An FDA Rejection, A Big Pharma Purchase, and Charting Watertown's Life Science Future -Watertown Bio News RoundUp

Big news from Watertown in the last week, but before diving in, we wanted to highlight our upcoming Event on March 10th Charting Watertown’s Life Science Future, in partnership with the Charles River Chamber of Commerce. We will be discussing Watertown’s recent life science growth (centered on our recent industry report) , where it stands today, and where it is heading, with remarks from Eric Paley, State Sec. of Economic Development and panel of municipal and industry leaders featuring Watertown City Manager George Proakis, Labshares CEO Philip Borden and President of Boylston Properties Mark Deschenes, moderated by Hannah Green from the Boston Business Journal. We have a special offer for Watertown Bio readers,use code DNA for complementary registration, and we hope to see you there! As always, if you find our content informative, consider supporting our work.

FDA fails to Approve Disc Medicine’s Bitopertin

On Friday 2/13, Disc Medicine (headquartered at the Arsenal on the Charles) received an FDA letter failing to approve their new drug application for Bitopertin, a small molecule designed to treat those with erythropoietic protoporphyria, a rare genetic disease that causes pain when exposed to sunlight. In their letter, the FDA said that while they agreed Bitopertin’s phase 2 trials did show a decrease in the target biomarker protoporphyrin IX (PPIX), it is still unclear if the decrease in PPIX is likely to predict a clinical benefit.

Bitopertin had previously received one of nine initial Commissioner’s National Priority Voucher (CNPV), a new program that aims to bring drugs “aligned with national priorities” to market sooner by reducing FDA review time from 11-12 months down to 1-2 months. The program has come under scrutiny from lawmakers for potential political interference in the review process, and a report from Stat News alleged that involved in the CNPV program had become personally involved in the bitopertin review committee’s decision.

In a press release from Disc Medicine on Friday, the company stated that it believes its Phase 3 Apollo trial will provide the necessary data to meet the FDA’s concerns as outlined in their letter, with top line data available in Q4 2026 for re-submission to the FDA, and an updated FDA decision anticipated in mid-2027. Shares in the company fell Friday by nearly 22%, closing at $55.95.

Orna Therapeutics to Be Acquired By Eli Lilly

Last week, Orna Therapeutics (headquartered at Arsenal Yards) and Eli Lilly announced a definitive agreement for Eli Lilly to acquire Orna for $2.4 billion including incentives and milestone payments. Orna is using their circular mRNA and lipid nanoparticle delivery technology to develop cell therapies for a range of diseases. Its not clear if the merger means Orna will be leaving Watertown for Lilly’s Fort Point headquarters, and neither company responded to inquiries from Watertown Bio.

Orna’s lead candidate ORN-252 is being developed to treat B-cell driven autoimmune diseases such as lupus or rheumatoid arthritis. Arsenal on the Charles-based Kymera Therapeutics is also developing an oral degrader (KT-579) to treat these diseases, which plans to enter clinical trials this year. Orna also had recently signed an agreement in early 2025 with Vertex Pharmaceuticals to develop a cell therapy for sickle cell disease and transfusion-dependent beta thalassemia. Its unclear how the Lilly acquisition will impact this research pact.

Windgap Medical, Pathcision Medicine and Stellular Bio raise an aggregated $11.85 million in funding.

Windgap Medical, based out of Cambridge Scientific Labs, reported a $10,350,000 fundraising round to the SEC at the end of January. The company develops a number of products based on their auto-injection drug delivery platform, such as their Dualflo platform that can automatically inject two different medicines in sequence, or their OneMix platform that can automatically reconstitute and inject a medcine.

Pathcision Medicine, based out of BioLabs, reported a $1,500,000 fundraising round to the SEC in the first week of February. The company is using their proprietary drug discovery engine to develop new precision medicines for oncology and other diseases.

Stellular Bio, (formerly known as Platelet Bio) headquartered at 65 Grove St, reported a Series 1 fundraising round (amount undisclosed) led by Ziff Capital Partners, who also helped launch Watertown based Corner Therapeutics. The company aims to generate a Platelet-derived regenerative biologic (PRB) that is similar to platelet-rich plasma but is produced by pluripotent stem cells in a controlled process, rather than harvested and refined from the patient. The series 1 fundraising will be used to advance their lead candidate STLR-201, which is being developed to treat treatment for dry eye from Sjogren's disease , an autoimmune disease where the immune system attacks saliva producing glands, resulting in dry eyes and dry mouth.

ICYM: Soufflé Therapeutics Signs Research Agreement with Bayer

Last month Soufflé Therapeutics (headquartered at the Genesis Building) announced a research agreement with Bayer to develop an siRNA therapeutic for a form of dilated cardiomyopathy, a disease where the heart chambers thin and stretch, reducing their ability to pump blood. The financial terms of the deal were not announced. Soufflé Therapeutics launched from stealth last year with a $200 million Series A raise, (the second largest in Watertown in 2025), with a mission to develop siRNA therapies and associated delivery methods, with an initial focus on muscular and cardiovascular diseases.

Quick Updates

Arrivals

• Cidnam, Inc (Cambridge Scientific Labs) which is focused on large DNA synthesis without using large volume bacterial fermentation methods.

In the News

• The Boston Business Journal has more details on the Orna-Eli Lilly Deal.

People

• Remix Therapeutics named Chip Michaud, as their Vice President and Head of Finance.