On May 2, 2019 Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) reported financial results for the three months ended March 31, 2019 and provided an operating forecast and program updates (Press release, Ligand, MAY 2, 2019, View Source [SID1234535543]). Ligand management will host a conference call today beginning at 4:30 p.m. Eastern time to discuss this announcement and answer questions.

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"The first quarter of 2019 was a great start to the year for Ligand. We saw an important new drug approved that will generate royalties for Ligand, we completed six new OmniAb and Captisol licensing deals, invested in a new technology company called Dianomi and saw our first OmniAb partnered program enter Phase 3 testing. This past quarter we divested Promacta for $827 million, converting the remaining years of royalty cash to be generated into capital today that can be reinvested into growing our business. Our partnered programs experienced significant value-enhancing events in the first quarter with a calendar for the rest of the year stacked with several substantial events," said John Higgins, Chief Executive Officer of Ligand. "At our analyst day we laid out our strategic priorities and cash investment agenda, which centers on M&A, project-financing royalty purchases, seed investments into technology companies and share repurchases. We have a highly diversified portfolio and robust financial outlook with attractive growth of top and bottom lines projected and meaningful expansion of our operating margins anticipated."

First Quarter 2019 Financial Results

Total revenues for the first quarter of 2019 were $43.5 million, compared with $56.2 million for the same period in 2018. Royalties were $19.5 million, compared with $20.8 million for the same period in 2018 and primarily consisted of royalties from Promacta, Kyprolis and EVOMELA. Royalties reflect Ligand’s sale of Promacta to Royalty Pharma as of March 6, 2019, resulting in a partial quarter of Promacta royalties, and Ligand will not receive Promacta royalties going forward. Material sales were $9.0 million, compared with $4.4 million for the same period in 2018 due to the timing of Captisol purchases for use in clinical trials and commercial products. License fees, milestones and other revenues were $15.0 million, compared with $30.9 million for the same period in 2018, with the prior-year quarter including an upfront milestone payment upon the out-license of Ligand’s Glucagon Receptor Antagonist program to Roivant Sciences.

Cost of material sales was $3.9 million for the first quarter of 2019, compared with $0.8 million for the same period in 2018. Amortization of intangibles was $3.5 million, compared with $3.3 million for the same period in 2018. Research and development expense was $11.3 million, compared with $7.4 million for the same period of 2018, due to costs associated with recent acquisitions. General and administrative expense was $11.1 million, compared with $7.6 million for the same period in 2018, due to costs associated with recent acquisitions and non-cash stock-based compensation expense.

Net income for the first quarter of 2019 was $666.3 million, or $31.32 per diluted share, compared with net income of $45.3 million, or $1.83 per diluted share, for the same period in 2018. Net income for the first quarter of 2019 was impacted by an after-tax gain of just over $640 million on the sale of Ligand’s assets and royalty on Promacta to Royalty Pharma. Adjusted net income for the first quarter of 2019 was $24.8 million, or $1.16 per diluted share excluding the impact of the gain recognized on the sale of Promacta, compared with adjusted net income of $35.7 million, or $1.55 per diluted share, for the same period in 2018.

As of March 31, 2019, Ligand had cash, cash equivalents and short-term investments of approximately $1.4 billion. Cash generated from operations during the first quarter of 2019 was $45.3 million.

2019 Financial Guidance

Ligand is affirming existing revenue guidance for 2019 with total revenues expected to be approximately $118 million including royalties of approximately $48 million, material sales of approximately $27 million and license fees and milestones of approximately $43 million. Ligand is also affirming its existing adjusted earnings per share guidance of approximately $3.20, which excludes the impact of the gain recognized on the sale of Promacta of $30.09 per share compared to the initial estimate of the impact of the gain of $29.05 per share.

