Thursday, 9 Oct 2025
  • About Us
  • Contact Us
  • Terms and Conditions
  • Privacy Policy
Subscribe
Life Care News
  • Home
  • Business
    • Business Wire
    • Globenews Wire
  • News
    NewsShow More
    GEDU Group to Become Largest Foreign Education Investor in India
    GEDU Group to Become Largest Foreign Education Investor in India
    09/10/2025
    Signs of Change: Equipping Teachers with Tools for Inclusive Classrooms
    Signs of Change: Equipping Teachers with Tools for Inclusive Classrooms
    09/10/2025
    Nobel Prize 2025 in Medicine: Three Scientists Who Changed the Future of Immunology
    Nobel Prize 2025 in Medicine: Three Scientists Who Changed the Future of Immunology
    08/10/2025
    नोबेल पुरस्कार 2025 – मेडिसिन कैटेगरी में तीन वैज्ञानिकों की शानदार जीत!
    नोबेल पुरस्कार 2025 – मेडिसिन कैटेगरी में तीन वैज्ञानिकों की शानदार जीत!
    08/10/2025
    Comviva Fintech Platforms Hits  Billion Daily Transaction Milestone, Strengthening its Global Fintech Dominance
    Comviva Fintech Platforms Hits $1 Billion Daily Transaction Milestone, Strengthening its Global Fintech Dominance
    08/10/2025
  • Tech
  • Health
  • Sports
  • Entertainment
  • Automobile
  • 🔥
  • news
  • global
  • Business
  • june
  • announced
  • today
  •  and
  • july
  •  the
  • company
Font ResizerAa
Life Care NewsLife Care News
  • Home
  • Business
  • News
  • Tech
  • Health
  • Sports
  • Entertainment
  • Automobile
Search
  • Home
  • Business
    • Business Wire
    • Globenews Wire
  • News
  • Tech
  • Health
  • Sports
  • Entertainment
  • Automobile
Follow US
© 2022 Foxiz News Network. Ruby Design Company. All Rights Reserved.
Health

Skyhawk Therapeutics Announces Positive First Interim Results in Patients from its Phase 1 Clinical Trial of SKY-0515 as a Treatment for Huntington’s Disease

PRNW Agency
Last updated: 17/09/2025 8:32 PM
PRNW Agency
Share
6 Min Read
Skyhawk Therapeutics Announces Positive First Interim Results in Patients from its Phase 1 Clinical Trial of SKY-0515 as a Treatment for Huntington’s Disease
SHARE
Skyhawk Therapeutics Announces Positive First Interim Results in Patients from its Phase 1 Clinical Trial of SKY-0515 as a Treatment for Huntington’s Disease

SKY-0515 achieves dose-dependent reductions of mutant huntingtin (mHTT) protein, with 62% lowering at Day 84 on the 9mg daily oral dose

- Advertisement -

Additional findings include dose-dependent reductions in PMS1 mRNA, excellent brain penetration, and a favorable safety profile

- Advertisement -

SKY-0515’s Phase 2/3 FALCON-HD trial in patients in Australia and New Zealand is ongoing 

- Advertisement -

BOSTON, Sept. 17, 2025 /PRNewswire/ — Skyhawk Therapeutics, Inc., a clinical-stage biotechnology company developing novel small molecule therapies designed to modulate critical RNA targets, today announced positive results from the first interim analysis of the Part C patient cohort in its Phase 1 clinical trial of SKY-0515, an investigational treatment for Huntington’s disease (HD). At Day 84, patients receiving SKY-0515 demonstrate dose-dependent reductions of mHTT protein in blood including 62% at the 9mg dose. SKY-0515 has been generally well tolerated at both dose levels tested. 

- Advertisement -

Treatment with SKY-0515 also results in important dose-dependent PMS1 mRNA reduction and demonstrates excellent central nervous system penetration. The overall safety profile has been favorable, supporting the continued clinical development of the program.

- Advertisement -

“SKY-0515 is reducing mHTT protein to the most impressive extent we’ve seen so far in patients, and crucially the clinical and biomarker data show no safety concerns so far at any dose tested,” said Ed Wild, Professor of Neurology at University College London. “It is great that we are seeing substantial PMS1 reduction as well, which should be a potent combination for treating Huntington’s disease via two of its core pathogenic mechanisms. This is what success looks like at the 3-month timepoint, setting the stage for meaningful impact for people living with HD across the world – for whom an orally administered huntingtin-lowering treatment such as SKY-0515 would be truly transformative.”

