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Schrödinger's SGR-1505 Receives FDA Fast Track Designation for Relapsed Waldenström Macroglobulinemia

Orphan Drug
  • Schrödinger's MALT1 inhibitor SGR-1505 received FDA Fast Track designation for treating adult patients with Waldenström macroglobulinemia who have failed at least two lines of therapy, including a BTK inhibitor.
  • The designation addresses significant unmet medical need as treatment failure and disease progression due to BTK resistance remains a challenge for a growing number of patients with hematologic malignancies.
  • Phase 1 clinical data presented at recent conferences showed SGR-1505 has a favorable safety profile and encouraging preliminary efficacy across multiple B-cell malignancy subtypes.
  • The company plans to discuss Phase 1 study results and recommended Phase 2 dose with the FDA later this year, with potential for accelerated approval pathways.

Study of SGR-1505 in Mature B-Cell Neoplasms

NCT05544019RecruitingPhase 1
Schrödinger, Inc.
Posted 4/10/2023

First European Patient Receives Breakthrough Treatment for Ultra-Rare APDS Immune Disorder

Rare DiseaseTargeted TherapyDrug ApprovalOrphan Drug
  • Mary Catchpole, 19, becomes the first European patient to receive leniolisib (Joenja), a newly approved targeted treatment for activated PI3-kinase delta syndrome (APDS), a rare inherited immune disorder.
  • The drug works by inhibiting an overactive enzyme that disrupts immune function, offering a potential cure for patients who previously faced lifelong infections and invasive treatments.
  • APDS was discovered by Cambridge researchers in 2013 with help from Catchpole's family, who lost four members to the condition before this breakthrough treatment became available.
  • The NHS approved leniolisib at a list price of £352,000 per year with a confidential discount, potentially benefiting up to 50 patients over age 12 in England.

QBiotics' Tigilanol Tiglate Achieves 80% Response Rate in Soft Tissue Sarcoma Phase IIa Trial

Orphan Drug
  • QBiotics reported an 80% objective response rate in Stage 1 of its Phase IIa trial evaluating tigilanol tiglate in 10 patients with advanced soft tissue sarcoma.
  • Of 27 injected tumors, 81% showed complete or partial ablation, with 14 tumors achieving complete destruction and none recurring at 6-month follow-up.
  • The small molecule drug was well tolerated with mostly localized adverse events, supporting progression to Stage 2 expansion at Memorial Sloan Kettering Cancer Center.
  • Three patients demonstrated enhanced responses to subsequent systemic therapies, suggesting potential synergistic effects with standard treatments.

A Clinical Study to Investigate the Efficacy of Intratumoral Tigilanol Tiglate in Soft Tissue Sarcoma

NCT05755113RecruitingPhase 2
QBiotics Group Limited
Posted 4/13/2023

Sterotherapeutics Doses First Patient in Phase 2 Trial of ST-002 for Cushing's Syndrome

Orphan DrugRare Disease
  • Sterotherapeutics has successfully dosed the first patient in its Phase 2 clinical trial evaluating ST-002 for Cushing's Syndrome treatment.
  • The multicenter European study aims to assess safety, efficacy, and tolerability of ST-002 in patients with this rare endocrine condition caused by chronic excess cortisol exposure.
  • ST-002 has received FDA Orphan Drug Designation and addresses a significant unmet medical need, as no universally effective treatment currently exists for Cushing's Syndrome.
  • The trial will evaluate complications including diabetes and MASLD using magnetic resonance imaging and targeted measurements across multiple European sites.

Nuvalent to Present Pivotal Data for ROS1-Selective Inhibitor Zidesamtinib in Advanced Lung Cancer

Orphan Drug
  • Nuvalent will host a webcast on June 24, 2025, to discuss pivotal data for zidesamtinib in TKI pre-treated patients with advanced ROS1-positive non-small cell lung cancer from the ARROS-1 Phase 1/2 trial.
  • Zidesamtinib is a novel brain-penetrant ROS1-selective inhibitor designed to overcome resistance to current ROS1 inhibitors and address brain metastases in cancer patients.
  • The drug has received breakthrough therapy designation for ROS1-positive metastatic NSCLC patients previously treated with 2 or more ROS1 tyrosine kinase inhibitors.
  • The ARROS-1 trial's Phase 2 portion is designed with registrational intent for both TKI-naïve and TKI pre-treated patients with ROS1-positive NSCLC.

