DMPK

Dyne Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Recent Business Highlights

Retrieved on: 
Tuesday, March 5, 2024

WALTHAM, Mass., March 05, 2024 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today reported financial results for the fourth quarter and full year 2023 and recent business highlights.

Key Points: 
  • (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today reported financial results for the fourth quarter and full year 2023 and recent business highlights.
  • Net loss: Net loss for the quarter ended December 31, 2023 was $66.6 million, or $1.09 per basic and diluted share.
  • This compares with a net loss of $38.8 million, or $0.74 per basic and diluted share, for the quarter ended December 31, 2022.
  • Net loss for the year ended December 31, 2023 was $235.9 million, or $3.95 per basic and diluted share.

Arrowhead Pharmaceuticals Initiates Phase 1/2a Study of ARO-DM1 for Treatment of Type 1 Myotonic Dystrophy

Retrieved on: 
Friday, March 8, 2024

Patients with DM1 have muscle weakness and wasting, myotonia, cataracts, and often develop cardiac conduction abnormalities.

Key Points: 
  • Patients with DM1 have muscle weakness and wasting, myotonia, cataracts, and often develop cardiac conduction abnormalities.
  • Pathogenesis of DM1 is driven by an expanded CUG trinucleotide repeat in the 3’-untranslated region of DMPK transcripts.
  • These abnormal transcripts cause mis-regulated splicing, known as spliceopathy, for certain messenger RNAs which are directly linked to the clinical manifestations of DM1.
  • Presentation materials may be accessed on the Events and Presentations page under the Investors section of the Arrowhead website.

MBrace Therapeutics Appoints Steve Alley as Chief Scientific Officer

Retrieved on: 
Tuesday, March 5, 2024

MBrace™ Therapeutics, Inc. (“MBrace”), a clinical-stage biopharmaceutical company devoted to improving the lives of patients with cancer by developing novel antibody-drug conjugates (ADCs), today announced the appointment of Steve Alley, Ph.D., as chief scientific officer.

Key Points: 
  • MBrace™ Therapeutics, Inc. (“MBrace”), a clinical-stage biopharmaceutical company devoted to improving the lives of patients with cancer by developing novel antibody-drug conjugates (ADCs), today announced the appointment of Steve Alley, Ph.D., as chief scientific officer.
  • Dr. Alley joins MBrace with over 20 years of experience at Seagen, a world-leader in ADCs that was acquired by Pfizer in 2023.
  • “We are thrilled to welcome Dr. Alley to the MBrace team.
  • Dr. Alley has exceptional expertise in ADC development, and his vision will undoubtedly help propel the progress for our discovery and clinical programs,” said Isan Chen, M.D., president and chief executive officer at MBrace Therapeutics.

Ryvu Therapeutics to Present Preclinical Data on RVU120 and Synthetic Lethality Programs at the 2024 AACR Annual Meeting

Retrieved on: 
Wednesday, March 6, 2024

Ryvu's partner Menarini to present data on MEN1703 (SEL24), demonstrating promising anti-tumor activity in preclinical models of myelofibrosis both as a single agent and combined with ruxolitinib.

Key Points: 
  • Ryvu's partner Menarini to present data on MEN1703 (SEL24), demonstrating promising anti-tumor activity in preclinical models of myelofibrosis both as a single agent and combined with ruxolitinib.
  • "We are excited to present our latest preclinical data at the AACR Annual Meeting, showcasing our significant progress in advancing novel small molecule therapies for oncology.
  • This year, we will present data from our most advanced preclinical project on MTA-cooperative PRMT5 inhibitors, the lead program within Ryvu's synthetic lethality pipeline.
  • Ryvu has developed potentially best-in-class MTA-cooperative PRMT5 inhibitors showing favorable drug-like properties and effective PRMT5 inhibition dependent on MTA binding.

ARTHEx Biotech Receives IND Clearance from FDA to Initiate the Phase I-IIa ArthemiR™ Trial of ATX-01 for Myotonic Dystrophy Type 1 (DM1)

Retrieved on: 
Wednesday, February 28, 2024

VALENCIA, Spain, Feb. 28, 2024 /PRNewswire/ -- ARTHEx Biotech S.L., a clinical-stage biotechnology company focused on developing innovative medicines through the modulation of microRNAs, announced that the U.S. Food and Drug Administration (FDA) cleared the Company to initiate the Phase I-IIa ArthemiR™ study of ATX-01 for the treatment of Myotonic Dystrophy Type 1 (DM1). 

Key Points: 
  • The ArthemiR™ trial is a Phase I-IIa double-blind, placebo-controlled, dose escalation study expected to enroll participants with classic Myotonic Dystrophy Type 1 (DM1).
  • ARTHEx will also investigate target engagement at the muscle level through biomarkers, including MBNL levels and splicing index.
  • In addition, the clinical endpoints from the trial will include measures related to muscle function, patient-reported outcomes, and quality of life measures.
  • Patients are eager for new trials, and we are delighted to start enrolment in the coming months."

Systemic Bio to Present Innovative Human-relevant Datasets from h-VIOS™ Platform at SOT and AACR 2024 Annual Meetings

Retrieved on: 
Thursday, February 8, 2024

HOUSTON, Feb. 08, 2024 (GLOBE NEWSWIRE) -- Systemic Bio™ , a 3D Systems (NYSE:DDD) company, is excited to announce the acceptance of two abstracts for presentation at the prestigious Society of Toxicology (SOT) 2024 Annual Meeting (Salt Lake City, March 10-14, 2024) and American Association for Cancer Research (AACR) 2024 Annual Meeting (San Diego, April 5-10, 2024).