- Advertisement -

“The strength of SKY-0515’s biomarker response after just 84 days of treatment underscores its potential as a transformative therapy for HD,” said Sergey Paushkin, Head of R&D at Skyhawk Therapeutics. “These interim data represent an important milestone for SKY-0515 and highlight the power of Skyhawk’s platform to deliver first-in-class small molecules for devastating diseases with no approved disease-modifying therapies.”

- Advertisement -

Huntington’s disease is a rare, hereditary, and ultimately fatal neurodegenerative disorder that affects over 40,000 symptomatic patients in the United States, with hundreds of thousands estimated to be affected worldwide. There are currently no approved treatments which slow or halt disease progression. SKY-0515 is Skyhawk’s orally-administered, investigational small molecule RNA splicing modifier developed through the company’s novel RNA-splicing platform, SKYSTAR®.  SKY-0515 is designed to reduce both HTT protein and PMS1 protein, an additional key driver of somatic CAG repeat expansion and HD pathology.

- Advertisement -

SKY-0515 was developed through Skyhawk’s proprietary integrated platform, SKYSTAR®, and is the first Skyhawk drug in clinical trials. 

- Advertisement -

Skyhawk expects to put a series of additional novel drugs to treat rare neurological diseases with no presently approved disease modifying therapies in the clinic by the end of 2027. The first is on track to initiate trials in mid-2026.

- Advertisement -

About SKY-0515’s Phase 1 Clinical Study
SKY-0515’s Phase 1 clinical trial is a first-in-human trial designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics, specifically blood biomarker modulation activity, of SKY-0515 in healthy volunteers and individuals with early-stage Huntington’s disease (HD). The trial is separated into three parts. Parts A and B evaluated SKY-0515 in Healthy Volunteers. Part C is a double-blind placebo-controlled parallel design study of two dose levels of SKY-0515 and placebo of individuals with early-stage HD (HD-ISS Stage 1, 2, or mild Stage 3) for 84 days followed by a 12 month extension of active treatment where all participants will receive either a low or high dose of SKY-0515 in a blinded fashion. The objectives of the study are to evaluate safety, pharmacokinetic, and pharmacodynamic parameters including mutant HTT protein and PMS1 mRNA. Part C enrollment is complete, and topline data from the blinded active treatment extension are expected in mid-2026.

- Advertisement -

About SKY-0515’s Phase 2/3 FALCON-HD Clinical Study
FALCON-HD (NCT06873334) is a Phase 2/3 randomized, double-blind, placebo-controlled, dose ranging study to evaluate the pharmacodynamics, safety, and efficacy of SKY-0515 in 120 participants with Stage 2 and early Stage 3 HD across 10 sites in Australia and New Zealand. Eligible patients will receive a once-daily oral dose of SKY-0515 at one of three dose levels, or placebo, for a treatment period of at least 12 months. The trial aims to assess the potential of SKY-0515 to modulate RNA splicing and reduce mHTT and PMS1 proteins, which are implicated in the pathology of Huntington’s disease. Additional information about FALCON-HD, including participating sites and eligibility criteria, can be found at ClinicalTrials.gov and www.FALCON-HD.com.

- Advertisement -

About Skyhawk Therapeutics
Skyhawk Therapeutics is a clinical-stage biotechnology company which uses its proprietary platform, SKYSTAR®, to discover and develop small molecule RNA modulating therapies for the world’s most intractable diseases. For more information visit www.skyhawktx.com.  

- Advertisement -

Skyhawk Contact
Maura McCarthy
maura@skyhawktx.com

- Advertisement -

Photo – https://mma.prnewswire.com/media/2774749/Skyhawk_Therapeutics_Reduction_in_mHTT_protein_level.jpg
Logo – https://mma.prnewswire.com/media/710814/Skyhawk_Therapeutics_Logo.jpg 

- Advertisement -

 

- Advertisement -

View original content:https://www.prnewswire.co.uk/news-releases/skyhawk-therapeutics-announces-positive-first-interim-results-in-patients-from-its-phase-1-clinical-trial-of-sky-0515-as-a-treatment-for-huntingtons-disease-302559094.html

