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Clinical Trial News

FDA Grants Priority Review to Omidubicel for Severe Aplastic Anemia Treatment

Rare Disease
  • The FDA has accepted Ayrmid Ltd.'s priority review application for omidubicel as a treatment for severe aplastic anemia, with a PDUFA target action date of December 10, 2025.
  • Omidubicel is a nicotinamide modified allogeneic hematopoietic progenitor cell therapy derived from umbilical cord blood, already approved under the brand name Omisirge for hematologic malignancies.
  • Severe aplastic anemia affects 2-3 individuals per million and represents a significant unmet medical need for patients without matched sibling donors for transplantation.
  • The supplementary Biologics License Application is based on results from an investigator-sponsored study conducted at the National Heart, Lung, and Blood Institute of the National Institutes of Health.

Arnatar Therapeutics Emerges from Stealth with FDA Orphan Drug Designation for ART4 in Alagille Syndrome

Rare Disease
  • Arnatar Therapeutics has emerged from stealth mode and received FDA Orphan Drug and Rare Pediatric Disease designations for ART4, an antisense oligonucleotide therapy targeting Alagille Syndrome.
  • ART4 addresses the root cause of Alagille Syndrome by upregulating JAG1 protein expression through Arnatar's proprietary ACT-UP1 platform, with preclinical studies showing restored bile duct development and reduced liver injury markers.
  • The company completed a $52 million Series A financing round in 2024 to advance its DARGER platform, which uniquely combines siRNA gene silencing with upregulating antisense oligonucleotides.
  • Alagille Syndrome affects approximately 95% of cases through JAG1 gene mutations, causing life-threatening liver damage and systemic complications with no current curative treatment options.

Danforth Advisors Acquires PharmaDirections to Create Comprehensive Drug Development Platform

  • Danforth Advisors has acquired PharmaDirections, a pioneer of the virtual drug development model founded in 2003, bringing over 150 specialists to expand integrated capabilities from discovery through commercialization.
  • The acquisition enables Danforth to provide comprehensive drug development expertise across all stages, covering discovery research, non-clinical development, manufacturing, clinical operations, regulatory strategy, and medical writing.
  • PharmaDirections represents Danforth's sixth acquisition since 2022, following previous acquisitions of Elite BioPharma Consulting and Advyzom, as part of a strategic expansion to serve over 1,500 life science companies globally.
  • The combined platform aims to help biotech and pharmaceutical companies overcome complex drug development challenges while accelerating the delivery of new therapies to patients.

BriaCell Receives $2 Million NCI Grant to Advance Personalized Prostate Cancer Immunotherapy

  • BriaCell Therapeutics has been awarded a $2,054,651 non-dilutive grant from the National Cancer Institute to advance Bria-PROS+, a next-generation personalized off-the-shelf immunotherapy for prostate cancer.
  • The grant will fund manufacturing of clinical supplies and support a Phase 1/2a trial in patients with metastatic prostate cancer, led by Dr. William Oh at Yale Cancer Center.
  • The Bria-OTS+ platform is designed to overcome the complexity, cost, and delays of traditional personalized cancer treatments while addressing the urgent unmet medical need in advanced prostate cancer.
  • Despite recent advances in metastatic hormone-sensitive prostate cancer management, most patients still progress with significant pain and limited treatment options.

FDA Issues Not-Approvable Letter for enVVeno Medical's VenoValve, Shares Plummet 76%

  • enVVeno Medical's shares crashed 76.1% following the FDA's not-approvable letter for VenoValve, a surgical replacement venous valve for severe chronic venous insufficiency.
  • The FDA determined that data from the pivotal SAVVE trial was insufficient to establish a favorable benefit-to-risk profile for the device.
  • The regulatory setback affects the company's most advanced pipeline candidate and delays potential commercialization, impacting revenue generation prospects.
  • enVVeno Medical is reviewing FDA feedback and may pursue resubmission or appeal while advancing its non-surgical enVVe valve candidate.

Vaxxas Secures A$90 Million to Advance Needle-Free Vaccine Delivery Technology Toward Market

  • Vaxxas raised approximately A$90 million in Series D funding led by SPRIM Global Investments to accelerate commercialization of its HD-MAP needle-free vaccine delivery platform.
  • The HD-MAP technology has demonstrated safety and tolerability in over 750 clinical trial participants while achieving equal or greater immune responses at lower doses compared to traditional injections.
  • Funding will support scale-up of semi-automated manufacturing lines and later-stage clinical trials, with the company targeting market readiness by the second half of 2027.
  • CEO David Hoey will transition to Strategic Advisor after 14 years of leadership, as the company searches for new leadership to drive commercialization efforts.

