Priority review

EQS-News: AdrenoMed Receives FDA Fast Track Designation for Enibarcimab for Treatment of Septic Shock

Retrieved on: 
Wednesday, April 10, 2024
Sepsis, Communication, Patient, Public, Fast Track, Adrenomedullin, Biomarker, Accelerated approval (FDA), Mortality, Septic shock, Death, DPP3, Septic, Medicine, Priority review, Organ dysfunction, Intensive care medicine, CMO, Collection, FDA, Infection, RCI, Food, Pharmaceutical industry

AdrenoMed is now preparing a confirmatory Phase IIb/III clinical trial to confirm the reduced septic shock mortality under enibarcimab treatment employing a precision medicine approach.

Key Points: 
  • AdrenoMed is now preparing a confirmatory Phase IIb/III clinical trial to confirm the reduced septic shock mortality under enibarcimab treatment employing a precision medicine approach.
  • AdrenoMed’s CEO Dr. Richard Jones commented: “We are very pleased that enibarcimab has received Fast Track designation from the FDA, recognizing its potential as an innovative biomarker-guided treatment against septic shock to fill the unmet medical need in this very serious condition with a high death toll.
  • With a mortality rate of 20-30% for sepsis [1] and 30%-50% for septic shock in developed countries, [2] sepsis represents an enormous public health burden and is responsible for almost 20% of all deaths worldwide.
  • Dr. Stephan Witte, CMO of AdrenoMed, said: “We are very confident that the use of enibarcimab in combination with two biomarkers, Adrenomedullin (bio-ADM) and circulating dipeptidyl peptidase 3 (cDPP3), holds the promise to become the first effective targeted treatment against septic shock.

Cardiol Therapeutics Announces Year-End 2023 Update on Operations

Retrieved on: 
Tuesday, April 2, 2024
Hypertrophy, Heart, CRDL, Patient, Mayo Clinic, PPAR, Agent handling, Cleveland Clinic, Fibrosis, European, Therapy, Myocarditis, Analysis, American Heart Association, Human, Massachusetts General Hospital, Pericarditis, MMT, Heart failure, American College, Safety, ODD, Cardiology, Clinical professor, Disease, Pericardium, Research, World Heart Federation, Management, University, Death, Pericardial effusion, Maverick, Reactive oxygen species, TSX, Priority review, FDA, Conversation, Financial statement, Inflammation, Fast Track, Calcium, Wound healing, API, Pharmaceutical industry

"Cardiol Therapeutics made important progress in 2023 and early 2024 as we pursued our primary objective of providing new therapeutic options to patients with poorly served heart diseases," said David Elsley, President and Chief Executive Officer of Cardiol Therapeutics.

Key Points: 
  • "Cardiol Therapeutics made important progress in 2023 and early 2024 as we pursued our primary objective of providing new therapeutic options to patients with poorly served heart diseases," said David Elsley, President and Chief Executive Officer of Cardiol Therapeutics.
  • In January 2023, Cardiol announced the first patient had been enrolled in the Company's Phase II open-label pilot study ("MAvERIC-Pilot") investigating the tolerance, safety, and efficacy of CardiolRx™ in patients with recurrent pericarditis.
  • In November 2023, Cardiol announced that it had exceeded 50% of the patient enrollment target for the MAvERIC-Pilot study.
  • In January 2024, Cardiol announced that ARCHER had exceeded 50% patient enrollment and was progressing ahead of the original study timeline.

HUTCHMED and Innovent Jointly Announce NDA Acceptance in China for Fruquintinib Combination with Sintilimab for the Treatment of Advanced Endometrial Cancer with Priority Review Status

Retrieved on: 
Tuesday, April 2, 2024
Toxicity, Radiation, Sintilimab, NMPA, Endometrial cancer, Disease, DCR, New Drug Application, Patient, NDA, Breakthrough therapy, Senior, Priority review, HKEX, Dor, IRC, Prognosis, PFS, Pharmaceutical industry

HONG KONG, SHANGHAI, China & FLORHAM PARK, N.J., April 02, 2024 (GLOBE NEWSWIRE) -- HUTCHMED (China) Limited (“HUTCHMED”) (Nasdaq/AIM:​HCM; HKEX:​13) and Innovent Biologics, Inc. (“Innovent”) (HKEX:1801) today jointly announce that the New Drug Application (“NDA”) for the combination of fruquintinib and sintilimab for the treatment of patients with advanced endometrial cancer with pMMR1 or non-MSI-H2 tumors that have failed prior systemic therapy but are not candidates for curative surgery or radiation has been accepted and granted priority review by the China National Medical Products Administration (“NMPA”).