First Quarter 2019 and Recent Business Highlights

Promacta

In March 2019, Ligand announced the sale of Promacta to Royalty Pharma for $827 million in cash. As of March 6, 2019 Ligand will not receive any royalty on sales of Promacta.
Kyprolis (carfilzomib), an Amgen Product Utilizing Captisol

On April 30, 2019, Amgen reported first quarter 2019 net sales of Kyprolis of $245 million, a $23 million or 10% increase over the same period in 2018.
Recent Acquisitions, Targeted Investments and Partner Funding Events

Ligand announced a $3 million investment in Dianomi Therapeutics in exchange for 1) a tiered royalty of 2% or 3% based on level of net sales for the first five products to be approved using Dianomi’s patented Mineral Coated Microparticle (MCM) technology, and 2) a loan convertible into $1 million of equity at the next qualified financing.
Seelos Therapeutics completed a reverse merger with Apricus Biosciences and is now publicly traded on the Nasdaq Capital Market under the symbol "SEEL". In conjunction with the transaction, Seelos issued common stock and warrants in a private round led by a group of leading venture capital investors, for gross proceeds of $18 million.
CStone Pharmaceuticals listed shares on the Hong Kong Stock Exchange and raised $266 million in an equity offering that, in part, will be used to fund the company’s OmniAb-derived Phase 2/3 program CS1001.
Internal R&D

Ligand announced completion of enrollment of the Company’s Phase 1 clinical trial of its internal Captisol-enabled Iohexol program and announced that top-line data from the trial is expected in the third quarter of 2019.
Additional Pipeline and Partner Developments

Ligand’s portfolio of partnerships now includes more than 200 Shots on Goal driven by business development and licensing activity, and the expansion and advancement of OmniAb partnerships.
Sage Therapeutics announced U.S. Food and Drug Administration (FDA) approval of ZULRESSO (brexanolone) injection for the treatment of postpartum depression. Ligand received a $3 million milestone payment as a result of the approval. Sage Therapeutics plans to launch ZULRESSO in late June 2019.
Daiichi Sankyo announced receipt of marketing approval in Japan for MINNEBRO (esaxerenone) for the treatment of hypertension.
Melinta Therapeutics announced preparation of a supplemental new drug application (sNDA) for Baxdela in community-acquired bacterial pneumonia with the sNDA expected to be filed in the second quarter of 2019.
CStone Pharmaceuticals announced dosing of the first patient in a Phase 3 clinical trial assessing OmniAb-derived CS1001 in combination with chemotherapy for the treatment of gastric adenocarcinoma or gastro-esophageal junction adenocarcinoma.
Viking Therapeutics announced that additional VK2809 Phase 2 data was presented at the 2019 annual meeting of the European Association for the Study of Liver, and that results demonstrated promising efficacy at doses as low as 5 mg daily. Viking also announced plans to initiate a Phase 2b study of VK2809 in biopsy-confirmed NASH in the second half of 2019.
Metavant updated Ligand that they no longer plan to initiate a clinical proof-of-concept trial this year for RVT-1502 in Type 1 diabetes following requests from FDA for additional non-clinical studies. Metavant is evaluating its development plans for the program, and we expect them to provide an update on the status of the program.
Verona Pharma announced positive interim efficacy and safety data from part one of a two-part Phase 2 clinical trial of a dry powder inhaler formulation of ensifentrine in patients with moderate-to-severe chronic obstructive pulmonary disease (COPD).
Verona Pharma also announced the European Patent Office granted an additional key patent relating to the company’s lead development candidate ensifentrine.
Sermonix Pharmaceuticals announced a poster presentation on the performance of its lead investigational drug, lasofoxifene, at ENDO 2019.
Opthea Limited announced that the independent Data and Safety Monitoring Board for the company’s ongoing Phase 2b study of OPT-302 in wet age-related macular degeneration reaffirmed its positive recommendation that the trial continue without modification.
Aptevo Therapeutics provided an update on OmniAb-derived APVO436 and announced that Phase 1 data is anticipated in the fourth quarter of 2019. New preclinical data for APVO436 was also presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2019 Annual Meeting.
OmniAb partner xCella Biosciences presented high-throughput functional screening of antibody libraries, highlighting OmniRat and OmniChicken, at the 2019 Protein Engineering Summit (PEGS).
Business Development

