 1 CRB-402 clinical study of bb21217, as well as subgroup analyses of the pivotal Phase 2 KarMMa study of ide-cel. Ide-cel and bb21217 are investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immune therapies being studied, in partnership with Bristol-Myers Squibb, for the treatment of adult patients with MM.

Sickle Cell Disease Data at ASH (Free ASH Whitepaper)

Improvements in Health-Related Quality of Life for Patients Treated with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy
Presenting Author: Julie Kanter, MD, University of Alabama at Birmingham, Birmingham, AL
Date/Time: Oral #365, Sunday, December 6, 2020, 9:45 am PST

Resolution of Serious Vaso-occlusive Pain Crises and Reduction in Patient-Reported Pain Intensity: Results from the Ongoing Phase 1/2 HGB-206 Group C Study of LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy
Presenting Author: Alexis A. Thompson, MD, Hematology Section Head, Ann & Robert H. Lurie Children’s Hospital, Chicago, IL
Date/Time: Oral #677, Monday, December 7, 2020, 1:30 pm PST

The GRNDaD Registry: Contemporary Natural History data and an analysis of real-world patterns of use and limitations of Disease Modifying Therapy in adults with SCD
Presenting Author: Alexandra Boye-Doe, MD, University of North Carolina School of Medicine, Chapel Hill, NC
Date/Time: Poster #1730, Sunday, December 6, 2020, 7:00 am – 3:30 pm PST

Transfusion-Dependent β-Thalassemia Data at ASH (Free ASH Whitepaper)

Long-Term Efficacy and Safety of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia: Results in Patients with up to 6 Years of Follow-up
Presenting Author: Janet L. Kwiatkowski, MD, MSCE, Director, Thalassemia Center at Children’s Hospital of Philadelphia, Philadelphia, PA
Date/Time: Oral #153, Saturday, December 5, 2020, 12:00 pm PST

Favorable Outcomes in Pediatric Patients in the Phase 3 HGB-207 (Northstar-2) and HGB-212 (Northstar-3) Studies of betibeglogene autotemcel Gene Therapy for the Treatment of Transfusion-dependent β-thalassemia
Presenting Author: Alexis A. Thompson, MD, MPH, Hematology Section Head, Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL
Date/Time: Oral #154, Saturday, December 5, 2020, 12:15 pm PST

Improvement in Erythropoiesis Following Treatment with Betibeglogene Autotemcel Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia in the Phase 3 HGB-207 Study
Presenting Author: John B. Porter, MA, MD, FRCP, FRCPath, Head of Red Cell Unit, University College London Hospital, London, UK
Date/Time: Poster #776, Saturday, December 5, 2020, 7:00 am – 3:30 pm PST

Response of patients with transfusion-dependent β-thalassemia (TDT) to betibeglogene autotemcel (beti-cel; LentiGlobin for β-thalassemia) gene therapy based on HBB genotype and disease genetic modifiers
Presenting Author: Mark C. Walters MD, Medical Director, Jordan Family Center for BMT & Cellular Therapies Research, UCSF Benioff Children’s Hospital Oakland, Oakland, CA
Date/Time: Poster #1699, Sunday, December 6, 2020, 7:00 am – 3:30 pm PST

Multiple Myeloma Data at ASH (Free ASH Whitepaper)

Updated results from the Phase I CRB-402 study of anti-BCMA CAR-T cell therapy bb21217 in patients with relapsed and refractory myeloma: correlation of expansion and duration of response with T cell phenotypes
Presenting Author: Melissa Alsina, MD, Department of Blood and Marrow Transplantation and Cellular Immunotherapy, H. Lee Moffitt Cancer Center and Research Institute, Tampa, FL
Date/Time: Oral #130, Saturday, December 5, 2020, 9:45 am PST

Idecabtagene Vicleucel (ide-cel, bb2121), a BCMA-directed CAR T cell therapy, in patients with relapsed and refractory multiple myeloma: updated results from phase 1 CRB-401 study
Presenting Author: Yi Lin, MD, PhD, Division of Hematology, Mayo Clinic, Rochester, MN
Date/Time: Oral #131, Saturday, December 5, 2020, 10:00 am PST

Secondary Quality-of-Life Domains in Patients With Relapsed and Refractory Multiple Myeloma Treated With the BCMA-Directed CAR T Cell Therapy Idecabtagene Vicleucel (ide-cel; bb2121): Results from the KarMMa Clinical Trial
Author: Nina Shah, MD, University of California San Francisco, San Francisco, CA
Date/Time: Oral #437, Sunday, December 6, 2020, 12:15 pm PST

