Pharmacology

Krystal Biotech Announces Fourth Quarter and Full Year 2023 Financial Results and Provides Business Updates

Retrieved on: 
Monday, February 26, 2024

(the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company, today reported financial results and key business updates for the fourth quarter and year ending December 31, 2023.

Key Points: 
  • (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company, today reported financial results and key business updates for the fourth quarter and year ending December 31, 2023.
  • Financial results for the quarter ended December 31, 2023:
    Cash, cash equivalents, and investments totaled $594.1 million on December 31, 2023.
  • The Company recorded net product revenue of $42.1 million from sales of VYJUVEK during the quarter ended December 31, 2023.
  • For additional information on the Company’s financial results for the year ended December 31, 2023, please refer to the Form 10-K filed with the Securities and Exchange Commission.

Avenue Therapeutics Announces Publication in Drug Development Research Highlighting First-In-Class Preclinical Data of BAER-101 in a Translational Model of Absence Epilepsy

Retrieved on: 
Thursday, February 22, 2024

MIAMI, Feb. 22, 2024 (GLOBE NEWSWIRE) -- Avenue Therapeutics, Inc. (Nasdaq: ATXI) (“Avenue” or the “Company”), a specialty pharmaceutical company focused on the development and commercialization of therapies for the treatment of neurologic diseases, today announced the publication of preclinical in vivo data in Drug Development Research highlighting BAER-101’s full suppression of seizure activity using the Genetic Absence Epilepsy Rats from Strasbourg (“GAERS”) model of absence epilepsy.

Key Points: 
  • MIAMI, Feb. 22, 2024 (GLOBE NEWSWIRE) -- Avenue Therapeutics, Inc. (Nasdaq: ATXI) (“Avenue” or the “Company”), a specialty pharmaceutical company focused on the development and commercialization of therapies for the treatment of neurologic diseases, today announced the publication of preclinical in vivo data in Drug Development Research highlighting BAER-101’s full suppression of seizure activity using the Genetic Absence Epilepsy Rats from Strasbourg (“GAERS”) model of absence epilepsy.
  • The publication describes the extent of anti-seizure activity of BAER-101 in the GAERS model, a widely used and translationally relevant animal model.
  • The study demonstrated full suppression of seizure activity with a minimal effective dose (MED) of 0.3 mg/kg.
  • “The preclinical data published in Drug Development Research demonstrate BAER-101’s ability to fully suppress seizures in the GAERS model, a translational animal model for anti-seizure drug development with a documented high predictability of response in humans.

CRISPR Therapeutics Provides Business Update and Reports Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Wednesday, February 21, 2024

R&D Expenses: R&D expenses were $95.1 million for the fourth quarter of 2023, compared to $103.6 million for the fourth quarter of 2022.

Key Points: 
  • R&D Expenses: R&D expenses were $95.1 million for the fourth quarter of 2023, compared to $103.6 million for the fourth quarter of 2022.
  • G&A Expenses: General and administrative expenses were $16.5 million for the fourth quarter of 2023, compared to $21.2 million for the fourth quarter of 2022.
  • Collaboration Expense: Collaboration expense, net, was $20.0 million for the fourth quarter of 2023, compared to $6.8 million for the fourth quarter of 2022.
  • Net Income (Loss): Net income was $89.3 million for the fourth quarter of 2023, compared to a net loss of $110.6 million for the fourth quarter of 2022.

Seismic Therapeutic to Present Preclinical Data for Novel Immunoglobulin Sculpting Enzyme at AAN Annual Meeting

Retrieved on: 
Friday, March 8, 2024

Seismic Therapeutic, Inc ., the machine learning immunology company, today announced that the company will present preclinical data for its pan-immunoglobulin G (IgG) sculpting enzyme candidate, S-1117, at the American Academy of Neurology (AAN) annual meeting taking place on April 13-18 in Denver.