Ligand announced a worldwide license agreement with Genagon Therapeutics AB to use the OmniAb platform technologies to discover fully human antibodies. Genagon is an immuno-oncology biotechnology company located in Sweden. Ligand is eligible to receive development milestone payments and tiered royalties on future product sales.
Ligand disclosed a worldwide license agreement with a San Francisco Bay Area venture-stage biotechnology company to use the OmniAb platform technologies to discover fully human antibodies. Ligand is eligible to receive development and commercial milestone payments and royalties on future product sales.
Ligand entered into new Captisol clinical use or commercial license and supply agreements with Merck KGaA, reVision Therapeutics, Takeda and SQ Innovation.
Publications and Presentations

At PEGS 2019 Ligand scientists announced the launch of OmniClic, a novel next-generation common light chain OmniChicken-based antibody discovery technology focused on bispecific antibodies.
Preclinical data of the combination of the BCL-2 inhibitor S55746 and the MCL1 inhibitor S63845, compounds originating from the Servier collaboration and now partnered with Novartis, in models of AML were recently published in the journal Leukemia, demonstrating potential to rapidly suppress leukemia with limited toxicity to normal human bone marrow cells compared to chemotherapy.
Adjusted Financial Measures

The Company reports adjusted net income and adjusted net income per diluted share in addition to, and not as a substitute for, or superior to, financial measures calculated in accordance with GAAP. The Company’s financial measures under GAAP include stock-based compensation expense, amortization of debt-related costs, amortization related to acquisitions and intangible assets, changes in contingent liabilities, mark-to-market adjustments for amounts relating to its equity investments in public companies, unissued shares relating to the Senior Convertible Notes, gain on the sale of Promacta and others that are listed in the itemized reconciliations between GAAP and adjusted financial measures included at the end of this press release. However, other than with respect to total revenues, the Company only provides financial guidance on an adjusted basis and does not provide reconciliations of such forward-looking adjusted measures to GAAP due to the inherent difficulty in forecasting and quantifying certain amounts that are necessary for such reconciliation, including adjustments that could be made for changes in contingent liabilities, changes in the market value of its investments in public companies, stock-based compensation expense and effects of any discrete income tax items. Management has excluded the effects of these items in its adjusted measures to assist investors in analyzing and assessing the Company’s past and future core operating performance. Additionally, adjusted earnings per diluted share is a key component of the financial metrics utilized by the Company’s board of directors to measure, in part, management’s performance and determine significant elements of management’s compensation.

Conference Call

Ligand management will host a conference call today beginning at 4:30 p.m. Eastern time (1:30 p.m. Pacific time) to discuss this announcement and answer questions. To participate via telephone, please dial (833) 591-4752 from the U.S. or (720) 405-1612 from outside the U.S., using the conference ID 6375579. To participate via live or replay webcast, a link is available at www.ligand.com.

On May 2, 2019 Acorda Therapeutics, Inc. (NASDAQ: ACOR) reported a financial and pipeline update for the quarter ended March 31, 2019 (Press release, Acorda Therapeutics, MAY 2, 2019, View Source [SID1234535541]).

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"Since INBRIJA became commercially available at the end of February, the feedback from both healthcare professionals and people with Parkinson’s, or PWPs, has been enthusiastic, and our Prescription Support Services center has received approximately 2,000 prescription requests. Our market research has consistently shown that both physicians and PWPs consider OFF periods one of the most troubling and disruptive aspects of Parkinson’s, and that they regard INBRIJA as having the potential to address this significant unmet need," said Ron Cohen, M.D., Acorda’s President and CEO. "We are currently meeting with insurers to discuss formulary placement. We are also continuing to roll out in-person and digital programs to educate healthcare professionals and PWPs about INBRIJA."

