Forge Biologics Announces Closing of $120 Million Series B Financing

Forge Biologics, a gene therapy-focused contract development and manufacturing organization, reported that the closing of a $120 million Series B financing (Press release, Forge Biologics, APR 29, 2021, View Source [SID1234578742]). The financing was led by RA Capital Management with participation from Perceptive Advisors and related affiliates, Surveyor Capital (a Citadel company), Octagon Capital, and Marshall Wace. Existing investors Perceptive Xontogeny Venture Fund and Drive Capital also participated. In connection with the financing, Matthew Hammond, Ph.D., of RA Capital, and Fred Callori of the Perceptive Xontogeny Venture Fund will join the Company’s Board of Directors.

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This press release features multimedia. View the full release here: View Source

"We are very pleased to be working with RA Capital and a strong syndicate of top-tier life sciences investors who share our vision as we now advance our growth as a global gene therapy manufacturing and development company," said Timothy J. Miller, Ph.D., CEO, President and Co-Founder of Forge Biologics. "We have built Forge into a focused gene therapy development engine with a firm commitment to helping our clients provide potentially life-saving gene therapies to patients. We believe that focusing entirely on gene therapy will allow us to best serve our clients and patients by efficiently delivering high quality product."

Forge will use the proceeds of this Series B financing to accelerate the expansion of its AAV manufacturing CDMO capabilities with cGMP production capacity, as well as operate its subsidiaries that are advancing novel AAV gene therapy programs. Forge brings a patients-first approach to accelerate the development of transformative medicines for those who need them most and is addressing the growing demand for gene therapy manufacturing capacity. Through its currently-operational 175,000 square foot cGMP facility in Columbus, Ohio, dedicated to AAV viral vector manufacturing, Forge offers end-to-end manufacturing services, including research and toxicology grade AAV production, to accelerate gene therapy programs from preclinical through clinical and commercial stage manufacturing.

"The success of complex biologics like AAV is exhausting existing manufacturing capacity around the industry. This financing will help to address this industry-wide capacity shortage by properly capitalizing an emerging gene therapy-focused CDMO capable of producing high quality cGMP product for its clients," said Matthew Hammond, Ph.D., principal at RA Capital Management. "We are confident that Forge’s experienced team will become the trusted partner of innovative therapeutics companies, working collaboratively with clients to successfully deliver AAV manufacturing solutions."

Through its subsidiaries, Forge is also working to advance a proprietary pipeline of novel gene therapies, including its lead program FBX-101 for the treatment of patients with Krabbe disease, a first-in-human gene therapy utilizing an adeno-associated virus (AAV) to deliver a functioning copy of the GALC gene intravenously to cells in the central nervous system (CNS) and peripheral organs.

Chardan Capital Markets acted as exclusive placement agent for the offering, with Ice Miller acting as legal advisor.

BioMarin Announces First Quarter 2021 Financial Results and Corporate Updates

On April 29, 2021 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) (BioMarin or the Company) reported its financial results for the first quarter ended March 31, 2021 (Press release, BioMarin, APR 29, 2021, View Source [SID1234578779]).

"We mark the start of 2021 with strong financial results, and a number of exciting regulatory decisions ahead this year." said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin. "The potential approval in Europe this summer of vosoritide, the first potential therapeutic option for children with achondroplasia, will set the stage for our next significant phase of growth, especially considering the EMEA region is 3 times larger than the U.S. market. To date, the interactions with the review team at the European Medicines Agency have been very positive. Next, we plan to re-submit the application for valoctocogene roxaparvovec gene therapy, to treat hemophilia A, to the EMA in the second quarter, to be followed by potential approval of vosoritide in the United States later this year."

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Mr. Bienaimé continued, "We are well positioned to deliver strong results for the full-year 2021, and reaffirm our financial guidance provided early this year. This takes into consideration the uneven ordering patterns in our dynamic and global commercial business, as well as continued uncertainty outside of the United States from COVID-19, and further underscores the essential nature of our products to the people who rely on them. Taken together, our financial strength, including positive cash flows from operations of $113.5 million in the first quarter, combined with near-term opportunities from potential new products and a robust early-stage pipeline, we believe that 2021 will be pivotal to BioMarin’s evolution."

