The share prices of blue-chip biopharmaceutical companies closed out the month on a high note to contribute to their stellar collective performance during the second quarter, with the BioWorld Biopharmaceutical index increasing almost 20%. Small- and mid-cap biopharma companies also enjoyed a strong period fueled by positive clinical trial data and new drug approvals.
Although the Biopharmaceutical index closed out the month relatively flat, significant expectations that the sector will eventually provide a therapeutic solution, or solutions, to the COVID-19 pandemic was the catalyst for growth during the first two months of the second quarter. In addition, investors maintained a bullish outlook for the country’s economic restart that helped drive the general markets. As a result, the Dow Jones Industrial Average gained about 18% in the quarter, a performance that has helped erase a good portion of the bellwether index’ dramatic meltdown earlier in the year. (See BioWorld Biopharmaceutical Index, below.)
The leading gainer for the second quarter was Cambridge, Mass.-based Sarepta Therapeutics Inc. with its shares (NASDAQ:SRPT) surging almost 64% fueled by a steady flow of positive news. In the quarter, the company announced a 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 its 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.
Also, at the end of last week, Sarepta said it had completed the submission of a rolling new drug application to the FDA that is seeking accelerated approval for casimersen (SRP-4045), a phosphorodiamidate morpholino oligomer engineered to treat patients with Duchenne muscular dystrophy who have genetic mutations that are amenable to skipping exon 45 of the Duchenne gene.
The submission includes data from the casimersen arm of the Essence phase III study evaluating efficacy and safety in patients amenable to skipping exons 45 and 53. An interim analysis demonstrated a statistically significant increase in dystrophin production as measured by western blot in patients who received casimersen compared to baseline and placebo. The ongoing study is still blinded in order to collect additional efficacy and safety data. If the NDA is accepted and granted accelerated approval, the company said the completed study would serve as a postmarketing confirmatory study.
Seattle Genetics Inc. also saw its shares (NASDAQ:SGEN) push 47% higher in the second quarter based on positive pipeline progress, including top-line results from a phase II single-arm trial, known as innovaTV 204, evaluating tisotumab vedotin (TV), an investigational antibody-drug conjugate (ADC) directed to tissue factor, which is expressed on cervical cancer and can promote tumor growth, angiogenesis and metastases, administered every three weeks for the treatment of patients who have relapsed or progressed on or after prior treatment for recurrent or metastatic cervical cancer. Results showed a 24% confirmed objective response rate by independent central review with a median duration of response of 8.3 months. The drug is being developed by Seattle Genetics in collaboration with Genmab A/S.
Wainwright analyst Andrew Fein was impressed with the study and noted in a report that these “data should be approvable by benchmarking with pembrolizumab, the most recent approval in this population. We believe Seattle Genetics should still be able to seek a label at least in patients who are not eligible for pembrolizumab (e.g., PD-L1 negative), as well as those who progress following pembrolizumab.”
Abbvie Inc. was the top performing big pharma member in the group, with its shares (NYSE:ABBV) pushing 29% in the quarter. Its acquisition of Allergan plc for $63 billion was completed in May and represents, according to BioWorld data, the largest M&A so far this year and the fourth largest biopharma M&A of all time, behind Actavis plc’s acquisition of Allergan in 2015 for $77 billion (the combined company became Allergan), Bristol Myers Squibb Co.’s buyout of Celgene Corp. in 2019 for $74 billion and Pfizer Inc.’s purchase of Wyeth in 2009 for $68 billion.
In total, the Abbvie/Allergan acquisition accounts for 53% of the total M&A value for 2020, which is at $118.67 billion. The completed acquisition did not get off to a great start, however, as Allergan along with Molecular Partners AG, of Zurich, Switzerland, receiving a complete response letter from the FDA for the BLA for abicipar pegol, their VEGF-A inhibitor-Darpin therapy for patients with wet age-related macular degeneration.
In the letter, the FDA noted the rate of intraocular inflammation occurring after administering abicipar pegol 2 mg/0.05 mL results in an unfavorable benefit-risk ratio. Abbvie said it will meet with the FDA to discuss the comments and determine next steps.
Drug developers on a tear
It was a great quarter for biopharmas developing therapeutics, with the BioWorld Drug Developers index jumping 40%, led 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 380% in Q2. Investors continue to be impressed by its pipeline and are eagerly awaiting news of several upcoming catalysts, including a Dec. 18 PDUFA goal date for margetuximab, an Fc-engineered, monoclonal antibody targeting HER2, that is being evaluated in a phase III trial.
In a research note, SVB Leerink analyst Jonathan Chang commented on the product updates that Macrogenics presented at the American Association for Cancer Research Virtual Annual Meeting II saying that they “were a reminder of the considerable potential for MGNX's broad portfolio of antibody-based therapies.”
Cambridge, Mass.-based Moderna Inc.’s shares (NASDAQ:MRNA) closed out the quarter up 114%. 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.
Based on its experience with mRNA vaccines, the company has moved rapidly, and the first participants have already been dosed in its phase II study of mRNA-1273 that is being conducted under its own IND. 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.
Ahead of trial results, the company is collaborating with Catalent Inc., of Somerset, N.J., on large-scale, commercial fill-finish manufacturing of mRNA-1273, at Catalent’s biologics facility in Bloomington, Ind. Catalent will provide vial filling, packaging capacity and additional staffing required for round-the-clock manufacturing operations to produce an initial 100 million doses of the vaccine candidate intended to supply the U.S. market starting in the third quarter of 2020. The companies are in discussions to secure fill-finish capacity for continued production of hundreds of millions of additional doses. Catalent will also provide clinical supply services from its facilities in Philadelphia, including packaging and labeling, as well as storage and distribution to support Moderna’s phase III study of mRNA-1273.
New Haven, Conn.-based Biohaven Pharmaceutical Holding Co. Ltd. has had a great quarter, with its shares (NASDAQ:BHVN) doubling in the period, up 113%. The stock’s value shrugged off a phase II/III proof-of-concept study of troriluzole for treating obsessive-compulsive disorder (OCD) that failed to meet its primary outcome measure at week 12. Despite that, it said it would carry on and conduct a larger phase III trial.
Troriluzole, which modulates glutamate, the human body’s most abundant excitatory neurotransmitter, is being developed as an adjunctive OCD therapy. The study’s top-line data showed consistent numerical improvement over placebo at all the study’s timepoints, especially for those patients who were most severely ill at baseline.
Following an end-of-phase II meeting with the FDA, the company said it plans to initiate a pivotal phase III study of two doses of troriluzole vs. placebo.