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Clinical Trial News

Health Canada Extends Evkeeza Approval to Infants as Young as 6 Months for Rare Cholesterol Disorder

Drug ApprovalRare DiseaseMonoclonal Antibody
  • Health Canada has extended approval of Evkeeza (evinacumab) to treat children as young as 6 months old with homozygous familial hypercholesterolemia (HoFH), a devastating inherited cholesterol disorder.
  • HoFH affects 1 in 300,000 people globally and causes dangerously high LDL cholesterol levels exceeding 10 mmol/L, putting patients at risk for premature heart disease and cardiac events.
  • Evkeeza works by blocking the ANGPTL3 protein to reduce cholesterol levels and is administered monthly via infusion alongside low-fat diet and other cholesterol-lowering medications.
  • The therapy is now commercially available in multiple countries including Canada, the UK, US, Italy, Japan, and several European nations, with early access programs in 13 additional countries.

Sonnet BioTherapeutics Pivots to Cryptocurrency with $888 Million HYPE Token Treasury Strategy

  • Sonnet BioTherapeutics announced a definitive agreement to merge with Rorschach I LLC, creating Hyperliquid Strategies Inc. with an $888 million valuation focused on HYPE token treasury management.
  • The new entity will hold approximately 12.6 million HYPE tokens worth $583 million plus $305 million in cash, making it the largest U.S.-listed public company to hold HYPE tokens in its treasury.
  • Following the merger, Sonnet will continue as a wholly owned subsidiary developing its biotech assets including SON-1010, while legacy shareholders receive contingent value rights tied to future biotech asset value.
  • The transaction includes participation from prominent investors including Paradigm, Galaxy Digital, and Pantera Capital, with closing expected in the second half of 2025.

Sun Pharma Launches Deuruxolitinib for Severe Alopecia Areata in US Market

Drug Approval
  • Sun Pharmaceuticals has launched deuruxolitinib (Leqselvi) in the United States for treating severe alopecia areata in adults, offering 8 mg tablets to patients aged 18 and older.
  • Clinical trials demonstrated that approximately one-third of patients achieved 80% or more scalp hair coverage by week 24, with some reaching 90% coverage from an initial average of only 13%.
  • Long-term data showed sustained efficacy with 48.8% of patients achieving SALT scores ≤20 by week 68, and 99.6% of initial responders maintaining their response.
  • The company has introduced the Leqselvi Support Program offering eligible patients medication for as little as $0 for up to two years with personalized assistance.

Study to Evaluate the Efficacy and Safety of CTP-543 in Adults With Moderate to Severe Alopecia Areata (THRIVE-AA1)

NCT04518995CompletedPhase 3
Concert Pharmaceuticals
Posted 11/23/2020

Study to Evaluate the Efficacy and Safety of CTP-543 in Adults With Moderate to Severe Alopecia Areata (THRIVE-AA2)

NCT04797650CompletedPhase 3
Concert Pharmaceuticals
Posted 6/10/2021

PCI Pharma Services Secures Strategic Investment to Expand Biotherapeutics Manufacturing Capabilities

  • PCI Pharma Services, a global CDMO specializing in biotherapies, received strategic investment co-led by Bain Capital and existing investor Kohlberg, with support from Mubadala Investment Company.
  • The Philadelphia-based company has achieved over 450 successful product launches in the past five years and provides integrated end-to-end drug development and manufacturing services.
  • Investment will fund organic and inorganic growth initiatives, including expansion of sterile fill-finish capabilities for injectables and high-potency specialized manufacturing capacity.
  • The strategic funding aims to strengthen US pharmaceutical manufacturing infrastructure while enabling faster market access for life-changing biopharmaceutical therapies.

Endometriosis Pain Treatment Pipeline Shows Mixed Results as NHS Approves First Daily Oral Therapy

Monoclonal AntibodyDrug Approval
  • The NHS in England has approved relugolix combination therapy as the first long-term daily oral treatment for endometriosis, expected to benefit around 1,000 women annually whose symptoms persist despite previous treatments.
  • Organon discontinued development of OG-6219 after the drug candidate failed its Phase 2 trial in July 2025, despite being acquired for $75 million and previously described as the company's "biggest potential opportunity."
  • Hope Medicine's HMI-115, a first-in-class monoclonal antibody targeting the prolactin receptor, reported positive interim Phase II results and received Breakthrough Therapy Designation from China's NMPA.
  • Multiple companies including TiumBio, Lisata Therapeutics, and Gynica are advancing novel therapeutic approaches through various clinical trial phases, representing diverse mechanisms of action for endometriosis pain management.

