ItHome Cardiac Arrest JPM21: Gene editing for heart disease, a new date to watch in Alzheimer’s and Pfizer’s deep pockets

JPM21: Gene editing for heart disease, a new date to watch in Alzheimer’s and Pfizer’s deep pockets

Credits to the 👉🏾Source Link👉🏾 Olivia
JPM21: Gene editing for heart disease, a new date to watch in Alzheimer's and Pfizer's deep pockets

The second day of the J.P. Morgan Healthcare Conference didn’t bring any billion-dollar acquisitions or high-priced partnerships. But the light slate of news felt a little less existential than might be expected for a meeting that prides itself on market-moving developments.

Biotech stock indices continue to do well, boosted by a stretch of dealmaking that closed out 2020. Venture and IPO fundraising hit record highs last year. And the industry is one month removed from the success of coronavirus vaccines developed by Moderna and partners Pfizer and BioNTech.

While quiet, the 39th annual meeting seems to reflect a sector confident its strong run will continue, even as the world struggles with its response to the coronavirus pandemic.

Read on for updates from the meeting’s second day:

Gene editing for heart disease

Verve Therapeutics, a small two-year-old biotech startup, has a bold vision. Led by cardiologist Sekar Kathiresan, the company aims to develop a one-time treatment for coronary heart disease using a precise form of gene editing.

“We’re ultimately looking to treat and hopefully eradicate heart disease,” said Kathiresan, in an interview.

On Tuesday, Verve unveiled its lead drug candidate, dubbed VERVE-101, which it plans to put into clinical testing next year. The therapy uses what’s known as base editing, a tool that allows scientists to change a single “letter” in the DNA make-up of genes.

Verve is targeting the drug at heterozygous familial hypercholesterolemia, a genetic disease that results in extremely high levels of LDL cholesterol (LDL-C) in the blood and, eventually, early heart disease and stroke.

VERVE-101 edits a gene called PCSK9, inactivation of which has been proven to dramatically lower LDL-C in the blood. Several approved or near-to-market heart drugs inhibit the protein encoded by PCSK9, or block expression of the gene. VERVE-101, by contrast, promises to turn off PCSK9 permanently.

Data disclosed by Verve Tuesday show inactivating PCSK9 by base editing led to sharp reductions in both PCSK9 protein and LDL-C in monkeys, an effect that persisted past six months.

“These data give us confidence this kind of change is going to be durable for the lifetime of the animal,” Kathiresan said.

What works in animals doesn’t necessarily work in humans, of course. Verve will spend this year preparing to ask the Food and Drug Administration for permission to begin its first study of VERVE-101 in humans.

The goal, Kathiresan told investors Tuesday, is “curative, life-long lowering of LDL cholesterol.”

The next big date in Alzheimer’s research

By March 7, the Food and Drug Administration will choose whether or not to approve Biogen’s controversial Alzheimer’s drug — a decision that will have a wide impact on research into the progressive, deadly disease.

The beginning of March will also bring detailed results from Eli Lilly for an earlier-stage drug that it said this week succeeded in a small, Phase 2 study. The news boosted Lilly’s market value by nearly $20 billion and raised Biogen’s share price in the process, too.

But while the data appeared promising, all researchers have to go on currently is Lilly’s short press release. On Tuesday, Lilly’s CEO, David Ricks, said the company would present its findings at the Alzheimer’s & Parkinson’s Disease conference, with a simultaneous publication in a major medical journal.

The meeting, which will be held virtually, kicks off March 9.

Sage’s new CEO makes his debut

For nearly two decades, Barry Greene helped build Alnylam Pharmaceuticals from a small startup working on Nobel Prize-winning science to a commercial-stage drugmaker with three approved medicines.

In mid-December, three months after departing Alnylam, he joined Sage Therapeutics as its new CEO, replacing Jeff Jonas.

While Alnylam worked in rare diseases, Sage is focused on developing drugs for more common mood and movement disorders — medicines that are critically needed, Greene reminded investors in a presentation Tuesday.

“I believe we are in a brain health pandemic,” he said, while expressing optimism about research into central nervous system disorders, a notoriously challenging field of drug development. “CNS feels like oncology was ten or 15 years ago.”

Greene will be tasked with getting Sage back on track after a difficult year following a clinical trial setback for zuranolone, the biotech’s drug for depression. On Tuesday, he laid out the company’s plan to recover, which includes several zuranolone studies already underway.

A readout from one of those trials, in major depressive disorder, is expected in the first half of 2021, while data from two others, also in MDD, should arrive later in the year.

Positive results would rekindle investor optimism in Sage and validate a decision by Biogen last November to invest $3 billion in a collaboration with the biotech.

Analysts had questioned why Sage signed the deal before knowing results from that first zuranolone study this year. But Greene argued the money from the partnership would fuel Sage’s broader research agenda.

“The ability to accelerate the entirety of our pipeline six, nine, 12 months ahead of time really made [the deal] worth it,” he said.

No deal price too high for Pfizer?

The success of the coronavirus vaccine Pfizer developed this year with partner BioNTech is among the most significant achievements of the biopharmaceutical industry in decades. (As are other coronavirus vaccines.)

Accordingly, company CEO Albert Bourla’s conversation with J.P. Morgan healthcare analyst Christopher Schott on Tuesday dealt primarily with the vaccine’s impact on Pfizer’s business. But a J.P. Morgan session wouldn’t be complete without questions on what kind of dealmaking investors can expect in the new year.

Schott pressed Bourla on how Pfizer views acquisitions, particularly after spinning out its generic drug division Upjohn.

Companies working in Pfizer’s therapeutic areas of focus, such as oncology, with drugs ready for Phase 2 or Phase 3 testing would fit the bill, according to the CEO.

As for price, “I don’t have an upper bound,” Bourla said. “We have the ability to basically do everything that exists out there, if we wanted to.”

Source Link

related posts

Leave a Comment

This website uses cookies to improve your experience. We will assume you are ok with this, but you can opt-out if you wish. Accept Read More

%d bloggers like this: