Medication

Mirum Pharmaceuticals’ LIVMARLI (maralixibat oral solution) Receives Positive Reimbursement Recommendation by Canada’s CADTH for Patients with Cholestatic Pruritus in Alagille Syndrome

Retrieved on: 
Tuesday, April 2, 2024
Biotechnology, Pharmaceutical, Health, Clinical Trials, Itch, Medication, Alagille syndrome, Liver disease, Inflammation, Patient, Cholestasis, Bile, Cholestatic pruritus, CADTH, Liver injury, The Hospital, Liver, CDEC, Genetic disorder, PFIC, Pharmaceutical industry

“The positive reimbursement recommendation for LIVMARLI by CADTH is an important step forward for patients suffering from the debilitating effects of cholestatic pruritus related to Alagille syndrome,” said Chris Peetz, chief executive officer at Mirum.

Key Points: 
  • “The positive reimbursement recommendation for LIVMARLI by CADTH is an important step forward for patients suffering from the debilitating effects of cholestatic pruritus related to Alagille syndrome,” said Chris Peetz, chief executive officer at Mirum.
  • These patients suffer greatly from the debilitating and disruptive effects of Alagille syndrome, which often starts in early childhood.”
    “CADTH’s recommendation of reimbursement for LIVMARLI provides a meaningful advancement in the treatment of cholestatic pruritus for patients with Alagille syndrome.
  • LIVMARLI is also approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. and Europe, and for Progressive Familial Intrahepatic Cholestasis (PFIC) in the U.S. and in Europe.
  • Cholestasis in ALGS is associated with pruritus which is among the most common indications for liver transplant in ALGS.

Melt Pharmaceuticals Closes $24 Million Series B Preferred Stock Financing

Retrieved on: 
Tuesday, April 2, 2024
Medical Supplies, Biotechnology, FDA, Health, Pharmaceutical, Optical, Health Technology, Medical Devices, Hospitals, Surgery, Clinical Trials, Catalent, Series, Absorption, Midazolam, Ketamine, Medication

Melt Pharmaceuticals, Inc. (“Melt”), a clinical‑stage pharmaceutical company developing novel approaches for procedural sedation, today announced the completion of its Series B Preferred Stock financing of approximately $24 million from new and existing investors at a pre‑money valuation that increased nearly 150% from the pre-money valuation for Melt’s Series A Preferred Stock financing in 2019.

Key Points: 
  • Melt Pharmaceuticals, Inc. (“Melt”), a clinical‑stage pharmaceutical company developing novel approaches for procedural sedation, today announced the completion of its Series B Preferred Stock financing of approximately $24 million from new and existing investors at a pre‑money valuation that increased nearly 150% from the pre-money valuation for Melt’s Series A Preferred Stock financing in 2019.
  • The capital raised will primarily support the further development of MELT-300, including the MELT-300 Phase 3 program.
  • “With this round of financing, we’ve obtained the funding to further the development of MELT-300, including conducting our recently commenced Phase 3 program.
  • Their shared belief in the vision of MELT-300 and their confidence in its potential to revolutionize short-duration procedural sedation is incredibly valuable to us.”
    Newbridge Securities Corporation acted as the exclusive placement agent for the Series B Preferred Stock Offering.

Longboard Pharmaceuticals Announces Inducement Grants Under Nasdaq Listing Rule 5635(C)(4)

Retrieved on: 
Tuesday, April 2, 2024
Biotechnology, Neurology, Health, Pharmaceutical, Clinical Trials, Common stock, Longboard, Employment, Compensated emancipation, Medication, DSM-IV codes

Longboard Pharmaceuticals, Inc. (Nasdaq: LBPH), a clinical-stage biopharmaceutical company focused on developing novel, transformative medicines for neurological diseases, today announced the grant of inducement awards to three new employees.

Key Points: 
  • Longboard Pharmaceuticals, Inc. (Nasdaq: LBPH), a clinical-stage biopharmaceutical company focused on developing novel, transformative medicines for neurological diseases, today announced the grant of inducement awards to three new employees.
  • The Compensation Committee of the Board of Directors of Longboard approved the grants of non-qualified stock options to purchase an aggregate of 99,500 shares of its common stock (the “Common Stock”) as inducements material to the employees entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4).
  • The stock options were granted on March 31, 2024 (the “Grant Date”) and have an exercise price of $21.60 per share, which is equal to the closing price of the Common Stock on the last trading day preceding the Grant Date.
  • The stock options were granted as an inducement material to the employees entering into employment with Longboard in accordance with Nasdaq Listing Rule 5635(c)(4) and are subject to the terms and conditions of the applicable award agreement covering such grant.