Efficacy and Safety of Idecabtagene Vicleucel (ide-cel, bb2121) in Elderly Patients with Relapsed/Refractory Multiple Myeloma: KarMMa Subgroup Analysis
Presenting Author: Jesús Berdeja, MD, Sarah Cannon Research Institute and Tennessee Oncology, Nashville, TN
Date/Time: Poster #1367, Saturday, December 5, 2020, 7:00 am – 3:30 pm PST

Characterization of Cytokine Release Syndrome in the KarMMa Study of Idecabtagene Vicleucel (ide-cel, bb2121) For Relapsed and Refractory Multiple Myeloma
Presenting Author: Ankit Kansagra, MD, Simmons Comprehensive Cancer Center, UT Southwestern Medical Center, Dallas, TX
Date/Time: Poster #1378, Saturday, December 5, 2020, 7:00 am – 3:30 pm PST

Molecular and Phenotypic Profiling of Drug Product and Post-infusion Samples from CRB-402, an Ongoing: Phase I Clinical Study of bb21217 a BCMA-directed CAR T Cell Therapy
Presenting Author: Olivia Finney, PhD, Associate Director, Immunotherapy, bluebird bio
Date/Time: Poster #1401, Saturday, December 5, 2020, 7:00 am – 3:30 pm PST

Effects of Prior Alkylating Therapies on Preinfusion Patient Characteristics and Starting Material for CAR T Cell Product Manufacturing in Late-Line Multiple Myeloma
Presenting Author: Julie Rytlewski, PhD, Bristol Myers Squibb, Princeton, NJ
Date/Time: Poster #1405, Saturday, December 5, 2020, 7:00 am – 3:30 pm PST

KarMMa-4: Idecabtagene Vicleucel (ide-cel, bb2121), a BCMA-Targeted CAR T Cell Therapy, in High-Risk Newly Diagnosed Multiple Myeloma
Presenting Author: Saad Z. Usmani, MD, Director, Clinical Research in Hematologic Malignancies, Levine Cancer Institute/Atrium Health, Charlotte, NC
Date/Time: Poster #1418, Saturday, December 5, 2020, 7:00 am – 3:30 pm PST

Healthcare Resource Utilization and Cost of Cytokine Release Syndrome and Neurologic Events in Patients with Relapsed and Refractory Multiple Myeloma Receiving the BCMA-directed CAR T Cell Therapy Idecabtagene Vicleucel (ide-cel, bb2121) in the KarMMa Trial
Presenting Author: Parmeswaran Hari, MD, Medical College of Wisconsin, Milwaukee, WI
Date/Time: Poster #1598, Saturday, December 5, 2020, 7:00 am – 3:30 pm PST

A Matching-Adjusted Indirect Comparison of Efficacy Outcomes for Idecabtagene Vicleucel (ide-cel, bb2121), a BCMA-directed CAR T Cell Therapy Versus Conventional Care in Triple-Class Exposed Relapsed and Refractory Multiple Myeloma
Presenting Author: Nina Shah, MD, University of California San Francisco, San Francisco, CA
Date/Time: Poster #1653, Saturday, December 5, 2020, 7:00 am – 3:30 pm PST

Idecabtagene Vicleucel (ide-cel, bb2121) Responses Are Characterized by Early and Temporally Consistent Activation and Expansion of CAR T Cells With a T Effector Phenotype
Presenting Author: Nathan Martin, PhD, Bristol Myers Squibb, Princeton, NJ
Date/Time: Poster #2315, Sunday, December 6, 2020, 7:00 am – 3:30 pm PST

KarMMa-3: A Phase 3 Study of Idecabtagene Vicleucel (ide-cel, bb2121), a BCMA-Targeted CAR T Cell Therapy Versus Standard Regimens in Relapsed and Refractory Multiple Myeloma
Presenting Author: Michel Delforge, MD, PhD, University Hospital Leuven, Leuven, Belgium
Date/Time: Poster #2323, Sunday, December 6, 2020, 7:00 am – 3:30 pm PST

Idecabtagene Vicleucel (ide-cel, bb2121) in Relapsed and Refractory Multiple Myeloma: Analyses of High-Risk Subgroups in the KarMMa Study
Presenting Author: Noopur S. Raje, MD, Massachusetts General Hospital, Boston, MA
Date/Time: Poster #3234, Monday, December 7, 2020, 7:00 am – 3:00 pm PST

Health State Utility Valuation in Patients with Triple-Class Exposed Relapsed and Refractory Multiple Myeloma Treated with the BCMA‑directed CAR T Cell Therapy, Idecabtagene Vicleucel (ide‑cel, bb2121): Results from the KarMMa Trial
Presenting Author: Michel Delforge, MD, PhD, University Hospital Leuven, Leuven, Belgium
Date/Time: Poster #3465, Monday, December 7, 2020, 7:00 am – 3:00pm PST

Abstracts outlining bluebird bio’s accepted data at ASH (Free ASH Whitepaper) are available on the ASH (Free ASH Whitepaper) conference website.

