Home HealthStartup OMass Secures $310M to Advance Drug for Rare Endocrine Disorder

Startup OMass Secures $310M to Advance Drug for Rare Endocrine Disorder

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OMass Therapeutics now has $310 million in financing to support a pipeline led by a drug in development for a rare endocrine disorder with limited treatment options.

The disease is congenital adrenal hyperplasia (CAH), an inherited condition in which patients lack an enzyme the adrenal glands need to produce key hormones. The resulting hormonal imbalance leads to low levels of sodium that affect the brain and kidneys. Patients can also experience dehydration and adrenal crisis, a life-threatening complication stemming from the lack of the hormone cortisol from the adrenal glands.

Standard CAH treatment is high doses of glucocorticoids, synthetic versions of the hormone cortisol. But the required high doses introduce a range of side effects. The lead drug candidate of Oxford, U.K.-based OMass is OMS1620, a small molecule designed to block the melanocortin-2 (MC2) receptor in the adrenal glands. This approach is intended to decrease the production of hormones that leads to hormonal imbalance in CAH.

According to OMass, OMS1620 is designed to stay bound to the target receptor longer and maintain its activity in the presence of rising levels of hormones that get adrenal glands to produce more cortisol. The company says these properties allow more CAH patients to reach hormone levels closer to normal while they are taking glucocorticoids.

The only drug currently available for CAH drug is Crenessity, a Neurocrine Biosciences product approved by the FDA in 2024. This oral small molecule is designed to block a receptor called CRFR1 to inhibit the pituitary gland’s secretion of adrenocorticotropic hormone (ACTH), which in turn reduces the production of other hormones that lead to the hormonal imbalance in CAH. For 2025, Neurocrine reported $301.2 million in Crenessity sales. Vertex Pharmaceuticals is vying for a piece of the market with atumelnant, a small molecule inhibitor of ACTH. This drug came from Vertex’s recently closed $10 billion acquisition of Crinetics Pharmaceuticals.

OMass’s new capital will support plans to bring OMS1620 through Phase 2 testing in CAH and ACTH-dependent Cushing’s syndrome. The Series C round announced on Oct. 15 will also support OMass’s earlier-stage assets. A drug the company describes as a potential first-in-class SLC15A4 inhibitor is in development for lupus and other diseases associated with interferon overproduction and pathogenic B-cell activation.

OMass’s financing was co-led by new investors Bain Capital Life Sciences, Forbion, and Novo Holdings. Other participants include Janus Henderson Investors, SV Health Investors, and Jefferson Life Sciences as well as earlier investors Syncona, Oxford Science Enterprises, GV, Northpond Ventures, Sanofi Ventures, British Business Bank and University of Oxford.

Here’s a recap of other recent life sciences financing activity:

—Estuary Biotherapeutics launched, revealing $103 million in funding for two gene therapies in development for autosomal dominant polycystic kidney disease (ADPKD), an inherited progressive disorder that leads to the growth of cysts on the kidneys. The cash breaks down to a $10 million seed round co-led by 4BIO Capital and GV and a $93 million Series A round led by RA Capital Management.

—Altay Therapeutics raised $37 million to bring lead drug candidate DX057 to the clinic. This oral small molecule inhibitor of DUX4 is in development for facioscapulohumeral muscular dystrophy (FSHD), a rare inherited muscle disorder with no FDA-approved therapies. Sound Bioventures and HealthCap co-led Altay’s Series A round.

—RNA therapies developer Soufflé Therapeutics unveiled $135 million to bring its three lead programs into clinical testing and advance the rest of its pipeline. The startup’s therapies come from a technology platform that enables delivery of the therapy to specific types of cells and tissues. Juvia Group led Soufflé’s Series B financing.

—Biotech company Kallyope unveiled a new name and new leadership along with $94 million in funding. Now called TriGemx Bio, the biotech’s lead program is elismetrep, an oral TRPM8 channel blocker in Phase 3 development as a treatment for acute migraine. Data are expected in mid-2027. Results from early- and mid-stage tests of the drug were presented at medical conferences earlier this year. TriGemx’s new financing was co-led by The Column Group, TCGX, and Viking Global Investors.

—RougeTx launched with $58 million to support lead program RTX-001, a small molecule moving toward human testing as a treatment for hereditary hemorrhagic telangiectasia (HHT), an inherited bleeding disorder with no approved therapies. The Naarden, Netherlands-based startup’s Series A round was co-led by BioGeneration Ventures, Angelini Ventures, and Kurma Partners.

—Sensible Biotechnologies secured $47 million for its technology that enables the design and manufacturing messenger RNA. The startup says its naturally modified messenger RNA is produced in living cells, overcoming limitations of synthetic mRNA production. The financing encompasses a Series A round led by Oxford Science Enterprises and up to $20 million in non-dilutive funding from the government of Slovakia and the European Union.

—Startup ai3Bio revealed $48 million to support its new take on resetting the immune system. While many of the biopharma companies pursuing immune reset are developing cell therapies that address pathogenic B cells, ai3Bio’s is using messenger RNA to tap into self-destruct pathway in disease-driving T cells. Ziff Capital Partners and UPMC Enterprises led ai3Bio’s Series A financing.

—Varda Space Industries landed $251 million for “space-pharmaceutical manufacturing,” which is the production of drugs in space where zero gravity allows crystallization of active pharmaceutical ingredients in ways that are impossible on Earth. Lux Capital and Natural Capital led the Series D round for Varda, which has now raised a total of $598 million to date.

—Matchpoint Therapeutics secured $150 million for its “precision covalent medicines,” oral small molecules designed to address validated targets that have been historically hard to drug. The biotech is preparing to enter the clinic with MPT-062, an oral T cell modulator in development for autoimmune and inflammatory disorders.

