Degenerative disease

Eloxx Pharmaceuticals Announces First Patients Enrolled in Phase 2 Clinical Study Evaluating ELX-02 for the Treatment of Alport Syndrome

Retrieved on: 
Wednesday, January 25, 2023
RNA, Alport syndrome, Safety, Patient, Proteinuria, VII, Mutation, COL4, COL, Cell, Degenerative disease, Pharmaceutical industry, Alport

WATERTOWN, Mass., Jan. 25, 2023 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today announced that the first patients have now been enrolled in its Phase 2 study of ELX-02 for the treatment of Alport syndrome in patients with nonsense mutations.

Key Points: 
  • WATERTOWN, Mass., Jan. 25, 2023 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today announced that the first patients have now been enrolled in its Phase 2 study of ELX-02 for the treatment of Alport syndrome in patients with nonsense mutations.
  • “With enrollment of the first patients in our Phase 2 study of ELX-02 for the treatment of Alport syndrome in patients with nonsense mutations, we remain on track to deliver topline clinical results from this trial in the first half of 2023,” said Sumit Aggarwal, President and Chief Executive Officer of Eloxx.
  • “ELX-02 treatment has demonstrated restoration of full-length protein in multiple preclinical models, including collagen IV and collagen VII in cells, and also clinical activity in our Phase 2 cystic fibrosis trial.
  • For eligible patients, induction of COL IV will also be measured at the end of two months.

Catalyst Pharmaceuticals Completes the Acquisition of U.S. Rights to FYCOMPA® (Perampanel) CIII

Retrieved on: 
Wednesday, January 25, 2023
Neuroscience, Catalyst Pharmaceuticals, EPS, Acquisition, EBITDA, Patient, CIII, Growth, Neurology, Degenerative disease, CORAL, Fine chemical, Bank, Eisai, Catalysis

CORAL GABLES, Fla., Jan. 25, 2023 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst") (Nasdaq: CPRX), a commercial-stage biopharmaceutical company focused on in-licensing, developing, and commercializing novel medicines for patients living with rare diseases, today announced that it has successfully completed the acquisition of the U.S. rights for FYCOMPA® (perampanel) CIII, from Eisai Co., Ltd, ("Eisai"). The closing of the acquisition provides Catalyst with an increased U.S. commercial presence in neurology and an expanded product portfolio with an established, first-in-class, commercial stage epilepsy asset.

Key Points: 
  • The closing of the acquisition provides Catalyst with an increased U.S. commercial presence in neurology and an expanded product portfolio with an established, first-in-class, commercial stage epilepsy asset.
  • Net FYCOMPA revenues for the 2022 fiscal year ending March 31, 2023, are expected to be approximately $136 million.
  • As previously disclosed, Catalyst made a total all-cash purchase payment of $160 million to Eisai.
  • The completion of the acquisition does not impact Catalyst's previously reported 2022 full-year FIRDAPSE total net revenue guidance of $205 million to $210 million.

Atossa Therapeutics Issues Letter to Shareholders

Retrieved on: 
Wednesday, January 25, 2023
Exemestane, Breast cancer, Food, Safety, Patient, Lymphatic system, COVID-19, Cancer, Radiation-induced lung injury, SERM, Vaccine, Risk, Survival, Goserelin, Breast, DCT, Letter, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, FDA, Incidence, Patent, Lung cancer, Health, ER, Growth, Neoplasm, Woman, 1836 U.S. Patent Office fire, Pharmacotherapy, Axis, Degenerative disease, Therapy, MBD, Pharmaceutical industry, Medical imaging, Atossa

SEATTLE, Jan. 25, 2023 (GLOBE NEWSWIRE) -- Atossa Therapeutics, Inc. (Nasdaq: ATOS), a clinical stage biopharmaceutical company developing innovative proprietary medicines to address significant unmet need in cancer, today announces the issuance of the following Letter to Shareholders from Steven C. Quay, M.D., Ph.D., the Company’s CEO and Chairman:

Key Points: 
  • SEATTLE, Jan. 25, 2023 (GLOBE NEWSWIRE) -- Atossa Therapeutics, Inc. (Nasdaq: ATOS), a clinical stage biopharmaceutical company developing innovative proprietary medicines to address significant unmet need in cancer, today announces the issuance of the following Letter to Shareholders from Steven C. Quay, M.D., Ph.D., the Company’s CEO and Chairman:
    2022 was a year of significant progress for Atossa.
  • We currently have Phase 2 studies ongoing in the prevention and treatment settings, both focused on areas of unmet medical need.
  • Our investment, which totaled $4.7 million and resulted in Atossa owning approximately 19% of the outstanding capital stock of DCT, closed in December 2022.
  • On behalf of the board of directors, management, and employees of Atossa Therapeutics, we thank you for your investment and continued support of our Company.

Rewind Therapeutics Raises Additional Funding and Adds Sunstone Life Science Ventures A/S as New Investor

Retrieved on: 
Wednesday, January 25, 2023
Rewind, Remyelination, Neurodegeneration, CNS, General partnership, Neuron, Demyelinating disease, Multiple sclerosis, Therapy, Degenerative disease, Total survey error, Central nervous system, Depression, Patient, Anxiety, Irritability, Death, PMV, Doctor of Philosophy, Pharmaceutical industry

Leuven, Belgium, January 25, 2023 --- Rewind Therapeutics, a company developing first-in-class treatments for demyelination-associated diseases, today announced that it has raised additional funding, with Sunstone Life Science Ventures A/S joining as a new investor and with participation of existing investors Boehringer Ingelheim Venture Fund, M. Ventures, PMV, Gemma Frisius Fonds, and CD3/ KU Leuven.

Key Points: 
  • Leuven, Belgium, January 25, 2023 --- Rewind Therapeutics , a company developing first-in-class treatments for demyelination-associated diseases, today announced that it has raised additional funding, with Sunstone Life Science Ventures A/S joining as a new investor and with participation of existing investors Boehringer Ingelheim Venture Fund, M. Ventures, PMV, Gemma Frisius Fonds, and CD3/ KU Leuven.
  • Claus Andersson, PhD, General Partner at Sunstone Life Science Ventures A/S, will join Rewind Therapeutics´ Board of Directors.
  • "We are delighted to welcome Sunstone Life Science Ventures as a new investor alongside the continued support by our existing investor syndicate," said Anja Harmeier, Chief Executive Officer at Rewind Therapeutics.
  • "Moreover, Rewind has a strong patent estate and a team with long research and development expertise in the field of remyelination therapeutics.

The GastroIntestinal Research Foundation Awards Grants to Two Cancer Research Powerhouses

Retrieved on: 
Tuesday, January 24, 2023
Fatty liver disease, BRAF, Research, DNA, Science, DNA repair, Anderson, Immunotherapy, Cancer, Moses Austin, Vaccine, Survival, Therapy, DRS, Drug combination, Mayo Clinic, Immune system, CAR, University, Patient, Colorectal cancer, Neoplasm, Degenerative disease, Clinical trial, FDA, Ulcerative colitis, Precision medicine, Workflow, CRC, Monroe Dunaway Anderson, Biology, Crohn's disease, Toxicity, Pharmaceutical industry

The GastroIntestinal Research Foundation (the Foundation) has made bold and innovative grant awards to advance the development of immunotherapies and personalized vaccines for colorectal cancer.

Key Points: 
  • The GastroIntestinal Research Foundation (the Foundation) has made bold and innovative grant awards to advance the development of immunotherapies and personalized vaccines for colorectal cancer.
  • The Foundation's Executive Director, Jackie Casey, said, "The Foundation designed CA CURE to get vital research dollars quickly into the hands of investigators.
  • With focused funding on immunotherapies and vaccines and by taking multiple shots on goal with multiple research grants, we hope to see new successful treatments on the horizon."
  • We are grateful for the GI Research Foundation grant as it will allow us to take successes in the study of other cancers and apply them to colorectal cancer."

