JPM22: Pfizer’s big bet on mRNA, Biogen ‘proven wrong’ on Aduhelm price and the next cell therapies – BioPharma Dive

Biotechnology stocks ended the first day of the J.P. Morgan Healthcare Conference in the green, but only barely.By Monday afternoon, a closely followed index of the industry was up a fraction of a percent, marking a tepid start for a conference that's historically been able to drum up excitement in drug companies.

After a difficult end to 2021 for many biotechs, hopes were also high that dealmaking or major announcements might spark a rally. But, like last year's conference and the one before it, those hopes weren't met.

For those looking for a silver lining, Monday did see a slate of partnerships between large pharmas and smaller players at the cutting-edge of drugmaking science in gene and cell therapy, most notably Pfizer's trio of deals in messenger RNA technologies.

Biopharma Dive will be tracking updates and notable comments from the conference, which is being held virtually due to COVID-19, with a series of short reports this week. The first is below.

Aduhelm, the first new treatment for Alzheimer's disease to enter the U.S. market in nearly 20 years, has been controversial for several reasons, not least of which is its cost. Its developer, Biogen, initially set a price of $56,000 a year for the average patient, fueling concerns the drug might not be accessible to many, and that it could singularly bust insurer budgets if used in even a small fraction of the Alzheimer's population.

Speaking Monday during the company's virtual presentation, Biogen CEO Michel Vounatsos said his team thought it would be able to defend Aduhelm's price, especially when considering the comparative costs for treating Alzheimer's. But that hope quickly dissipated after the company received pushback from stakeholders on all sides.

"We were proven wrong by the reaction of the community, by the reaction of physicians, by, eventually, the reaction of patients and policy leaders," Vounatsos said.

Biogen last month announced it would cut Aduhelm's list price almost in half starting Jan. 1, a move meant to win over insurers and invigorate what have so far been disappointing sales. Alisha Alaimo, president of the company's U.S. organization, told investors Monday that this change has been "very well received" by payers and physicians. Biogen now estimates roughly 220 treatment sites are administering Aduhelm an increase from the approximately 120 it tallied as of late October, yet still far from its earlier target of 900.

The color on Aduhelm's pricing strategy proved timely as, earlier in the morning, the Centers for Medicare and Medicaid Services was told to reassess significant premium hikes it had set this year. In November, the CMS said premiums and deductibles in one part of Medicare would rise 15% in 2022, a historic increase that was made, at least in part, to offset the potential impact of Aduhelm.

"With the 50% price drop of Aduhelm on January 1, there is a compelling basis for CMS to reexamine the previous recommendation," said Xavier Becerra, who oversees the agency through his role as head of the U.S. Department of Health and Human Services, in a statement.

CMS has another important decision to make with regard to Aduhelm. The agency is expected to this week deliver a draft version of a policy known as a National Coverage Determination, which will hold substantial influence over how Aduhelm and Alzheimer's drugs like it are paid for. A final decision is due in April.

If Monday were the first time investors had ever heard Pfizer CEO Albert Bourla speak, they may not have realized the drugmaker has 15 potential new medicines in late-stage testing. That's because Bourla spent much of his time talking about the messenger RNA technology underlying the COVID-19 vaccine, Comirnaty, that was forecast to deliver Pfizer an incredible $36 billion in sales last year.

One can hardly blame Bourla. After all, Comirnaty's success helped drive the company's shares 60% higher in 2021. The shot's progress also emboldened Pfizer and its mRNA partner BioNTech to start clinical testing of an influenza vaccine and add an experimental shot for shingles to their longstanding partnership.

Pfizer's mRNA ambitions appear to be growing, too. Early Monday, just before the JPM meeting began, the company cut a gene editing partnership with Beam Therapeutics, a wide-ranging deal involving drugs that use mRNA molecules as well as the lipid nanoparticles that typically deliver them.

That deal was one of several mRNA-focused deals Pfizer revealed on Monday. One partnership with Acuitus, for example, will give the company greater access to lipid nanoparticle technology. Another with Codex DNA, focused on manufacturing, could help Pfizer get a new vaccine ready for human testing in 30 days, rather than 100. Pfizer executives still believe they are "just scratching the surface" of mRNA's drugmaking capabilities, Bourla said.

"We decided that we are going all in because we have developed the expertise and the infrastructure that allow us to be a leading player," Bourla said. The deals announced so far are "not the only ones we will work on."

Add CAR-M, iNK and gamma delta T to the list of abbreviations for cancer cell therapy. A trio of deals announced on the first day of the meeting show pharmaceutical companies are interested in taking the field in new directions.

So-called CAR-T treatments, based on patient T cells engineered in the lab to target and attack tumors, are already approved in leukemia, lymphoma and multiple myeloma. Researchers and drug companies are testing a similar approach with another type of immune defender called natural killer, or NK, cells.

In a new deal, Moderna plans to explore a third type, paying Carisma Therapeutics $45 million in cash to start a research collaboration based on the Philadelphia biotech's work with monocytes. The companies will work together to discover and develop chimeric antigen receptors therapies built around these white blood cells, which can transform into what's called a macrophage to remove dead cells or attack bacteria.

Bristol Myers Squibb, meanwhile, is teaming up with Century Therapeutics to develop natural killer and T cell therapies using induced pluripotent stem cells dubbed iNK or iT rather than extracting mature immune cells from each patient. Century will receive $100 million in cash from Bristol Myers, which will also invest $50 million in the biotech.

And while no major acquisitions were announced Monday, Takeda agreed to buy Adaptate Technologies for access to antibodies the London-based company is developing to engage a specific type of gamma delta T cells. Adaptate was spun out of another company, GammaDelta Therapeutics, that Takeda acquired in October.

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JPM22: Pfizer's big bet on mRNA, Biogen 'proven wrong' on Aduhelm price and the next cell therapies - BioPharma Dive