Bitopertin

Disc Medicine Advances Bitopertin NDA for Erythropoietic Protoporphyria as Pipeline Progress Continues

14 days ago

• Disc Medicine has initiated the APOLLO confirmatory trial for bitopertin in erythropoietic protoporphyria (EPP) and remains on track to submit an NDA under accelerated approval pathway in the second half of 2025. • The company is advancing its hematologic disease pipeline with DISC-0974 studies in myelofibrosis and chronic kidney disease anemia, with data readouts expected in H2 2025. • Following a successful $259 million public offering in January, Disc Medicine reports a strong financial position with $695 million in cash, providing runway into 2028.

FDA Aligns with Gemini Therapeutics on APOLLO Trial Design for Bitopertin in Rare Porphyria Disorders

4 months ago

• Gemini Therapeutics has successfully concluded a Type C meeting with the FDA, reaching agreement on the design of APOLLO confirmatory trial for bitopertin in EPP and XLP treatment. • The planned APOLLO trial will enroll approximately 150 patients across multiple continents in a randomized, double-blind, placebo-controlled study with a 1:1 allocation ratio. • The company aims to submit a New Drug Application for bitopertin under the Accelerated Approval Program in the second half of 2025, with trial initiation planned for mid-2025.

Boehringer Ingelheim's Iclepertin Fails to Meet Endpoints in Phase III Schizophrenia Trials

4 months ago

• Boehringer Ingelheim's iclepertin, a GlyT1 inhibitor, failed to demonstrate statistically significant benefits in improving cognitive function in schizophrenia patients. • The Phase III CONNEX program, involving over 1,800 patients across 41 countries, did not meet primary or key secondary endpoints at six months. • As a result of the disappointing outcomes, Boehringer Ingelheim has discontinued the CONNEX-X long-term extension trial. • The company remains committed to developing solutions for serious mental illnesses, with over 20 investigative therapies in its pipeline.

Boehringer Ingelheim Expands Schizophrenia Pipeline with Sosei Heptares and Autifony Collaborations

4 months ago

• Boehringer Ingelheim licenses GPR52 agonists from Sosei Heptares for €755m, targeting a range of schizophrenia symptoms including positive, negative, and cognitive dysfunction. • The collaboration with Autifony Therapeutics focuses on Kv3.1/3.2 positive modulators, with Boehringer funding R&D and holding an option to purchase AUT00206, currently in Phase 1b. • These deals complement Boehringer's existing schizophrenia pipeline, including iclepertin (GlyT1 inhibitor) in Phase 3 and a digital therapeutic (DTx) in late-stage testing.

Disc Medicine Advances Hematology Pipeline with Bitopertin NDA Plans and Phase 2 Trials

4 months ago

• Disc Medicine is preparing for an NDA submission for bitopertin in erythropoietic protoporphyria (EPP) following positive FDA feedback, potentially leading to accelerated approval. • Phase 2 trials for DISC-0974 in anemia of myelofibrosis (MF) and chronic kidney disease (CKD) are progressing, with initial data expected in the second half of 2025. • A Phase 2 study of DISC-3405 in polycythemia vera (PV) is set to begin in H1 2025, expanding Disc Medicine's portfolio in hematologic diseases. • Disc Medicine's strengthened financial position, with a cash runway into 2027, supports the advancement of its clinical programs and strategic objectives.

Disc Medicine Secures $200M to Advance Clinical Trials of Hematologic Disease Therapies

6 months ago

• Disc Medicine has obtained a $200 million non-dilutive term loan to support its clinical trials and enhance financial flexibility. • The funding will facilitate a confirmatory study of bitopertin for erythropoietic protoporphyria (EPP) and Phase II trials for DISC-0974 and DISC-3405 in anemia. • A multiple-dose trial in anemia of non-dialysis dependent chronic kidney disease (NDD-CKD) is also planned with the secured financing. • The company anticipates initiating a confirmatory trial of bitopertin in EPP by mid-2025, supported by this financial boost.