First Quarter 2019 Financial Results

For the quarter ended March 31, 2019, the Company reported INBRIJA net revenue of $1.3 million. INBRIJA became commercially available on February 28, 2019.

For the quarter ended March 31, 2019, the Company reported AMPYRA net revenue of $40.1 million compared to $102.8 million for the same quarter in 2018. In September 2018, AMPYRA lost its exclusivity and generics entered the market. Consequently, the Company expects AMPYRA revenue to continue to decline.

Research and development (R&D) expenses for the quarter ended March 31, 2019 were $16.0 million, including $0.7 million of share-based compensation compared to $30.6 million, including $1.7 million of share-based compensation for the same quarter in 2018.

Sales, general and administrative (SG&A) expenses for the quarter ended March 31, 2019 were $52.7 million, including $2.8 million of share-based compensation compared to $47.6 million, including $4.2 million of share-based compensation for the same quarter in 2018.

Benefit from income taxes for the quarter ended March 31, 2019 was $0.7 million compared to a provision for income taxes of $3.5 million for the same quarter in 2018.

The Company reported a GAAP net loss of $47.6 million for the quarter ended March 31, 2019, or $1.00 per diluted share. GAAP net loss in the same quarter of 2018 was $8.2 million, or $0.18 per diluted share.

Non-GAAP net loss for the quarter ended March 31, 2019 was $26.5 million, or $0.56 per diluted share. Non-GAAP net income in the same quarter of 2018 was $6.8 million, or $0.14 per diluted share. This quarterly non-GAAP net (loss) income measure, more fully described below under "Non-GAAP Financial Measures," excludes share-based compensation charges, non-cash interest charges on our debt, and changes in the fair value of acquired contingent consideration. A reconciliation of the GAAP financial results to non-GAAP financial results is included with the attached financial statements.

At March 31, 2019, the Company had cash, cash equivalents and short-term investments of $343.3 million compared to $445.6 million at year end 2018. The decline in cash was due in large part to non-recurring payments of approximately $45 million that were made in the first quarter, primarily related to AMPYRA inventory purchases. The Company does not expect to purchase additional AMPYRA inventory in 2019.

For the remainder of 2019, the Company expects cash expenditures to align with normal operating activities, and continues to believe that it can become cash flow positive without raising additional capital, based on its long-term projections.

2019 Financial Guidance

During INBRIJA’s launch year, the Company does not expect to provide INBRIJA revenue guidance.

The Company will no longer provide revenue guidance for AMPYRA, due to the unpredictable trajectory of revenue decline given the entrance of generics.

R&D expenses for the full year 2019 are expected to be $70-$80 million and SG&A expenses for the full year 2019 are expected to be $200-$210 million. This guidance is a non-GAAP projection that excludes share-based compensation as more fully described below under "Non-GAAP Financial Measures."

First Quarter 2019 Highlights

INBRIJA (levodopa inhalation powder)

In March, the Company submitted responses to the INBRIJA Marketing Authorization Application (MAA) Day 120 list of questions. A final decision from the European Commission is expected before the end of 2019. Acorda is seeking approval to market INBRIJA in the European Union.

In March, new one-year safety and exploratory efficacy outcomes data from the extension of the Phase 3 SPANSM-PD trial were presented at the Academy of Managed Care Pharmacy (AMCP) Managed Care & Specialty Pharmacy Annual Meeting.

Webcast and Conference Call

The Company will host a conference call today at 8:30 a.m. ET. To participate in the conference call, please dial (866) 393-4306 (domestic) or (734) 385-2616 (international) and reference the access code 4783943. The presentation will be available on the Investors section of www.acorda.com.

A replay of the call will be available from 11:30 a.m. ET on May 2, 2019 until 11:59 p.m. ET on June 2, 2019. To access the replay, please dial (855) 859-2056 (domestic) or (404) 537-3406 (international); reference code 4783943. The archived webcast will be available in the Investor Relations section of the Acorda website at www.acorda.com.

