Phase 3

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

Retrieved on: 
Wednesday, February 28, 2024

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.

NewAmsterdam Pharma Reports Full Year 2023 Financial Results and Provides Corporate Update

Retrieved on: 
Wednesday, February 28, 2024

NAARDEN, The Netherlands and MIAMI, Feb. 28, 2024 (GLOBE NEWSWIRE) -- NewAmsterdam Pharma Company N.V. (Nasdaq: NAMS or “NewAmsterdam” or the “Company”), a late-stage, clinical biopharmaceutical company developing oral, non-statin medicines for patients at risk of cardiovascular disease with elevated low-density lipoprotein cholesterol (“LDL-C”), for whom existing therapies are not sufficiently effective or well-tolerated, today announced financial results for the fourth quarter and full year ended December 31, 2023 and provided a corporate update.

Key Points: 
  • "2023 was a year of remarkable progress for NewAmsterdam, marked by consistent clinical execution and substantial corporate development.
  • NewAmsterdam completed enrollment of over 350 patients in April 2023 and expects to report topline data in the third quarter of 2024.
  • NewAmsterdam completed enrollment of over 2,500 patients in July 2023 and expects to report topline data in the fourth quarter of 2024.
  • Cash Position: As of December 31, 2023, NewAmsterdam recorded cash of $340.5 million, compared to $467.7 million as of December 31, 2022.

Capricor Therapeutics Announces Upcoming Type-B Meeting with the FDA to Discuss Commercial Manufacturing Planning with an Aim to Expedite BLA Pathway for CAP-1002 in Duchenne Muscular Dystrophy

Retrieved on: 
Tuesday, February 27, 2024

SAN DIEGO, Feb. 27, 2024 (GLOBE NEWSWIRE) -- – Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment and prevention of rare diseases, announced today that the Company has been granted an in-person Type-B meeting with the U.S. Food & Drug Administration (FDA) scheduled for late March.

Key Points: 
  • SAN DIEGO, Feb. 27, 2024 (GLOBE NEWSWIRE) -- – Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment and prevention of rare diseases, announced today that the Company has been granted an in-person Type-B meeting with the U.S. Food & Drug Administration (FDA) scheduled for late March.
  • In the upcoming meeting with the FDA, the Company intends to discuss its proposed chemistry, manufacturing and controls (CMC) plans for commercial launch, if approved which may enable opportunities to expedite the pathway to a Biologics License Application (BLA) submission.
  • The Company has previously been granted Regenerative Medicine Advanced Therapy (RMAT) and orphan drug designations for the use of CAP-1002 in Duchenne muscular dystrophy (DMD) and these designations enable sponsors to work closely with the FDA and receive its guidance on potential expedited pathways for approval.
  • “At Capricor, we remain focused on getting to approval as quickly as possible and we look forward to providing more details after this meeting.”

HARMONY BIOSCIENCES RECEIVES U.S. FOOD AND DRUG ADMINISTRATION ORPHAN DRUG DESIGNATION FOR PITOLISANT IN PRADER-WILLI SYNDROME

Retrieved on: 
Tuesday, February 20, 2024

PLYMOUTH MEETING, Pa., Feb. 20, 2024 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY), announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to pitolisant for the treatment of Prader-Willi syndrome (PWS).

Key Points: 
  • PLYMOUTH MEETING, Pa., Feb. 20, 2024 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY), announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to pitolisant for the treatment of Prader-Willi syndrome (PWS).
  • "The decision to grant Orphan Drug designation to pitolisant indicates that it could be a promising treatment option for people living with Prader-Willi syndrome," said Kumar Budur, M.D., M.S., Chief Medical Officer at Harmony Biosciences.
  • FDA Orphan Drug designation incentivizes the advancement of promising therapies for rare diseases by providing tax credits for clinical development, waivers for user fees, and seven years of market exclusivity following drug approval.
  • In the upcoming Phase 3 registrational TEMPO study, Harmony will assess the safety and efficacy of pitolisant in treating EDS and behavioral disturbances in PWS.

Acasti Announces Poster Detailing its GTX-104 STRIVE-ON Trial

Retrieved on: 
Thursday, February 1, 2024

The poster, Safety and Tolerability of Intravenous Versus Enteral Nimodipine in Patients with Aneurysmal Subarachnoid Hemorrhage, will be presented Thursday February 8th during the Ongoing Clinical Trials session of the conference.

Key Points: 
  • The poster, Safety and Tolerability of Intravenous Versus Enteral Nimodipine in Patients with Aneurysmal Subarachnoid Hemorrhage, will be presented Thursday February 8th during the Ongoing Clinical Trials session of the conference.
  • The STRIVE-ON trial is a prospective, open-label, randomized (1:1 ratio), parallel group trial of GTX-104 compared with oral nimodipine, in patients hospitalized for aSAH.
  • Key trial design features include:
    Approximately 100 patients are expected to be enrolled at an estimated 25 hospitals in the U.S.
  • The primary endpoint is safety and will be measured as comparative adverse events, including hypotension, between the two groups.

