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Clinical Trial News

FDA Appoints Biotech Veteran George Tidmarsh to Lead Drug Regulation Center Amid Agency Restructuring

  • The FDA has appointed Dr. George Tidmarsh, a former pharmaceutical executive and cancer specialist, to direct the Center for Drug Evaluation and Research, the agency's largest division responsible for reviewing drug safety and effectiveness.
  • Tidmarsh brings extensive industry experience, having founded multiple pharmaceutical companies including Horizon Pharmaceuticals and developed seven FDA-approved drugs, providing stability during significant agency upheaval.
  • The appointment comes as the FDA faces major workforce reductions with about 2,000 staff layoffs and over 1,000 departures through buyouts or early retirement under Health Secretary Robert F. Kennedy Jr.'s restructuring efforts.
  • Tidmarsh has previously criticized key regulatory decisions, including the accelerated approval of controversial treatments like Biogen's Alzheimer's drug Aduhelm and Sarepta's Duchenne muscular dystrophy therapy Elevidys.

Corteria Pharmaceuticals Advances First-in-Class CRF2 Agonists into Phase 2 Heart Failure and Phase 1 Obesity Trials

  • Corteria Pharmaceuticals has initiated the CRAFT-WHF Phase 2 trial of COR-1167 in 300 patients with worsening heart failure, targeting an unmet medical need affecting over 20% of heart failure patients.
  • The company's COR-1389 obesity treatment has completed single ascending dose testing and entered a 12-week multiple ascending dose phase, with preclinical data showing weight loss comparable to GLP-1 agonists while preserving muscle mass.
  • Both first-in-class CRF2 agonists demonstrated positive safety profiles and target engagement in Phase 1 studies, with topline results expected by the end of 2026.
  • The novel mechanism targets corticotropin-releasing factor receptor 2, potentially addressing significant gaps in current treatments for heart failure and obesity-related cardiovascular complications.

CRF2 Agonist for the Treatment of Worsening Heart Failure

NCT06815471RecruitingPhase 2
Corteria Pharmaceuticals
Posted 8/7/2025

NICE Approves First-in-Class Birch Pollen Immunotherapy for Severe Hayfever on NHS

Drug Approval
  • NICE has recommended Itulazax 12 SQ-Bet, an under-the-tongue immunotherapy tablet containing birch pollen extract, for NHS use in England for patients with severe hayfever unresponsive to conventional treatments.
  • The treatment works by training the immune system to tolerate tree pollen through three years of daily use, offering long-term relief rather than symptom management.
  • Approximately 27,000 people in England are expected to benefit from this recommendation, with birch pollen allergy affecting about 25% of hayfever sufferers.
  • The NHS will provide the treatment within three months of NICE publishing its final guidance, marking a significant advancement for the UK allergy community.

ALK's Tree Pollen Tablet ITULAZAX Receives NICE Recommendation for UK NHS Reimbursement

Drug Approval
  • ALK's tree pollen tablet ITULAZAX has been recommended by NICE for use in the UK health system, making it eligible for general reimbursement through the NHS in England, Wales, and Northern Ireland.
  • This marks ALK's second NICE approval for sublingual allergy immunotherapy products in 2025, following ACARIZAX approval in March, addressing significant underutilization of AIT in the UK compared to other European countries.
  • The approval comes as scientists warn of an early tree pollen season with higher counts, affecting approximately 16 million people with hay fever in the UK, including 4 million with moderate to severe tree pollen allergy.
  • NICE published Final Draft Guidance on July 21, 2025, with Final Guidance expected in August, after which NHS systems will be required to implement ITULAZAX in treatment practices.

Abu Dhabi Investment Authority Invests $200 Million in Indian MedTech Company Meril

  • Abu Dhabi Investment Authority (ADIA) will acquire a 3% stake in Indian medical device company Micro Life Sciences (Meril) for $200 million, valuing the company at $6.6 billion.
  • Meril specializes in clinically advanced medical technology solutions across cardiovascular, structural heart, orthopedics, endo-surgery, in-vitro diagnostics, and surgical robotics.
  • The investment will enable Meril to accelerate growth, attract world-class talent, and strengthen clinical research efforts to improve healthcare solutions globally.
  • The transaction is subject to regulatory approval by India's Competition Commission and will make Meril backed by two globally recognized investors, ADIA and Warburg Pincus.

