New Drug Application

GH Research Reports Full Year 2023 Financial Results and Provides Business Updates

Retrieved on: 
Thursday, February 29, 2024
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DUBLIN, Ireland, Feb. 29, 2024 (GLOBE NEWSWIRE) -- GH Research PLC (Nasdaq: GHRS), a clinical-stage biopharmaceutical company dedicated to transforming the treatment of psychiatric and neurological disorders, today reported financial results for the year ended December 31, 2023, and provided updates on its business.

Key Points: 
  • DUBLIN, Ireland, Feb. 29, 2024 (GLOBE NEWSWIRE) -- GH Research PLC (Nasdaq: GHRS), a clinical-stage biopharmaceutical company dedicated to transforming the treatment of psychiatric and neurological disorders, today reported financial results for the year ended December 31, 2023, and provided updates on its business.
  • As announced in November 2023, both trials were recruiting slower than anticipated, in part due to the closure, for business reasons, of one of the two sites activated in each trial.
  • Subsequently, we have implemented measures to strengthen recruitment of both trials, including the addition of further clinical trial sites.
  • For the trial in patients with PPD (GH001-PPD-203), we now expect completion and availability of top-line data in the third quarter of 2024.

Achieve Life Sciences Announces Pricing of up to $124.2 Million Registered Direct Offering and Concurrent Private Placement

Retrieved on: 
Thursday, February 29, 2024
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SEATTLE, Wash. and VANCOUVER, British Columbia, Feb. 29, 2024 (GLOBE NEWSWIRE) -- Achieve Life Sciences, Inc. (Nasdaq: ACHV), a late-stage pharmaceutical company committed to the global development and commercialization of cytisinicline for smoking cessation and nicotine dependence, today announced that it has entered into a securities purchase agreement with new and existing investors to raise up to approximately $124.2 million in gross proceeds that includes initial upfront funding of $60.0 million and up to an additional approximately $64.2 million upon exercise of milestone-driven warrants.

Key Points: 
  • Lake Street Capital Markets, LLC and JonesTrading Institutional Services LLC are acting as joint placement agents for the registered direct offering and concurrent private placement.
  • The registered direct offering and the concurrent private placement are each expected to close on or about March 4, 2024, subject to customary closing conditions.
  • The aggregate gross proceeds from the issuance of common stock in the registered direct offering are expected to be approximately $60.0 million, prior to deducting placement agent fees and estimated offering expenses.
  • If the milestone-driven warrants issued in the private placement are exercised in full, the company would receive $64.2 million in additional proceeds.

Pacira BioSciences Reports Fourth Quarter and Full-Year 2023 Financial Results

Retrieved on: 
Thursday, February 29, 2024
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TAMPA, Fla., Feb. 29, 2024 (GLOBE NEWSWIRE) -- Pacira BioSciences, Inc. (Nasdaq: PCRX), the industry leader in its commitment to non-opioid pain management and regenerative health solutions, today reported financial results for the fourth quarter and full-year of 2023.

Key Points: 
  • ET —
    TAMPA, Fla., Feb. 29, 2024 (GLOBE NEWSWIRE) -- Pacira BioSciences, Inc. (Nasdaq: PCRX), the industry leader in its commitment to non-opioid pain management and regenerative health solutions, today reported financial results for the fourth quarter and full-year of 2023.
  • Fourth quarter 2023 iovera° net product sales were $6.0 million, a 32% increase over the $4.6 million reported in the fourth quarter of 2022.
  • Total operating expenses were $148.1 million in the fourth quarter of 2023, versus the $181.8 million reported for the fourth quarter of 2022.
  • Cash provided by operations was $47.6 million in the fourth quarter of 2023, compared to $42.0 million in the fourth quarter of 2022.

Intercept Announces FDA Acceptance of Supplemental New Drug Application for Ocaliva® (obeticholic acid) for the Treatment of PBC

Retrieved on: 
Thursday, February 29, 2024
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The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of October 15, 2024.

Key Points: 
  • The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of October 15, 2024.
  • In this communication, the agency informed Intercept that they are planning to hold an Advisory Committee meeting to discuss the application.
  • The precedent-setting sNDA for Ocaliva is intended to satisfy the post-marketing requirements to confirm a clinical benefit in patients with PBC.
  • Intercept and Alfasigma remain committed to supporting people living with PBC and look forward to advancing discussions with the agency.

Verona Pharma Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Corporate Update

Retrieved on: 
Thursday, February 29, 2024
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LONDON and RALEIGH, N.C., Feb. 29, 2024 (GLOBE NEWSWIRE) -- Verona Pharma plc (Nasdaq: VRNA) ("Verona Pharma" or the "Company"), a clinical-stage biopharmaceutical company focused on respiratory diseases, announces its financial results for the fourth quarter and full year ended December 31, 2023, and provides a corporate update.

Key Points: 
  • The debt facility provides non-dilutive capital and further financial flexibility to support Verona Pharma’s continued growth, including the commercialization of ensifentrine.
  • Net loss: Net loss was $14.1 million for the fourth quarter ended December 31, 2023 (Q4 2022: net loss $10.5 million).
  • Verona Pharma will host an investment community webcast and conference call at 9:00 a.m. EST / 2:00 p.m. GMT on Thursday, February 29, 2024, to discuss the fourth quarter and full year 2023 financial results and the corporate update.
  • An electronic copy of the fourth quarter and full year 2023 results press release will also be made available today on the Company’s website.

