A-T

Quince Therapeutics Provides Business Update and Reports Fourth Quarter and Fiscal 2023 Financial Results

Retrieved on: 
Monday, April 1, 2024
Health, Clinical Trials, Research, Pharmaceutical, Science, Biotechnology, A-T, DSP, Disease, SPA, Half-life, Hematology, Patient, NDA, EU4, Acquisition, Standard of care, Pivotal, Cancer, Quality of life, Biology, AIDE, Investment, Clinical trial, Therapy, NEAT, FDA, Pharmaceutical industry

Quince Therapeutics, Inc. (Nasdaq: QNCX), a late-stage biotechnology company developing an innovative drug delivery technology designed to leverage a patient’s own biology to deliver rare disease therapeutics, today provided an update on the company’s development pipeline and reported financial results for the fourth quarter and fiscal year ended December 31, 2023.

Key Points: 
  • Quince Therapeutics, Inc. (Nasdaq: QNCX), a late-stage biotechnology company developing an innovative drug delivery technology designed to leverage a patient’s own biology to deliver rare disease therapeutics, today provided an update on the company’s development pipeline and reported financial results for the fourth quarter and fiscal year ended December 31, 2023.
  • Pivotal Phase 3 NEAT clinical trial will be conducted under a Special Protocol Assessment (SPA) agreement with the U.S. Food & Drug Administration (FDA).
  • Expect to report Phase 3 NEAT topline results in the second half of 2025 with a potential NDA submission in 2026, assuming positive study results.
  • Quince estimates there are an aggregate of approximately 10,000 patients with A-T in the U.S., U.K., and EU4 countries.

Quince Therapeutics Completes Acquisition of EryDel S.p.A.

Retrieved on: 
Monday, October 23, 2023
Health, Clinical Trials, Research, Pharmaceutical, Science, Biotechnology, European Investment Bank, A-T, FDA, Ageing, Therapy, Neurodegenerative disease, Acquisition, DSP, AIDE, Disease, Clinical trial, Biology, NDA, Safety, SPA, Patient, EIB, Pharmaceutical industry, Quince

Quince Therapeutics, Inc. (Nasdaq: QNCX), a late-stage biotechnology company developing an innovative drug delivery technology that leverages a patient’s own biology to deliver rare disease therapeutics, announced the successful completion of its acquisition of EryDel S.p.A., a privately-held, late-stage biotech company.

Key Points: 
  • Quince Therapeutics, Inc. (Nasdaq: QNCX), a late-stage biotechnology company developing an innovative drug delivery technology that leverages a patient’s own biology to deliver rare disease therapeutics, announced the successful completion of its acquisition of EryDel S.p.A., a privately-held, late-stage biotech company.
  • Currently, there are no approved treatments for A-T and the market represents a $1+ billion estimated peak sales opportunity globally.
  • “The successful closing of the EryDel acquisition is an exciting step forward in fulfilling our vision to build a leading rare disease biotechnology company,” said Dirk Thye, M.D., Quince’s Chief Executive Officer.
  • The acquisition of EryDel was completed with no upfront cash payment, using a stock-for-stock exchange and potential downstream milestone cash payments.

U.S. FDA Partial Clinical Hold Lifted on IND for EryDel’s Lead Phase 3 Asset EryDex for the Treatment of Ataxia-Telangiectasia

Retrieved on: 
Thursday, September 28, 2023
Science, Neurology, Biotechnology, Research, Pharmaceutical, Health, FDA, Clinical Trials, A-T, Plastic, Therapy, New Drug Application, Clinical trial, NDA, Acquisition, Patient, Agency, SPA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, IND, Food, Safety, Pharmaceutical industry, Quince

Currently, there are no approved treatments for patients with A-T and the market represents a $1+ billion estimated peak sales opportunity.

Key Points: 
  • Currently, there are no approved treatments for patients with A-T and the market represents a $1+ billion estimated peak sales opportunity.
  • Dirk Thye, M.D., Quince’s Chief Executive Officer, said, “We are pleased with the FDA’s decision to lift the partial clinical hold related to EryDel’s lead asset, EryDex.
  • We look forward to completing the clinical and regulatory activities necessary to advance EryDex into the Phase 3 NEAT study – with patient enrollment beginning as soon as the second quarter of 2024.
  • The commercial version of the EryKit treatment consumables is already approved for clinical trial use in Europe.

Quince Therapeutics Appoints Dr. Charles S. Ryan as President

Retrieved on: 
Wednesday, September 6, 2023
Biotechnology, Health, Professional Services, Pharmaceutical, Legal, Archives of Oral Biology, DSM-IV codes, Patent, A-T, College, Therapy, Western New England University, Senior, New York State Bar Association, Stony Brook University, Research, IP, CNS, Acquisition, AbbVie, Forest Laboratories, Patient, Pathology, Forest, College of Wooster, Pharmaceutical industry, Quince, Chemistry

Quince Therapeutics, Inc. (Nasdaq: QNCX), a biotechnology company focused on acquiring, developing, and commercializing innovative therapeutics that transform patients’ lives, today announced the appointment of Charles S. Ryan, J.D., Ph.D. as President.