—KymaThera raised $80 million for K-1728, a small molecule PI3K alpha inhibitor on track to startup Phase 1 testing as a monotherapy and as part of drug combinations for the treatment of HR-positive, HER2-negative breast cancers. Alta Partners led the San Diego-based biotech’s Series B round.

—Consano Bio raised $15 million for lead program C-1101, a platelet-derived multi-protein biologic in development for chronic painful lumbosacral radiculopathy, also called chronic sciatica. Participants in the financing were not disclosed. The capital will support the completion of Phase 1 testing; data are expected in the first half of 2027.

—Breye Therapeutics raised €67.5 million (about $76 million) to support its development of oral drugs for eye diseases. Lead program danegaptide is being readied for Phase 2 testing in non-proliferative diabetic retinopathy (NPDR). Breye’s Series A round was co-led by new investor Mission Biocapital and earlier investor Novo Holdings.

—Stockholm, Sweden-based AnaCardio now has €62.5 million (about $70.9 million) for lead program AC01, which is heading to Phase 2b testing for chronic advanced heart failure with reduced ejection fraction. Novo Holdings and the Ljungström family office co-led AnaCardio’s Series B round.

—Enveda raised $313 million to support development of three oral small molecules from its proprietary AI drug discovery technology. ENV-294 is in Phase 2 testing for eczema; ENV-308 is in early clinical development for weight loss and metabolic health; and ENV-6946 is in Phase 1 testing for inflammatory bowel disease. Catalio Capital Management led Enveda’s Series E financing.

—Basecamp Research has $140 million to advance toward the clinic with its in vivo cell therapies designed with the startup’s AI technology. S32 led Basecamp’s Series C financing.

—Sling Therapeutics secured $123 million for to bring its drug, linsitinib into Phase 3 testing for thyroid eye disease (TED). Sling’s small molecule inhibitor of the IGF-1 receptor could provide an oral alternative to infused or injected TED drugs. Forbion led Sling’s Series C financing.

—Circle Pharma secured $92.5 million to enter the clinic with a drug for ER-positive breast cancer. The company’s CID-165 is an oral macrocyclic cyclin D1 RxL inhibitor. In preclinical research, the drug candidate showed anti-tumor activity on its own and in combination with currently available therapies for breast cancer. The Column Group led Circle’s Series E financing.

—RegCell raised $44 million to support the development of regulatory T cell (Treg) therapies for immune tolerance. The startup’s approach converts pathogenic T cells into “stable, epigenetically reprogrammed, disease-relevant Tregs that restore immune tolerance at the source.” Playground Global led RegCell’s Series A financing.

—Encoded Therapeutics raised $275 million to finance Phase 3 testing of ETX101, an experimental treatment for SCN1A-positive Dravet syndrome in infants and young children. The capital will also support plans to advance ETX301 toward the clinic for post-amputation neuroma pain. GV and an unnamed healthcare fund co-led Encoded’s Series F round.

—Solstice Oncology launched with $225 million to support porustobart, a second-generation CTLA-4 inhibitor in mid-stage development for colon cancer. This antibody drug was licensed from Harbour BioMed. RA Capital Management led Solstice’s Series A financing.

—TwoStep Therapeutics secured $62.5 million as the startup prepares for Phase 1 testing of TS-104, a peptide drug conjugate that targets five tumor-associated integrins expressed across a range of solid tumors. Insight Partners and Medical Excellence Capital led TwoStep’s Series A financing.

—Tectora Therapeutics emerged with $55 million to support two early-stage programs in development for immunology and inflammation disorders. The small molecules came from Schrödinger, which formed the startup with New Enterprise Associates and RA Capital Management. The two investment firms led Tectora’s Series A financing. Schrödinger holds an equity stake in Tectora and could receive milestone payments and royalties tied to the progress of the drug candidates.

—Caspian Therapeutics launched from Kura Oncology with $50 million and the Kura program KO-7246, a next-generation menin inhibitor developed specifically for metabolic diseases. Kura currently markets Kozifti, a menin inhibitor approved last year for treating acute myeloid leukemia. BVF Partners led the new financing for Caspian, which is working to advance KO-7246 to Phase 1 testing.

—BrainChild Bio closed $116 million to support Phase 3 testing of BCB-276, a CAR T cell therapy in development for diffuse pontine glioma, a rare and aggressive pediatric brain cancer. This drug targets the cancer protein B7-H3. An undisclosed private family fund and foundation led BrainChild’s Series A round.

—Moonwalk Biosciences raised $70 million as it look to bring obesity drug candidate MW101 into the clinic next year. The South San Francisco- and San Diego-based biotech develops small-interfering RNA that is delivered to adipose tissue to precisely modulate pathways that are different than the ones addressed by GLP-1 drugs. Alpha Wave and YK Bioventures co-led the Series B round; Eli Lilly also participated in the financing.

—AI drug discovery startup Superluminal Medicines reeled in $60 million to bring an experimental metabolic medicine into the clinic. The small molecule is an agonist of the MC4 receptor, a G protein-coupled receptor that’s the same target as Imcivree, a Rhythm Pharmaceuticals drug with approvals in rare forms of obesity. Superluminal claims its molecule maximizes therapeutic activity and minimizes side effects. BVF Partners led the biotech’s Series B financing, which comes two years after a Series A round led by RA Capital Management.

—AusperBio closed $120 million in financing to support late-stage clinical testing and commercialization preparation for AHB-137, an antisense oligonucleotide in development for chronic hepatitis B. The capital will also support AHB-171, a small-interfering RNA therapy in early clinical testing for chronic hepatitis B. AusperBio’s Series C round was led by an unnamed strategic investor with participation from new investor RA Capital Management; they were joined by earlier investors in the biotech.

Photo: RomoloTavani, Getty Images 

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