Non-GAAP Financial Measures

This press release includes financial results prepared in accordance with accounting principles generally accepted in the United States (GAAP), and also certain historical and forward-looking non-GAAP financial measures. In particular, Acorda has provided non-GAAP net loss, adjusted to exclude the items below, and has provided 2019 guidance for R&D and SG&A expenses on a non-GAAP basis. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. However, the Company believes the presentation of non-GAAP net loss, when viewed in conjunction with our GAAP results, provides investors with a more meaningful understanding of our ongoing and projected operating performance because this measure excludes (i) non-cash compensation charges and benefits that are substantially dependent on changes in the market price of our common stock, (ii) non-cash interest charges related to the accounting for our outstanding convertible debt which are in excess of the actual interest expense owing on such convertible debt, as well as non-cash interest related to the Fampyra monetization, and acquired Biotie debt, and (iii) changes in the fair value of acquired contingent consideration which do not correlate to our actual cash payment obligations in the relevant periods. The Company believes its non-GAAP net loss measure helps indicate underlying trends in the Company’s business and is important in comparing current results with prior period results and understanding projected operating performance. Also, management uses this non-GAAP financial measure to establish budgets and operational goals, and to manage the Company’s business and to evaluate its performance.

In addition to non-GAAP net loss, we have provided 2019 guidance for R&D and SG&A expenses on a non-GAAP basis. Due to the forward looking nature of this information, the amount of

compensation charges and benefits needed to reconcile these measures to the most directly comparable GAAP financial measures is dependent on future changes in the market price of our common stock and is not available at this time. The Company believes that these non-GAAP measures, when viewed in conjunction with our GAAP results, provide investors with a more meaningful understanding of our ongoing and projected R&D and SG&A expenses. Also, management uses these non-GAAP financial measures to establish budgets and operational goals, and to manage the Company’s business and to evaluate its performance.

On May 2, 2019 Apollo Endosurgery, Inc. ("Apollo") (Nasdaq: APEN), a global leader in less invasive medical devices for gastrointestinal and bariatric procedures, reported financial results for the first quarter ended March 31, 2019 (Press release, Lpath, MAY 2, 2019, View Source [SID1234535539]).

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First Quarter 2019 Highlights
•Endo-bariatric sales, representing our core business moving forward, increased 5% from $10.3 million to $10.8 million, with ESS sales increasing 13% and IGB sales declining 5%
•On a constant currency basis, endo-bariatric sales increased 9% with ESS sales increasing 17% and IGB sales equal to the prior year
•U.S. ESS sales increased 20% year-over-year in the quarter
Todd Newton, CEO of Apollo, commented, "We were pleased with our ESS product sales growth this quarter. We are also pleased with our Sx launch progress to date. The activity and resulting new account pipeline is encouraging to us that Sx will roll out very well as the year progresses. Similarly, we think our IGB product is positioned well for a return to growth."
First Quarter 2019 Results
U.S. Endo-bariatric sales increased 9% to $4.5 million, while outside the U.S. ("OUS") Endo-bariatric sales of $6.4 million increased 3% as reported and 10% in constant currency. Worldwide, total Endo-bariatric sales of $10.8 million increased 5% as reported and 9% constant currency. Of our Endo-bariatric product sales in the first quarter, 60% related to our Endoscopic Suturing System ("ESS" or "OverStitch") and 40% related to our Intragastric Balloon ("IGB" or "Orbera").
U.S. ESS product sales increased 20% to $3.0 million, while OUS ESS product sales of $3.5 million increased 7% as reported and 14% in constant currency. Worldwide ESS sales of $6.5 million grew 13% as reported and 17% constant currency. Growth was due to continuing new user adoption and greater product utilization in existing accounts.
U.S. IGB product sales decreased $0.2 million, or 9%, to $1.5 million in the first quarter of 2019. OUS IGB product sales were unchanged at $2.9 million, on an as reported basis, and increased 5% in constant currency as higher sales of Orbera365 were partially offset by weaker six-month balloon sales in other OUS markets. On a worldwide basis, IGB sales of $4.3 million declined 5% as reported and were flat in constant currency terms.
The divestiture of our Surgical products in December of 2018 affects the comparability of our total revenues in the first quarter of 2019 versus 2018. Total revenues in the first quarter of 2019 only include Surgical product sales in certain OUS markets where we continue to temporarily distribute these products. Total revenues in the first quarter of 2019 were $13.2 million, which included $1.7 million of Surgical product sales, compared to $15.7 million in the first quarter of 2018, which included $5.2 million of Surgical product sales, for a decrease of 16% or $2.5 million.
Gross margin for the first quarter 2019 was 55%, compared to 58% for the first quarter 2018 due to lower Surgical product sales following their divestiture and a greater proportion of our overall product sales coming from our ESS products, which realize a lower gross margin than our other products. Partially offsetting the gross margin effects of these changes in our product sales mix were the gross margin improvement projects we completed in late 2018.
Total operating expenses decreased $7.0 million to $9.8 million in the first quarter 2019, compared to the first quarter 2018. The decrease was primarily due to a one-time non-cash settlement gain of $5.6 million resulting from the resolution of a dispute regarding amounts charged for inventory purchases and transition services through 2016 following our December 2013 acquisition of the obesity intervention division of Allergan, Inc. Excluding this gain, total operating expenses decreased $1.4 million due largely to lower amortization expense resulting from the reduction in our intangible assets after the recent divestiture of our Surgical products.