Key Points: 
  • Seismic Therapeutic, Inc ., the machine learning immunology company, today announced that the company will present preclinical data for its pan-immunoglobulin G (IgG) sculpting enzyme candidate, S-1117, at the American Academy of Neurology (AAN) annual meeting taking place on April 13-18 in Denver.
  • S-1117 is a novel engineered pan-IgG protease for the potential treatment of a range of chronic and acute autoantibody mediated diseases, including the chronic neuromuscular autoimmune disorder, myasthenia gravis.
  • The presentation at the AAN annual meeting will outline key preclinical in vitro and in vivo results supporting the differentiated profile of S-1117 compared to therapeutic benchmarks in chronic autoantibody mediated diseases.
  • Title: Preclinical Pharmacology of S-1117, a Novel Engineered Fc-fused IgG Cleaving Enzyme, for Chronic Treatment of Autoantibody-mediated Diseases

NTx Appoints Steven Evans as Chief Scientific Officer

Retrieved on: 
Tuesday, February 27, 2024

Nature’s Toolbox (NTx) a life sciences company building next generation platforms to manufacture RNA and protein, today announced the appointment of Steven Evans, Ph.D. as Chief Scientific Officer and Senior Vice President of R&D.

Key Points: 
  • Nature’s Toolbox (NTx) a life sciences company building next generation platforms to manufacture RNA and protein, today announced the appointment of Steven Evans, Ph.D. as Chief Scientific Officer and Senior Vice President of R&D.
  • View the full release here: https://www.businesswire.com/news/home/20240227674119/en/
    NTx has appointed Steven Evans, Ph.D. as Chief Scientific Officer and Senior Vice President of R&D.
  • At NTx, Steven will leverage his extensive skills in executive leadership, strategic planning, and cross-collaboration to drive the company’s R&D initiatives to new heights.
  • He has built an outstanding team of scientists and positioned the company for accelerated growth,” said Jamie Coffin, CEO of NTx.

AceLink Therapeutics Announces Publication of Phase 1 Clinical Trial Data Evaluating AL01211 in Healthy Volunteers

Retrieved on: 
Monday, February 26, 2024

AL01211 is a potent, oral, glucosylceramide synthase (GCS) inhibitor being developed for the treatment of Fabry disease and Type 1 Gaucher disease.

Key Points: 
  • AL01211 is a potent, oral, glucosylceramide synthase (GCS) inhibitor being developed for the treatment of Fabry disease and Type 1 Gaucher disease.
  • “These results highlight the transformative potential of AL01211 as a best-in-class GCS inhibitor,” said Jerry Shen, Ph.D., Chief Executive Officer and Founder of AceLink Therapeutics.
  • “Our Phase 1 study provided the critical safety and biomarker data to support our ongoing Phase 2 clinical trial in patients with Fabry Disease.
  • “In our trial, AL01211 demonstrated dose-dependent PK and PD effects with a clear correlation between AL01211 exposure and reduction in disease biomarkers.

CIBC Innovation Banking Provides Growth Capital to InsightRX

Retrieved on: 
Friday, February 23, 2024

CIBC Innovation Banking announced today that it has provided growth financing to InsightRX, a San Francisco-based software company leveraging quantitative pharmacology and artificial intelligence (AI) to improve therapeutic decision-making from phase I drug development to the point of patient care.

Key Points: 
  • CIBC Innovation Banking announced today that it has provided growth financing to InsightRX, a San Francisco-based software company leveraging quantitative pharmacology and artificial intelligence (AI) to improve therapeutic decision-making from phase I drug development to the point of patient care.
  • InsightRX applies patient-specific data and quantitative models to tailor dosing for complex treatments, including antibiotics, chemotherapeutic agents, and immunosuppressants.
  • To help us achieve our strategic vision of transforming patient care, we worked with CIBC for the necessary financing to accelerate our growth.
  • CIBC showed intellectual curiosity about the intricacies of our business and were thoughtful in their approach,” said Sirj Goswami, Ph.D., CEO and co-founder of InsightRX.