Disc Medicine's Bitopertin Poised for Potential Accelerated FDA Approval in Erythropoietic Protoporphyria

7 months ago

• Disc Medicine's bitopertin may receive accelerated FDA approval for erythropoietic protoporphyria (EPP) treatment based on existing clinical data. • The FDA has agreed to the design of Disc's Phase III APOLLO trial, which will serve as a confirmatory study for bitopertin. • Bitopertin aims to reduce protoporphyrin IX (PPIX) levels, potentially alleviating pain and burns from sun exposure in EPP and XLP patients. • The Phase III APOLLO trial is set to begin in mid-2025 and will assess bitopertin's safety and efficacy in patients aged 12 and older.

Disc Medicine's Bitopertin Gains Momentum with Positive Regulatory Feedback and Analyst Upgrades

7 months ago

• Disc Medicine received positive feedback from the FDA regarding bitopertin for erythropoietic protoporphyria (EPP), potentially allowing for accelerated approval based on PPIX reduction. • Analyst ratings for Disc Medicine have been upgraded to 'Strong Buy' by Raymond James, with a price target of $110, citing the accelerated timeline for bitopertin's launch. • The company is expected to submit a New Drug Application (NDA) for bitopertin in the second half of 2025, with potential approval and commercial launch anticipated in 2026. • Phase 1b study of DISC-0974 showed significant reduction in hepcidin levels and improvement in iron mobilization and hemoglobin levels in patients with NDD-CKD and anemia.

Disc Medicine Gains FDA Alignment on Bitopertin Trial Design for Erythropoietic Protoporphyria

7 months ago

• Disc Medicine's Type C meeting with the FDA resulted in alignment on the design of the APOLLO post-marketing confirmatory trial for bitopertin in EPP. • The APOLLO trial will assess bitopertin's efficacy using co-primary endpoints focusing on sunlight tolerance and PPIX reduction over a 6-month treatment period. • The FDA agreed to the potential for accelerated approval based on existing data and utilizing reduction of PPIX as a surrogate endpoint. • Disc Medicine plans to initiate the APOLLO trial by mid-2025, studying a 60 mg dose of bitopertin in EPP and XLP patients aged 12 and older.

Disc Medicine Announces End-of-Phase 2 Meeting with FDA for Bitopertin in Erythropoietic Protoporphyria

7 months ago

• Disc Medicine will host a conference call on November 4 to discuss feedback from the FDA regarding the end-of-Phase 2 meeting for bitopertin. • Bitopertin is an investigational, orally administered inhibitor of GlyT1, designed to modulate heme biosynthesis for hematologic diseases. • The company is developing bitopertin as a potential disease-modifying therapy for erythropoietic porphyrias, including EPP and XLP. • Current treatments for EPP and XLP are limited to managing symptoms, highlighting the need for new therapies like bitopertin.

Patient-Researcher's Journey Leads to Breakthrough Treatment for Rare Light Sensitivity Disorder

7 months ago

• Dr. Jasmin Barman-Aksözen's personal experience with erythropoietic protoporphyria (EPP) led her to contribute to the development and approval of Scenesse, the first treatment for this rare genetic disorder. • Clinical trials demonstrated Scenesse allows EPP patients to spend up to 20 additional minutes in sunlight daily without pain, with some achieving three hours of sun exposure while using the treatment. • The drug's approval marked a historic milestone as the first treatment approved through EMA's patient experience pilot program, though limitations remain and new therapeutic candidates are under development.

Disc Medicine Presents Positive Clinical Data Across Portfolio

a year ago

Disc Medicine announced positive clinical data from its portfolio, including bitopertin for erythropoietic protoporphyria (EPP), DISC-0974 for myelofibrosis (MF) anemia, and DISC-3405 in healthy volunteers, showcasing significant advancements in treatment options for hematologic diseases.

Boehringer Ingelheim Plans 25 New Treatments by 2030, Bolstered by Oncology and Metabolic Disease Programs

a year ago

• Boehringer Ingelheim aims to launch 25 new treatments by 2030, focusing on oncology, mental health, cardiovascular, renal, and metabolic diseases. • Key candidates include Brigimadlin, an MDM2-p53 agonist for dedifferentiated liposarcoma, and zongertinib, a HER2 tyrosine kinase inhibitor, showing promising early data. • Survodutide, a glucagon/GLP-1 candidate, demonstrated significant improvement in liver disease due to metabolic dysfunction-associated steatohepatitis in Phase II trials. • Despite slow sales of its Humira biosimilar, Cyltezo, Boehringer reported a 9.7% net sales increase in 2023, driven by Jardiance and Ofev.

Drug Development Updates