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Cardiol Therapeutics Secures $11M Funding to Advance Heart Disease Pipeline Following Breakthrough ARCHER Trial Results

Orphan Drug
  • Cardiol Therapeutics completed an $11 million private placement offering that extends the company's cash runway into Q3 2027 and fully funds the Phase III MAVERIC trial through FDA submission.
  • The ARCHER trial demonstrated significant reduction in left ventricular mass in myocarditis patients, marking the first evidence of structural and remodeling improvement in this condition.
  • The funding will accelerate development of next-generation therapy CRD-38 for heart failure, with partnership discussions advancing with leading pharmaceutical companies.
  • CardiolRx has received FDA Orphan Drug Designation for pericarditis treatment, with the company pursuing additional designation for myocarditis based on ARCHER findings.

CardiolRx in Recurrent Pericarditis Following IL-1 Blocker Cessation

NCT06708299RecruitingPhase 3
Cardiol Therapeutics Inc.
Posted 4/7/2025

Impact of CardiolRx on Myocardial Recovery in Patients With Acute Myocarditis

NCT05180240CompletedPhase 2
Cardiol Therapeutics Inc.
Posted 6/22/2022

Impact of CardiolRxTM on Recurrent Pericarditis (MAvERIC-Pilot)

NCT05494788CompletedPhase 2
Cardiol Therapeutics Inc.
Posted 11/30/2022

Esperion Advances ESP-2001 for Primary Sclerosing Cholangitis, Targeting $1 Billion Market Opportunity

Rare DiseaseOrphan Drug
  • Esperion has nominated ESP-2001, a highly-specific allosteric ATP citrate lyase inhibitor, as its preclinical development candidate for treating primary sclerosing cholangitis (PSC).
  • The company plans to file an Investigational New Drug application with the FDA in 2026 to initiate first-in-human clinical studies for this rare liver disease with no approved treatments.
  • ESP-2001 represents a potential blockbuster market opportunity exceeding $1 billion annually, targeting approximately 76,000 diagnosed PSC patients across the U.S. and Europe.
  • Preclinical studies demonstrate ESP-2001's ability to reduce markers of liver and bile duct injury, inflammation, and fibrosis associated with PSC pathogenesis.

US Orphan Drug Market Poised to Exceed $190 Billion by 2030 as FDA Designations Accelerate Rare Disease Therapy Development

Orphan DrugRare DiseaseDrug Approval
  • The US orphan drug market is projected to surpass $190 billion by 2030, driven by increasing demand for rare disease therapies and regulatory incentives.
  • More than 850 FDA-designated orphan drugs are currently in clinical trials, with over 500 already approved for clinical use in the United States.
  • Recent FDA orphan drug designations include Transpire Bio's TRB-ILD1 for idiopathic pulmonary fibrosis and TRB-ONC1 for glioblastoma, along with Minovia Therapeutics' MNV-201 for myelodysplastic syndrome.

UK's Innovative Licensing and Access Pathway Awards First Innovation Passports to Three Rare Disease Therapies

Rare DiseaseOrphan Drug
  • Three investigational therapies targeting rare diseases have become the first recipients of Innovation Passports under the UK's new Innovative Licensing and Access Pathway (ILAP), designed to accelerate promising medicines to NHS patients.
  • The selected therapies include treatments for neonatal onset ornithine transcarbamylase deficiency, Duchenne muscular dystrophy affecting 1 in 3,500-5,000 boys, and a rare pediatric neurodegenerative condition.
  • ILAP represents a world-first end-to-end pathway where healthcare developers, regulators, the NHS, and health technology assessment bodies collaborate from the earliest development stages.
  • The pathway addresses critical unmet needs in rare disease treatment, where 5,000-8,000 rare conditions collectively affect 3 million people in the UK but often lack effective therapies.

Innate Pharma Advances Lacutamab Toward Accelerated Approval for Rare T-Cell Lymphomas

Monoclonal AntibodyOrphan Drug
  • Innate Pharma will host an investor event on October 28, 2025, to discuss lacutamab's path to potential accelerated approval in Sézary syndrome based on Phase 2 TELLOMAK trial data.
  • The first-in-class anti-KIR3DL2 antibody has received FDA Fast Track, EMA PRIME, and Breakthrough Therapy designations for treating cutaneous T-cell lymphoma patients with high unmet medical needs.
  • Real-world claims data reveals a larger eligible CTCL patient population in the U.S. than previously reported, strengthening the commercial opportunity as the company prepares for Phase 3 initiation.