Key Points: 
  • The NDA is supported by data from FRUSICA-1, the endometrial cancer registration cohort of a multi-center, open-label Phase II study investigating fruquintinib in combination with sintilimab in endometrial cancer patients who experienced disease recurrence, disease progression or intolerable toxicity with treatment on platinum-based doublet chemotherapy.
  • Data from FRUSICA-1 will be submitted for presentation at an upcoming medical conference.
  • “This is the first regulatory filing for the combination of fruquintinib and the immune checkpoint inhibitor sintilimab.
  • “Endometrial cancer remains one of the most common gynecological malignancies.

Akari Therapeutics Reports Full-Year 2023 Financial Results and Recent Highlights

Retrieved on: 
Monday, April 1, 2024
Diagnosis, Civil service commission, Research, AATD, Growth, Akari, C5, Patient, Choroidal neovascularization, Sale, B4, PRV, Mortality, Hot game, Clinical trial, Therapy, Priority review, Safety, NEI, European Commission, ADC, Geographic atrophy, Hematopoietic stem cell transplantation, CNV, Pharmaceutical industry

BOSTON and LONDON, April 01, 2024 (GLOBE NEWSWIRE) -- Akari Therapeutics, Plc (Nasdaq: AKTX), a late-stage biotechnology company developing advanced therapies for autoimmune and inflammatory diseases, has reported financial results for the full year 2023 as well as recent company highlights.

Key Points: 
  • BOSTON and LONDON, April 01, 2024 (GLOBE NEWSWIRE) -- Akari Therapeutics, Plc (Nasdaq: AKTX), a late-stage biotechnology company developing advanced therapies for autoimmune and inflammatory diseases, has reported financial results for the full year 2023 as well as recent company highlights.
  • The combined entity will operate as Akari Therapeutics, Plc, which is expected to continue to be listed and trade on the Nasdaq Capital Market as AKTX.
  • Akari is continuing to recruit patients into the Part A portion of the Phase 3 clinical trial that has treated 10 patients to date.
  • During 2023, Akari also advanced the long-acting version of nomacopan (PASylated nomacopan) into the final stages of pre-clinical development as a treatment for geographic atrophy (GA).

Zevra Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Corporate Updates

Retrieved on: 
Thursday, March 28, 2024
Narcolepsy, Exercise, Somnolence, New Drug Application, Patient, Visual analogue scale, Sale, NDA, G&A, Arimoclomol, Nature, IND, Bank statement, Microsoft Access, Investment, Therapy, Priority review, Corporation, Audit committee, Research, Niemann–Pick disease, Medical science liaison, Sleep disorder, Brain, Trial of the century, Safety, Interim, Food, Sleep, QuickStart, Disease, FY, Acquisition, Emetine, UCD, COL3A1, Celiprolol, Syndrome, FDA, Marketing, Conference call, Conference, EAP, SDX, NPC, Epworth Sleepiness Scale, Idiopathic hypersomnia, Physician, PDUFA, Pharmaceutical industry

CELEBRATION, Fla., March 28, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today provided corporate updates and reported its financial results for the fourth quarter and year ended December 31, 2023. 

Key Points: 
  • ET
    CELEBRATION, Fla., March 28, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today provided corporate updates and reported its financial results for the fourth quarter and year ended December 31, 2023.
  • “We made solid progress on our key priorities in 2023,” said Neil F. McFarlane, President and Chief Executive Officer of Zevra.
  • Overview of Q4 2023 and FY 2023 Financial Results:
    Net revenue for Q4 2023 was $13.2 million, compared to prior year Q4 net revenue of $2.2 million.
  • ET, to discuss its corporate and financial results for Q4 and FY 2023.

Applied Therapeutics Provides FDA Update on PDUFA Target Action Date for Govorestat for the Treatment of Classic Galactosemia

Retrieved on: 
Thursday, March 28, 2024
Galactitol, MAA, New Drug Application, Patient, Ageing, NDA, PRV, EMA, Doctor of Philosophy, Marketing, Priority review, Therapy, ARI, Food, FDA, CHMP, PDUFA, Pharmaceutical industry

The FDA has set a new Prescription Drug User Fee Act (PDUFA) target action date of November 28, 2024.

Key Points: 
  • The FDA has set a new Prescription Drug User Fee Act (PDUFA) target action date of November 28, 2024.
  • In February 2024, the Company announced that the FDA accepted and granted Priority Review to the NDA.
  • “While the PDUFA action date extension represents a delay, we remain confident in the potential for govorestat approval for Galactosemia and we will continue to work closely with the FDA throughout the review process,” said Shoshana Shendelman, PhD, Founder and CEO of Applied Therapeutics.
  • If approved, govorestat would be the first medication indicated for the treatment of Galactosemia and would be Applied Therapeutics’ first commercial product.

Humacyte to Host Virtual KOL Event “Hemodialysis Access: A Crossroads of Care,” on March 28, 2024

Retrieved on: 
Tuesday, March 26, 2024
AV, Food, Priority review, FDA, BLA, HAV

In addition, in April 2023 Humacyte completed enrollment in a Phase 3 trial in AV Access comparing the HAV to the current standard of care, autologous arteriovenous fistula.