GENFIT Announces Revenues and Cash Position as of December 31, 2023

Retrieved on: 
Thursday, February 29, 2024
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As of December 31, 2023, the Company’s cash and cash equivalents amounted to €77.8 million compared with €140.2 million as of December 31, 2022.

Key Points: 
  • As of December 31, 2023, the Company’s cash and cash equivalents amounted to €77.8 million compared with €140.2 million as of December 31, 2022.
  • As of September 30, 2023, cash and cash equivalents totaled €93.9 million.
  • Revenues for 2023 amounted to €28.6 million compared to €20.2 million for the same period in 2022.
  • The 2023 Universal Registration Document, the 2023 Annual Financial Report (included in the 2023 Universal Registration Document), and the Annual Report on Form 20-F will be published by the end of April 2024.

Catalyst Pharmaceuticals Reports Strong Fourth Quarter and Full Year 2023 Financial Results and Provides Business Update

Retrieved on: 
Wednesday, February 28, 2024
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Research and development expenses: In the fourth quarter of 2023, research and development expenses were $2.0 million, compared to $4.1 million in the fourth quarter of 2022.

Key Points: 
  • Research and development expenses: In the fourth quarter of 2023, research and development expenses were $2.0 million, compared to $4.1 million in the fourth quarter of 2022.
  • Selling, general, and administrative expenses: Selling, general, and administrative expenses for the fourth quarter of 2023 were $42.0 million compared to $14.1 million in the fourth quarter of 2022.
  • Amortization of intangible assets was $9.1 million in the fourth quarter of 2023), compared to $0.6 million in the fourth quarter of 2022.
  • Operating income: Operating income for the fourth quarter of 2023 was $41.7 million, compared to $30.8 million in the fourth quarter of 2022, representing an increase of approximately 35.4%.

CymaBay Reports Fourth Quarter and Year Ended December 31, 2023 Financial Results and Provides Corporate Update

Retrieved on: 
Wednesday, February 28, 2024
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NEWARK, Calif., Feb. 28, 2024 (GLOBE NEWSWIRE) -- CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet need, today announced corporate updates and financial results for the year and fourth quarter ended December 31, 2023.

Key Points: 
  • NEWARK, Calif., Feb. 28, 2024 (GLOBE NEWSWIRE) -- CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet need, today announced corporate updates and financial results for the year and fourth quarter ended December 31, 2023.
  • “2023 was a seminal year for CymaBay with critical achievements in the development of our investigational therapeutic, seladelpar.
  • Net loss for the year ended December 31, 2023 and 2022 was $105.4 million and $106.0 million, or ($0.99) and ($1.21) per share, respectively.
  • Net loss for the three months ended December 31, 2023 was higher than the three months ended December 31, 2022 primarily due to higher operating expenses.

Applied Therapeutics Announces FDA Acceptance and Priority Review of New Drug Application for Govorestat for the Treatment of Classic Galactosemia

Retrieved on: 
Wednesday, February 28, 2024
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NEW YORK, Feb. 28, 2024 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (Nasdaq: APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, today announced that the U.S. Food and Drug Administration (FDA) has accepted the filing of the New Drug Application (NDA) for govorestat (AT-007) for the treatment of Classic Galactosemia. The NDA was granted Priority Review status, and the FDA assigned a Prescription Drug User Free Act (PDUFA) target action date of August 28, 2024. The FDA also noted that it is planning to hold an advisory committee meeting to discuss the application. Govorestat was previously granted Pediatric Rare Disease designation, and will qualify for a Priority Review Voucher (PRV) upon approval.

Key Points: 
  • The NDA was granted Priority Review status, and the FDA assigned a Prescription Drug User Free Act (PDUFA) target action date of August 28, 2024.
  • Govorestat was previously granted Pediatric Rare Disease designation, and will qualify for a Priority Review Voucher (PRV) upon approval.
  • “The FDA’s acceptance of the NDA for govorestat for the treatment of Galactosemia represents a critical milestone for Applied Therapeutics and more importantly, for patients with Galactosemia and their families.
  • If approved, govorestat would be the first medication indicated for the treatment of Galactosemia and would be Applied Therapeutics’ first commercial product.

Silo Pharma Completes Successful Dose-Ranging Study of SPC-15 Intranasal Therapeutic for PTSD

Retrieved on: 
Wednesday, February 28, 2024
Therapy, Anxiety, Toxicology, New Drug Application, Data analysis, Absorption, FDA, GLP, Safety, Administration, Post-traumatic stress disorder, SILO, PTSD, IND, Pharmaceutical industry

SARASOTA, FL, Feb. 28, 2024 (GLOBE NEWSWIRE) -- Silo Pharma, Inc. (Nasdaq: SILO) (“Silo” or the “Company”), a developmental stage biopharmaceutical company focused on developing novel formulations and drug delivery systems for traditional therapeutics and psychedelic treatments, today announced positive results from its non-GLP small animal dose-ranging study of SPC-15, an intranasal prophylactic treatment for anxiety and post-traumatic stress disorder (PTSD).

Key Points: 
  • The purpose of the non-GLP small animal study was to identify the maximum tolerated intranasal dose of the drug pharmacokinetic and determine the therapeutically relevant dose range to be studied in clinical trials.
  • Data analysis demonstrated rapid absorption of SPC-15 with good exposure over a 24-hour period, suggesting a once-per-day human intranasal dosing regimen would be optimal.
  • “Non-GLP and GLP toxicology and safety pharmacology studies are required steps before seeking approval for our first-in-human clinical-stage trials of SPC-15,” said Eric Weisblum, CEO of Silo.
  • This is an exciting step forward for our Company as we advance our lead therapeutic, SPC-15.”