Financial Highlights:
•Net Product Revenues for the first quarter of 2021 decreased to $467.8 million, compared to $489.0 million for the same period in 2020. The decrease in Net Product Revenues was primarily attributed to the following:
•Lower Kuvan product revenues primarily due to known generic competition in the U.S.; partially offset by
•Higher Vimizim product revenues primarily due to timing of orders from Europe, Middle East and North Africa; and
•Higher Palynziq product revenues primarily driven by a combination of revenue from more U.S. patients achieving maintenance dosing and new patients initiating therapy.

•GAAP Net Income decreased $64.0 million to $17.4 million for the first quarter of 2021 compared to $81.4 million for the same period in 2020. The decrease was primarily due to the absence of the gain on sale the Firdapse commercial assets totaling $59.5 million in the first quarter of 2020 and a decrease in gross profits due to the lower Kuvan product revenues due to generic competition in the U.S. This decrease was partially offset by lower selling, general and administrative (SG&A) expenses primarily due to a decrease in foreign currency exchange losses in the first quarter of 2021.

•Non-GAAP Net Income for the first quarter of 2021 decreased to $104.4 million compared to Non-GAAP Income of $116.5 million for the same period in 2020. The decline in Non-GAAP Income for the quarter, compared to the same period in 2020, was primarily attributed to lower gross profits, partially offset by lower SG&A expenses primarily due to a decrease in foreign currency exchange losses in the first quarter of 2021.
Commercial Portfolio (Naglazyme, VIMIZIM, Brineura, Palynziq, Kuvan and Aldurazyme)
•Naglazyme, VIMIZIM and Brineura maintained robust patient compliance in the quarter and continue to grow based on strong underlying demand.
◦Patients on commercial Naglazyme and Vimizim therapy both increased approximately 10% year-over-year.
2

◦Patients on commercial Brineura therapy increased by more than 30% year-over-year.
•Order timing for both Naglazyme and VIMIZIM are expected to be more concentrated in the first half of 2021 as compared to the second half of 2021.
•Palynziq top-line results in 2021 are expected to increase approximately 35% based on the mid-point of full-year 2021 guidance as compared to full-year 2020 results.
•Palynziq growth in European, Middle East and African regions (EMEA) has been impacted by ongoing challenges due to COVID-19 with Palynziq revenue from EMEA expected to increase when PKU clinics have more freedom to operate and start additional patients.
◦The number of U.S. Patients on commercial Palynziq therapy increased more than 20% year-over-year and is expected to continue to grow as U.S. PKU clinics re-open over the coming quarters.
•Loss of Kuvan U.S. market share, due to the introduction of generics following BioMarin’s loss of U.S. market exclusivity, is consistent with expectations.
•Aldurazyme contributions were driven by the timing of product released and transfer of control to Genzyme, who is responsible for marketing Aldurazyme worldwide.
Late-stage Regulatory Portfolio (Vosoritide and valoctocogene roxaparvovec)
•In Europe, with respect to vosoritide for the treatment of achondroplasia, BioMarin is in the final stages of the review procedure ahead of the anticipated June 2021 Committee for Medicinal Products for Human Use (CHMP) opinion. Assuming a positive CHMP opinion, the European Commission could potentially grant marketing authorization for vosoritide in the third quarter of 2021.
•In Europe, with respect to valoctocogene roxaparvovec for the treatment of severe hemophilia A, based on positive pre-submission feedback in the current quarter, BioMarin reaffirms its plan to submit the Marketing Authorization Application with one-year results from the Phase 3 GENEr8-1 study to the European Medicines Agency (EMA) in June 2021.
•In the U.S., BioMarin provided the U.S. Food and Drug Administration (FDA) with the two-year results from the Phase 3 extension study with vosoritide to supplement the New Drug Application (NDA) already under review. As anticipated, the FDA designated this submission as a major amendment to the application, thus extending the Prescription Drug User Fee Act (PDUFA) target action date by three months to November 20, 2021 to provide time for a full review of the submission.
◦Also in the first quarter of 2021, the FDA pre-approval inspection of BioMarin’s Novato facility for the manufacture of vosoritide drug substance was completed, representing another important milestone as vosoritide advances through the review process.
•During the current quarter, the FDA reiterated their recommendation that BioMarin submit two-year follow-up safety and efficacy data on all study participants from the GENEr8-1 study to support their benefit/risk assessment of valoctocogene roxaparvovec. BioMarin is targeting a Biologics License Application (BLA) submission in the second quarter of 2022 assuming favorable results, followed by an expected 6-month review procedure by the FDA.
◦The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to valoctocogene roxaparvovec. The RMAT designation is complementary to Breakthrough Therapy Designation, which BioMarin received in 2017.
3

