Clinical trial

Pharming announces US FDA approval of Joenja® (leniolisib) as the first and only treatment indicated for APDS

Retrieved on: 
Friday, March 24, 2023
US Foods, PIK3CD, Fetus, Senior counsel, Birth, New Drug Application, Atopic dermatitis, Autoimmunity, Associate, Committee, Incidence, MD, Patient, Risk, Ageing, Disease, Priority review, Hope, EMA, Primary immunodeficiency, Diagnosis, FDA, Headache, Caregiver, Safety, Rheumatology, Liver, PASLI disease, Marketing Authorisation Application, Lymph, University, European Medicines Agency, MAA, PIK3R1, Splenomegaly, CHMP, Lymphoma, ANC, PRV, Euronext Amsterdam, Infection, Clinical trial, NDA, Sale, Sinusitis, Physician, Immunodeficiency, Genetic testing, Gene, Food, APDS, Pharmaceutical industry, Management, Vaccine, Pharming, Novartis, Medicine

LEIDEN, The Netherlands, March 24, 2023 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or "the Company") (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces that the US Food and Drug Administration (FDA) has approved Joenja® (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older. Joenja®, an oral, selective PI3Kδ inhibitor, is the first and only treatment approved in the US for APDS, a rare and progressive primary immunodeficiency. The FDA evaluated the Joenja® application for APDS under Priority Review, which is granted to therapies that have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions. Joenja® is expected to launch in the US in early April and will be available for shipment in mid-April.

Key Points: 
  • Joenja®, an oral, selective PI3Kδ inhibitor, is the first and only treatment approved in the US for APDS, a rare and progressive primary immunodeficiency.
  • The FDA approval of a treatment option for one of the more than 450 primary immunodeficiencies is also a key moment for the broader primary immunodeficiency community.
  • Until now, management of APDS has relied on the treatment of the diverse symptoms associated with APDS.
  • With the approval of Joenja®, as a treatment for a rare pediatric disease, the FDA granted Pharming a priority review voucher ("PRV").

Pharming announces US FDA approval of Joenja® (leniolisib) as the first and only treatment indicated for APDS

Retrieved on: 
Friday, March 24, 2023
US Foods, PIK3CD, Fetus, Senior counsel, Birth, New Drug Application, Atopic dermatitis, Autoimmunity, Associate, Committee, Incidence, MD, Patient, Risk, Ageing, Disease, Priority review, Hope, EMA, Primary immunodeficiency, Diagnosis, FDA, Headache, Caregiver, Safety, Rheumatology, Liver, PASLI disease, Marketing Authorisation Application, Lymph, University, European Medicines Agency, MAA, PIK3R1, Splenomegaly, CHMP, Lymphoma, ANC, PRV, Euronext Amsterdam, Infection, Clinical trial, NDA, Sale, Sinusitis, Physician, Immunodeficiency, Genetic testing, Gene, Food, APDS, Pharmaceutical industry, Management, Vaccine, Pharming, Novartis, Medicine

LEIDEN, The Netherlands, March 24, 2023 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or "the Company") (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces that the US Food and Drug Administration (FDA) has approved Joenja® (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older. Joenja®, an oral, selective PI3Kδ inhibitor, is the first and only treatment approved in the US for APDS, a rare and progressive primary immunodeficiency. The FDA evaluated the Joenja® application for APDS under Priority Review, which is granted to therapies that have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions. Joenja® is expected to launch in the US in early April and will be available for shipment in mid-April.

Key Points: 
  • Joenja®, an oral, selective PI3Kδ inhibitor, is the first and only treatment approved in the US for APDS, a rare and progressive primary immunodeficiency.
  • The FDA approval of a treatment option for one of the more than 450 primary immunodeficiencies is also a key moment for the broader primary immunodeficiency community.
  • Until now, management of APDS has relied on the treatment of the diverse symptoms associated with APDS.
  • With the approval of Joenja®, as a treatment for a rare pediatric disease, the FDA granted Pharming a priority review voucher ("PRV").

Evolution of Oncology Treatments Giving New Hope to Those Diagnosed with Breast Cancer

Retrieved on: 
Friday, March 24, 2023
Survival rate, Treatment, Survival, Fulvestrant, CDK2, Patient, Risk, News, Breast Cancer Awareness Month, Breast, Minerva Surgical, Inc. v. Hologic, Inc., ONC, TSX, Diagnosis, FDA, Cancer, Immunotherapy, PFS, Șaeș, HR, Lymph, Therapy, ORR, HOLX, Progression-free survival, Clinical trial, Paclitaxel, Woman, Fast track (FDA), OFS, Nature, Breast cancer, American Cancer Society, Recurrence, BCT, Tamoxifen, Pharmaceutical industry, Birth control, Medical imaging, Nursing, Hologic, Oncolytics Biotech

VANCOUVER, British Columbia, March 24, 2023 /PRNewswire/ -- USA News Group - Treatments for various forms of breast cancer continue to evolve, with several new breakthroughs being labeled as landmark in nature and giving hope to families dealing with a fateful diagnosis. According to the American Cancer Society in 2022, the 5-year relative survival rate for localized breast cancer, cancer that has not spread outside the breast, is 99%, and for those whose cancer has spread outside the breast to nearby structures or lymph nodes, the survival rate is 86%. Over the last year, the biotech sector has increased its efforts and delivered very optimistic data from such groups as Oncolytics Biotech Inc. (NASDAQ: ONCY) (TSX: ONC), CytomX Therapeutics, Inc. (NASDAQ: CTMX), BriaCell Therapeutics (NASDAQ: BCTX) (NASDAQ: BCTXW) (TSX: BCT), Relay Therapeutics (NASDAQ: RLAY), and Hologic, Inc. (NASDAQ: HOLX).