Net loss for the first quarter 2019 was $2.8 million compared to $8.1 million for the first quarter 2018. The reduction in net loss was primarily due to the one-time settlement gain. Excluding this one-time gain, net loss would have been $8.4 million, an increase of $0.3 million due largely to lower Surgical product sales following our December 2018 divestiture, partially offset by the reduced amortization expense mentioned previously.

Cash, cash equivalents and restricted cash were $31.0 million as of March 31, 2019.
Conference Call
Apollo will host a conference call on May 2, 2019 at 3:30 p.m. Central Time / 4:30 p.m. Eastern Time to discuss Apollo’s operating results for the first quarter ended March 31, 2019.
To participate in the conference call dial (866) 393-4306 for domestic callers and (734) 385-2616 for international callers. The conference ID number is 7094276. A live webcast of the conference call will be made available on the "Events and Presentations" section of our Investor Relations website: www.ir.apolloendo.com.
A replay of the webcast will be made available on Apollo’s website, www.apolloendo.com following the event.
Non-GAAP Financial Measures
To supplement our financial results we are providing a non-GAAP financial measure, percentage revenue change in constant currency, which removes the impact of changes in foreign currency exchange rates that affect the comparability and trend of revenues. Percentage revenue change in constant currency is calculated by translating current foreign currency sales at last year’s exchange rate. This supplemental measure of our performance is not required by, and is not determined in accordance with GAAP.
We believe the non-GAAP financial measure included herein is helpful in understanding our current financial performance. We use this supplemental non-GAAP financial measure internally to understand, manage and evaluate our business, and make operating decisions. We believe that making non-GAAP financial information available to investors, in addition to GAAP financial information, may facilitate more consistent comparisons between the company’s performance over time with the performance of other companies in the medical device industry, which may use similar financial measures to supplement their GAAP financial information. However, our non-GAAP financial measure is not meant to be considered in isolation or as a substitute for the comparable GAAP metric.

On May 2, 2019 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and reported financial results for the first quarter ending March 31, 2019 (Press release, PTC Therapeutics, MAY 2, 2019, View Source [SID1234535538]).

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"We believe we are making great strides towards our vision of having many more treatments for patients of rare disorders," said Stuart Peltz, Ph.D., CEO of PTC Therapeutics. "The approval of Translarna expands our footprint in Brazil. We are now preparing for the launches for multiple products, including Tegsedi for hATTR and Waylivra for FCS in Latin America. These differentiated therapies for rare disorders have the potential to be part of growing our revenue base to $1.5 billion by 2023."