About LentiGlobin for SCD (bb1111)

SCD is a serious, progressive and debilitating genetic disease caused by a mutation in the β-globin gene that leads to the production of abnormal sickle hemoglobin (HbS), causing red blood cells (RBCs) to become sickled and fragile, resulting in chronic hemolytic anemia, vasculopathy and painful vaso-occlusive events (VOEs). For adults and children living with SCD, this means unpredictable episodes of excruciating pain due to vaso-occlusion as well as other acute complications—such as acute chest syndrome (ACS), stroke, and infections, which can contribute to early mortality in these patients.

LentiGlobin for SCD (bb1111) is an investigational gene therapy being studied as a potential treatment for SCD. bluebird bio’s clinical development program for LentiGlobin for SCD includes the ongoing Phase 1/2 HGB-206 study and the ongoing Phase 3 HGB-210 study.

LentiGlobin for SCD was designed to add functional copies of a modified form of the β-globin gene (βA-T87Q-globin gene) into a patient’s own hematopoietic (blood) stem cells (HSCs). Once patients have the βA-T87Q-globin gene, their red blood cells can produce anti-sickling hemoglobin (HbA-T87Q) that decreases the proportion of HbS, with the goal of reducing sickled red blood cells, hemolysis and other complications.

As of March 3, 2020, a total of 37 patients have been treated with LentiGlobin for SCD to-date in the HGB-205 (n=3) and HGB-206 (n=34) clinical studies. The HGB-206 total includes: Group A (n=7), B (n=2) and C (n=25).

LentiGlobin for SCD received orphan medicinal product designation from the European Commission for the treatment of SCD, and Priority Medicines (PRIME) eligibility by the European Medicines Agency (EMA) in September 2020.

The U.S. Food and Drug Administration (FDA) granted orphan drug designation, fast track designation, regenerative medicine advanced therapy (RMAT) designation and rare pediatric disease designation for LentiGlobin for SCD. LentiGlobin for SCD continues to be evaluated in the ongoing Phase 1/2 HGB-206 and Phase 3 HGB-210 studies.

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of LentiGlobin for SCD. For more information visit: View Source or clinicaltrials.gov and use identifier NCT02633943 for LTF-303.

LentiGlobin for SCD is investigational and has not been approved in any geography.

About betibeglogene autotemcel

Transfusion dependent beta-thalassemia (TDT) is a severe genetic disease caused by mutations in the β-globin gene that result in reduced or significantly reduced hemoglobin (Hb). In order to survive, people with TDT require chronic blood transfusions to maintain adequate Hb levels. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload.

Betibeglogene autotemcel (beti-cel) adds functional copies of a modified form of the β-globin gene (βA-T87Q-globin gene) into a patient’s own hematopoietic (blood) stem cells (HSCs). Once a patient has the βA-T87Q-globin gene, they have the potential to produce HbA-T87Q, which is gene therapy-derived adult hemoglobin, at levels that may eliminate or significantly reduce the need for transfusions.

The European Commission granted conditional marketing authorization (CMA) for beti-cel, marketed as ZYNTEGLO gene therapy, for patients 12 years and older with transfusion-dependent β-thalassemia (TDT) who do not have a β0/β0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate, but a human leukocyte antigen (HLA)-matched related HSC donor is not available.

As of March 3, 2020, a total of 60 pediatric, adolescent and adult patients, including 11 patients with at least 5 years of follow-up, across genotypes of TDT have been treated with beti-cel in the Phase 1/2 Northstar (HGB-204) and HGB-205 studies, and the Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies. In studies of beti-cel, patients were assessed for transfusion independence, defined as no longer needing red blood cell transfusions for at least 12 months while maintaining a weighted average Hb of at least 9 g/dL.

