Medication

Alnylam Reports Positive KARDIA-2 Topline Study Results Demonstrating Clinically Significant Blood Pressure Reductions When Zilebesiran is Added to Standard of Care Antihypertensives

Retrieved on: 
Tuesday, March 5, 2024

Zilebesiran demonstrated an encouraging safety and tolerability profile when added to these standard of care antihypertensives.

Key Points: 
  • Zilebesiran demonstrated an encouraging safety and tolerability profile when added to these standard of care antihypertensives.
  • “We are thrilled that a single dose of zilebesiran achieved clinically significant, additional reductions in systolic blood pressure when administered to patients who are not adequately controlled with commonly prescribed antihypertensives,” said Simon Fox, Ph.D., Vice President, Zilebesiran Program Lead at Alnylam.
  • “These KARDIA-2 results, showing durable additional levels of blood pressure reduction on top of what is achieved by standard of care first-line antihypertensives with an encouraging safety profile, reinforce our confidence in zilebesiran’s differentiated profile.
  • The primary endpoint is the change from baseline in mean SBP at Month 3, assessed by 24-hour ABPM.

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

Retrieved on: 
Tuesday, March 5, 2024

Marinus Pharmaceuticals , Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today reported business highlights and financial results for the fourth quarter and year ended December 31, 2023.

Key Points: 
  • Marinus Pharmaceuticals , Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today reported business highlights and financial results for the fourth quarter and year ended December 31, 2023.
  • For the fiscal year 2024, the Company expects ZTALMY U.S. net product revenues of between $32 and $34 million.
  • At December 31, 2023, the Company had cash, cash equivalents and short-term investments of $150.3 million, compared to cash and cash equivalents of $240.6 million at December 31, 2022.
  • Selected Financial Data (in thousands, except share and per share amounts)

Biomunex Signs Exclusive Licensing Agreement With Institut Curie to Develop MAIT Engagers, a New Class of Bispecific Antibodies in Immuno-oncology

Retrieved on: 
Monday, March 4, 2024

This agreement covers the development of a new class of antibodies, capable of specifically targeting and engaging MAIT cells to kill cancer cells.

Key Points: 
  • This agreement covers the development of a new class of antibodies, capable of specifically targeting and engaging MAIT cells to kill cancer cells.
  • Biomunex, which was already co-inventor and a 50% co-owner of this unique innovative approach, now holds full worldwide rights.
  • In addition, MAIT cells present the MDR-12 protein on their surface, providing them with a natural resistance to some major chemotherapies.
  • “The research performed at Institut Curie has led to a major discovery which now opens up promising new therapeutic options.

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

Retrieved on: 
Friday, March 1, 2024

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 four 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 four 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 184,000 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 February 29, 2024 (the “Grant Date”) and have an exercise price of $22.18 per share, which is equal to the closing price of the Common Stock on 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.

Amneal Reports Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Friday, March 1, 2024

Amneal Pharmaceuticals, Inc. (Nasdaq: AMRX) (“Amneal” or the “Company”) today announced its results for the fourth quarter and full year ended December 31, 2023.

Key Points: 
  • Amneal Pharmaceuticals, Inc. (Nasdaq: AMRX) (“Amneal” or the “Company”) today announced its results for the fourth quarter and full year ended December 31, 2023.
  • Net revenue in the fourth quarter of 2023 was $617 million, an increase of 1% compared to $610 million in the fourth quarter of 2022.
  • Adjusted EBITDA(1) in the fourth quarter of 2023 was $142 million, a decrease of 8% compared to the fourth quarter of 2022, primarily due to investments in research and development and commercial to drive future growth.
  • Adjusted diluted EPS(1) in the fourth quarter of 2023 was $0.14, a decrease of 39% from $0.23 in the fourth quarter of 2022.

Hepatology Publishes Six-Year Data Demonstrating Improved Clinical Outcomes in Patients with Alagille Syndrome Treated with Mirum’s LIVMARLI

Retrieved on: 
Thursday, February 29, 2024

The analysis evaluated time to clinical outcome from the pooled LIVMARLI clinical studies versus a control cohort from the Global Alagille Alliance (GALA) clinical database, the largest global ALGS natural history database.