Key Points: 
  • In addition, in April 2023 Humacyte completed enrollment in a Phase 3 trial in AV Access comparing the HAV to the current standard of care, autologous arteriovenous fistula.
  • Top-line results are planned to be available during 2024.
  • A live question and answer session will follow the formal presentations.
  • A replay will be made available here after the call.

Tonix Pharmaceuticals Receives Rare Pediatric Disease Designation from the FDA for TNX-2900 for the Treatment of Prader-Willi Syndrome

Retrieved on: 
Monday, March 25, 2024
Eating, Prader–Willi syndrome, Obesity, Orphan drug, Appetite, Ageing, Tonix Pharmaceuticals, IND, Polyphagia, Priority review, Oxytocin, Marketing, FDA, Genetic disorder, PWS, Food, Pharmaceutical industry

CHATHAM, N.J., March 25, 2024 (GLOBE NEWSWIRE) -- Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a biopharmaceutical company with marketed products and a pipeline of development candidates, today announced the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to TNX-2900* (intranasal potentiated oxytocin), a proprietary magnesium (Mg2+)-potentiated formulation of intranasal oxytocin, to treat Prader-Willi syndrome (PWS) in children and adolescents. TNX-2900 was previously granted Orphan Drug designation by the FDA in 2022 for the treatment of PWS and the investigational new drug (IND) application was cleared by the FDA in 2023. The Company may be eligible to receive a transferable Priority Review Voucher if TNX-2900 for PWS is approved for marketing. Recently, vouchers have sold for approximately $100 million.

Key Points: 
  • TNX-2900 was previously granted Orphan Drug designation by the FDA in 2022 for the treatment of PWS and the investigational new drug (IND) application was cleared by the FDA in 2023.
  • The Company may be eligible to receive a transferable Priority Review Voucher if TNX-2900 for PWS is approved for marketing.
  • Recently, vouchers have sold for approximately $100 million.
  • “The Rare Pediatric Disease Designation is an important regulatory milestone in the development of TNX-2900.

Humacyte Fourth Quarter and Year End 2023 Financial Results and Business Update

Retrieved on: 
Friday, March 22, 2024
Diagnosis, Research, U.S. FDA, Peripheral artery disease, Infection, Patent, Buddha's hand, Evaluation, PAD, Journal, Sale, University, Doctor of Philosophy, Efficacy, Priority review, Regenerative medicine, Clinical trial, Trial of the century, Vascular surgery, HAV, Hospital, Injury, Nationwide Children's Hospital, HUMA, FACS, Amputation, Food, FDA, Defense, BLA, PDUFA, Pharmaceutical industry

DURHAM, N.C., March 22, 2024 (GLOBE NEWSWIRE) -- Humacyte, Inc. (Nasdaq: HUMA), a clinical-stage biotechnology platform company developing universally implantable, bioengineered human tissue at commercial scale, today announced financial results for the fourth quarter and year ended December 31, 2023 and highlighted recent corporate accomplishments in advancing the investigational Human Acellular Vessel (HAV) closer to planned U.S. market launch.

Key Points: 
  • There was no revenue for either the fourth quarter of 2023 or the fourth quarter of 2022, and there was no revenue for the year ended December 31, 2023.
  • Research and development expenses were $20.2 million for the fourth quarter of 2023, compared to $15.0 million for the fourth quarter of 2022, and were $76.6 million for the year ended December 31, 2023, compared to $63.3 million for the year ended December 31, 2022.
  • General and administrative expenses were $6.0 million for the fourth quarter of 2023, compared to $5.8 million for the fourth quarter of 2022, and were $23.5 million for the year ended December 31, 2023, compared to $22.9 million for the year ended December 31, 2022.
  • The 2023 increases in net loss resulted from the non-cash remeasurement of the contingent earnout liability, and operating expense increases, described above.

X4 Pharmaceuticals Reports Fourth-Quarter and Full-Year 2023 Financial Results and Provides Corporate Update

Retrieved on: 
Thursday, March 21, 2024
Security (finance), WHIM, WHIM syndrome, Conference call, Conference, RPD, New Drug Application, Ageing, Orphan, Wart, NDA, Priority review, Research and development, Immune system, File, ID, FDA, X4, PDUFA, Pharmaceutical industry

BOSTON, March 21, 2024 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today reported financial results for the fourth quarter and full year ended December 31, 2023, and highlighted key 2023 events and expected upcoming milestones.

Key Points: 
  • “Following an incredibly productive 2023, we are expecting a transformative year in 2024,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals.
  • X4 will host a conference call and webcast today at 8:30 am ET to discuss these financial results and business highlights.
  • The live webcast can be accessed on the investor relations section of X4 Pharmaceuticals’ website at www.x4pharma.com .
  • Following the completion of the call, a webcast replay of the conference call will be available on the company website.