Key Points: 
  • HOUSTON, Feb. 08, 2024 (GLOBE NEWSWIRE) -- Systemic Bio™ , a 3D Systems (NYSE:DDD) company, is excited to announce the acceptance of two abstracts for presentation at the prestigious Society of Toxicology (SOT) 2024 Annual Meeting (Salt Lake City, March 10-14, 2024) and American Association for Cancer Research (AACR) 2024 Annual Meeting (San Diego, April 5-10, 2024).
  • At the SOT 2024 meeting, Systemic Bio will present its latest research on an advanced liver model that demonstrates several-fold enhanced albumin production, CYP (cytochrome P450) activity, and urea production compared to traditional models.
  • The second abstract, accepted for AACR 2024, showcases a novel model that simultaneously assesses on-target anti-tumor therapeutic efficacy and off-target liver injury of therapeutics across different modalities.
  • To delve deeper into this research, all conference attendees and fellow researchers can visit booth #4452 to engage directly with Systemic Bio’s team and discover the full potential of the h-VIOS platform.

Sai Life Sciences augments DMPK capabilities to ace large-scale collaborations

Retrieved on: 
Thursday, February 1, 2024

HYDERABAD, India, Feb. 01, 2024 (GLOBE NEWSWIRE) -- To offer global big pharma clients cutting-edge, end-to-end drug metabolism and pharmacokinetics (DMPK) services, Sai Life Sciences, a global Contract Research, Development & Manufacturing Organization (CRO-CDMO) , has expanded its DMPK capabilities .

Key Points: 
  • HYDERABAD, India, Feb. 01, 2024 (GLOBE NEWSWIRE) -- To offer global big pharma clients cutting-edge, end-to-end drug metabolism and pharmacokinetics (DMPK) services, Sai Life Sciences, a global Contract Research, Development & Manufacturing Organization (CRO-CDMO) , has expanded its DMPK capabilities .
  • Making the announcement, Krishna Kanumuri, CEO & Managing Director, Sai Life Sciences, said, “The focus of innovator companies on diversifying their supply chains bodes well for Indian CROs-CDMOs like ours.
  • Sauri Gudlavalleti, Chief Operating Officer said, “The latest expansion of DMPK capabilities augments our integrated drug discovery (IDD) offerings.
  • Sai Life Sciences also has plans to invest in niche areas such as peptides, oligos and large molecules to strengthen its discovery capabilities.

DP Technology Announces Nomination of Development Candidate, a Potential Best-in-Class Kv1.3 Inhibitor for Immunological Diseases Including IBD and AD

Retrieved on: 
Tuesday, January 30, 2024

The nomination of novel Kv1.3 inhibitor DPT0218 for the treatment of various immunological diseases including IBD and AD.

Key Points: 
  • The nomination of novel Kv1.3 inhibitor DPT0218 for the treatment of various immunological diseases including IBD and AD.
  • Inhibition of Kv1.3 therefore offers a potential opportunity to specifically impair inflammatory immune cell activity and proliferation, including in antigen-activated T cells.
  • Long-lived memory T-cell populations, particularly tissue resident subsets, may contribute to the chronicity of IBD and represent a potential target for therapy[4].
  • It has shown positive results in preclinical animal models of various autoimmune diseases such as IBD and AD.

QPS Significantly Enhances Mass Spectrometry Laboratory Capacity

Retrieved on: 
Wednesday, January 10, 2024

QPS Holdings, LLC (QPS), a global contract research organization (CRO) that provides discovery, preclinical, bioanalysis, clinical trials, and clinical research services, is rapidly expanding its mass spectrometry capacity, adding 13 LC-MS machines in just 15 months at their laboratories in Newark, Delaware (USA) and Groningen, The Netherlands (NL).

Key Points: 
  • QPS Holdings, LLC (QPS), a global contract research organization (CRO) that provides discovery, preclinical, bioanalysis, clinical trials, and clinical research services, is rapidly expanding its mass spectrometry capacity, adding 13 LC-MS machines in just 15 months at their laboratories in Newark, Delaware (USA) and Groningen, The Netherlands (NL).
  • View the full release here: https://www.businesswire.com/news/home/20240110198636/en/
    Zamas Lam, PhD, Global Head of Bioanalytical (Mass Spec) & Preclinical Development, QPS LLC.
  • (Photo: Business Wire)
    This expansion includes the impressive growth seen in 2023, when QPS installed seven additional UPLC-(HR)MS/MS tandem instruments - four triple quadrupoles, one QTRAP, and two quadrupole time-of-flight high-resolution mass spectrometers (HRMS).
  • QPS Subject Matter Experts are some of the best in the industry and this new equipment will provide the capacity for them to expand their relationships with our important biotech and pharmaceutical clients,” said Zamas Lam, PhD, Global Head of Bioanalytical (Mass Spec) & Preclinical Development, QPS LLC.

Dyne Therapeutics Announces Positive Initial Clinical Data from ACHIEVE Trial in DM1 Patients and DELIVER Trial in DMD Patients Demonstrating Promise of the FORCE™ Platform in Developing Therapeutics for Rare Muscle Diseases

Retrieved on: 
Wednesday, January 3, 2024

“We are excited that Dyne’s first clinical data in two programs have demonstrated proof-of-concept and validated the promise of the FORCE™ platform in developing targeted therapeutics for rare muscle diseases.

Key Points: 
  • “We are excited that Dyne’s first clinical data in two programs have demonstrated proof-of-concept and validated the promise of the FORCE™ platform in developing targeted therapeutics for rare muscle diseases.
  • Dyne anticipates providing its next clinical data update from the ACHIEVE trial in the second half of 2024.
  • Key initial findings from DELIVER include:
    Muscle delivery: DYNE-251 showed a mean 657 ng/g PMO muscle drug concentration at 6 months.
  • Dyne anticipates providing its next clinical data update from the DELIVER trial in the second half of 2024.