Rocket Pharmaceuticals Announces European Medicines Agency Acceptance of RP-L102 Marketing Authorization Application for the Treatment of Fanconi Anemia

Retrieved on: 
Tuesday, April 2, 2024
Biotechnology, Pharmaceutical, Oncology, General Health, Health, Genetics, Clinical Trials, Other Health, Transplant, DNA repair, Medication, Biologics license application, MAA, Risk, FANCA, Patient, Fanconi anemia, Pharming, DNA, BMF, HSCT, EMA, Bone marrow failure, Death, Birth, Life, MBA, European Medicines Agency, Marketing, FA, Disorder, FDA, BLA, Genetic disorder, Food

“The acceptance of the MAA for RP-L102 marks an important step forward in our goal of bringing this potential gene therapy treatment to patients impacted by this devastating childhood disorder.

Key Points: 
  • “The acceptance of the MAA for RP-L102 marks an important step forward in our goal of bringing this potential gene therapy treatment to patients impacted by this devastating childhood disorder.
  • Currently, there are no existing options to potentially prevent BMF for patients with FA,” said Kinnari Patel, Pharm.D., MBA, President, Head of R&D and Chief Operating Officer, Rocket Pharma.
  • The safety profile was highly favorable with no significant safety signals, and the treatment, administered without any cytotoxic conditioning, was well tolerated.
  • The Biologics License Application (BLA) for FA remains on track for submission to the U.S. Food and Drug Administration (FDA) in the first half of 2024.

Otsuka and Click Therapeutics Announce the U.S. Food and Drug Administration (FDA) Clearance of Rejoyn™, the First Prescription Digital Therapeutic Authorized for the Adjunctive Treatment of Major Depressive Disorder (MDD) Symptoms

Retrieved on: 
Monday, April 1, 2024
Biotechnology, FDA, Health, Pharmaceutical, Clinical Trials, Depression, Psychiatry, Brain, Mirai, Medication, MDD, Patient, DSM-IV codes, Therapy, Cognition, Regulation, Otsuka Pharmaceutical, MADRS, Multimedia, Clinical Global Impression, Emotion, Food, Pharmaceutical industry

Unlike wellness apps, the Rejoyn app is a medical device authorized by the FDA for prescription by a healthcare professional.

Key Points: 
  • Unlike wellness apps, the Rejoyn app is a medical device authorized by the FDA for prescription by a healthcare professional.
  • “While traditional approaches are often effective, many are left with only a partial response to treatment.
  • “The clearance of Rejoyn signals a fundamental change in how clinicians can treat symptoms of major depressive disorder.
  • Rejoyn does not monitor the patient’s symptoms or clinical status and cannot send or receive alerts or warnings to the prescriber.

Pyros Pharmaceuticals Announces VIGPODER™ (vigabatrin) is Now Available

Retrieved on: 
Monday, April 1, 2024
Parenting, FDA, Neurology, Children, Biotechnology, Family, Health, Consumer, Pharmaceutical, Diagnosis, Epilepsy, Medication, USP, Pyros, Caregiver, Patient, Epileptic spasms, Child, Vigabatrin, Spasm, Safety, Multimedia, Central Time, Pharmaceutical industry

Pyros Pharmaceuticals, Inc. (Pyros or the Company), a leader in the development of enhanced specialty pharmaceuticals for rare diseases, announced today that VIGPODER™ (vigabatrin) for oral solution, USP, a treatment for appropriate patients with infantile spasms (IS), is now available.

Key Points: 
  • Pyros Pharmaceuticals, Inc. (Pyros or the Company), a leader in the development of enhanced specialty pharmaceuticals for rare diseases, announced today that VIGPODER™ (vigabatrin) for oral solution, USP, a treatment for appropriate patients with infantile spasms (IS), is now available.
  • As a therapeutically equivalent product, VIGPODER™ is anticipated to offer the same safety and efficacy profile as SABRIL® (vigabatrin) for oral solution.
  • Additionally, Pyros Total Care™, the Company’s personalized comprehensive support program, is available to assist families throughout the treatment journey.
  • "The launch of VIGPODER™ and the Pyros Total Care™ program underscores our dedication to supporting families facing the challenges of infantile spasms," stated Michael Smith, Chief Executive Officer at Pyros.

Bristol Myers Squibb Announces Pivotal KRYSTAL-12 Confirmatory Trial Evaluating KRAZATI (adagrasib) Meets Primary Endpoint of Progression-Free Survival for Patients with Pretreated KRASG12C-Mutated Locally Advanced or Metastatic Non-Small Cell Lung…

Retrieved on: 
Thursday, March 28, 2024
Oncology, FDA, Health, Clinical Trials, Pharmaceutical, Biotechnology, Civil service commission, BMY, NSCLC, Cetuximab, Progression-free survival, Patient, Neoplasm, Shanda, Health care, News, Bristol Myers Squibb, MHRA, Pancreatic cancer, CRC, Safety, European Commission, PFS, PDUFA, Medication, Pharmaceutical industry

The study remains ongoing to assess the additional key secondary endpoint of overall survival.

