Gene family

Interim Management Statement Q1 2024 of Molecular Partners: Pipeline Progressing with Two Additional Programs to Enter the Clinic in 2025, Update to MP0533 Program

Retrieved on: 
Donnerstag, Mai 16, 2024

For our emerging pipeline, we plan to announce preclinical data from our Switch-DARPin Platform at EHA and anticipate translational efficacy data in the second half of 2024.

Key Points: 
  • For our emerging pipeline, we plan to announce preclinical data from our Switch-DARPin Platform at EHA and anticipate translational efficacy data in the second half of 2024.
  • Of this figure, approximately CHF 8 million will be non-cash effective costs for share-based payments, IFRS pension accounting and depreciation.
  • With CHF 174.1 million in cash and short-term time deposits and no debt as of March 31, 2024, the Company expects to be funded well into 2026.
  • The first Switch-DARPin program (cKIT x CD16a x CD47) was introduced at the annual J.P. Morgan Healthcare Conference in January 2024.

Capricor Therapeutics to Present Exosome Platform Updates at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting

Retrieved on: 
Donnerstag, Mai 9, 2024

SAN DIEGO, May 09, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced that an abstract featuring new preclinical data highlighting the therapeutic potential of its StealthX™ exosome platform technology has been selected for an oral presentation at the ASGCT 27th Annual Meeting taking place in Baltimore, Maryland from May 7–11, 2024. The findings highlight a potential exosome-based approach for the treatment of arginase-1 deficiency (ARG1-D), a rare genetic metabolic disease characterized by complete or partial lack of the enzyme arginase in the liver and red blood cells.

Key Points: 
  • The findings highlight a potential exosome-based approach for the treatment of arginase-1 deficiency (ARG1-D), a rare genetic metabolic disease characterized by complete or partial lack of the enzyme arginase in the liver and red blood cells.
  • “The findings from this preclinical study have further characterized our StealthX™ exosome platform and provide a novel approach for exosome-based enzyme-replacement therapies which aim to replace a deficient or absent enzyme,” said Linda Marbán, Ph.D., Capricor’s chief executive officer.
  • “We believe our StealthX™ platform provides an opportunity with potentially broad applications and our data continues to support the concept of exosomes as a suitable delivery vehicle for a variety of payloads.
  • Results showed the Arg1-exosomes were enzymatically active and able to convert arginine into urea in vitro.

Molecular Partners Reports Corporate Highlights From Q4 2023 and Key Financials for Full Year 2023

Retrieved on: 
Donnerstag, März 14, 2024

ZURICH-SCHLIEREN, Switzerland and CONCORD, Mass., March 14, 2024 (GLOBE NEWSWIRE) -- Ad hoc announcement pursuant to Art. 53 LR Molecular Partners AG (SIX: MOLN; NASDAQ: MOLN), a clinical-stage biotech company developing a new class of custom-built protein drugs known as DARPin therapeutics, today announced its corporate highlights and audited financial results for the full year 2023.

Key Points: 
  • 53 LR Molecular Partners AG (SIX: MOLN; NASDAQ: MOLN), a clinical-stage biotech company developing a new class of custom-built protein drugs known as DARPin therapeutics, today announced its corporate highlights and audited financial results for the full year 2023.
  • “2023 was a year of successful innovation and execution on our strategy, focusing on novel mechanisms that we believe only DARPin therapies can deliver.
  • In addition to the above updates, Molecular Partners continued to progress its RDT portfolio of projects in partnership with Novartis.
  • In the financial year 2023, Molecular Partners recognized total revenues and other income of CHF 7.0 million (2022: CHF 189.6 million) and incurred total expenses of CHF 68.1 million (2022: CHF 73.0 million).

Cogent Biosciences Announces Planned 2024 Milestones for Bezuclastinib and Emerging Portfolio of Selective and Potent Targeted Therapeutics

Retrieved on: 
Dienstag, Januar 9, 2024

ET

Key Points: 
  • ET
    WALTHAM, Mass.
  • and BOULDER, Colo., Jan. 09, 2024 (GLOBE NEWSWIRE) -- Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, today highlighted the company’s key 2024 milestones ahead of its presentation at J.P. Morgan’s 42nd annual healthcare conference.
  • “Given the foundation that was established in 2023, we are well positioned to move Cogent forward aggressively this year, including a cash runway expected to carry us into 2026,” said Andrew Robbins, President and CEO of Cogent Biosciences.
  • A live webcast will be accessible in the “Investors & Media” section of the company’s website, www.cogentbio.com , and will be archived for 30 days following the event.