Key Points: 
  • Quince Therapeutics, Inc. (Nasdaq: QNCX), a biotechnology company focused on acquiring, developing, and commercializing innovative therapeutics that transform patients’ lives, today announced the appointment of Charles S. Ryan, J.D., Ph.D. as President.
  • Dirk Thye, M.D., Quince’s Chief Executive Officer, said, “Charles is a valuable addition to our management team.
  • His considerable expertise will help enhance efficiency and effectiveness across our research programs and business operations.
  • Most recently, Dr. Ryan served as President, Chief Executive Officer, and Chairman of Travecta Therapeutics, a private biopharmaceutical company pioneering transformative treatments for serious neurological conditions.

Quince Therapeutics to Acquire EryDel SpA and its Phase 3 Asset Targeting Ataxia-Telangiectasia with No Currently Approved Treatments and Estimated $1+ Billion Peak Sales Opportunity

Retrieved on: 
Monday, July 24, 2023
Health, Medical Devices, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, News, EU4, A-T, Therapy, AIDE, Bank statement, Clifford Chance, Clinical trial, NDA, Toxicity, DSM-IV codes, Quality, Dexamethasone, EMA, Cooley LLP, QOL, Acquisition, EQ-5D, HIV disease progression rates, Patient, Investment, Vineland Adaptive Behavior Scale, Quality of life, SPA, Steroid, Growth, Ageing, MAA, European Investment Bank, FDA, EIB, ITT, Deficit spending, Enzyme replacement therapy, OLE, International Cooperative Ataxia Rating Scale, Safety, Korea International Cooperation Agency, Pharmaceutical industry, Cryptocurrency, Medical imaging, Quince

EryDel has developed an autologous intracellular drug encapsulation (AIDE) technology and a Phase 3 lead asset, EryDex, targeting a rare fatal pediatric neurological disease, Ataxia-Telangiectasia (A-T), which currently has no approved treatments.

Key Points: 
  • EryDel has developed an autologous intracellular drug encapsulation (AIDE) technology and a Phase 3 lead asset, EryDex, targeting a rare fatal pediatric neurological disease, Ataxia-Telangiectasia (A-T), which currently has no approved treatments.
  • Commercial version of EryKit treatment consumables approved in Europe and currently under partial clinical hold pending response to FDA query.
  • A-T population estimated to be approximately 10,000 patients in the U.S., U.K., and EU4 countries with no currently approved therapies and $1+ billion estimated peak sales opportunity globally.
  • MTS Health Partners, L.P. is serving as financial advisor and Cooley LLP is serving as legal counsel to Quince.

Acasti Pharma Reports Third Quarter 2023 Operational Results

Retrieved on: 
Tuesday, February 14, 2023
Bank statement, Subarachnoid hemorrhage, Toxicity, Caregiver, Brain, Dizziness, Nimodipine, NDA, AUC, Bupivacaine, Trial of the century, OS, Human, Blood, Postherpetic neuralgia, PK, Cmax, TSX, Hypoesthesia, Aes, Investment, SAH, Safety, Literature, Acquisition, Tmax, Anesthesiology, Patient, PHN, Telephone, AE, Skin, Food, Skull, Research, Government, Aneurysm, Risk, IM, SubArachnoid Space, Betamethasone, Plasma, Tongue, FDA, Nausea, A-T, Food and Drug Administration Amendments Act of 2007, Calendar, Bleeding, Type, Ataxia–telangiectasia, ACST, Pharmacokinetics, Rupture, Headache, Pharmaceutical industry, Vaccine, Cryptocurrency

LAVAL, Québec, Feb. 14, 2023 (GLOBE NEWSWIRE) -- Acasti Pharma Inc. (“Acasti” or the “Company”) (Nasdaq: ACST and TSX-V: ACST), a late-stage, specialty pharma company advancing three clinical stage drug candidates addressing rare and orphan diseases, today announced financial and operational results for the third quarter ended December 31, 2022

Key Points: 
  • These positive results allow us to advance both programs to the next stage of clinical development in 2023.
  • We expect 2023 to be very exciting for Acasti with two of our drug candidates ready to enter Phase 3.
  • Topline results from this study were reported on December 23, 2022, and the results met all primary outcome measures.
  • Acasti will host a conference call on Tuesday, February 14, 2023, at 1:00 PM Eastern Time to discuss the Company’s corporate progress and other developments, as well as financial results for its quarter ended December 31, 2022.

Adrestia Therapeutics Partners with Leading Patient Advocacy Groups to Form Ataxia Telangiectasia Research Consortium

Retrieved on: 
Wednesday, February 8, 2023
Health, Medical Devices, Genetics, Research, Science, Pharmaceutical, Biotechnology, DNA, Biology, Infectious Diseases Society of America, DSM-IV codes, American Cancer Society Cancer Action Network, Diabetes, Therapy, Disease, Trustee, DNA repair, A-T, AT, Conditional sentence, Cardiovascular disease, Ataxia–telangiectasia, Genome, Pharmaceutical industry, Vaccine

Adrestia Therapeutics , a leader in synthetic rescue therapies for genetic diseases, today announced it has co-founded a research consortium to find new treatments for ataxia telangiectasia (AT), a fatal, inherited, progressive neurodegenerative disorder that is typically diagnosed in young children.