Cassava Sciences Announces Positive Top-Line Clinical Results in Phase 2 Study Evaluating Simufilam in Alzheimer’s Disease

Retrieved on: 
Tuesday, January 24, 2023
Disease, FAS, Food, Safety, Patient, Geriatric Depression Scale, Science, City University of New York, Trial of the century, COVID-19, Cognition, SPA, Behavior, Biomarker, Government budget balance, Headache, Tablet, ADAS-Cog, MMSE, Neuropsychiatry, Cerebrospinal fluid, Degenerative disease, City University, CSF, GDS, Clinical trial, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Dementia, Mini–Mental State Examination, Alzheimer's disease, CUNY, Urinary tract infection, Data, Brain, Doctor of Philosophy, Pharmaceutical industry, Medical imaging, Vaccine, Copper

AUSTIN, Texas, Jan. 24, 2023 (GLOBE NEWSWIRE) -- Cassava Sciences, Inc. (Nasdaq: SAVA), a biotechnology company, today announced positive top-line Phase 2 results for simufilam, its oral drug candidate for Alzheimer’s disease dementia.

Key Points: 
  • AUSTIN, Texas, Jan. 24, 2023 (GLOBE NEWSWIRE) -- Cassava Sciences, Inc. (Nasdaq: SAVA), a biotechnology company, today announced positive top-line Phase 2 results for simufilam, its oral drug candidate for Alzheimer’s disease dementia.
  • Top-line Results – mean scores, baseline to month 12 (lower is better, except for MMSE):
    Alzheimer’s is a degenerative disease of the brain.
  • ADAS-Cog scores that change minimally (or improve) over 1 year is a highly desirable outcome in a clinical study of mild-to-moderate Alzheimer’s disease.
  • Cassava Sciences is currently evaluating simufilam tablets for Alzheimer’s disease dementia in two Phase 3 clinical studies.

KemPharm Issues Letter to Shareholders

Retrieved on: 
Tuesday, January 24, 2023
Disease, Narcolepsy, Idiopathic hypersomnia, IH, Food, Safety, Somnolence, Arimoclomol, CNS, Cognition, KMPH, Acquisition, Pediatrics, Neurodegeneration, Niemann–Pick disease, Special access program, Glycogen storage disease, Therapy, Death, European Medicines Agency, Lipid, NDA, Child development stages, New Drug Application, Central nervous system, Letter, SDX, Learning, Patient, RICH, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, FDA, Methylphenidate, Interim, NPC, Investment, Growth, Speech, EAPS, Cell, Degenerative disease, Brain, Pharmaceutical industry, Management, Risk management, Shareholder

CELEBRATION, Fla., Jan. 24, 2023 (GLOBE NEWSWIRE) -- KemPharm, Inc. (NasdaqGS: KMPH) (KemPharm, or the Company), a rare disease therapeutics company focused on the development of treatments for rare central nervous system (CNS) disorders, neurodegenerative diseases, lysosomal storage disorders and related treatment areas, today announced that Richard W. Pascoe, Chief Executive Officer of KemPharm, has issued a Letter to Shareholders.

Key Points: 
  • CELEBRATION, Fla., Jan. 24, 2023 (GLOBE NEWSWIRE) -- KemPharm, Inc. (NasdaqGS: KMPH) (KemPharm, or the Company), a rare disease therapeutics company focused on the development of treatments for rare central nervous system (CNS) disorders, neurodegenerative diseases, lysosomal storage disorders and related treatment areas, today announced that Richard W. Pascoe, Chief Executive Officer of KemPharm, has issued a Letter to Shareholders.
  • KemPharm is a company founded on solid science and this will continue to be a pillar upon which we stand.
  • However, solid science alone will not be enough to propel KemPharm into its next stage of growth and create the value that our shareholders deserve.
  • Our move to rare diseases is an opportunity to build upon what is good at KemPharm and make it great.

MBX Biosciences to Participate in the SVB Securities Global Biopharma Conference

Retrieved on: 
Tuesday, January 24, 2023
Conference, Degenerative disease, Therapy, Pharmaceutical industry

CARMEL, Ind., Jan. 24, 2023 (GLOBE NEWSWIRE) -- MBX Biosciences, Inc. , a clinical stage biopharmaceutical company developing Precision Endocrine Peptide™ (PEP™) therapeutics to treat an array of endocrine disorders, today announced its participation at the virtual SVB Securities Global Biopharma Conference – Private Company Track.