Non-serious adverse events (AEs) observed during clinical studies that were attributed to beti-cel included abdominal pain, thrombocytopenia, leukopenia, neutropenia, hot flush, dyspnoea, pain in extremity, tachycardia and non-cardiac chest pain. One serious adverse event (SAE) of thrombocytopenia was considered possibly related to beti-cel.

Additional AEs observed in clinical studies were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan, including SAEs of veno-occlusive disease. On April 28, 2020, the European Medicines Agency (EMA) renewed the CMA for beti-cel. The CMA for beti-cel is valid in the 27 member states of the EU as well as UK, Iceland, Liechtenstein and Norway. For details, please see the Summary of Product Characteristics (SmPC).

The U.S. FDA granted beti-cel orphan drug designation and Breakthrough Therapy designation for the treatment of TDT. Beti-cel is not approved in the United States. Beti-cel continues to be evaluated in the ongoing Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies.

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of beti-cel.

About idecabtagene vicleucel (ide-cel, bb2121)

Ide-cel is a B-cell maturation antigen (BCMA)-directed genetically modified autologous chimeric antigen receptor (CAR) T cell immunotherapy. The ide-cel CAR is comprised of a murine extracellular single-chain variable fragment (scFv) specific for recognizing BCMA, attached to a human CD8 α hinge and transmembrane domain fused to the T cell cytoplasmic signaling domains of CD137 4-1BB and CD3-ζ chain, in tandem. Ide-cel recognizes and binds to BCMA on the surface of multiple myeloma cells leading to CAR T cell proliferation, cytokine secretion, and subsequent cytolytic killing of BCMA-expressing cells.

Ide-cel is being developed as part of a Co-Development, Co-Promotion and Profit Share Agreement between Bristol Myers Squibb and bluebird bio. Ide-cel was granted accelerated assessment by the European Medicines Agency (EMA) on March 26, 2020, and the Marketing Authorization Application (MAA) was validated by the EMA on May 20, 2020. The FDA accepted the ide-cel Biologics License Application (BLA) for priority review on September 22, 2020.

KarMMa (NCT03361748) is a pivotal, open-label, single-arm, multicenter, multinational, Phase 2 study evaluating the efficacy and safety of ide-cel in adults with RRMM in North America and Europe. The primary endpoint of the study is overall response rate as assessed by an independent review committee (IRC) according to the International Myeloma Working Group (IMWG) criteria. Complete response rate is a key secondary endpoint. Other secondary endpoints include time to response, duration of response, progression-free survival, overall survival, minimal residual disease evaluated by Next-Generation Sequencing (NGS) assay and safety. The study enrolled 140 patients, of whom 128 received ide-cel across the target dose levels of 150-450 x 106 CAR+ T cells after receiving lymphodepleting chemotherapy. All enrolled patients had received at least three prior treatment regimens, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody, and were refractory to their last regimen, defined as progression during or within 60 days of their last therapy.

CRB-401 (NCT02658929) is an open-label Phase 1 study evaluating the preliminary safety and efficacy of ide-cel in patients with relapsed and refractory multiple myeloma (RRMM). The primary endpoint of the study is safety. CRB-401 was designed as a two-part (dose escalation and dose expansion) study to determine the maximum tolerated dose and further evaluate the safety, tolerability and clinical activity at the recommended Phase 2 dose; these findings established the recommended dose of the Phase 2 KarMMa trial. All patients have been treated in the study and follow-up is ongoing.

In addition to the pivotal KarMMa and CRB-401 trials, bluebird bio and Bristol Myers Squibb’s broad clinical development program for ide-cel includes clinical studies (KarMMa-2, KarMMa-3, KarMMa-4) exploring ide-cel combinations and activity in earlier lines of treatment for patients with multiple myeloma, including newly diagnosed multiple myeloma. For more information visit clinicaltrials.gov.

Ide-cel is not approved for any indication in any geography.

About bb21217

bb21217 is an investigational BCMA-targeted CAR T cell therapy that uses the ide-cel CAR molecule and is cultured with the PI3 kinase inhibitor (bb007) to enrich for T cells displaying a memory-like phenotype with the intention to increase the in vivo persistence of CAR T cells. bb21217 is being studied for patients with multiple myeloma in partnership with Bristol Myers Squibb.

bluebird bio’s clinical development program for bb21217 includes the ongoing Phase 1 CRB-402 study. CRB-402 is the first-in-human study of bb21217 in patients with relapsed and refractory multiple myeloma (RRMM), designed to assess safety, pharmacokinetics, efficacy and duration of effect. CRB-402 is a two-part (dose escalation and dose expansion), open-label, multi-site Phase 1 study of bb21217 in adults with RRMM. For more information visit: clinicaltrials.gov using identifier NCT03274219.

bb21217 is not approved for any indication in any geography.