Key First Quarter and Other Corporate Highlights:

Expanding commercial platform

Translarna received approval from the Brazilian health regulatory authority (ANVISA) which will allow for expanded market access.

Waylivra received a positive CHMP opinion from the European medical authorities in the first quarter 2019 and will be referred to the European Commission for consideration. After ratification by the European Commission, PTC plans to initiate early access programs in Latin America in 2019.

Tegsedi application submitted to the Brazilian health regulatory authority (ANVISA) was granted priority review, with expected approval by year end 2019. PTC has initiated early access programs for Tegsedi to support patient diagnosis and monitoring in Latin America.

PTC has submitted a sNDA with the FDA for Emflaza for patients 2 to 5 years old and received an approval action date of July 4, 2019.

Advancing gene therapy portfolio

As previously disclosed, PTC plans to submit a BLA with the FDA in late 2019 followed by an MAA in Europe for the AADC deficiency gene therapy program with anticipated commercial launch in the United States in 2020.

Friedreich ataxia program is advancing with an expected IND submission in late 2019 and subsequent entry in the clinic.

PTC is in the process of finalizing a long-term lease on an existing biologics manufacturing facility for the gene therapy pipeline.

Risdiplam SMA data expected at American Academy of Neurology (AAN)

As previously disclosed, based on regulatory interactions an NDA and MAA is planned for the second half of this year with the intention to support a broad label to treat SMA Types 1, 2, & 3 patients.

The SMA program is a collaboration between PTC, Roche and SMA Foundation.

Net sales over $1B would be subject to mid-teens royalties to PTC from Roche, resulting in potential royalties to PTC in excess of $200M per year. Potential remaining regulatory and sales-based milestones are approximately $400M.

Growing pipeline and R&D capabilities

As previously disclosed, PTC’s splicing platform has generated another development candidate, PTC258, for Familial dysautonomia, a rare genetic neurological disorder causing life-threatening medical complications from birth. PTC258 is advancing through IND-enabling studies to enter the clinic in late 2019. This program is in collaboration with MGH and NYU

Translarna’s dystrophin study in currently enrolling for potential U.S. regulatory re-submission for accelerated approval in 2020.

PTC’s oncology portfolio continues to advance with studies in AML with PTC299 and DIPG & LMS for PTC596. PTC expects these studies to fully enroll by the end of 2019.

PTC Re-iterates Full Year 2019 Guidance:

PTC anticipates full year DMD franchise net product revenues to be between $285 and $305 million.

PTC anticipates GAAP R&D and SG&A expense for the full year 2019 to be between $395 and $405 million.

PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2019 to be between $360 and $370 million, excluding estimated non-cash, stock-based compensation expense of approximately $35 million.

First Quarter 2019 Financial Highlights:

Total revenues were $53.6 million for the first quarter of 2019, compared to $56.1 million for the first quarter of 2018.

Translarna net product revenues were $35.3 million for the first quarter of 2019, compared to $36.8 million for the first quarter of 2018. These results reflect lumpiness in ordering patterns from Latin America.

Emflaza net product revenues were $17.8 million for the first quarter of 2019, compared to $19.2 million for the first quarter of 2018. These results reflect first quarter dynamics including seasonality and a planned transition to a new specialty pharmacy distributor.

GAAP R&D expenses were $52.6 million for the first quarter of 2019, compared to $31.4 million for the first quarter of 2018. The increase in R&D expenses reflects costs associated with advancing the gene therapy platform and increased investment in research programs as well as advancement of the clinical pipeline.

Non-GAAP R&D expenses were $47.9 million for the first quarter of 2019, excluding $4.7 million in non-cash, stock-based compensation expense, compared to $27.6 million for the first quarter of 2018, excluding $3.7 million in non-cash, stock-based compensation expense.

GAAP SG&A expenses were $40.6 million for the first quarter of 2019, compared to $33.0 million for the first quarter of 2018. The increase in SG&A expenses were primarily due to continued investment to support our commercial activities.

