Blue chip public biopharmaceutical companies continued their positive trajectory in May, with the BioWorld Biopharmaceutical index recording an 8% jump in valuation and contributing to its year-to-date performance of approximately 17%. Expectations that the sector will provide a therapeutic solution, or solutions, to the COVID-19 pandemic has certainly been the catalyst for growth during the past two months. (See BioWorld Biopharmaceutical Index, below.)
Interestingly, the leading gainer for the month was not driven by COVID-19 news. The shares of Cambridge, Mass.-based Sarepta Therapeutics Inc. (NASDAQ:SRPT) surged almost 30%, helped by the clinical results announced by fellow index member Pfizer Inc. Like Sarepta, the pharma company is developing a gene therapy for Duchenne muscular dystrophy (DMD). At the American Society of Gene & Cell Therapy meeting mid-month, Pfizer presented updated phase Ib data on PF-06939926 from nine ambulatory boys with DMD, ages 6 to 1,2 indicating that the intravenous administration of the therapy was well-tolerated during the infusion period, with encouraging efficacy and manageable safety events. The treatment provided durable and statistically significant improvements across multiple efficacy-related endpoints measured at 12 months post-infusion, including sustained levels of mini-dystrophin expression and improvements on the North Star Ambulatory Assessment (NSAA) rating scale, which is a validated measure of muscle function.
Brian Abrahams, a senior analyst with RBC Capital Markets LLC, commented on Pfizer’s update, saying, “The data are still evolving, but our initial impression is that while it still looks viable, and will be moved into [phase III in the second half of 2020] as expected, the safety and efficacy profile continues to look less favorable than SRPT.”
Investors also reacted positively to Sarepta’s deal with Dyno Therapeutics Inc., a biotech company applying artificial intelligence to gene therapy, to develop next-generation adeno-associated virus (AAV) vectors for muscle diseases, using Dyno’s Capsidmap platform. The technology will be used for the design and discovery of AAV capsids with improved functional properties for gene therapy, and Sarepta will be responsible for conducting preclinical, clinical and commercialization activities for gene therapy product candidates using those capsids.
Partner sells shares
It was also an active period for Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. At the end of the month, it reported it had initiated an underwritten public secondary offering of its common shares to allow for the exit of investment by long-time partner Sanofi SA, which intends to sell approximately 12.8 million of its roughly 23.2 million Regeneron shares, or about 20.6% ownership in the company. Regeneron said it plans to repurchase approximately $5 billion of its common stock directly from Sanofi at a price equal to the net offering price per share, funding the purchase with a combination of $3.5 billion of cash on hand and $1.5 billion of fully committed bridge financing from Goldman Sachs Bank. Following the offering and Regeneron's share repurchase, and assuming full exercise by underwriters of their option to purchase additional shares, Sanofi said it will have disposed of all but 400,000 Regeneron shares, which it intends to retain. Regeneron’s shares (NASDAQ:REGN) closed the period up 16.5% at $612.81, while Sanofi’s shares (NASDAQ:SNY) closed up about 5%.
The two companies also reported that they had achieved FDA approval for Dupixent (dupilumab) for children ages 6 to 11 with moderate to severe atopic dermatitis whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable. The drug is already approved in the U.S. to treat patients 12 and older with moderate to severe atopic dermatitis.
Drug Developers on a tear
The BioWorld Drug Developers index also pushed 12% higher in May driven by a strong performance from Rockville, Md.-based Macrogenics Inc. (See BioWorld Drug Developers Index, below.)
The company’s shares (NASDAQ:MGNX) jumped a whopping 167% in May. It reported, at the end of the month, that during a mid-cycle communication with the FDA, the agency notified the firm that it is no longer planning to hold an Oncologic Drugs Advisory Committee meeting to discuss the BLA for margetuximab and continues to anticipate meeting the PDUFA goal date for the application review, which has been set for Dec. 18. Margetuximab, an Fc-engineered, monoclonal antibody that targets HER2, is being evaluated in SOPHIA, a phase III trial testing the combination with chemotherapy compared to trastuzumab plus chemotherapy in patients with HER2-positive metastatic breast cancer.
Also pushing 59% higher are shares of New York-based TG Therapeutics Inc. (NASDAQ:TGTX). That enabled the company to complete a follow-on offering, pricing 8.5 million shares of common stock at $18 each, with gross proceeds expected to be $153 million. A further $23 million could be added to the total if the underwriters exercise in full their option to purchase an additional 1.275 million shares. The company anticipates using net proceeds to help support the continued development of ublituximab and umbralisib (U2). This month, the independent data safety monitoring board recommended that the UNITY-CLL study be stopped early for superior efficacy after an interim analysis showed its U2 combination helped patients with chronic lymphocytic leukemia (CLL).
Cambridge, Mass.-based Moderna Inc.’s shares (NASDAQ:MRNA) closed out the month up 33%. Investors have warmed to the company’s mRNA vaccine candidate, mRNA-1273, targeting coronavirus (SARS-CoV-2). The vaccine encodes for a prefusion stabilized form of the Spike (S) protein and was selected in collaboration with investigators from the Vaccine Research Center at the National Institute of Allergy and Infectious Diseases (NIAID), a part of the NIH.
Last Friday, the company reported that the first participants have been dosed in its phase II study of mRNA-1273 that is being conducted under its own investigational new drug application. It will evaluate the safety, reactogenicity and immunogenicity of two vaccinations of mRNA-1273 given 28 days apart. It is planned that 600 healthy participants will be enrolled across two cohorts of adults ages 18 to 55 (n=300) and older adults ages 55 and above (n=300). Each participant will be assigned to receive placebo, a 50-μg or a 100-μg dose at both vaccinations. They will be followed through 12 months after the second vaccination.
Midmonth, Moderna reported initial data from a phase I study of mRNA-1273 led by NIAID, and it anticipates collaborating with the agency to implement the phase III study in July, subject to finalization of the clinical trial protocol.