Earlier-stage Development Portfolio (BMN 307, BMN 255, BMN 331, DiNA-001, Allen Institute Collaboration)
◦BMN 307: Dose escalation in PHEarless, the Phase 1/2 study of BMN 307 continues based on encouraging Phe lowering and safety profile observed in study participants who were treated with the lowest dose.
◦BMN 255 for a subset of chronic renal disease: On January 11, 2021 BioMarin announced that it had filed an IND in 2020 for BMN 255, a small molecule for a subset of chronic renal disease. BMN 255 was driven by genetic discoveries for both mechanism and for identifying individuals for treatment.

◦BMN 331 gene therapy product candidate for Hereditary Angioedema (HAE): IND-enabling studies are ongoing with BMN 331 for the treatment of HAE, BioMarin’s third gene therapy candidate. BioMarin plans to leverage its broad expertise in developing gene therapies for severe hemophilia A and PKU to improve efficiencies in the development process of BMN 331. BioMarin is on track to file an IND for BMN 331 mid-year 2021.

◦BMN 351 for Duchenne Muscular Dystrophy (DMD): IND-enabling studies are underway with BMN 351, an antisense oligonucleotide therapy that has demonstrated dystrophin expression levels of 30-50% of wild-type levels in the quadriceps in a DMD mouse model treated at 18.7 mg/kg/week for 13 weeks (measured 2 weeks following last administration). If results from the ongoing pre-clinical studies are supportive, BioMarin anticipates filing an IND for BMN 351 in the first half of 2022.

◦DiNA-001 for MYBPC3 hypertrophic cardiomyopathy (HCM): Pre-clinical studies are underway with DiNA-001 following a collaboration announced in 2020 with DiNAQOR, a gene therapy platform company, to develop novel gene therapies to treat rare genetic cardiomyopathies. DiNAQOR received an undisclosed upfront payment and is eligible to receive development, regulatory and commercial milestones on product sales in addition to tiered royalties on worldwide sales.

◦On April 28, 2021, BioMarin and the Allen Institute announced a collaboration to create new gene therapies aimed at rare genetic diseases of the central nervous system (CNS). The goal is to combine the Allen Institute’s leadership in large-scale genomic science in the CNS therapeutic area with BioMarin’s expertise in developing transformational gene therapies.
2021 Full-Year Financial Guidance (in millions, except %)

Item

2021 Guidance * (reaffirmed)
Total Revenues

$1,750

to

$1,850
Vimizim Net Product Revenues

$570

to

$610
Kuvan Net Product Revenues

$250

to

$290
Naglazyme Net Product Revenues

$365

to

$395
Palynziq Net Product Revenues

$210

to

$250
Brineura Net Product Revenues

$120

to

$140

Cost of Sales (% of Total Revenues)

23
%

to

25%
Research and Development Expense

$645

to

$695
Selling, General and Administrative Expense

$725

to

$775

GAAP Net Loss

($130)

to

($80)
Non-GAAP Income (1)

$170

to

$220

IMV Inc. to Announce First Quarter 2021 Results and Host a Conference Call and Webcast on May 12, 2021

On April 29, 2021 IMV Inc. ("IMV" or the "Corporation") (Nasdaq: IMV; TSX: IMV), a clinical-stage biopharmaceutical company pioneering a novel class of cancer immunotherapies and vaccines to fight against infectious diseases, reported that it will hold a conference call and webcast on Wednesday, May 12, 2021 at 8:00 a.m. ET to discuss the company’s first quarter 2021 financial and operational results (Press release, IMV, APR 29, 2021, View Source [SID1234578796]).