On November 5, 2020 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, reported operational highlights and financial results for the third quarter ended September 30, 2020 (Press release, Intellia Therapeutics, NOV 5, 2020, View Source [SID1234570118]).

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"We are very pleased with the recent regulatory authorization to begin our Phase 1 study of NTLA-2001, which keeps us on track to dose our first patient by year-end. This is an important step toward improving the lives of ATTR patients with a potentially curative treatment, and marks our transition into a clinical-stage company. Further, advancing NTLA-2001 is a major milestone for the field of genome editing, as this is the first clinical trial of a systemically delivered CRISPR/Cas9-based therapy," said Intellia President and Chief Executive Officer John Leonard, M.D. "In parallel, we are progressing NTLA-5001 and NTLA-2002 for the treatment of AML and HAE, respectively, each to an IND or equivalent regulatory submission next year. We also continue to develop innovative capabilities across our platform, based on the Nobel Prize-winning CRISPR/Cas9 technology, to generate our next wave of therapeutic candidates."

Third Quarter 2020 and Recent Operational Highlights

ATTR Program: Intellia recently announced the authorization of its Clinical Trial Application (CTA) by the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a first-in-human clinical trial of NTLA-2001, an investigational therapy in development for the treatment of all clinical manifestations of ATTR. By applying the Company’s in vivo liver gene knockout technology, NTLA-2001 allows for the possibility of lifelong transthyretin (TTR) protein reduction after a single course of treatment. Intellia’s first-in-human study will evaluate NTLA-2001 in adults with hereditary ATTR with polyneuropathy (hATTR-PN). The Phase 1 study will be a two-part, open label, multi-center study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-2001, which will include the measurement of serum TTR levels following a single intravenous infusion. Intellia is on track to dose its first patient by the end of 2020, subject to the impact of the COVID-19 pandemic, and is submitting additional regulatory applications in other countries as part of its ongoing, global development strategy. Once safety and an optimal dose have been determined in the first-in-human study, Intellia intends to further evaluate NTLA-2001 in a broader ATTR patient population of both polyneuropathy and cardiomyopathy patients. NTLA-2001 is part of a co-development/co-promotion agreement between Intellia, the lead development and commercialization party, and Regeneron Pharmaceuticals, Inc. (Regeneron).
AML Program: NTLA-5001 is a wholly owned, T cell receptor (TCR)-T cell therapy development candidate targeting the Wilms’ Tumor 1 (WT1) antigen for the treatment of AML. The Company seeks to develop NTLA-5001 as a broadly applicable treatment for AML patients, regardless of the mutational subtypes of the cancer. Intellia continues to advance Investigational New Drug application (IND)-enabling activities and remains on track to submit an IND or IND-equivalent for NTLA-5001 in the first half of 2021. At the upcoming 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, taking place virtually from December 5 – 8, 2020, the Company will present new preclinical results in support of NTLA-5001, showing high anti-tumor activity of its lead WT1-directed TCR-T therapy in a mouse model of AML. The preclinical data will also highlight the advantages of its proprietary T cell engineering process to produce multiple, highly efficient sequential edits in T cells that have superior function and minimal translocations compared to results from standard T cell engineering approaches. Additional efforts are underway to evaluate the potential use of NTLA-5001 to treat WT1-positive solid tumors.
HAE Program: NTLA-2002 is a wholly owned, in vivo development candidate for the treatment of HAE. Today, Intellia announced results from its completed non-human primate (NHP) study of its lead lipid nanoparticle (LNP) formulation for NTLA-2002. Following a single dose, the knockout of the prekallikrein B1 (KLKB1) gene resulted in a year-long therapeutically relevant reduction of serum kallikrein protein levels and activity. Building on Intellia’s modular LNP delivery system, NTLA-2002 is designed to knock out the KLKB1 gene in the liver after a single course of treatment. This approach is expected to prevent improperly regulated bradykinin production and therefore reduce HAE attacks. During the third quarter, the Company initiated Good Laboratory Practices (GLP) toxicology studies in preparation for an IND or IND-equivalent submission for NTLA-2002, which remains on track for the second half of 2021.
Modular Platform: Intellia continues to make significant progress across its platform technologies, broadening the in vivo and ex vivo application of genome editing. This includes developing innovative CRISPR/Cas9-mediated targeted transgene insertion and allogeneic cell solutions. At the 16th Annual Meeting of the Oligonucleotide Therapeutics Society, held September 27-30, 2020, Intellia presented new data highlighting the potential to develop single-course therapies that may have a lifelong effect for a variety of genetic diseases. The data showed the persistence of both in vivo knockout and insertion CRISPR/Cas9 edits and corresponding durability of effect following a partial hepatectomy (PHx) and liver regrowth in a murine model. Unlike traditional gene therapy, for which a significant loss (over 80%) in transgene expression was observed in the insertion PHx model, Intellia’s targeted gene insertion approach yielded durable edits, with no significant loss in expression in the same model. Intellia and Regeneron are co-developing potential hemophilia A and B CRISPR/Cas9-based treatments using their jointly developed insertion capabilities. Intellia is also continuing to develop its proprietary platform to advance its wholly-owned programs.
Board of Directors: In October 2020, Intellia appointed John F. Crowley to its Board of Directors. Mr. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, is a well-established leader in biotech and pharmaceuticals and a visionary advocate for the advancement of treatments for people living with rare diseases.
Scientific Co-Founder Awarded Nobel Prize: Jennifer Doudna, Ph.D., one of Intellia’s scientific co-founders, was awarded the 2020 Nobel Prize in Chemistry for inventing the revolutionary CRISPR/Cas9 genome editing technology. Dr. Doudna shares the award with her research collaborator, Dr. Emmanuelle Charpentier.
Upcoming Events