Molecular Partners Provides Updates at 42nd Annual J.P. Morgan Healthcare Conference

Retrieved on: 
Sonntag, Januar 7, 2024

53 LR Molecular Partners AG (SIX: MOLN; NASDAQ: MOLN), a clinical-stage biotech company developing a new class of custom-built protein drugs known as DARPin therapeutics, today announced it will present a business overview and provide its 2024 outlook at the 42nd Annual J.P. Morgan Healthcare Conference.

Key Points: 
  • 53 LR Molecular Partners AG (SIX: MOLN; NASDAQ: MOLN), a clinical-stage biotech company developing a new class of custom-built protein drugs known as DARPin therapeutics, today announced it will present a business overview and provide its 2024 outlook at the 42nd Annual J.P. Morgan Healthcare Conference.
  • Data were presented at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition in December 2023.
  • Molecular Partners continues to progress its RDT platform and portfolio of projects, both in-house and in partnership with Novartis.
  • In addition to these updates, Novartis has returned the rights to the ensovibep program, previously under investigation for the treatment of COVID-19, to Molecular Partners.

Biomea Fusion Announces Two Poster Presentations at Upcoming ASH Annual Meeting 2023

Retrieved on: 
Donnerstag, November 2, 2023

Both BMF-219 and BMF-500 were originated in-house with Biomea’s proprietary FUSION™ system platform, which discovers and designs next-generation covalent-binding small molecule product candidates.

Key Points: 
  • Both BMF-219 and BMF-500 were originated in-house with Biomea’s proprietary FUSION™ system platform, which discovers and designs next-generation covalent-binding small molecule product candidates.
  • Methods: Doses of BMF-219 are escalated independently for each indication, initially in single-subject cohorts followed by a “3 + 3” design.
  • A subsequent amendment introduced quotas for KMT2Ar (MLL1r), NPM1 and other known menin-dependent mutations: CEBP/A, MLL1-PTD, MN1, NUP98, NUP214, PICALM-AF10, SETBP1.
  • The study was initiated in July 2023 and will enroll ~110 participants at approximately 30 sites.

Biomea Fusion Announces First Patient Dosed with Covalent FLT3 Inhibitor BMF-500 in Relapsed or Refractory Acute Leukemia in Phase I Clinical Trial (COVALENT-103)

Retrieved on: 
Dienstag, Oktober 17, 2023

REDWOOD CITY, Calif., Oct. 17, 2023 (GLOBE NEWSWIRE) -- Biomea Fusion, Inc. (“Biomea” or “the company”) (Nasdaq: BMEA), a clinical-stage biopharmaceutical company dedicated to discovering and developing novel covalent small molecules to treat and improve the lives of patients with genetically defined cancers and metabolic diseases, today announced that the first patient has been dosed in COVALENT-103, the company’s Phase I trial of BMF-500, an investigational covalent FLT3 inhibitor developed using the proprietary FUSIONTM System, in adult patients with relapsed or refractory acute leukemia.

Key Points: 
  • “Despite several late stage and approved therapies targeting FLT3, the majority of patients with AML harboring FLT3 mutations have not achieved durable remissions.
  • We believe that BMF-500, which is designed to have key attributes of covalency, potency, and selectivity, has the capacity to fully extinguish FLT3-driven disease and the potential to combine with other targeted therapies and standard-of-care agents,” said Steve Morris, MD, Biomea’s Chief Development Officer.
  • “Today, we have taken another important step to bring novel covalent drug candidates into the clinic by exploring the potential of BMF-500, an investigational covalent FLT3 inhibitor, in treating adult patients with relapsed or refractory acute leukemia.
  • BMF-500 is designed to be a potent molecule and is the second covalent inhibitor we have developed in-house, demonstrating the potential of the FUSIONTM platform and our team in bringing novel assets to the clinic.