- Advertisement -
Young voices drive SCO innovation and culture
Applied Intuition and Komatsu Strike Deal to Transform Mining Industry with Intelligent, Autonomous Machines
Swiggy Launches ‘Mom’entum Policy: A Multi-Year Maternity Program Supporting Mothers at Every Stage
Alamar Biosciences and the German Center for Neurodegenerative Diseases (DZNE) partner for Landmark Proteomic Profiling study in the Rhineland Study Cohort
Stride, Inc. Signs Multi-District Agreements to Operate K12-Powered Online School in New Mexico
TAGGED: for fromachievesadditionalannouncesaustraliabrainclinicaldailydaydiseasedosedosedependentexcellentfalconhdfavorablefindingsfirsthuntingtinhuntington’sincludeinterimits loweringmhttmrnamutantnewsoralpatientspenetrationphasepmspositiveprofileproteinreductionsresultssafetyskysky-0515skyhawkskystherapeuticstreatmenttrial
Share This Article
Facebook Copy Link Print
- Advertisement -

Your Trusted Source for Accurate and Timely Updates!

Our commitment to accuracy, impartiality, and delivering breaking news as it happens has earned us the trust of a vast audience. Stay ahead with real-time updates on the latest events, trends.
FacebookLike
XFollow
PinterestPin
InstagramFollow
YoutubeSubscribe
LinkedInFollow
MediumFollow

Most Selling Products

Top Picks, Trending Now – Discover the Best Sellers!
Tecno Camon 20 Premier 5G

Tecno Camon 20 Premier 5G

Dark Welkin | 8GB RAM + 512GB Storage (Expandable RAM up to 16GB) | Industry’s 1st 50MP RGBW-Pro Camera | Segment-First 108MP Ultra-Wide Macro Lens | 6.67" 120Hz 10-bit AMOLED In-Display

iQOO Z10 Lite 5G

iQOO Z10 Lite 5G

Titanium Blue | 6GB RAM + 128GB Storage | Dimensity 6300 5G with 433K+ AnTuTu Score | Robust 6000mAh Battery | IP64 Rated + Military-Grade Shock Resistance

OnePlus 13s

OnePlus 13s

Black Velvet | 12GB RAM + 256GB Storage | Flagship Snapdragon® 8 Elite Chipset | Exceptional Battery Life in a Compact Form | Lifetime Display Warranty Included

Samsung Galaxy A55 5G

Samsung Galaxy A55 5G

Awesome Iceblue | 8GB RAM + 256GB Storage | Premium Metal Frame | 50MP OIS Main Camera with Nightography | IP67 Water & Dust Resistance | Gorilla Glass Victus+ | sAMOLED Display with Vision Booster

You Might Also Like

Odyssey Investment Partners to Sell Applied Technical Services to SGS for .325 Billion
Business

Odyssey Investment Partners to Sell Applied Technical Services to SGS for $1.325 Billion

02/07/2025

GENESIS GV60 TAKES A CLASS WIN AT THE CARAVAN AND MOTORHOME CLUB TOWCAR OF THE YEAR AWARDS

27/09/2025
LECTRA: Monthly declaration of the total number of shares and voting rights composing the company’s capital (at June 30th, 2025)
Tech

LECTRA: Monthly declaration of the total number of shares and voting rights composing the company’s capital (at June 30th, 2025)

08/07/2025

Lamborghini Day UK celebrates the marques present, past and future

30/09/2025
Life Care News
Facebook Twitter Youtube Rss Medium

Life Care News:


We increase the awareness of millions of users through our news networks. We are one of the most trusted news networks in the world.

Top Categories
  • Home
  • Business
  • News
  • Tech
  • Health
  • Sports
  • Entertainment
  • Automobile
Usefull Links
  • About Us
  • Contact Us
  • Terms and Conditions
  • Privacy Policy
Copyright © 2015 – 2025 LifeCareNews Network. All Rights Reserved. LIFE CARE IS REGISTERED MAGAZINE IN RNI, NO.GUJGUJ/2015/71283
Life Care NewsLife Care News
Copyright © 2015 - 2025 LifeCareNews Network. All Rights Reserved. LIFE CARE IS REGISTERED MAGAZINE IN RNI, NO.GUJGUJ/2015/71283
Welcome Back!

Sign in to your account

Username or Email Address
Password

Lost your password?