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Tonix Pharmaceuticals Advances TNX-2900 to Phase 2 Trial for Prader-Willi Syndrome Following FDA Clearance

Rare Disease
  • Tonix Pharmaceuticals received FDA clearance to initiate a Phase 2 trial of TNX-2900, a magnesium-potentiated intranasal oxytocin formulation for Prader-Willi syndrome in children and adolescents aged 8 to 17.5 years.
  • The drug received both Orphan Drug and Rare Pediatric Disease designations from the FDA, potentially qualifying Tonix for a Priority Review Voucher upon approval.
  • The 12-week randomized, double-blind, placebo-controlled study will evaluate TNX-2900's efficacy using the Hyperphagia Questionnaire for Clinical Trials as the primary endpoint.
  • Prader-Willi syndrome affects 1 in 10,000 to 1 in 30,000 births and has an average life expectancy of 22.1 years, representing a significant unmet medical need.

Kedrion Biopharma Secures FDA Approval for QIVIGY, First Proprietary IVIG Therapy for Primary Immunodeficiency

Drug ApprovalRare Disease
  • Kedrion Biopharma received FDA approval for QIVIGY, a 10% intravenous immune globulin therapy for treating adults with primary humoral immunodeficiency disorders.
  • The therapy demonstrated zero acute serious bacterial infections in a 12-month clinical study of 47 patients, meeting its primary efficacy endpoint.
  • QIVIGY represents over 10 years of internal development and will be available in the U.S. in early 2026, with global expansion planned.
  • Kedrion is investing over $415 million in U.S. infrastructure expansion, including 40 new plasma collection centers and manufacturing capacity increases.

Clinical Assessment of Pharmacokinetics, Efficacy, and Safety of 10% IVIg in PID Patients

NCT03961009CompletedPhase 3
Kedrion S.p.A.
Posted 4/30/2019

Soligenix Raises $7.5 Million in Public Offering to Advance Rare Disease Pipeline

Rare Disease
  • Soligenix priced a public offering of 5.56 million shares and warrants at $1.35 per unit, raising approximately $7.5 million in gross proceeds.
  • The late-stage biopharmaceutical company will use funds for research and development activities, commercialization efforts, and general corporate purposes.
  • The company's pipeline includes HyBryte for cutaneous T-cell lymphoma treatment and vaccine candidates for biodefense applications.
  • Existing warrants from previous offerings will be amended to reduce exercise price to $1.35 per share to align with the new offering terms.

FDA Issues Three Draft Guidances to Accelerate Cell and Gene Therapy Development Through Innovative Trial Designs

Rare DiseaseReal World Evidence
  • The FDA released three draft guidances on September 24, 2025, aimed at promoting innovative clinical trial designs for cell and gene therapies (CGTs) in small populations as part of PDUFA VII commitments.
  • The guidance outlines six innovative trial design approaches including single-arm trials, disease progression modeling, externally controlled studies, adaptive designs, Bayesian designs, and master protocol designs.
  • New recommendations emphasize the use of real-world evidence and digital health technologies for post-approval safety monitoring and data collection in CGT development.
  • The guidances specifically target regenerative medicine advanced therapies (RMATs) for serious conditions, replacing previous 2019 guidance with enhanced flexibility and collaborative regulatory approaches.

A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 50 Years of Age With Non-congenital Myotonic Dystrophy Type 1

NCT06844214RecruitingPhase 1
Sanofi
Posted 7/23/2025

A Safety and Efficacy Study of One-time SAR402663 in Adults With Neovascular Age-related Macular Degeneration

NCT06660667RecruitingPhase 1
Sanofi
Posted 11/21/2024

Harmony Biosciences' ZYN002 Phase 3 Trial Fails to Meet Primary Endpoint in Fragile X Syndrome

Rare DiseaseOrphan Drug
  • Harmony Biosciences announced that its Phase 3 RECONNECT study of ZYN002 in Fragile X syndrome failed to meet the primary endpoint of improvement in social avoidance due to higher than expected placebo response rates.
  • The randomized, double-blind, placebo-controlled trial enrolled 215 patients aged 3 to under 30 years with Fragile X syndrome, testing the transdermal cannabidiol gel over an 18-week treatment period.
  • Despite the setback, Harmony Biosciences remains committed to advancing its late-stage pipeline, including upcoming Phase 3 trials for pitolisant HD in narcolepsy and idiopathic hypersomnia planned for Q4 2025.
  • The failure represents a significant blow to addressing the unmet medical need in Fragile X syndrome, a rare genetic disorder affecting up to 80,000 patients in the US with no FDA-approved treatments currently available.