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Financial analysts are invited to join the conference call by dialing (866) 211-3204 (U.S. and Canada) or (647) 689-6600 (international) using the conference ID: 9284231

Other interested parties will be able to access the live audio webcast at this link: View Source The webcast will be recorded and will then be available on the IMV website for 30 days following the call.

Labcorp Announces 2021 First Quarter Results Company Raises Full Year Guidance

On April 29, 2021 Labcorp (NYSE: LH), a leading life sciences company, reported results for the first quarter ended March 31, 2021 and raised 2021 guidance (Press release, LabCorp, APR 29, 2021, View Source [SID1234578816]).

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"We delivered very strong results in the first quarter driven by revenue growth across both our Diagnostics and Drug Development businesses," said Adam H. Schechter, chairman and CEO, Labcorp. "Overall revenue in our base business grew 14.6% as people continued to return to their pre-pandemic healthcare routines and our biopharmaceutical clients resumed their important research and development. Our drug development pipeline remained robust, with a book-to-bill of 1.47 on a trailing twelve-month basis driven by strong demand across major therapeutic areas."

In the quarter, Labcorp continued to bring science and technology innovations to market quickly to improve health and improve lives. The company opened a fully automated kit production facility in Belgium to support its Central Lab customers, ultimately improving access and cost efficiency for biopharma and clinical trial clients across Europe, the Middle East and Africa. In the fight against COVID-19, Labcorp expanded its work with the CDC to identify variants to the virus, and now offers Pixel by Labcorp COVID-19 home collection kits in thousands of pharmacies across the United States.

"We are pleased with our strong first quarter performance and improved outlook, and are raising our full year adjusted EPS guidance range to between $20.00 and $24.00. I am proud of our more than 70,000 employees and their commitment to our patients and customers during this pandemic and the difference they are making in the lives of people around the world," said Schechter.

Consolidated Results

First Quarter Results
Revenue for the quarter was $4.16 billion, an increase of 47.4% over $2.82 billion in the first quarter of 2020. The increase in revenue was due to organic growth of 45.0%, acquisitions of 0.9%, and favorable foreign currency translation of 1.4%. The 45.0% increase in organic revenue includes a 32.9% contribution from PCR and antibody testing (COVID-19 Testing) and a 12.2% increase in the company’s organic Base Business. Base Business includes Labcorp’s business operations except for COVID-19 Testing.

Operating income for the quarter was $1,057.9 million, or 25.4% of revenue, compared to ($192.6) million, or (6.8%), in the first quarter of 2020. The increase in operating income and margin was primarily due to COVID-19 Testing, organic Base Business growth, acquisitions, and LaunchPad savings, partially offset by higher personnel costs. The company recorded amortization, restructuring charges, and special items, which together totaled $124.0 million in the quarter, compared to $558.5 million during the same period in 2020. This decrease was primarily due to the goodwill impairment recorded in the first quarter of 2020. Adjusted operating income (excluding amortization, restructuring charges, and special items) for the quarter was $1,181.9 million, or 28.4% of revenue, compared to $365.9 million, or 12.9%, in the first quarter of 2020.

Net earnings (losses) for the quarter were $769.6 million, compared to ($317.2) million in the first quarter of 2020. Diluted EPS were $7.82 in the quarter, up from ($3.27) in the same period in 2020. Adjusted EPS (excluding amortization, restructuring charges, and special items) were $8.79 in the quarter, up from $2.37 in the first quarter of 2020.

Operating cash flow for the quarter was $1,157.6 million, compared to $203.8 million in the first quarter of 2020. The increase in operating cash flow was due to higher cash earnings and lower working capital. Capital expenditures totaled $95.4 million, down from $106.6 million a year ago. As a result, free cash flow (operating cash flow less capital expenditures) was $1,062.2 million, up from $97.2 million in the first quarter of 2020.

