The exceptional and speedy response in bringing safe and effective vaccines and therapeutics to combat COVID-19 has kept investors engaged and supportive. As a result, not only have companies involved in this research and development benefited, but so has the sector as a whole, in particular small- and medium-sized drug developers. Those biopharma companies have enjoyed significant jumps in their share values, with the BioWorld Drug Developers index closing up 4.6% in December and up 29% for 2020, well ahead of the general markets. (See BioWorld Drug Developers 2020 index, below.)
Cambridge, Mass.-based Moderna Inc., which received FDA emergency use authorization (EUA) for its COVID-19 vaccine, mRNA-1273, the second to receive an EUA, following the Dec. 11 award to Pfizer Inc. and Biontech SE for BNT-162b2, has enjoyed significant investor support, with its shares (NASDAQ:MRNA) soaring 434% in 2020, to close Dec. 31 at $104.47. The performance ranks the company in the top 10 of major market movers for the year.
The prospects for the company going forward are also bright, according to Piper Sandler analyst Edward Tenthoff, referencing the company’s sizeable order book of 510.5 million doses of mRNA-1273. That includes a 40 million dose order from South Korea and the European Commission’s confirmed order commitment of 160 million doses. The first deliveries to European countries from Modena’s dedicated European supply chain are expected to begin early this year, subject to regulatory approval by the EMA, whose scientific committee for human medicines plans to meet Jan. 6 to discuss the candidate.
Tenthoff estimates that the revenues generated from those orders could be approximately $7.8 billion this year. In addition, he noted, “we believe Moderna's rich pipeline is validated with multiple blockbuster vaccines in development. We reiterate our overweight rating and $166 target.”
Non-COVID-19-related innovation also received strong support throughout the year. Ranking just outside the top 10 marker movers is Denali Therapeutics Inc., whose shares (NASDAQ:DNLI) closed the year up 381%. The South San Francisco-based company received a positive market reaction when it announced it had closed a lucrative leucine-rich repeat kinase 2 (LRRK2) collaboration and license agreement and a right of first negotiation, option and license agreement with Biogen Inc.
Ranking just behind Denali was TG Therapeutics Inc., of New York, with its shares (NASDAQ:TGTX) up 369% for the year. After reporting positive top-line results from two phase III studies, called Ultimate I and II, evaluating ublituximab, the company’s glycoengineered anti-CD20 monoclonal antibody, compared to teriflunomide in patients with relapsing forms of multiple sclerosis (RMS), the company priced an upsized underwritten public offering in December that raised $275 million in gross proceeds. The net proceeds will go to support continued development of ublituximab, TG-1101, and for developing umbralisib, which is being reviewed by the FDA for accelerated approval as a therapy for patients with previously treated marginal zone lymphoma who have received at least one prior anti-CD20-based regimen or follicular lymphoma who have received at least two prior systemic therapies.
Following those data, J.P. Morgan analyst Eric Joseph said that “we see a strengthened argument for ublituximab being best-in-class among the CD20 [monoclonal antibody] landscape in RMS, [and] accordingly we have raised our U.S. peak sales estimates in the indication to [about] $2.2 billion, in addition to raising assumptions on the likelihood of approval.”
Two companies in the group saw their share prices double in 2020. San Diego-based Halozyme Therapeutics Inc.’s shares (NASDAQ:HALO) closed the year up 141%. Its recent news flow included the fact that the European Commission had approved Roche Holdings AG's Phesgo, a fixed-dose combination of Perjeta (pertuzumab) and Herceptin (trastuzumab) with the company's Enhanze technology, administered by subcutaneous injection for the treatment of patients with early and metastatic HER2-positive breast cancer.
The company also entered a global collaboration and license agreement with Horizon Therapeutics plc, of Dublin, providing the latter with exclusive access to its drug delivery technology for subcutaneous formulation of medicines targeting IGF-1R. Horizon said it plans to develop a formulation of Tepezza (teprotumumab-trbw) for treating thyroid eye disease. Horizon paid $30 million up front and is obligated to make potential future payments of up to $160 million, subject to achievement of specified development, regulatory and sales-based milestones.
Shares of Editas Medicine Inc. (NASDAQ:EDIT) posted an annual gain of 138%. It has been a year of progress for the Cambridge, Mass.-based genome editing company, which is developing a therapy targeting sickle cell disease (SCD). The FDA granted rare pediatric disease designation for EDIT-301, an experimental, autologous cell medicine, being developed as a potentially best-in-class, durable medicine for SCD. EDIT-301 comprises sickle patient CD34-positive cells genetically modified using a CRISPR/Cas12a (also known as Cpf1) ribonucleoprotein to edit the HBG1/2 promoter region in the beta-globin locus.
In December, it submitted an IND application to the FDA for the initiation of a phase I/II trial of EDIT-301.
Another gene therapy company targeting SCD, Bluebird Bio Inc., had a rougher time, with its shares (NASDAQ:BLUE) falling 51% in 2020. In November, the shares took a hit when it announced the U.S. regulatory filing for Lentiglobin in SCD would be delayed until late 2022.
At the American Society of Hematology meeting last month, it announced that new data from group C of its ongoing phase I/II HGB-206 study of Lentiglobin gene therapy (bb-1111) for adult and adolescent patients with SCD showed a complete elimination of severe vaso-occlusive events (VOEs) and VOEs between six and 24 months of follow-up.
Mizuho analyst Difei Yang said, “We believe these data set a high bar for other gene therapy programs in sickle cell disease, some of which have shown encouraging early data but also raised questions on the safety side of the equation.”