A Study of BPN14770 in Male Adults (Aged 18 to 45) With Fragile X Syndrome

NCT05358886CompletedPhase 3
Tetra Discovery Partners
Posted 11/1/2022

A Randomized Study of BPN14770 in Male Adolescents (Aged 9 to < 18 Years) With Fragile X Syndrome

NCT05163808Active, Not RecruitingPhase 2
Tetra Discovery Partners
Posted 3/29/2022

Study of SPG601 in Adult Men With Fragile X Syndrome

NCT06413537CompletedPhase 2
Spinogenix
Posted 7/5/2024

Aro Biotherapeutics Completes Enrollment in Phase 1b Trial of Novel siRNA Therapy for Late-Onset Pompe Disease

Rare DiseaseOrphan DrugTargeted Therapy
  • Aro Biotherapeutics has completed enrollment in its Phase 1b clinical trial of ABX1100, a first-in-class targeted siRNA therapy for late-onset Pompe disease patients currently receiving enzyme replacement therapy.
  • ABX1100 represents a novel therapeutic approach using Centyrin-siRNA conjugate technology to inhibit glycogen synthase 1 (GYS1), targeting the root cause of glycogen accumulation in muscle tissue.
  • The trial evaluates safety, tolerability, and preliminary therapeutic activity over 20 weeks, with results expected to inform later-phase clinical studies for this orphan drug-designated treatment.
  • Current enzyme replacement therapy requires intravenous infusions lasting up to 6 hours multiple times monthly, highlighting significant unmet medical need in this rare neuromuscular disorder.

Study to Assess the Safety, Tolerability, PK and PD of ABX1100

NCT06109948RecruitingEarly Phase 1
Aro Biotherapeutics
Posted 10/19/2023

ResVita Bio Receives FDA Clearance to Advance Novel Bacterial Therapy for Rare Skin Disease

Orphan DrugRare Disease
  • ResVita Bio completed a successful Pre-IND meeting with the FDA for RVB-003, a first-in-class therapy for Netherton Syndrome, a life-threatening genetic skin disorder with no approved treatments.
  • The company's innovative approach uses genetically engineered bacteria to continuously produce therapeutic proteins directly on the skin surface, overcoming limitations of conventional protein drugs.
  • FDA provided positive feedback on the development program, clearing the path for IND submission in the first half of 2026 and clinical efficacy readout by early 2027.

Acadia Pharmaceuticals' Phase 3 Trial of Intranasal Carbetocin for Prader-Willi Syndrome Fails to Meet Primary Endpoint

Rare DiseaseOrphan Drug
  • Acadia Pharmaceuticals announced that its Phase 3 COMPASS PWS trial of intranasal carbetocin (ACP-101) failed to demonstrate statistically significant improvement over placebo for hyperphagia in Prader-Willi syndrome patients.
  • The 12-week randomized, placebo-controlled trial enrolled 175 children and adults aged 5-30 years with PWS, testing carbetocin 3.2 mg three times daily against placebo.
  • Despite the setback, Acadia maintains its growth outlook with two approved products projected to generate over $1 billion in net sales in 2025 and a robust pipeline including eight disclosed programs.
  • The company will discontinue further investigation of intranasal carbetocin but plans to share study data with the PWS community for future learning.

FDA Breaks Precedent with Literature-Based Approval for Leucovorin in Cerebral Folate Deficiency

Drug ApprovalRare Disease
  • The FDA is initiating approval of leucovorin calcium tablets for cerebral folate deficiency based on systematic literature analysis rather than traditional clinical trials, marking a departure from the agency's standard evidence requirements.
  • The neurological condition affects folate transfer into the brain and causes developmental delays with autistic features, seizures, and movement coordination problems in patients.
  • President Trump referenced leucovorin as "a potential drug for some autism symptoms" during a recent media briefing, highlighting the connection between cerebral folate deficiency and autism spectrum features.
  • This approval represents a significant shift in FDA policy, as the agency has historically maintained that observational studies generate hypotheses but are insufficient for drug approvals.

Santhera Secures CHF 20 Million Growth Funding to Accelerate Global AGAMREE Rollout for Duchenne Muscular Dystrophy

Rare Disease
  • Santhera Pharmaceuticals secured approximately CHF 20 million in additional funding from existing investors Highbridge and R-Bridge to accelerate the global rollout of AGAMREE (vamorolone) for Duchenne muscular dystrophy treatment.
  • Strong demand for AGAMREE has exceeded expectations across the US, Europe, and China, with over 1,000 patients treated worldwide and US sales reaching USD 49.4 million in the first half of 2025.
  • The funding comprises USD 13 million from a royalty monetization with R-Bridge and CHF 10 million from Highbridge through a convertible bond extension, supporting inventory expansion and launch acceleration.
  • AGAMREE represents a novel dissociative anti-inflammatory drug that demonstrated efficacy in the pivotal VISION-DMD study while potentially avoiding growth restrictions and bone metabolism issues associated with traditional corticosteroids.

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