Key Points: 
  • The analysis evaluated time to clinical outcome from the pooled LIVMARLI clinical studies versus a control cohort from the Global Alagille Alliance (GALA) clinical database, the largest global ALGS natural history database.
  • The GALA control group was identified based on a pre-specified and blinded selection process to align with eligibility criteria from the LIVMARLI clinical studies.
  • Data from the analysis demonstrated a significant improvement in six-year event-free survival with a p-value of
  • The full publication including additional data from the analysis is available on the Hepatology website.

Ironwood Pharmaceuticals Announces Positive Topline Results from Global Phase III Trial of Once-Weekly Apraglutide in Adults with Short Bowel Syndrome with Intestinal Failure (SBS-IF)

Retrieved on: 
Thursday, February 29, 2024

Ironwood Pharmaceuticals, Inc. (Nasdaq: IRWD), a GI-focused healthcare company, today announced positive topline results from its pivotal Phase III STARS trial, which evaluated the efficacy and safety of once-weekly subcutaneous apraglutide in reducing parenteral support (PS) dependency in adult patients with short bowel syndrome with intestinal failure (SBS-IF).

Key Points: 
  • Ironwood Pharmaceuticals, Inc. (Nasdaq: IRWD), a GI-focused healthcare company, today announced positive topline results from its pivotal Phase III STARS trial, which evaluated the efficacy and safety of once-weekly subcutaneous apraglutide in reducing parenteral support (PS) dependency in adult patients with short bowel syndrome with intestinal failure (SBS-IF).
  • Based on these results, Ironwood plans to submit a new drug application (NDA) and other regulatory filings for apraglutide for use in adult patients with SBS who are dependent on PS.
  • The global, multicenter, double-blind, randomized, placebo-controlled trial evaluated the efficacy and safety of weekly subcutaneous injections of apraglutide in adult patients with SBS-IF.
  • Topline safety results were generally consistent with the safety profile demonstrated in apraglutide studies to date.

Mirum Pharmaceuticals Reports Fourth Quarter and Year-End 2023 Financial Results and Provides Business Update

Retrieved on: 
Wednesday, February 28, 2024

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported financial results for the fourth quarter and year-end 2023 and provided a business update.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported financial results for the fourth quarter and year-end 2023 and provided a business update.
  • 2023 LIVMARLI net product sales totaled $141.8 million, representing approximately 89% growth over 2022 net product sales.
  • As of December 31, 2023, Mirum had cash and cash equivalents of $286.3 million.
  • The foregoing financial information is unaudited and subject to change, and actual results may vary from the foregoing.

Alnylam to Webcast Presentations at Upcoming March Investor Conferences

Retrieved on: 
Tuesday, February 27, 2024

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that management will present company overviews at the following conferences:

Key Points: 
  • Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that management will present company overviews at the following conferences:
    TD Cowen 44th Annual Health Care Conference on Tuesday, March 5, 2024 at 9:10 am ET in Boston
    Barclays 26th Annual Global Healthcare Conference on Tuesday, March 12, 2024 at 10:15 am ET in Miami
    Stifel 2024 CNS Day on Wednesday, March 20, 2024 at 10:00 am ET being held virtually
    Canaccord Genuity “Genetic Medicine for Generalists” on Monday, March 25, 2024 at 1:00 pm ET being held virtually
    A live audio webcast of each presentation will be available on the Investors section of the Company’s website at www.alnylam.com/events .
  • A replay will be available on the Alnylam website within 48 hours after each event.

Amneal Enters into Exclusive European Licensing Agreement with Zambon Biotech for IPX203

Retrieved on: 
Tuesday, February 27, 2024

Amneal Pharmaceuticals, Inc. (NASDAQ: AMRX) (“Amneal” or the “Company”) today announced that it has entered into an exclusive licensing agreement with Zambon Biotech SA, part of the Zambon group (“Zambon”), for IPX203 in the European Union, United Kingdom, and Switzerland.

Key Points: 
  • Amneal Pharmaceuticals, Inc. (NASDAQ: AMRX) (“Amneal” or the “Company”) today announced that it has entered into an exclusive licensing agreement with Zambon Biotech SA, part of the Zambon group (“Zambon”), for IPX203 in the European Union, United Kingdom, and Switzerland.
  • Zambon will seek regulatory approval and commercialize IPX203 in Europe.
  • Today’s announcement of a licensing agreement with a European partner is part of Amneal’s broader strategy to bring IPX203 to Parkinson’s patients around the world.
  • Earlier this year, Amneal also signed a license agreement with Knight Therapeutics Inc. granting exclusive rights to seek regulatory approval and commercialize IPX203 in Canada and Latin America.