Key Points: 
  • Adrestia Therapeutics , a leader in synthetic rescue therapies for genetic diseases, today announced it has co-founded a research consortium to find new treatments for ataxia telangiectasia (AT), a fatal, inherited, progressive neurodegenerative disorder that is typically diagnosed in young children.
  • The consortium’s other founding members include the A-T Children’s Project, the AT Society and Action for AT.
  • Adrestia is applying its synthetic rescue technology to systematically mine the human genome for novel targets which could be used as the basis for therapies for AT and related diseases.
  • Our ultimate goal is to develop effective medicines which rebalance the underlying biology, unlocking new ways of treating intractable genetic diseases.

Acasti to Host Conference Call on Tuesday, January 10, 2023 to Discuss Results from Recent Phase 1 PK Studies for GTX-101 and GTX-102 That Met All Outcome Measures

Retrieved on: 
Thursday, January 5, 2023
Bupivacaine, Calendar, Betamethasone, Pain, Pharmacokinetics, Telephone, Ataxia–telangiectasia, PK, Neuropathic pain, TSX, ACST, Trial of the century, Nerve Damage, A-T, Safety, FDA, Human, Pharmaceutical industry, Internet service provider, Postherpetic neuralgia

ET to review the recently reported preliminary topline results from two separate Phase 1 pharmacokinetic (PK) studies for GTX-101 and GTX-102.

Key Points: 
  • ET to review the recently reported preliminary topline results from two separate Phase 1 pharmacokinetic (PK) studies for GTX-101 and GTX-102.
  • Both studies successfully met all target outcome measures.
  • The conference call will be available via telephone by dialing toll free 844-836-8745 for U.S. callers or +1 412-317-6797 for international callers.
  • On December 28, 2022, Acasti announced that the preliminary topline results of the pharmacokinetic (PK) bridging study for GTX-102 met all primary outcome measures.

Acasti Announces Preliminary Topline Results Met All Outcome Measures in the Pharmacokinetic Bridging Study for GTX-102, the Company’s Drug Candidate for the Treatment of Ataxia Telangiectasia

Retrieved on: 
Wednesday, December 28, 2022
Calendar, OS, AE, Phase 3, Pharmacokinetics, Ataxia–telangiectasia, PK, TSX, ACST, Caregiver, NDA, Patient, A-T, AUC, ClinicalTrials.gov, Data, Safety, FDA, Genetic disorder, COVID-19, Literature, IM, Cmax, Tongue, Pharmaceutical industry, Generic drug, Betamethasone

This PK study was the next step in the proposed 505(b)(2) regulatory pathway for GTX-102.

Key Points: 
  • This PK study was the next step in the proposed 505(b)(2) regulatory pathway for GTX-102.
  • We are very pleased to report the results of this study, which we expect will now support the advancement of the program directly into Phase 3.
  • The primary objective of this PK bridging study was to evaluate and characterize the PK profile of GTX-102 as an oral spray.
  • GTX-102 PK study outcome measures definitions and preliminary topline findings are as follows:
    Primary outcome measures and their definitions include:
    Cmax is the maximum concentration occurring between 0 hour to 72 hours after study drug administration.

Acasti Pharma Announces Preliminary Topline Results Met All Primary Outcome Measures in the Single Dose Pharmacokinetic Study for GTX-101, the Company’s Drug Candidate for the Treatment of Postherpetic Neuralgia (PHN)

Retrieved on: 
Thursday, December 22, 2022
Standard of care, Bupivacaine, Calendar, Betamethasone, Pain, Blood, Pharmacokinetics, Gabapentin, Lidocaine, PHN, Ataxia–telangiectasia, PK, Neuropathic pain, Hydrogen chloride, TSX, Tmax, ACST, Skin, NDA, Toxicity, Risk, Trial of the century, Irritation, Nerve Damage, Patient, Plasma, Type, A-T, AUC, Safety, Physician, FDA, Headache, Human, Hypoesthesia, Postherpetic neuralgia, Cmax, Pharmaceutical industry, Risk management, Generic drug

The final clinical study report is anticipated to be received by the Company in the first half of 2023.

Key Points: 
  • The final clinical study report is anticipated to be received by the Company in the first half of 2023.
  • The potential benefits of GTX-101 could include faster onset of action and a longer duration of pain relief as compared to the lidocaine patch.
  • The study enrolled 48 healthy adult subjects (24 males/24 females, mean age = 36 years), in 12 subjects per cohort.
  • Following GTX-101 topical application: headache (3%) and numbness (3%) at the sprayed area, and
    In conclusion, this Single Dose PK study was conducted successfully, and it achieved all its primary outcome measures.