Oryzon Receives EMA Approval for First LSD1 Inhibitor Trial in Sickle Cell Disease

Orphan Drug
  • Oryzon Genomics received European Medicines Agency approval to initiate the RESTORE Phase Ib trial of iadademstat in sickle cell disease, marking the first clinical investigation of an LSD1 inhibitor in this indication.
  • The study will enroll 40 adult patients across multiple Spanish sites to evaluate safety, tolerability, and the drug's ability to induce fetal hemoglobin production.
  • Preclinical data showed iadademstat significantly increased fetal hemoglobin levels in baboons after a single dose, targeting a clinically meaningful endpoint recognized by the FDA.
  • The sickle cell disease treatment market is projected to grow from approximately $3 billion in 2025 to around $8 billion by 2032, highlighting significant commercial potential.

Theravance Biopharma Completes Phase 3 Enrollment for Ampreloxetine in Rare Neurological Disorder

Orphan DrugRare Disease
  • Theravance Biopharma has completed enrollment in the pivotal Phase 3 CYPRESS study of ampreloxetine for neurogenic orthostatic hypotension in multiple system atrophy patients.
  • The investigational once-daily norepinephrine reuptake inhibitor could become the first therapy to provide durable benefit for 40,000 U.S. patients with this devastating condition.
  • Topline results are expected in Q1 2026, with plans for expedited NDA submission and priority FDA review if data prove supportive.
  • Previous Study 0170 showed a 72% reduction in treatment failure odds for MSA patients, demonstrating compelling symptom improvement without worsening supine hypertension.

Phase 3 Efficacy and Durability of Ampreloxetine for the Treatment of Symptomatic NOH in Participants with Multiple System Atrophy

NCT05696717RecruitingPhase 3
Theravance Biopharma
Posted 6/27/2023

Clinical Effect of Ampreloxetine (TD-9855) for Treating Symptomatic nOH in Subjects With Primary Autonomic Failure

NCT03750552CompletedPhase 3
Theravance Biopharma
Posted 1/24/2019

Phase 3 Clinical Effect Durability of TD-9855 for Treating Symptomatic nOH in Subjects With Primary Autonomic Failure

NCT03829657TerminatedPhase 3
Theravance Biopharma
Posted 2/22/2019

Incyclix Bio Raises $11.25 Million to Advance CDK2 Inhibitor INX-315 for Treatment-Resistant Breast and Ovarian Cancers

Precision Medicine
  • Incyclix Bio secured $11.25 million in Series B extension funding from investors including Eli Lilly and Company, Eshelman Ventures, and Pharmacosmos to advance its Phase 1/2 clinical trial.
  • The funding will support completion of the ongoing INX-315-01 trial evaluating the novel CDK2 inhibitor in patients with CDK4/6 inhibitor-resistant ER+/HER2- breast cancer and CCNE1-amplified solid tumors.
  • INX-315 represents a precision treatment approach targeting aberrant cell cycle proliferation in advanced and treatment-resistant cancers, with study completion expected by mid-2026.
  • The company has already presented interim clinical data from the dose escalation portion at the 2024 San Antonio Breast Cancer Symposium.

Open-Label Study to Evaluate the Safety, Tolerability, PK, and Efficacy of INX-315 in Patients With Advanced Cancer

NCT05735080RecruitingPhase 1
Incyclix Bio
Posted 3/28/2023

ProVerum Raises $80 Million Series B to Commercialize Minimally Invasive BPH Treatment

  • ProVerum Limited secured $80 million in Series B financing led by MVM Partners to advance commercialization of its ProVee System for treating benign prostatic hyperplasia.
  • The ProVee System utilizes a nitinol stent deployed through a flexible delivery system to reshape enlarged prostates and alleviate lower urinary tract symptoms.
  • New investors OrbiMed and Ireland Strategic Investment Fund joined the round alongside existing backers including Gilde Healthcare Partners and Lightstone Ventures.
  • The minimally invasive device is deployed using the same size endoscope routinely used for BPH diagnosis, potentially enabling office-based treatment procedures.

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