Key Points: 
  • The study remains ongoing to assess the additional key secondary endpoint of overall survival.
  • Results of the confirmatory trial showed that KRAZATI demonstrated a statistically significant and clinically meaningful benefit in PFS and ORR compared to standard-of-care chemotherapy as a second-line or later treatment for these patients.
  • “Today’s news is an important reinforcement of the power of a targeted therapy for patients with locally advanced or metastatic KRASG12C-mutated lung cancer.
  • Bristol Myers Squibb thanks the patients and investigators involved in the KRYSTAL-12 clinical trial.

Ironwood Pharmaceuticals Announces Positive Results from its Phase II Exploratory STARGAZE Trial of Apraglutide in Patients with Steroid-Refractory Gastrointestinal Acute Graft-versus-Host Disease (SR GI aGVHD)

Retrieved on: 
Thursday, March 28, 2024
Biotechnology, Pharmaceutical, Health, Clinical Trials, Principal, Disease, GVHD, Regeneration, Medication, Ironwood Pharmaceuticals, Patient, University, GI, Mortality, Standard of care, HSCT, Senior, NRM, Skin, Safety, MAGIC, Hematology, Liver, Phase II, Pharmaceutical industry

Ironwood Pharmaceuticals, Inc. (Nasdaq: IRWD), a GI-focused healthcare company, today announced positive, primary results up to Day 91 for its Phase II exploratory STARGAZE trial evaluating apraglutide in patients with steroid-refractory gastrointestinal acute Graft-versus-Host Disease (SR GI aGVHD).

Key Points: 
  • Ironwood Pharmaceuticals, Inc. (Nasdaq: IRWD), a GI-focused healthcare company, today announced positive, primary results up to Day 91 for its Phase II exploratory STARGAZE trial evaluating apraglutide in patients with steroid-refractory gastrointestinal acute Graft-versus-Host Disease (SR GI aGVHD).
  • This was a first-of-its-kind study combining a GLP-2 analog with immunosuppressive therapy in patients with SR GI aGVHD.
  • GI aGVHD, is a leading cause of morbidity and mortality following HSCT with approximately 70% of aGVHD cases having GI involvement.
  • The STARGAZE study will continue through its two-year endpoint, where apraglutide will be re-evaluated for safety and efficacy.

Amneal to Ring the Nasdaq Closing Bell on April 2, 2024

Retrieved on: 
Thursday, March 28, 2024
Health, General Health, Research, Pharmaceutical, Science, Biotechnology, Growth, Medication

Amneal Pharmaceuticals, Inc. (Nasdaq: AMRX) (“Amneal” or the “Company”), a global pharmaceutical company, today announced that Chirag and Chintu Patel, Co-founders and Co-Chief Executive Officers, will ring the Nasdaq closing bell on Tuesday, April 2, 2024.

Key Points: 
  • Amneal Pharmaceuticals, Inc. (Nasdaq: AMRX) (“Amneal” or the “Company”), a global pharmaceutical company, today announced that Chirag and Chintu Patel, Co-founders and Co-Chief Executive Officers, will ring the Nasdaq closing bell on Tuesday, April 2, 2024.
  • “As entrepreneurs who founded Amneal in 2002, we are pleased to join the esteemed roster of Nasdaq-listed companies.
  • Today Amneal is a growing global pharmaceutical leader with a diverse portfolio of essential medicines that help make healthy possible.
  • We thank the entire Amneal family for their tremendous dedication and commitment to those we serve, and we are excited for our next era of growth and success,” said Chirag and Chintu Patel.

Merrimack Receives $225 Million Milestone Payment from Ipsen

Retrieved on: 
Wednesday, March 27, 2024
Biotechnology, FDA, Health, Pharmaceutical, Oncology, ANY, Medication, Name, Security (finance), Proxy, Pancreas, Special, Food, Person

Merrimack Pharmaceuticals, Inc. (Nasdaq: MACK) (“Merrimack” or the “Company”) today announced that it has received a $225 million payment which was due from Ipsen, S.A. as a result of its receipt of approval from the U.S. Food and Drug Administration, or FDA, to market ONIVYDE as a first-line treatment of metastatic adenocarcinoma on the pancreas.

Key Points: 
  • Merrimack Pharmaceuticals, Inc. (Nasdaq: MACK) (“Merrimack” or the “Company”) today announced that it has received a $225 million payment which was due from Ipsen, S.A. as a result of its receipt of approval from the U.S. Food and Drug Administration, or FDA, to market ONIVYDE as a first-line treatment of metastatic adenocarcinoma on the pancreas.
  • Merrimack’s Board of Directors has evaluated the likelihood of receiving additional milestone payments under the Ipsen Agreement and from the 2019 Agreement with Elevation Oncology and has concluded that it is unlikely that any additional milestone payments from either agreement will become payable.
  • We currently anticipate the initial liquidating dividend to be in the range of between approximately $14.68 and $15.30 per share.
  • The Plan of Dissolution will include establishment of a liquidating trust for the benefit of stockholders in the unlikely event that Merrimack might receive any future milestone payments from Ipsen or Elevation Technology.