Non-GAAP SG&A expenses were $36.0 million for the first quarter of 2019, excluding $4.6 million in non-cash, stock-based compensation expense, compared to $29.0 million for the first quarter of 2018, excluding $4.0 million in non-cash, stock-based compensation expense.

Change in the fair value of deferred and contingent consideration was $21.2 million for the first quarter of 2019. The change in fair value of deferred and contingent consideration is related to the fair valuation of potential future consideration to be paid to former equity holders of Agilis Biotherapeutics, Inc. (Agilis) in connection with PTC’s acquisition of Agilis, which closed in August 2018.

Net loss was $72.1 million for the first quarter of 2019, compared to net loss of $19.3 million for the first quarter of 2018.

Cash, cash equivalents, and marketable securities were $407.2 million at March 31, 2019, compared to $227.6 million at December 31, 2018.

Shares issued and outstanding as of March 30, 2019 were 58,418,790 .

Non-GAAP Financial Measures:
In this press release, the financial results and financial guidance of PTC are provided in accordance with accounting principles generally accepted in the United States (GAAP) and using certain non-GAAP financial measures. In particular, the non-GAAP financial measures exclude non-cash, stock-based compensation expense. These non-GAAP financial measures are provided as a complement to financial measures reported in GAAP because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management’s opinion, these non-GAAP financial measures are useful to investors and other users of PTC’s financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company’s future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. Quantitative reconciliations of the non-GAAP financial measures to their respective closest equivalent GAAP financial measure are included in the table below.

Today’s Conference Call and Webcast Reminder:
Today’s conference call will take place at 4:30 pm ET and can be access by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the call and providing the passcode 1559069. A live, listen-only webcast of the conference call can be accessed on the investor relations section of the PTC website at www.ptcbio.com. The accompanying slide presentation will be posted on the investor relations section of the PTC website. A webcast replay of the call will be available approximately two hours after completion of the call and will be archived on the company’s website for two weeks.

On May 1, 2019 MaxCyte, the global clinical-stage cell-based therapies and life sciences company, reported that Robert Keefe, Director of Technical Operations, provided an oral presentation on April 29, titled "Novel mRNA-based Autologous CAR Therapies in Oncology," at the annual meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) in Washington, DC (Press release, MaxCyte, MAY 1, 2019, View Source [SID1234537622]).

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Dr. Keefe highlighted the CARMA platform’s key differentiating features, including manufacture and delivery to a patient in a fraction of the time compared to traditional chimeric antigen receptor (CAR) therapies and without a viral component. He also described the manufacturing feasibility data for MaxCyte’s first CARMA drug candidate, MCY-M11, a mesothelin-targeting chimeric antigen receptor (CAR), which is currently being evaluated in a Phase I clinical trial in mesothelin-expressing solid tumors at the National Cancer Institute (NCI) and Washington University in St Louis.

"The advancement of our first CARMA clinical trial, which is consistently showing the feasibility of our rapid manufacturing process, is significant for MaxCyte and the application of our technology," said Claudio Dansky Ullmann, MD, MaxCyte’s Chief Medical Officer. "Development of a cell therapy with application in solid tumors is impactful for patients with unmet needs in a variety of cancers and we look forward to further advancing this program."

The CARMA platform offers an innovative approach to cell-based therapies that can be applied in a broad range of diseases, including solid tumors. The manufacturing process for MCY-M11 utilizes MaxCyte’s proprietary Flow Electroporation technology to transfect mRNA into fresh (i.e., unexpanded) peripheral blood mononuclear cells (PBMCs). The entire CARMA manufacturing process allows for streamlined manufacturing providing a much needed faster turnaround of autologous cell therapy to patients. CARMA cell therapy was engineered with the intention of reducing potential adverse events that have been evident with other CAR technologies, and allowing for multiple dosing, a feature that may be key in the treatment of solid tumors with cellular therapies.

Last month, this trial was featured as a "trial in progress" at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting , via a poster highlighting key aspects of the study design.