NewAmsterdam Pharma Announces 2024 Strategic Priorities

Retrieved on: 
Thursday, January 4, 2024

-- Well-capitalized with cash to support operations through BROADWAY, BROOKLYN, and PREVAIL readouts --

Key Points: 
  • “NewAmsterdam is entering 2024 on the precipice of a major transformation, with the potential to deliver significant benefit to patients globally and create value for our shareholders,” said Michael Davidson, M.D., Chief Executive Officer of NewAmsterdam.
  • NewAmsterdam completed enrollment of approximately 350 patients in April 2023 and expects to report topline data in the third quarter of 2024.
  • NewAmsterdam completed enrollment of over 2,500 patients in July 2023 and expects to report topline data in the fourth quarter of 2024.
  • NewAmsterdam expects to complete patient enrollment in PREVAIL in the first quarter of 2024 and to report topline data in 2026.

Clarity Child Guidance Center Breaks Ground on Vital Renovations to Increase Capacity for Children facing Mental Health Challenges

Retrieved on: 
Friday, December 29, 2023

SAN ANTONIO, Dec. 29, 2023 /PRNewswire-PRWeb/ -- Clarity Child Guidance Center broke ground during a special ceremony on Wednesday, September 20, 2023, on renovations being made possible through its "HEROES: The Campaign for Clarity." When complete, this $16 million capital campaign will allow Clarity to serve an additional 1,000 kids a year—bringing the annual total to an annual average of 9,000 children and teens--ensuring that every child and family who needs behavioral health services can receive them. Currently, about 60% of children treated at Clarity are uninsured, underinsured or low-income. San Antonio-based Clarity Child Guidance Center--the only nonprofit in South Texas providing a continuum of mental health care for children ages 3-17 and their families to manage conditions ranging from ADHD and anxiety to suicidal ideation, bipolar disorder and/or schizophrenia—is indeed experiencing a 27% increase in inpatient hospitalizations, 19% increase in children who were suicidal, a 65% increase in inpatient treatment of kids that have never been seen in the mental health system before, and a 159% increase in the crisis services department. (More on Clarity below.)

Key Points: 
  • SAN ANTONIO, Dec. 29, 2023 /PRNewswire-PRWeb/ -- Clarity Child Guidance Center broke ground during a special ceremony on Wednesday, September 20, 2023, on renovations being made possible through its "HEROES: The Campaign for Clarity."
  • Currently, about 60% of children treated at Clarity are uninsured, underinsured or low-income.
  • "School violence, isolation of the pandemic, and return to the classroom for the increase in demand.
  • Bexar County Judge Peter Sakai and District 8 Councilman Manny Pelaez publicly reaffirmed their commitment to youth mental health for San Antonio area.

Reviva Announces Closing of $30 Million Registered Direct Offering Priced At-the-Market Under Nasdaq Rules

Retrieved on: 
Monday, November 20, 2023

The warrants have an exercise price of $5.00 per share, are immediately exercisable and will expire five years following the date of issuance.

Key Points: 
  • The warrants have an exercise price of $5.00 per share, are immediately exercisable and will expire five years following the date of issuance.
  • H.C. Wainwright & Co. acted as the exclusive placement agent for the offering.
  • The gross proceeds to Reviva from this offering were approximately $30,000,000, before deducting the placement agent’s fees and other offering expenses.
  • The offering of the securities was made only by means of a prospectus, including a prospectus supplement, forming a part of an effective registration statement.

Web3 Infrastructure Player WalletConnect Shares Decentralization Roadmap for Web3 Communications Protocol

Retrieved on: 
Wednesday, December 20, 2023

Today, Pedro Gomes, CEO and founder of WalletConnect – a leading web3 communications protocol – announces an ambitious new roadmap to build an open, permissionless, and decentralized network that will both improve performance of the WalletConnect Protocol and achieve functionality that better aligns with the tenets of web3.

Key Points: 
  • Today, Pedro Gomes, CEO and founder of WalletConnect – a leading web3 communications protocol – announces an ambitious new roadmap to build an open, permissionless, and decentralized network that will both improve performance of the WalletConnect Protocol and achieve functionality that better aligns with the tenets of web3.
  • Over the last five years, the WalletConnect Protocol successfully established paths of interoperability linking millions of users across more than 3,000 decentralized applications and over 500 wallets.
  • Now, the WalletConnect Protocol will be able to safely transition to the purely decentralized, permissionless, and verifiable WalletConnect Network and still maintain the same reliability and low-latency that users experience today.
  • After establishing WalletConnect and building the proper infrastructure and architecture of the protocol, we’re excited for this long-awaited update,” said Gomes.

Reviva Announces $30 Million Registered Direct Offering Priced At-the-Market Under Nasdaq Rules

Retrieved on: 
Thursday, November 16, 2023

The closing of the offering is expected to occur on or about November 20, 2023, subject to the satisfaction of customary closing conditions.

Key Points: 
  • The closing of the offering is expected to occur on or about November 20, 2023, subject to the satisfaction of customary closing conditions.
  • H.C. Wainwright & Co. is acting as the exclusive placement agent for the offering.
  • The gross proceeds to Reviva from this offering are expected to be approximately $30,000,000, before deducting the placement agent’s fees and other offering expenses.
  • A prospectus supplement and accompanying prospectus relating to the offering will be filed with the SEC.