Coya Therapeutics to Present Novel Biomarker Survival Data in Large Cohort of ALS (Amyotrophic Lateral Sclerosis) Patients at Society of Neuroimmune Pharmacology Conference

Retrieved on: 
Thursday, February 22, 2024

Coya Therapeutics, Inc. (Nasdaq: COYA) (“Coya” or the “Company”), a clinical-stage biotechnology company developing biologics intended to enhance regulatory T cell (Treg) function, announces that Dr. Stanley Appel, M.D., Chairman of Coya’s Scientific Advisory Board, will present data on its novel oxidative stress biomarker candidate from a large cohort of Amyotrophic Lateral Sclerosis (ALS) patients at the upcoming Society of Neuroimmune Pharmacology Conference being held on March 10-13, 2024 in Charleston, SC.

Key Points: 
  • Coya Therapeutics, Inc. (Nasdaq: COYA) (“Coya” or the “Company”), a clinical-stage biotechnology company developing biologics intended to enhance regulatory T cell (Treg) function, announces that Dr. Stanley Appel, M.D., Chairman of Coya’s Scientific Advisory Board, will present data on its novel oxidative stress biomarker candidate from a large cohort of Amyotrophic Lateral Sclerosis (ALS) patients at the upcoming Society of Neuroimmune Pharmacology Conference being held on March 10-13, 2024 in Charleston, SC.
  • Dr. Appel’s presentation will be on Tuesday, March 12th, 2024, and is titled Immunomodulatory Therapy in Neurodegenerative Disease: Lessons from ALS.
  • Dr. Fred Grossman, Coya’s Chief Medical Officer, stated: “Neurofilament Light Chain (NFL), despite its lack of specificity for ALS, is currently the only robust and validated biomarker used for diagnostic and prognostic performance in symptomatic ALS patients.
  • We believe that our preliminary novel biomarker data may facilitate new opportunities to better predict patient survival, monitor disease progression, and track efficacy of therapies and will be an important part of the analysis in the upcoming Ph.

Manatt Adds Renowned Provider Executive to National Health Care Industry Group

Retrieved on: 
Tuesday, February 20, 2024

Manatt, Phelps & Phillips, LLP, a multidisciplinary, integrated professional services firm, announced today that Managing Director Mark Wietecha has joined the Firm’s national Health Care industry group in Washington, D.C.

Key Points: 
  • Manatt, Phelps & Phillips, LLP, a multidisciplinary, integrated professional services firm, announced today that Managing Director Mark Wietecha has joined the Firm’s national Health Care industry group in Washington, D.C.
  • Additionally, Wietecha has a career interest in and deep global knowledge of children’s hospitals from both a policy and delivery systems perspective.
  • “In addition to his leadership with CHA, Mark has worked with hundreds of hospitals to understand the opportunities and challenges associated with their missions to increase access to and improve the delivery of care for children,” said Manatt Health Leader Bill Bernstein .
  • Prior to joining CHA, Wietecha served as a senior partner and executive chairman of a global consulting firm and founded its academic medical center consultancy within the firm’s health care sector.

"Double Life" of Key Immune Protein Reveals New Strategies for Treating Cancer and Autoimmune Diseases

Retrieved on: 
Friday, March 8, 2024

The same findings also support experimental treatment strategies for autoimmune diseases, in which the immune system attacks the body, because stimulating the action of PD-1, as opposed to restricting it, can potentially block an overactive immune response.

Key Points: 
  • The same findings also support experimental treatment strategies for autoimmune diseases, in which the immune system attacks the body, because stimulating the action of PD-1, as opposed to restricting it, can potentially block an overactive immune response.
  • The study results revolve around the body's immune system, which is primed to attack virally infected and cancerous cells while leaving normal cells alone.
  • The immune system recognizes tumors as abnormal, but cancer cells can hijack checkpoints to turn off immune responses.
  • At the same time, PD-1 signaling is slowed in autoimmune diseases like rheumatoid arthritis, lupus, and type 1 diabetes, such that the action of unchecked immune cells creates inflammation that can damage tissues.