Key Points: 
  • CARMEL, Ind., Jan. 24, 2023 (GLOBE NEWSWIRE) -- MBX Biosciences, Inc. , a clinical stage biopharmaceutical company developing Precision Endocrine Peptide™ (PEP™) therapeutics to treat an array of endocrine disorders, today announced its participation at the virtual SVB Securities Global Biopharma Conference – Private Company Track.
  • The conference will take place January 31 – February 2, 2023 and management will be available for one-on-one meetings with institutional investors during that time.

Altamira Therapeutics Provides Update on Clinical Trials with Bentrio

Retrieved on: 
Tuesday, January 24, 2023
Nasal congestion, Virus, Particle-size distribution, Safety, Rhinitis, ANCOVA, Sudden death, COVID-19, Sneeze, Rhinorrhea, Allergen, Therapy, Omicron, Degenerative disease, CT, SAR, Research, Allergic rhinitis, Patient, Nasal mucosa, PCR, Incidence, NASAR, Severe acute respiratory syndrome coronavirus 2, Nasal spray, Pharmaceutical industry

The treatment effect of 1.55 points in favor of Bentrio was statistically significant in the ANCOVA model (LSmeans; p = 0.015; 95% confidence interval -2.78 to -0.32).

Key Points: 
  • The treatment effect of 1.55 points in favor of Bentrio was statistically significant in the ANCOVA model (LSmeans; p = 0.015; 95% confidence interval -2.78 to -0.32).
  • The interim analysis further showed good tolerability and safety both for Bentrio and the comparator.
  • Participants were randomized at a 1:1 ratio to either receive Bentrio or saline spray via self-administration three times per day, or as needed.
  • “We are very excited about the strong efficacy signals observed with Bentrio at the halfway point of the NASAR seasonal allergic rhinitis trial in Australia,” commented Thomas Meyer, Altamira Therapeutics’ founder, Chairman, and CEO.

Amolyt Pharma Announces Research Agreement and Licensing Option with XOMA

Retrieved on: 
Tuesday, January 24, 2023
Hypercalcaemia, Antibody, Malignancy, PHPT, Hypoparathyroidism, Degenerative disease, PTHR1, Parathyroid hormone, Primary hyperparathyroidism, Patient, Calcium, Parathyroid hormone-related protein, Biology, HHM, PTH, Bone loss, Research, Hypophosphatemia, Pharmaceutical industry, Vaccine

LYON, France and CAMBRIDGE, Mass., Jan. 24, 2023 (GLOBE NEWSWIRE) -- Amolyt Pharma (“Amolyt,” “Amolyt Pharma” or the “Company”), a global company specialized in developing innovative therapies for rare endocrine and related diseases, today announced that the Company has entered into a research agreement and licensing option with XOMA Corporation (“XOMA”) for the pre-clinical evaluation of a select set of monoclonal antibodies that arose from XOMA’s legacy discovery efforts as potential treatments for primary hyperparathyroidism (“PHPT”) and humoral hypercalcemia of malignancy (“HHM”). Amolyt has the option to license one or more of these candidates from XOMA for further clinical development worldwide.

Key Points: 
  • LYON, France and CAMBRIDGE, Mass., Jan. 24, 2023 (GLOBE NEWSWIRE) -- Amolyt Pharma (“Amolyt,” “Amolyt Pharma” or the “Company”), a global company specialized in developing innovative therapies for rare endocrine and related diseases, today announced that the Company has entered into a research agreement and licensing option with XOMA Corporation (“XOMA”) for the pre-clinical evaluation of a select set of monoclonal antibodies that arose from XOMA’s legacy discovery efforts as potential treatments for primary hyperparathyroidism (“PHPT”) and humoral hypercalcemia of malignancy (“HHM”).
  • Amolyt has the option to license one or more of these candidates from XOMA for further clinical development worldwide.
  • “We are continuing to build our portfolio for rare endocrine and related diseases while moving, in parallel, our lead program for hypoparathyroidism into a pivotal trial.
  • We look forward to watching Amolyt’s progress, as PHPT and HHM patients are underserved by current standards of care.”