January 3, 2024

The need-to-know this morning

Goldman Sachs Asset Management raised $650 million for a new life sciences fund that will invest in biotech, tools, and diagnostic startups.
Agios Pharmaceuticals said its drug, called mitapivat, significantly increased hemoglobin levels compared to a placebo in patients with non-tranfusion-dependent alpha and beta thalassemia. The results achieved the goals of a Phase 3 study.

Markets

Welcome back to biotech

In the time since last we sent this newsletter, the banner biotech index closed the year 8% above where it began, a late-year rally driven by yet another billion-dollar deal and the revelation that 2023's last big catalyst was a major success.

Biotech's performance in December, in which the closely watched XBI rose about 17%, papered over what was otherwise a volatile year for the sector. While dollars spent on buyouts hit their highest level since before the pandemic, the number of biotech IPOs fell to their lowest number in a decade.

Going into 2024 — and next week's J.P. Morgan Healthcare Conference — biotech is on somewhat uncertain ground. It's clear that major drugmakers are still willing to write sizable checks for promising companies, and the macroeconomic winds seem to be finally tilting in biotech's direction. But no one can be certain that the IPO market, which is vital to the whole ecosystem, can click back into gear.

And speaking of J.P. Morgan, STAT will be on the ground covering the conference all week, both on the site and in this newsletter. Stay tuned.

Biotech

Here's what lies ahead

We're in for what looks like a momentous first quarter in biotech, led by a pair of hotly anticipated data readouts that will swing billions of dollars in value.

Vertex Pharmaceuticals will present Phase 3 data on VX-548, a potential blockbuster pain treatment, from studies enrolling people recovering from surgery. The treatment, developed as a non-addictive alternative to opioids, has succeeded in earlier trials in acute and chronic pain, but its future as a product — and estimated $10 billion in peak annual revenue — depends on Vertex scoring a victory in Phase 3.

Also this quarter, Alnylam Pharmaceuticals is expected to disclose the results of HELIOS-B, a pivotal study testing whether vutrisiran, a subcutaneous medicine, has long-term benefits for patients with ATTR-CM, a progressive heart disease that is fatal if untreated. Once thought to be rare, ATTR-CM is now understood to affect about 400,000 people around the world, making it a burgeoning market for pharmaceutical companies.

Read more.

Neuroscience

Anavex's slow journey to failure

Back in June, Anavex Life Sciences said all patients in its rare disease study had completed treatment, suggesting the results would be made public in a month or two. Instead, the disclosure took seven months, but the result was the same as Anavex's earlier trials.

As STAT's Adam Feuerstein writes, Anavex's drug failed to beat placebo in a study enrolling patients with Rett syndrome, a genetic disease that causes severe neurologic impairments. The treatment, blarcamesine, showed no significant benefits on two measures of Rett syndromes, failing despite Anavex's decision to omit data from 15 participants in its analysis.

The negative study follows blarcamesine's similar disappointments in Alzheimer's disease and Parkinson's disease, leaving the company's only drug in clinical development with little in the way of a future.

Read more.

Regulatory

Remembering Sid Wolfe

In a career that spanned five decades, Sid Wolfe prodded regulators, skewered drugmakers, and became the most influential watchdog in medicine. And, as STAT's Ed Silverman writes, he also made time to painstakingly explain his logic to reporters.

Wolfe, who died Monday at the age of 86, founded Public Citizen's Health Research Group in 1971, forgoing a career practicing medicine for a life of advocacy. Over the years, Wolfe's work helped force the removal of 28 medications from the market, limiting the use of 10 more, and adding strong warnings to dozens of others, according to Public Citizen.

That career also included countless phone calls to Ed, who recalls that "nearly each one was the equivalent of a tutorial about medical products and regulatory oversight, or lack thereof, from someone who — more than anyone else, in my opinion — earned the right to be called a watchdog."

Read more.

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