Aptose Presents Highlights from Clinical Update

Retrieved on: 
Samstag, Juni 10, 2023

SAN DIEGO and TORONTO, June 10, 2023 (GLOBE NEWSWIRE) -- Aptose Biosciences Inc. (“Aptose” or the “Company”) (NASDAQ: APTO, TSX: APS), a clinical-stage precision oncology company developing highly differentiated oral kinase inhibitors to treat hematologic malignancies, today released highlights from a clinical update event held today, June 10, 2023, in conjunction with EHA 2023 International Congress of the European Hematology Association in Frankfurt, Germany. The event included an up-to-date review of clinical data for Aptose’s two investigational products under development for hematologic malignancies: tuspetinib, an oral, myeloid kinase inhibitor in the Phase 1/2 APTIVATE trial in patients with relapsed or refractory acute myeloid leukemia (AML); and luxeptinib, an oral, dual lymphoid and myeloid kinase inhibitor in Phase 1 a/b stage development for the treatment of patients with relapsed or refractory hematologic malignancies. The webcast of the presentation is available on Aptose’s website here.

Key Points: 
  • The webcast of the presentation is available on Aptose’s website here .
  • Aptose provided updated clinical findings with tuspetinib, a potent suppressor of FLT3, SYK, JAK 1/2, mutant forms of cKIT, and the RSK1/2 kinases operative in AML:
    Completed tuspetinib dose escalation and dose exploration Phase 1/2 trial in 77 R/R AML patients.
  • Tuspetinib is being administered as a monotherapy and as a combination doublet with tuspetinib + venetoclax (TUS/VEN), and enrollment has been brisk.
  • 50mg G3 formulation with continuous dosing achieves roughly equivalent PK profile as 900mg original G1 formulation.

Biomea Fusion to Present Preclinical Data for BMF-500, an Investigational Oral Covalent FLT3 Inhibitor, at the 64th American Society of Hematology (ASH) Annual Meeting

Retrieved on: 
Donnerstag, November 3, 2022

We believe the preclinical data we will present at ASH has the potential to establish BMF-500 as the most potent and selective FLT3 inhibitor reported to date.

Key Points: 
  • We believe the preclinical data we will present at ASH has the potential to establish BMF-500 as the most potent and selective FLT3 inhibitor reported to date.
  • The potent covalent inhibition of FLT3 by BMF-500 manifested in effective and durable cellular response that was improved over gilteritinib.
  • In cells harboring FLT3 activating mutations, BMF-500 induced dose-dependent inhibition of FLT3 phosphorylation and downstream signaling, including phospho-STAT5 and phospho-ERK.
  • Biomea Fusion explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

Aptose Presents Highlights from Corporate Update and KOL Event

Retrieved on: 
Donnerstag, Juni 2, 2022

SAN DIEGO and TORONTO, June 02, 2022 (GLOBE NEWSWIRE) -- Aptose Biosciences Inc. (“Aptose” or the “Company”) (NASDAQ: APTO, TSX: APS), a clinical-stage precision oncology company developing highly differentiated oral kinase inhibitors to treat hematologic malignancies, today released highlights from a key opinion leader (KOL) and corporate update event held today, June 2, 2022. The event included an up-to-date review of clinical data for Aptose’s two investigational products under development for hematologic malignancies: HM43239, an oral, myeloid kinome inhibitor in an international Phase 1/2 trial in patients with relapsed or refractory acute myeloid leukemia (AML); and luxeptinib, an oral, dual lymphoid and myeloid kinome inhibitor in a Phase 1 a/b trial in patients with relapsed or refractory B-cell malignancies, and in a separate Phase 1 a/b trial in patients with relapsed or refractory AML or high risk myelodysplastic syndrome (MDS).

Key Points: 
  • SAN DIEGO and TORONTO, June 02, 2022 (GLOBE NEWSWIRE) -- Aptose Biosciences Inc. (Aptose or the Company) (NASDAQ: APTO, TSX: APS), a clinical-stage precision oncology company developing highly differentiated oral kinase inhibitors to treat hematologic malignancies, today released highlights from a key opinion leader (KOL) and corporate update event held today, June 2, 2022.
  • The webcast of the presentation, including the Q&A with the guest KOLs, is available on Aptoses website here .
  • Weve identified three doses and target patient populations for our next stage of Expansion Clinical Trials with HM43239, as we move along a pathway toward registrational studies.
  • Aptose Biosciences is a clinical-stage biotechnology company committed to developing precision medicines addressing unmet medical needs in oncology, with an initial focus on hematology.