🤝 John Maraganore and Clive Meanwell have been around the block. They’ve each built successful biopharmas in Alnylam and the Novartis-acquired The Medicines Company, respectively. And they’ve both had second arcs of their careers, with Maraganore joining a litany of biotech boards as an advisor and Meanwell running the investment firm Population Health Partners, which incubated Metsera. 

Now the two will share the CEO post at a new company called Corsera Health. They’ve funded it with about $50 million of their own money so far, and expect to be in the clinic before the end of 2025. That’s a fast timeline — the two had been working on the company in secret for only two years. 

What is Corsera working on? The end goal is a once-a-year tool to prevent cardiovascular disease, combining drugs and an AI that helps predict a person’s heart health. Right now, the company has two siRNA drugs that they plan to combine into one medicine. Corsera wants to file an IND by next month for a PCSK9-targeting RNAi medicine designed to lower LDL cholesterol. They’re also working on another RNAi medicine aimed at angiotensinogen for lowering blood pressure, with an IND aimed for the early part of 2026.

The pair is leaning on CDMOs to manufacture their drug product, while staying agile by using Meanwell’s firm for legal, finance, IT and operations needs — a model previously used by Metsera before it closed two private megarounds. Read more about Corsera from biotech correspondent Kyle LaHucik here. 

✉️The FDA published redacted complete response letters on Thursday for drugs that are still under consideration by the agency despite initial rejection, or whose applications have been withdrawn. Until now, it has been up to companies to decide what they share about the rejections they receive, often raising doubts about whether they’re being fully transparent.

In a similar move, the FDA released about 200 CRLs in July, but only for products that had since been approved. The latest batch of releases includes some recent high-profile rejections of drugs from Replimune, Capricor Therapeutics, Stealth BioTherapeutics and many others.

In a statement, the agency said that it will "promptly release newly issued CRLs, and when approving applications will release all CRLs associated with that application." Zachary Brennan has more details here. 

In other news, the FDA proposed a new process for facilitating the approval of therapies for exceptionally rare diseases. The Rare Disease Evidence Principles would allow gene therapies for certain ultra-rare populations to rely on a single-arm study with supportive data for approval. The work builds on efforts by former CBER Director Peter Marks, who championed accelerated approvals for gene therapies, and a previous bipartisan legislative push, Zachary reported.

Submissions to the process must be made prior to the launch of a pivotal trial, and must be focused on a genetic defect that affects a very small population: generally fewer than 1,000 Americans, the FDA said this week. It also said the clinical course of the disease must be significant disability or death, with no adequate alternative therapies. 

🏅 Every year since 2018, Endpoints has selected a group of 11 companies making the big, ambitious bets that we believe have the potential to be tomorrow’s next great biotech. This year is no different, and we’ll be unveiling our selections live in Boston on Sept. 18. But what’s happened to winners from past years?

Pharma’s M&A spree has helped a lot of our winners, with Capstan, Scorpion and Verve all getting acquired within the last 12 months. Other honorees have made deals of their own. BioNTech, our most successful pick, bought out its mRNA rival CureVac. And two of our recent picks — Metsera and Maze Therapeutics — went public in January. 

But not all our picks have succeeded. Kronos Bio, an E11 winner from 2020, shuttered this year and was acquired by Concentra Biosciences. Several others, including Prime Medicine, insitro, Tessera and BioNTech, laid off staff this year. What else was in store for the E11 alumni? Read more from biotech correspondent Kyle LaHucik here. 

🧠 The companies will team up on neuro diseases, with Novartis getting a license to a preclinical drug codenamed ARO-SNCA. It’s designed to treat Parkinson’s disease and other conditions that Arrowhead described as “synucleinopathies.” In addition to the hefty upfront payment, Arrowhead could also get up to $2 billion in other milestones. 

Novartis has been on a neuro splurge lately, as the Swiss pharma signed deals with BioArctic and Sironax in the last two months for drugs that can cross the blood-brain barrier. And for Arrowhead, it’s their second major siRNA partnership in the last year after the company started working with Sarepta. That deal paid Arrowhead $500 million upfront and included $10 billion in biobucks.