At the end of the quarter, the company’s cash balance and total debt were $1.9 billion and $5.4 billion, respectively. During the quarter, the company invested $34.1 million on acquisitions, repurchased $68.5 million of stock representing approximately 0.3 million shares, and paid down $375.0 million of debt. As of March 31, 2021, the company had $731.5 million of authorization remaining under its share repurchase program.
3

First Quarter Segment Results

The following segment results exclude amortization, restructuring charges, special items, and unallocated corporate expenses.

Diagnostics
Revenue for the quarter was $2.76 billion, an increase of 62.0% over $1.70 billion in the first quarter of 2020. The increase in revenue was primarily due to organic growth of 60.8%, acquisitions of 0.9%, and favorable foreign currency translation of 0.4%. The increase in organic revenue was due to a 54.5% contribution from COVID-19 Testing and a 6.3% increase in the Base Business, which includes the unfavorable impact of weather of approximately (2.0%).

Total volume (measured by requisitions) increased by 27.3% as organic volume increased by 26.6% and acquisition volume contributed 0.7%. The organic volume growth was due to a 27.9% contribution from COVID-19 Testing demand, partially offset by a (1.3%) reduction in organic Base Business, which includes the unfavorable impact from weather of approximately (2.0%). Price / mix increased by 34.7% primarily due to COVID-19 Testing of 26.6% and organic Base Business of 7.5%.

Adjusted operating income for the quarter was $991.6 million, or 36.0% of revenue, compared to $254.2 million, or 14.9%, in the first quarter of 2020. The increase in adjusted operating income and adjusted operating margin were primarily due to the increase in COVID-19 Testing, organic Base Business growth and LaunchPad savings, partially offset by higher personnel costs. The company remains on track to deliver approximately $200 million of net savings from its three-year Diagnostics LaunchPad initiative by the end of 2021.

Drug Development
Revenue for the quarter was $1.44 billion, an increase of 25.7% over $1.14 billion in the first quarter of 2020. The increase in revenue was due to organic growth of 21.9%, acquisitions of 1.0%, and favorable foreign currency translation of 2.9%. The increase in organic revenue was due to a 19.7% increase in the Base Business and a 2.2% contribution from COVID-19 Testing performed through its Central Laboratories business. Drug Development benefited from broad-based demand across businesses, including COVID-19 vaccine and therapeutic work.

4

Adjusted operating income for the quarter was $234.1 million, or 16.3% of revenue, compared to $150.8 million, or 13.2%, in the first quarter of 2020. The increase in adjusted operating income and adjusted operating margin were primarily due to organic Base Business growth, COVID-19 Testing, and LaunchPad savings, partially offset by higher personnel costs. The company continues to develop and execute new LaunchPad programs to support profitable growth in Drug Development.

Net orders and net book-to-bill during the trailing twelve months were $7.61 billion and 1.47, respectively. Backlog at the end of the quarter was $13.97 billion, compared to $13.76 billion last quarter, and the company expects approximately $4.62 billion of its backlog to convert into revenue in the next twelve months.

5

Outlook for 2021

Labcorp is raising its 2021 full year guidance to reflect the improved recovery in the Diagnostics and Drug Development base businesses, while the COVID-19 Testing contribution remains within the original guidance range provided. The following guidance assumes foreign exchange rates effective as of March 31, 2021 for the remainder of the year. Enterprise level guidance includes the estimated impact from currently anticipated capital allocation, including acquisitions and share repurchases.

