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Biotech Briefs: Scholar Rock, Vaderis, Tolerance Bio & More 

The latest from small and Emerging Pharma companies featuring Scholar Rock, Vaderis Therapeutics, Tolerance Bio, NeoImmuneTech, Fulcrum Therapeutics, Slate Medicines, Infinimmune and Replimmune. 

For news on the large and mid-sized bio/pharmaceutical companies, see Global Briefs. 

* Scholar Rock Provides Update on Contract Fill–Finish Facility for BLA 
* Vaderis Raises $152 M for Rare-Disease Vascular Drug 
* Tolerance Bio, NeoImmuneTech in $260-M Pact for Fusion Protein for Thymic Disease 
* Fulcrum Therapeutics, Slate Medicines To Merge 
* Infinimmune Raises $75 M To Support mAbs for Inflammatory Disease 
* Replimmune Gets FDA Nod for Melanoma Drug 

Scholar Rock Provides Update on Contract Fill-Finish Facility for BLA 
Scholar Rock, a Cambridge, Massachusetts-based bio/pharmaceutical company focused on neuromuscular diseases, has announced that the US Food and Drug Administration’s (FDA) review of the company’s biologics license application (BLA) for apitegromab, a drug for treating children and adults with spinal muscular atrophy, a rare genetic neuromuscular disorder, is progressing with a new second fill–finish facility for the product following the removal of a facility of Catalent Indiana LLC (part of Novo Nordisk) as part of the application.  

Scholar Rock’s apitegromab BLA submission in March 2026 included two fill–finish facilities, in alignment with FDA guidance, the Catalent Indiana LLC facility and a second facility, which provided two independent paths to a potential FDA approval. The company received a customer notification from Catalent Indiana on August 7, 2026 that FDA has classified the April 2026 site inspection of Catalent Indiana as Official Action Indicated, a classification that indicates a facility is in an unacceptable state of compliance 

Scholar Rock will continue to collaborate closely with the FDA and under its guidance, will remove Catalent Indiana from the apitegromab BLA. FDA review of the apitegromab BLA will progress solely with the second fill–finish facility. 

The BLA remains on track for potential FDA approval by the September 30, 2026, Prescription Drug User Free Act (PDUFA) action date. 

Source: Scholar Rock 

Vaderis Raises $152 M for Rare-Disease Vascular Drug 
Vaderis Therapeutics, a Basel, Switzerland-based clinical-stage bio/pharmaceutical company focused on rare vascular diseases, has announced the closing of a private $152.5-million series B financing and initiation of the company’s global Phase III clinical study evaluating its lead candidate, engasertib, in patients with hereditary hemorrhagic telangiectasia, a rare genetic vascular disorder characterized by recurrent bleeding and arteriovenous malformations, a tangle of blood vessels that creates irregular connections between arteries and veins.

Source: Vaderis Therapeutics 

Tolerance Bio, NeoImmuneTech in $260-M Pact for Fusion Protein for Thymic Disease 
Tolerance Bio, a Philadelphia, Pennsylvania-based bio/pharmaceutical company focused on thymic diseases, has announced an exclusive license agreement in the Americas and Europe with NeoImmuneTech, a Rockville, Maryland-based bio/pharmaceutical company, for the development and commercialization of efineptakin alfa, a clinical-stage long-acting IL-7 fusion protein, for certain thymus-related indications, in a deal worth up to $260 million. 

Source: Tolerance Bio 

Fulcrum Therapeutics, Slate Medicines To Merge 
Fulcrum Therapeutics, a Cambridge, Massachusetts-based bio/pharmaceutical company, and Slate Medicines, a bio/pharmaceutical company developing therapeutics for the treatment of migraines, have entered into a definitive agreement to combine the companies in an all-stock transaction.  

The resulting entity will focus on advancing Slate’s pipeline of therapeutics, including SLTE-1009, a clinical-stage subcutaneous anti-PACAP/VIP monoclonal antibody for the prevention of migraines and other headache disorders. Upon completion of the merger, the combined company plans to operate under the name Slate Medicines. 

Source: Fulcrum Therapeutics 

Infinimmune Raises $75 M To Support mAbs for Inflammatory Disease 
Infinimmune, a bio/pharmaceutical company focused on antibody discovery and design, has announced the closing of a $75-million Series A financing to accelerate clinical development of Infinimmune’s two lead programs in atopic dermatitis, support advancement of additional programs leveraging human-first biology, and enable continued deployment of the company’s Anthrobody discovery platform. Infinimmune will initiate first-in-human studies for its two lead programs in 2027. 

Source: Infinimmune 

Replimmune Gets FDA Nod for Melanoma Drug 
The US Food and Drug Administration (FDA) has granted accelerated approval for vusolimogene oderparepvec-wtpg, a genetically modified oncolytic viral therapy of Replimmune, a Woburn, Massachusetts-based bio/pharmaceutical company. The approval for vusolimogene oderparepvec-wtpg, was in combination with nivolumab for the treatment of adult patients with unresectable advanced cutaneous melanoma who experienced disease progression on a programmed death receptor-1 (PD-1)-blocking antibody-based regimen. Nivolumab is the active ingredient of Bristol-Myers Squibb’s immuno-oncology drug, Opdivo.  

Source: US Food and Drug Administration