A Study of NVL-520 in Patients With Advanced NSCLC and Other Solid Tumors Harboring ROS1 Rearrangement (ARROS-1)

NCT05118789RecruitingPhase 1
Nuvalent Inc.
Posted 1/4/2022

A Study of NVL-655 in Patients With Advanced NSCLC and Other Solid Tumors Harboring ALK Rearrangement or Activating ALK Mutation (ALKOVE-1)

NCT05384626RecruitingPhase 1
Nuvalent Inc.
Posted 6/9/2022

Neladalkib (NVL-655) for TKI-naive Patients With Advanced ALK-Positive NSCLC

NCT06765109RecruitingPhase 3
Nuvalent Inc.
Posted 3/1/2025

Arcturus Therapeutics to Present Phase 2 Data for ARCT-810 mRNA Therapy in Rare Genetic Disorder

Rare DiseaseOrphan Drug
  • Arcturus Therapeutics will host a virtual presentation on June 30, 2025, featuring Phase 2 interim data for ARCT-810, an investigational mRNA therapeutic for ornithine transcarbamylase deficiency.
  • The presentation will review safety and pharmacodynamics data from multiple doses of ARCT-810 in adolescent and adult participants with OTC deficiency, the most common urea cycle disorder.
  • Leading experts Dr. Marshall Summar and Dr. Johannes Häberle will participate in the presentation, bringing decades of expertise in urea cycle disorders and metabolic medicine.
  • ARCT-810 has received multiple regulatory designations including Orphan Drug status and Fast Track designation from the FDA for treating this rare genetic condition affecting approximately 10,000 people in Europe and the U.S.

Cumberland Pharmaceuticals Reports Positive Phase 2 Results for Ifetroban in Duchenne Muscular Dystrophy Heart Disease

Rare DiseaseOrphan Drug
  • Cumberland Pharmaceuticals' Phase 2 FIGHT DMD trial demonstrated that high-dose ifetroban treatment resulted in a significant 5.4% improvement in left ventricular ejection fraction compared to control groups.
  • The study showed reduced blood levels of cardiac damage markers (NT-proBNP and cardiac troponin I) in patients receiving ifetroban, while these markers increased in placebo-treated patients.
  • Ifetroban represents a potential breakthrough for DMD heart disease, which is the leading cause of death in DMD patients and currently has no approved targeted treatments.
  • All patients who completed the 12-month study opted to continue with the open-label extension, demonstrating confidence in the treatment approach.

Oral Ifetroban in Subjects with Duchenne Muscular Dystrophy

NCT03340675Active, Not RecruitingPhase 2
Cumberland Pharmaceuticals
Posted 10/19/2020

Novadip Receives FDA RMAT Designation for NVD003 Regenerative Therapy in Rare Pediatric Bone Disease

Orphan DrugRare Disease
  • Novadip Biosciences received FDA Regenerative Medicine Advanced Therapy (RMAT) designation for NVD003, an autologous stem cell therapy for congenital pseudarthosis of the tibia (CPT).
  • Clinical data showed 88% of patients achieved fracture healing with NVD003, including children whose prior surgeries had failed.
  • The company plans to launch a pivotal phase 3 trial this month with recruitment in the US and Europe, targeting market entry by 2027.
  • NVD003 represents potential peak sales of $1.4 billion for large bone defects in pediatric and adult patients.

NVD-003 in the Treatment of Congenital Pseudarthrosis of the Tibia

NCT05693558Active, Not RecruitingPhase 1
Novadip Biosciences
Posted 11/24/2022

FDA Approves First Treatment for Alkaptonuria: Nitisinone Shows Clinical Benefits in Rare Metabolic Disorder

Drug ApprovalRare DiseaseOrphan Drug
  • The FDA approved nitisinone (Harliku) as the first and only treatment for alkaptonuria, a rare genetic metabolic disorder affecting 1 in 250,000 to 1 million Americans.
  • A 3-year randomized clinical trial of 40 patients demonstrated that nitisinone improved pain, energy levels, and physical functioning compared to untreated controls.
  • Alkaptonuria causes buildup of homogentisic acid leading to joint problems, ochronosis, and complications in kidneys and heart, with the medication expected to launch in July 2025.
  • The approval represents a significant breakthrough for patients who previously had no approved therapeutic options for this debilitating condition.

Long-Term Study of Nitisinone to Treat Alkaptonuria

NCT00107783CompletedPhase 2
National Human Genome Research Institute (NHGRI)
Posted 1/1/2005

ExCellThera's Zemcelpro Receives Positive CHMP Opinion for Blood Cancer Patients Without Suitable Donors

Orphan Drug
  • The European Medicines Agency's CHMP has recommended conditional marketing authorization for Zemcelpro, a novel stem cell therapy for blood cancer patients lacking suitable donor cells.
  • If approved, Zemcelpro would become the first and only EU-authorized therapy for adults with haematological malignancies requiring stem cell transplantation when no suitable donor cells are available.
  • The therapy addresses a significant unmet need, as over 10,000 new cases of patients with blood cancers requiring bone marrow transplant occur annually in Europe.
  • European Commission approval is expected within two months, with additional regulatory filings planned for the US, Canada, UK, and Switzerland.

Expanded Cord Blood in Patients in Need of an Allogeneic Stem Cell Transplant

NCT02668315CompletedPhase 1
Maisonneuve-Rosemont Hospital
Posted 12/16/2015

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