HDAC

Muscular Dystrophy Association’s Funding of Foundational Research Leads to New FDA Approved Treatment Duvyzat (givinostat) for Duchenne Muscular Dystrophy

Retrieved on: 
Thursday, March 21, 2024

"The approval of Duvyzat (givinostat) provides another significant treatment option for people living with Duchenne muscular dystrophy,” said Sharon Hesterlee, PhD, Chief Research Officer, MDA.

Key Points: 
  • "The approval of Duvyzat (givinostat) provides another significant treatment option for people living with Duchenne muscular dystrophy,” said Sharon Hesterlee, PhD, Chief Research Officer, MDA.
  • “Muscular Dystrophy Association’s funding was fundamental for the discovery of HDACi in the treatment of Duchenne muscular dystrophy.
  • “We’re excited to celebrate this additional treatment option for people living with Duchenne muscular dystrophy.
  • My son is an example,” said Jessica Curran, MDA family member and mother of Conner who lives with Duchenne muscular dystrophy.

Avnos and Deep Sky Forge Path to Gigaton-Scale Carbon Removal in Canada

Retrieved on: 
Thursday, March 28, 2024

Deep Sky , a Montreal-based carbon removal project developer, and Avnos , the Los Angeles-based company pioneering novel Hybrid Direct Air Capture (HDAC™) technology for carbon dioxide removal, have joined forces to deploy HDAC technology in Canada.

Key Points: 
  • Deep Sky , a Montreal-based carbon removal project developer, and Avnos , the Los Angeles-based company pioneering novel Hybrid Direct Air Capture (HDAC™) technology for carbon dioxide removal, have joined forces to deploy HDAC technology in Canada.
  • As part of its partnership with Deep Sky, Avnos will build and install a Hybrid DAC Air Handling Unit (AHU) at Deep Sky Labs in Canada, with the capacity to remove 450 tons of CO2 per year.
  • View the full release here: https://www.businesswire.com/news/home/20240328504068/en/
    Deep Sky, the world's first gigaton-scale carbon removal project developer, will operate Avnos’ AHU at Deep Sky Labs, the world’s first carbon removal innovation center.
  • “Our partnership with Deep Sky demonstrates our commitment to achieving gigaton scale carbon removal and steering the nascent CDR industry toward creating opportunity and shared benefits.”
    As a project developer, Deep Sky is actively constructing expansive carbon removal and storage infrastructure across Canada.

Italfarmaco Receives FDA Approval for Duvyzat™ (givinostat) in Duchenne Muscular Dystrophy

Retrieved on: 
Friday, March 22, 2024

“We are grateful for the support of those living with DMD and their dedicated caregivers, which played a central role in helping us reach this landmark FDA approval.

Key Points: 
  • “We are grateful for the support of those living with DMD and their dedicated caregivers, which played a central role in helping us reach this landmark FDA approval.
  • The FDA approval highlights the dedication of Italfarmaco’s research and clinical teams to achieve this milestone for the company.”
    The approval is based on the results of the pivotal multicentre, randomised, double-blind, placebo-controlled phase 3 EPIDYS trial ( NCT02851797 ).
  • Italfarmaco has significantly expanded its U.S. presence through the formation of a new fully owned subsidiary, ITF Therapeutics LLC.
  • Italfarmaco has a global presence and is also working with other regulatory agencies.

PPMD Applauds FDA for Approval of DUVYZAT™ (Givinostat) for Duchenne Patients Ages Six Years and Older

Retrieved on: 
Friday, March 22, 2024

DUVYZAT™ marks a significant milestone as the first nonsteroidal drug approved for Duchenne that is applicable regardless of the specific genetic variant.

Key Points: 
  • DUVYZAT™ marks a significant milestone as the first nonsteroidal drug approved for Duchenne that is applicable regardless of the specific genetic variant.
  • "PPMD is thrilled that the FDA has approved DUVYZAT™, an HDAC inhibitor therapy that works by targeting pathogenic processes to reduce inflammation and loss of muscle.
  • Today's approval expands the therapeutic options for individuals with Duchenne," said PPMD Founding President and CEO Pat Furlong.
  • PPMD also takes a lead role in advocating for national newborn screening efforts for Duchenne, Becker, and carrier identification, as well as policy efforts around access and reimbursement.

FDA Approves Nonsteroidal Treatment for Duchenne Muscular Dystrophy

Retrieved on: 
Thursday, March 21, 2024

SILVER SPRING, Md., March 21, 2024 /PRNewswire/ -- Today, the U.S. Food and Drug Administration approved Duvyzat (givinostat) oral medication for the treatment of Duchenne Muscular Dystrophy (DMD) in patients six years of age and older.