(Dollars in billions, except per share data) Previous Updated Results 2021 Guidance 2021 Guidance 2020 Low High Low High Revenue
Total Labcorp Enterprise (1)(2)
$ 13.98 (1.0%) 4.5% 2.0% 6.5%
Base Business (2)
$ 11.19 11.0% 13.5% 13.5% 16.0%
COVID-19 Testing (2)
$ 2.78 (50.0%) (35.0%) (50.0%) (35.0%)
Total Diagnostics (3)
$ 9.25 (7.5%) (0.5%) (5.0%) 0.0% Base Business $ 6.47 11.0% 14.0% 13.5% 16.0% COVID-19 Testing $ 2.78 (50.0%) (35.0%) (50.0%) (35.0%)
Total Drug Development (4)
$ 4.88 8.0% 10.5% 12.0% 14.0% Base Business $ 4.76 9.5% 12.0% 14.0% 16.0% Adjusted EPS $ 23.94 $ 19.00 $ 23.00 $ 20.00 $ 24.00
Free Cash Flow (5)
$ 1.75 $ 1.70 $ 1.90 $ 1.80 $ 2.00 (1) 2021 Updated Guidance includes a benefit from foreign currency translation of 0.7%, Previous 2021 Guidance was 0.9% (2) Enterprise level revenue is presented net of intersegment transaction eliminations, including Drug Development COVID-19 Testing revenue (3) 2021 Updated Guidance includes a benefit from foreign currency translation of 0.3%, Previous 2021 Guidance was 0.1% (4) 2021 Updated Guidance includes a benefit from foreign currency translation of 1.4%, Previous 2021 Guidance was 2.2% (5) Free Cash Flow consists of operating cash flow less capital expenditures

6Use of Adjusted Measures

The company has provided in this press release and accompanying tables "adjusted" financial information that has not been prepared in accordance with GAAP, including adjusted net income, adjusted EPS (or adjusted net income per share), adjusted operating income, adjusted operating margin, free cash flow, and certain segment information. The company believes these adjusted measures are useful to investors as a supplement to, but not as a substitute for, GAAP measures, in evaluating the company’s operational performance. The company further believes that the use of these non-GAAP financial measures provides an additional tool for investors in evaluating operating results and trends, and growth and shareholder returns, as well as in comparing the company’s financial results with the financial results of other companies. However, the company notes that these adjusted measures may be different from and not directly comparable to the measures presented by other companies. Reconciliations of these non-GAAP measures to the most comparable GAAP measures and an identification of the components that comprise "special items" used for certain adjusted financial information are included in the tables accompanying this press release.

The company today is providing an investor relations presentation with additional information on its business and operations, which is available in the investor relations section of the company’s website at View Source Analysts and investors are directed to the website to review this supplemental information.

A conference call discussing Labcorp’s quarterly results will be held today at 9:00 a.m. ET and is available by dialing 877-898-8036 (720-634-2811 for international callers). The conference ID is 6566853. A telephone replay of the call will be available through May 13, 2021, and can be heard by dialing 855-859-2056 (404-537-3406 for international callers). The conference ID for the replay is 6566853. A live online broadcast of Labcorp’s quarterly conference call on April 29, 2021, will be available at Labcorp Investor Relations website beginning at 9:00 a.m. ET. This webcast will be archived and accessible through April 15, 2022.

Hexagon Interim Report 1 January – 31 March 2021

On April 29, 2021 Hexagon reported that Interim Report 1 January – 31 March 2021 (Press release, Hexagon Bio, APR 29, 2021, View Source;31-march-2021-301280107.html [SID1234578853]).

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First quarter 2021

Net sales increased by 10 per cent to 977.9 MEUR (889.9). Using fixed exchange rates and a comparable group structure (organic growth), net sales increased by 11 per cent
Adjusted operating earnings (EBIT1) increased by 34 per cent to 257.9 MEUR (192.4)
Earnings before taxes, excluding adjustments, amounted to 250.3 MEUR (186.6)
Net earnings, excluding adjustments, amounted to 205.2 MEUR (153.0)
Earnings per share, excluding adjustments, amounted to 0.56 EUR (0.41)
Operating cash flow increased to 211.9 MEUR (136.9)
For further information please contact:

Maria Luthström, Head of Sustainability and Investor Relations, Hexagon AB, +46 8 601 26 27, [email protected]

This information is information that Hexagon AB is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out above, at 13:00 CET on 29 April 2021.

This information was brought to you by Cision View Source

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