The Company will participate in the following events during the fourth quarter of 2020:

Credit Suisse Healthcare Conference, November 11, Virtual
Barclays Gene Editing and Therapy Summit, November 16, Virtual
62nd ASH (Free ASH Whitepaper) Annual Meeting, December 5-8, Virtual
Upcoming Milestones

The Company has set forth the following for pipeline progression:

ATTR: Dose first patient in Phase 1 study by year-end
AML: Submit an IND or IND-equivalent for NTLA-5001 in 1H 2021
HAE: Submit an IND or IND-equivalent for NTLA-2002 in 2H 2021
Third Quarter 2020 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $407.9 million as of September 30, 2020, compared to $284.5 million as of December 31, 2019. The increase was driven by net proceeds of $107.7 million from the June follow-on public offering, $100.0 million upfront payment from the Regeneron collaboration expansion, which included a $30.0 million equity investment, $14.7 million of net equity proceeds raised from the Company’s "At the Market" (ATM) agreement, $18.2 million of funding received under the Regeneron and Novartis collaborations and $2.7 million in proceeds from employee-based stock plans. These increases were offset in part by cash used to fund operations of approximately $119.8 million.
Collaboration Revenue: Collaboration revenue increased by $11.6 million to $22.2 million during the third quarter of 2020, compared to $10.6 million during the third quarter of 2019. The increase was mainly driven by a $15.3 million amount recognized for the transfer of control of the license to develop the Factor VIII target for hemophilia A associated with the extension of the Regeneron collaboration.
R&D Expenses: Research and development expenses increased by approximately $12.2 million to $39.8 million during the third quarter of 2020, compared to $27.5 million during the third quarter of 2019. This increase was primarily driven by the advancement of our lead programs, research personnel growth to support these programs, and the expansion of the development organization.
G&A Expenses: General and administrative expenses increased by approximately $2.1 million to $10.6 million during the third quarter of 2020, compared to $8.4 million during the third quarter of 2019. This increase was primarily related to employee related expenses, including stock-based compensation, of $2.0 million.
Net Loss: The Company’s net loss was $27.8 million for the third quarter of 2020, compared to $23.6 million during the third quarter of 2019.
Financial Guidance

Intellia expects that its cash, cash equivalents and marketable securities as of September 30, 2020 will enable the Company to fund its anticipated operating expenses and capital expenditure requirements for at least the next 24 months. This expectation excludes any strategic use of capital not currently in the Company’s base-case planning assumptions.

Conference Call to Discuss Third Quarter 2020 Earnings

The Company will discuss these results on a conference call today, November 5, 2020, at 8 a.m. ET.

To join the call:

U.S. callers should dial 1-877-317-6789 and international callers should dial 1-412-317-6789, approximately five minutes before the call.
All participants should ask to be connected to the Intellia Therapeutics conference call.
A replay of the call will be available through the Events and Presentations page of the Investors & Media section on Intellia’s website at www.intelliatx.com, beginning on November 5, 2020 at 12 p.m. ET.