UK first to approve Pfizer/BioNTech COVID-19 vaccine
The UK spiked a political football on Wednesday by granting emergency authorisation of Pfizer and BioNTech's COVID-19 vaccine BNT162b2, becoming the first country in the world to do so.
The integrity of the review process conducted by the Medicines and Healthcare products Regulatory Agency (MHRA) was called into question by the European Medicines Agency (EMA) and UK approval of the vaccine has invoked inevitable political posturing ahead of the country formally leaving the European Union at the end of the year.
It will also put the FDA under increased pressure to approve BNT162b2 and Moderna's COVID-19 vaccine mRNA-1273 quickly after public advisory committee meetings, scheduled in the coming weeks to discuss respective emergency-use authorisations, have taken place.
What role for AZD1222?
Emergency approval of BNT162b2 also sharpens focus on the potential role to be played by AZD1222, the COVID-19 vaccine being co-developed by AstraZeneca and the University of Oxford.
Like many other countries, the UK is banking heavily on this vaccine, which appears at this stage to be less efficacious than BNT162b2 and mRNA-1273. To put this into context, the UK's currently secured supply of BNT162b2 will vaccinate about 30% of the population, presumably to be targeted at those most vulnerable to developing severe COVID-19.
Well documented over the past two weeks or so is the hint of higher efficacy (90% effectiveness) when a lower dose of AZD1222 is administered first as part of the two-dose immunisation regimen. Data supporting this claim is limited, however, and it will be fascinating to see how regulators – the UK in particular given the precedent it has now set – pick these results apart.
In the meantime, a majority of infectious disease specialists we surveyed this week think AZD1222 should be approved on an effectiveness rate as low as 62%. With the logistical utility of this vaccine being much greater and it expected to cost less, AZD1222 is still likely to become a key component of the vaccine-based response to the pandemic, said respondents.
Biogen bolsters pipeline as aducanumab decision looms
Biogen faces an uncertain few months as it waits to see if the FDA approves its investigational Alzheimer's disease treatment aducanumab. A panel of experts recently convened by the agency voted near unanimously not to support approval, but the regulatory outlook for aducanumab is muddied by the tone of the FDA's pre-meeting briefing notes, which appeared to suggest support for its approval.
With Biogen's share price and growth outlook heavily leveraged to this outcome, it was not surprising to see the company strike a significant co-development agreement with Sage Therapeutics this week at an upfront cost of $1.5 billion. In return, Biogen will co-develop zuranolone, currently in Phase III studies for major depressive disorder and postpartum depression, and SAGE-324, which is in earlier-stage development for essential tremor.
The deal has appeased analysts covering Biogen, but Sage investors appear to be notably less enamoured with the agreement, viewing it as a potential hedge against the possibility that zuranolone fails to demonstrate required efficacy in late-stage studies. To that end, Biogen continues to embrace a high-risk, high-reward strategy.
Analysis – ViewPoints: Biogen ups the ante
Gene therapy updates
Big Pharma's appetite for gene therapy development remains considerable, as evidenced by Johnson & Johnson's deal this week to acquire the rights to Hemera Biosciences' investigational gene therapy HMR59, which is designed to help preserve vision in patients with geographic atrophy.
HMR59 is designed to increase the ability of retina cells to make a soluble form of CD59, a protective protein typically available in low levels among patients with age-related macular degeneration (AMD) that blocks the complement cascade from further damaging the retina, thus helping to preserve vision. The company said a Phase I study of HMR59 for patients with geographic atrophy is complete, while a second early-stage trial evaluating it in patients with wet AMD is currently conducting follow-up visits to determine long-term safety.
Elsewhere, Bayer announced on Wednesday the launch of a cell and gene therapy (C>) platform within its pharmaceuticals division as the drugmaker looks to bolster its presence in the field. Stefan Oelrich, president of pharmaceuticals at Bayer, said the move would "complement our existing C> pipeline," which so far includes five advanced assets and over 15 preclinical candidates, "with at least three investigational new drugs annually for the next years."
Last year, Bayer acquired BlueRock Therapeutics, which gave it a foothold in cell therapies through the latter's induced pluripotent stem cell (iPSC) technology, and the recent takeover of adeno-associated virus (AAV) gene therapy firm Asklepios Biopharmaceutical. The two companies will both be integrated into the new C> platform.
Looking ahead to ASH
The annual meeting of the American Society of Hematology (ASH), which will take place in a virtual format this year, begins on Saturday (Dec 5) and runs through to Tuesday (Dec 8).
Oncology will as usual play a prominent role at the conference, though key cancer presentations at this year's meeting appear to be incremental rather than ground-breaking. Focal points will include various modalities targeting BCMA in myeloma (CAR-T, ADCs and bi-specifics) and the continued accumulation of data for CD20-targeting bi-specific monoclonal antibodies.
As a result, a lot of attention will be on significant presentations outside of cancer, most notably updated results for Uniqure's haemophilia B gene therapy etranacogene dezaparvovec and early data for CTX001, the sickle cell disease therapy being co-developed by Vertex Pharmaceuticals and CRISPR Therapeutics.
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