Key Points: 
  • SILVER SPRING, Md., March 21, 2024 /PRNewswire/ -- Today, the U.S. Food and Drug Administration approved Duvyzat (givinostat) oral medication for the treatment of Duchenne Muscular Dystrophy (DMD) in patients six years of age and older.
  • Duvyzat is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD.
  • "This approval provides another treatment option to help reduce the burden of this progressive, devastating disease for individuals impacted by DMD regardless of genetic mutation."
  • DMD is the most common childhood form of muscular dystrophy and typically affects males.

Edison issues initiation on Cereno Scientific (CRNO B): Differentiated approach in CVD with potential

Retrieved on: 
Wednesday, March 13, 2024

Cereno Scientific is a clinical-stage biotech, developing treatments for cardiovascular diseases (CVDs) using novel approaches.

Key Points: 
  • Cereno Scientific is a clinical-stage biotech, developing treatments for cardiovascular diseases (CVDs) using novel approaches.
  • Lead asset CS1, a delayed immediate release formulation of valproic acid, is an HDAC inhibitor, aiming to deliver disease-modifying results in pulmonary arterial hypertension (PAH).
  • Preclinical candidates include CS014, for thrombosis prevention without increased risk of bleeding (seen with current antithrombotics), and CS585 for CVD (specific indication not yet determined).
  • As with all Edison publications, Edison controls the editorial and timings of publications and we wish to make it clear that Cereno Scientific had no involvement in the aforementioned issue.

Tenaya Therapeutics Announces Publication of Preclinical HDAC6 Inhibitor Data for Heart Failure with Preserved Ejection Fraction in Nature Communications

Retrieved on: 
Monday, February 26, 2024

SOUTH SAN FRANCISCO, Calif., Feb. 26, 2024 (GLOBE NEWSWIRE) -- Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease, today announced the publication of preclinical research related to Tenaya’s small molecule inhibitors of histone deacetylase 6 (HDAC6), including TN-301, in the February 26, 2024, issue of Nature Communications. The article, titled “Targeting HDAC6 to Treat Heart Failure with Preserved Ejection Fraction in Mice,” details the potential of inhibiting HDAC6 for the treatment of Heart failure with preserved ejection fraction (HFpEF), a form of heart failure that effects more than three million people in the U.S. alone1.

Key Points: 
  • The article, titled “Targeting HDAC6 to Treat Heart Failure with Preserved Ejection Fraction in Mice,” details the potential of inhibiting HDAC6 for the treatment of Heart failure with preserved ejection fraction (HFpEF), a form of heart failure that effects more than three million people in the U.S. alone1.
  • Tenaya’s highly selective small molecule inhibitors of the enzyme HDAC6 were discovered using the company’s modality-agnostic target discovery and validation capabilities.
  • For preclinical studies, Tenaya researchers used TYA-018, an HDAC6 inhibitor structurally and functionally similar to the company’s clinical candidate, TN-301.
  • The selective effects of HDAC6 inhibition were reaffirmed through genetic deletion studies, in which treatment of Hdac6 knockout mice did not display any of the beneficial effects that wild-type HFpEF mice did following treatment.

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.

Avnos, Inc. Secures $36 Million in Series A Funding Led by NextEra Energy

Retrieved on: 
Tuesday, February 6, 2024

Avnos , Inc. (Avnos), the Los Angeles-based company developing novel Hybrid Direct Air Capture (HDAC™) technology for carbon dioxide removal, today announced it has closed $36 million in Series A funding.

Key Points: 
  • Avnos , Inc. (Avnos), the Los Angeles-based company developing novel Hybrid Direct Air Capture (HDAC™) technology for carbon dioxide removal, today announced it has closed $36 million in Series A funding.
  • NextEra Energy , one of America's largest utilities and investors in clean energy infrastructure, led the round.
  • Our investment in Avnos is a part of NextEnergy’s commitment to attain Real Zero and enable a carbon-free future,” said NAME, TITLE at NextEra Energy.
  • Avnos’ unique approach and energy efficient solution for CO2 capture will enable optimization of the economics and the footprint of synthetic aviation fuels production.”

Vanda Pharmaceuticals Receives FDA Approval to Proceed with Investigational New Drug VTR-297 a Topical Antifungal Candidate for the Treatment of Onychomycosis

Retrieved on: 
Wednesday, January 31, 2024

WASHINGTON, Jan. 31, 2024 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced that the U.S. Food and Drug Administration (FDA) has approved the Investigational New Drug (IND) application to evaluate VTR-297 for the treatment of onychomycosis.

Key Points: 
  • WASHINGTON, Jan. 31, 2024 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced that the U.S. Food and Drug Administration (FDA) has approved the Investigational New Drug (IND) application to evaluate VTR-297 for the treatment of onychomycosis.
  • Onychomycosis, or tinea unguium, is a fungal infection of the nail.
  • Onychomycosis can result in discoloration of the nail, onycholysis (nail separation from the nail bed), and nail plate thickening.
  • There have not been any new onychomycosis treatments approved by the FDA since 2014.4
    Centers for Disease Control and Prevention.