NexEos Bio Receives US FDA IND Clearance to Advance NTX-1024 into P-II/III Study for Vernal Keratoconjunctivitis

Shots: The US FDA has cleared NexEos Bio’s IND application to initiate a P-II/III clinical study of NTX-1024 ophthalmic solution for the treatment of vernal keratoconjunctivitis (VKC) The P-II/III study will evaluate the safety and efficacy of NTX-1024 in VKC, following positive results from an open-label investigator-initiated study showing improvements in symptoms and ocular inflammation…

ByDipanshu DixitFebruary 20, 2026

NEWS

Santhera Pharmaceuticals Reports Swissmedic’s Approval of Agamree (Vamorolone) to Treat Duchenne Muscular Dystrophy

Shots: Swissmedic has approved Agamree for the treatment of pts (≥4yrs) with Duchenne muscular dystrophy and expects launch in H2’26 Approval was based on pivotal P-IIb (VISION-DMD) data and EMA assessments, granting 15-year Swiss orphan drug exclusivity; Santhera retains exclusive Swiss distribution. Trial met its 1EP of Time to Stand (TTSTAND) velocity vs PBO at…

ByDipanshu DixitJanuary 15, 2026

NEWS

Genethon Signs Worldwide Licensing Agreement with AskBio (Bayer AG) to Advance Gene Therapy for Pompe Disease

Shots: Genethon has entered into an exclusive, worldwide licensing agreement with AskBio for the use of a patented component of AskBio’s investigational gene therapy AB-1009 for the treatment of Pompe disease The licensed component is based on a transgene encoding a truncated form of GAA, developed by Genethon, which demonstrated preclinical efficacy in correcting glycogen…

ByPrince GiriJanuary 14, 2026

NEWS

Agios Reports the US FDA Approval of Aqvesme for Anemia in Alpha or Beta Thalassemia

Shots: The US FDA has approved Agios’ Aqvesme (mitapivat) for the treatment of anemia in adults with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia Approval was based on the global P-III (ENERGIZE) and (ENERGIZE-T) studies (n=452), which met all 1EPs and 2EPs, demonstrating significant improvements in hemoglobin, fatigue, and reductions in transfusion burden vs PBO Aqvesme…

ByPrince GiriDecember 25, 2025

NEWS

Shionogi to Acquire Tanabe Pharma’s Radicava Franchise in $2.5B Rare Disease Deal

Shots: Shionogi to acquire a newly established company that will be created by Tanabe Pharma to hold the rights to Radicava ORS (edaravone) & IV Radicava As per the deal, Tanabe Pharma will establish a new entity, & Shionogi will acquire 100% of its shares, with Tanabe’s US Radicava business company becoming a wholly owned…

ByRidhi RastogiDecember 23, 2025

NEWS

Mirum Pharmaceuticals to Acquire Bluejay Therapeutics, Strengthening Rare Disease Portfolio

Shots: Mirum Pharmaceuticals has entered into an agreement to acquire Bluejay Therapeutics, gaining global rights to brelovitug with Breakthrough Therapy and PRIME designations, and expanding Mirum’s rare liver disease portfolio As per the deal, Mirum will acquire all Bluejay shares for $250M in cash and $370M in Mirum stock, plus up to $200M in sales-based…

ByDipanshu DixitDecember 9, 2025

INSIGHTS+

Disease of the Month – Telangiectasia

Shots: Telangiectasia refers to the presence of small, visible red blood vessels, often called broken capillaries, that appear close to the surface of the skin. While common, they are often misunderstood, making awareness and early understanding essential At PharmaShots, our Disease of the Month report is crafted to make complex medical information accessible and relevant…

ByDipanshu DixitNovember 26, 2025

NEWS

Glaukos Receives FDA Approval for Epioxa

Shots: Glaukos reported the US FDA approval of Epioxa HD/Epioxa, an incision-free, topical drug therapy for keratoconus, offering a non-surgical alternative to traditional corneal cross-linking The approval was based on two P-III pivotal studies (>400 pts) that met its pre-specified 1EPs strong efficacy and safety; Epioxa uses a UV-activated, oxygen-enriched formulation that preserves the corneal…

ByPrince GiriOctober 22, 2025

NEWS

VectorY Therapeutics Collaborates with Shape Therapeutics to Develop Vectorized Antibodies for Neurodegenerative Diseases

Shots: VectorY has entered into an option & license deal with Shape to assess Shape’s SHP-DB1 (AAV capsid) for vectorized Abs payloads against 3 targets & may secure an exclusive license for their delivery if evaluation succeeds As per the deal, VectorY will handle development & commercialization of resulting therapies in exchange for an upfront…

ByRidhi RastogiSeptember 18, 2025

NEWS

Neurocrine Biosciences Initiates P-I Study of NBIP-01435 to Treat CAH

Neurocrine initiated a P-I first-in-human study of NBIP-01435 to assess safety, PK/PD, tolerability, and immunogenicity in healthy adults via SC injection for the potential treatment of congenital adrenal hyperplasia (CAH) NBIP-01435 (CRF1 antagonist) aims to improve androgen control and reduce glucocorticoid dosing. In Dec’24, the FDA approved Neurocrine's crinecerfont, an oral CRF1 antagonist, to treat…

ByPrince GiriJuly 1, 2025

NexEos Bio Receives US FDA IND Clearance to Advance NTX-1024 into P-II/III Study for Vernal Keratoconjunctivitis

Santhera Pharmaceuticals Reports Swissmedic’s Approval of Agamree (Vamorolone) to Treat Duchenne Muscular Dystrophy

Genethon Signs Worldwide Licensing Agreement with AskBio (Bayer AG) to Advance Gene Therapy for Pompe Disease

Agios Reports the US FDA Approval of Aqvesme for Anemia in Alpha or Beta Thalassemia

Shionogi to Acquire Tanabe Pharma’s Radicava Franchise in $2.5B Rare Disease Deal

Mirum Pharmaceuticals to Acquire Bluejay Therapeutics, Strengthening Rare Disease Portfolio

Disease of the Month – Telangiectasia

Glaukos Receives FDA Approval for Epioxa

VectorY Therapeutics Collaborates with Shape Therapeutics to Develop Vectorized Antibodies for Neurodegenerative Diseases

Neurocrine Biosciences Initiates P-I Study of NBIP-01435 to Treat CAH

Contact US

FOLLOW US