A Study of E7386 in Combination With Other Anticancer Drug(s) in Participants With Solid Tumor

NCT04008797RecruitingPhase 1
Eisai Inc.
Posted 7/11/2019

Uniquity Bio Appoints Will Kane as CEO to Lead Anti-TSLP Therapy Through Phase III Development

Monoclonal AntibodyLeadership Change
  • Uniquity Bio has appointed Will Kane as President and CEO to advance solrikitug, a monoclonal antibody targeting TSLP, through completion of three Phase II trials and preparation for Phase III development.
  • Kane brings over three decades of biopharmaceutical leadership experience from companies including Anthos Therapeutics, Karuna Therapeutics, and Pfizer, succeeding co-founder Brian Lortie who stepped down in June 2025.
  • Solrikitug is currently being evaluated in separate Phase II trials for asthma, chronic obstructive pulmonary disease, and eosinophilic esophagitis, with the company pursuing best-in-class efficacy and convenient dosing optimization.
  • The company is also developing bispecific candidates that combine solrikitug with other inflammatory targets to enhance efficacy and broaden patient populations across immunology and inflammation indications.

ESSA Pharma Agrees to $1.91 Per Share Acquisition by XenoTherapeutics in Wind-Down Transaction

  • ESSA Pharma Inc. has entered into a definitive agreement to be acquired by XenoTherapeutics Inc. for approximately $1.91 per share in cash as the company discontinues operations and winds down its prostate cancer drug development business.
  • The transaction is structured with XOMA Royalty Corporation providing financing and includes contingent value rights worth up to $0.06 per share, with shareholders also receiving an initial cash distribution prior to closing.
  • ESSA's board unanimously approved the deal following a comprehensive review, determining it maximizes shareholder value compared to liquidation while delivering more certain returns in an expedited timeframe.
  • The acquisition requires approval from 66⅔% of ESSA shareholders and securityholders, with closing expected in the second half of 2025 pending court approval and other customary conditions.

Vivos Inc. Submits FDA Application for RadioGel Hydrogel-Based Cancer Therapy

  • Vivos Inc. has submitted an Investigational Device Exemption (IDE) application to the FDA for RadioGel, a novel hydrogel-based radioactive therapy designed to deliver targeted radiation to solid tumors.
  • The comprehensive submission is supported by extensive animal and human data and was developed through months of collaboration with the FDA under the Breakthrough Device sprint process.
  • RadioGel enables precise radiation delivery to tumors while minimizing damage to surrounding healthy tissue, addressing life-threatening conditions with unmet medical needs.
  • The Breakthrough Device designation expedites development for devices that address critical medical conditions, bringing the company closer to delivering transformative cancer therapy.

ViiV Healthcare Expands Generic License for Long-Acting HIV Treatment to 133 Countries

  • ViiV Healthcare has expanded its voluntary licensing agreement with the Medicines Patent Pool to allow generic production of cabotegravir for HIV treatment in 133 countries, including all low-income and Sub-Saharan African nations.
  • The updated license enables three generic manufacturers—Aurobindo, Cipla, and Viatris—to develop and supply the long-acting injectable treatment for use with Johnson & Johnson's rilpivirine.
  • The World Health Organization recently recommended long-acting cabotegravir and rilpivirine as a treatment option for people who are virologically suppressed but struggle with adherence to daily oral regimens.
  • This expansion builds on ViiV's existing 2022 agreement covering cabotegravir for HIV prevention and represents the only approved long-acting injectable treatment for HIV.

Indiana University Receives $11.3 Million NIH Grant to Advance SHIP1 Inhibitor for Alzheimer's Disease

Drug Discovery
  • Indiana University School of Medicine researchers received a five-year, $11.3 million NIH grant to develop SHIP1 inhibitors targeting the INPP5D gene, which acts as a brake on microglia function in Alzheimer's disease.
  • The TREAT-AD drug discovery center has narrowed down hundreds of potential drug targets to five promising candidates, with SHIP1/INPP5D leading as the most advanced therapeutic target.
  • Researchers aim to optimize small molecules that block SHIP1 protein, potentially making microglia more effective at clearing harmful amyloid plaques in the brain.
  • The team will test inhibitors in Alzheimer's mouse models and develop biomarkers to track drug activity and effectiveness for future clinical trials in humans.

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