Alkermes' Alixorexton Shows Promise in Phase 2 Narcolepsy Trial, Achieving Normal Wakefulness Levels

  • Alkermes reported positive Phase 2 results for alixorexton, an investigational daily pill that helped type 1 narcolepsy patients achieve statistically significant improvements in daytime wakefulness over six weeks.
  • Patients taking all tested doses of alixorexton reached normal wakefulness levels and experienced significant decreases in daytime sleepiness compared to baseline measurements.
  • Only the middle dose demonstrated statistically significant reduction in cataplexy frequency, though CEO Richard Pops attributed this to methodological limitations in patient self-reporting.
  • The results position Alkermes to compete in the predicted multibillion-dollar narcolepsy treatment market, with plans to refine methodology for future trials.

A Study to Evaluate the Safety and Effectiveness of ALKS 2680 in Subjects With Narcolepsy Type 2

NCT06555783RecruitingPhase 2
Alkermes, Inc.
Posted 7/26/2024

A Study to Evaluate the Safety and Effectiveness of ALKS 2680 in Subjects With Idiopathic Hypersomnia

NCT06843590RecruitingPhase 2
Alkermes, Inc.
Posted 5/22/2025

A Study to Evaluate the Safety and Effectiveness of ALKS 2680 in Subjects With Narcolepsy Type 1 (ALKS 2680-201)

NCT06358950CompletedPhase 2
Alkermes, Inc.
Posted 3/28/2024

Omega Funds Closes Oversubscribed $647M Fund VIII to Accelerate Life Sciences Innovation

Precision Medicine
  • Omega Funds successfully closed its eighth fund with $647 million in capital commitments, exceeding its $600 million target despite challenging fundraising conditions.
  • The firm has achieved significant commercial success with 52 products brought to market, 50 M&A exits, and 47 public listings since 2004.
  • Fund VIII will continue supporting innovative life sciences companies in the US and Europe targeting severe unmet medical needs across oncology, rare diseases, and precision medicine.
  • Recent notable exits include Scorpion Therapeutics acquired by Eli Lilly for potentially $2.5 billion and EyeBio purchased by Merck for $1.3 billion.

Japanese Researchers Develop GAI-17, Novel Stroke Drug with Six-Hour Treatment Window

  • Researchers at Osaka Metropolitan University developed GAI-17, a novel stroke treatment that significantly reduces brain cell death and paralysis in animal models by inhibiting GAPDH protein aggregation.
  • The drug demonstrated effectiveness even when administered six hours after stroke onset, potentially extending treatment windows well beyond current therapeutic standards.
  • GAI-17 showed no major side effects in preclinical studies and may have broader applications for treating other neurological diseases including Alzheimer's disease.
  • The compound targets a multifunctional protein linked to various intractable brain disorders, offering potential for a new class of neuroprotective therapies.

Donidalorsen Shows 62% Reduction in HAE Attacks When Patients Switch from Other Prophylactics in Phase 3 Study

Rare Disease
  • Patients with hereditary angioedema who switched to donidalorsen from other prophylactic treatments experienced a 62% further reduction in mean monthly attack rate compared to baseline.
  • 84% of patients surveyed preferred donidalorsen over their prior treatment, citing better disease control, less administration time, and reduced injection site reactions.
  • The investigational RNA-targeted therapy is currently under FDA review with a PDUFA date of August 21, 2025, as a potential first-in-class prophylactic treatment for HAE.
  • Results from the Phase 3 OASISplus prospective switch study were published in The Journal of Allergy and Clinical Immunology In Practice.

A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)

NCT05392114Active, Not RecruitingPhase 3
Ionis Pharmaceuticals, Inc.
Posted 7/13/2022

OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)

NCT05139810CompletedPhase 3
Ionis Pharmaceuticals, Inc.
Posted 12/3/2021

OncoZenge Partners with Meribel Pharma for BupiZenge Phase 3 Manufacturing

  • OncoZenge has appointed Meribel Pharma Solutions as its Contract Development and Manufacturing Organization (CDMO) for the BupiZenge Phase 3 project.
  • The partnership will support OncoZenge's multi-country Phase 3 study aimed at securing European approval for BupiZenge, a novel bupivacaine lozenge for oral pain.
  • BupiZenge targets oral mucositis in cancer patients, representing a significant unmet medical need affecting millions of patients worldwide.
  • The collaboration leverages Meribel's expertise in complex oral solid dose manufacturing and their prior experience with BupiZenge formulations.

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