4DMT Secures $11 Million CF Foundation Investment to Advance Gene Therapy 4D-710 into Phase 2 for Cystic Fibrosis

Orphan Drug
  • The Cystic Fibrosis Foundation will provide up to $11 million in additional funding to 4D Molecular Therapeutics to accelerate development of 4D-710, a gene therapy for cystic fibrosis lung disease.
  • Phase 2 enrollment in the AEROW clinical trial is currently underway with 2.5E14 vg selected as the anticipated pivotal and commercial dose for the variant-agnostic genetic medicine.
  • The funding supports redosing studies and Phase 3 readiness, with interim Phase 1 data including functional durability results expected by year-end 2025.
  • 4D-710 represents the first known genetic medicine to demonstrate successful CFTR transgene delivery and expression throughout CF patient airways via aerosol delivery.

AnnJi's AJ201 Shows Clinical Promise for Kennedy's Disease in Phase 2a Trial

Orphan DrugRare Disease
  • AnnJi Pharmaceutical's Phase 2a clinical trial results for AJ201 in treating spinal and bulbar muscular atrophy (Kennedy's disease) were selected as a Late-Breaking Abstract at the World Muscle Society 2025 International Congress.
  • AJ201, an oral suspension containing the novel curcumin analog Rosolutamide, demonstrated positive functional outcomes in SBMA patients and represents a potential first-in-class therapy for the condition in two decades.
  • The drug has received Orphan Drug Designation from both the U.S. FDA and EMA, with AnnJi advancing toward Phase 2/3 clinical development for this rare neuromuscular disorder affecting approximately 1 in 40,000 males.

Regeneron's DB-OTO Gene Therapy Restores Hearing in 11 of 12 Children with Genetic Hearing Loss

Orphan DrugRare Disease
  • Regeneron's investigational gene therapy DB-OTO demonstrated clinically meaningful hearing improvements in 11 of 12 children with profound genetic hearing loss due to OTOF gene variants in the pivotal CHORD trial.
  • Three participants achieved normal hearing levels, with one able to identify words without visual cues and respond to distant sounds in noisy environments after treatment.
  • The company plans to submit a U.S. regulatory application later this year, with DB-OTO having received multiple FDA designations including Orphan Drug and Fast Track status.
  • Eight participants with longer follow-up showed stability or continued improvement in their hearing, representing a potential breakthrough for a condition previously considered permanent.

A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations

NCT05788536RecruitingPhase 1
Regeneron Pharmaceuticals
Posted 6/27/2023

Aardvark Therapeutics Expands Phase 3 HERO Trial for Prader-Willi Syndrome Treatment to Include Younger Patients

Rare DiseaseOrphan Drug
  • Aardvark Therapeutics received FDA alignment on a protocol amendment to expand its Phase 3 HERO trial of ARD-101 for Prader-Willi Syndrome, lowering the minimum age eligibility from 13 to 10 years old.
  • The expansion allows the company to reach a larger segment of the PWS patient population, with historical data suggesting younger patients are more likely to benefit from early intervention.
  • ARD-101 is a gut-restricted small molecule that activates bitter taste receptors to stimulate release of satiety hormones including GLP-1 and CCK, targeting the insatiable hunger characteristic of PWS.
  • The company expects topline data readout from this potentially pivotal trial in the third quarter of 2026.

FDA Authorizes Expanded Access Program for First-in-Class Tourette Syndrome Drug Ecopipam

Orphan Drug
  • The FDA has authorized Emalex Biosciences' Expanded Access Program for ecopipam, an investigational first-in-class treatment for Tourette syndrome that selectively blocks dopamine-1 receptors.
  • The program allows physicians to request access for eligible patients who have failed current FDA-approved treatments or experienced tolerability issues with existing therapies.
  • Emalex plans to submit a New Drug Application to the FDA in late 2025 for this novel therapeutic approach that differs from current treatments targeting dopamine-2 receptors.
  • Clinical studies in pediatric patients showed ecopipam was generally well tolerated, with the most common adverse events being headache, insomnia, fatigue, and anxiety.

Expanded Access Program for Treatment With Ecopipam for Tourette's Disorder

NCT07093541Unknown Status
Emalex Biosciences Inc.

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