Key Points: 
  • According to the American Cancer Society in 2022, the 5-year relative survival rate for localized breast cancer, cancer that has not spread outside the breast, is 99%, and for those whose cancer has spread outside the breast to nearby structures or lymph nodes, the survival rate is 86%.
  • This pivotal registration study will be enrolling advanced metastatic breast cancer patients for whom no approved treatment options exist.
  • Hologic's data showed that the Breast Cancer Index test identified which premenopausal patients with early-stage HR+ breast cancer benefited from the addition of ovarian function suppression (OFS) to primary adjuvant endocrine therapy.
  • The Breast Cancer Index test is the first biomarker to be evaluated in a cohort from the landmark Suppression of Ovarian Function Trial (SOFT).

Evolution of Oncology Treatments Giving New Hope to Those Diagnosed with Breast Cancer

Retrieved on: 
Friday, March 24, 2023
Survival rate, Treatment, Survival, Fulvestrant, CDK2, Patient, Risk, News, Breast Cancer Awareness Month, Breast, Minerva Surgical, Inc. v. Hologic, Inc., ONC, TSX, Diagnosis, FDA, Cancer, Immunotherapy, PFS, Șaeș, HR, Lymph, Therapy, ORR, HOLX, Progression-free survival, Clinical trial, Paclitaxel, Woman, Fast track (FDA), OFS, Nature, Breast cancer, American Cancer Society, Recurrence, BCT, Tamoxifen, Pharmaceutical industry, Birth control, Medical imaging, Nursing, Hologic, Oncolytics Biotech

VANCOUVER, British Columbia, March 24, 2023 /PRNewswire/ -- USA News Group - Treatments for various forms of breast cancer continue to evolve, with several new breakthroughs being labeled as landmark in nature and giving hope to families dealing with a fateful diagnosis. According to the American Cancer Society in 2022, the 5-year relative survival rate for localized breast cancer, cancer that has not spread outside the breast, is 99%, and for those whose cancer has spread outside the breast to nearby structures or lymph nodes, the survival rate is 86%. Over the last year, the biotech sector has increased its efforts and delivered very optimistic data from such groups as Oncolytics Biotech Inc. (NASDAQ: ONCY) (TSX: ONC), CytomX Therapeutics, Inc. (NASDAQ: CTMX), BriaCell Therapeutics (NASDAQ: BCTX) (NASDAQ: BCTXW) (TSX: BCT), Relay Therapeutics (NASDAQ: RLAY), and Hologic, Inc. (NASDAQ: HOLX).

Key Points: 
  • According to the American Cancer Society in 2022, the 5-year relative survival rate for localized breast cancer, cancer that has not spread outside the breast, is 99%, and for those whose cancer has spread outside the breast to nearby structures or lymph nodes, the survival rate is 86%.
  • This pivotal registration study will be enrolling advanced metastatic breast cancer patients for whom no approved treatment options exist.
  • Hologic's data showed that the Breast Cancer Index test identified which premenopausal patients with early-stage HR+ breast cancer benefited from the addition of ovarian function suppression (OFS) to primary adjuvant endocrine therapy.
  • The Breast Cancer Index test is the first biomarker to be evaluated in a cohort from the landmark Suppression of Ovarian Function Trial (SOFT).

Spectral Medical Announces Fourth Quarter and Fiscal 2022 Results and Provides Corporate Update

Retrieved on: 
Friday, March 24, 2023
International joint venture, Cras, De novo, Marketing, Public, Dialysis, Hemoperfusion, ESS, Medical University of South Carolina, Patient, Medical device, Intensive care unit, Software, University, Baxter, Research, MDSAP, Sepsis, Certification, UCSF, TSX, EDEN, FDA, SAMI, Observational study, COVID-19, PMX, Contract research organization, MUSC, Mortality, Breakthrough, Lipopolysaccharide, University of California, San Francisco, Clinical trial, Medical school, Septic shock, Joint venture, Infomed, ICU, Pharmaceutical industry, Silviculture, Nursing, Spectral

TORONTO, March 24, 2023 (GLOBE NEWSWIRE) -- Spectral Medical Inc. (“Spectral” or the “Company”) (TSX: EDT), a late-stage theranostic company advancing therapeutic options for sepsis and septic shock, today announced its financial results for the fourth quarter and for the year ended December 31, 2022 and provided a corporate update.

Key Points: 
  • Importantly, we are in the process of engaging a new Contract Research Organization (“CRO”) to oversee the Tigris trial.
  • The new company, i-Dialco, is exclusively focused on advancing commercialization of the SAMI and DIMI dialysis devices in the North American markets.
  • We anticipate our initiatives aimed at enhancing trial recruitment and enrollment will assist in completion of Tigris as expeditiously as possible.
  • With the establishment of i-Dialco, the previous Dialco operations were considered discontinued as at December 31, 2022.

Enrollment Begins in FORESEE Clinical Trial Using Biocept’s CNSide™ to Evaluate Patients with Leptomeningeal Metastases

Retrieved on: 
Friday, March 24, 2023
Oncology, Medical Supplies, Health, Genetics, Clinical Trials, Research, Science, Cancer, LDT, Central nervous system, Hematology, Feinberg School of Medicine, Clinical trial, Breast, NSCLC, Department of Neurology, Xuanwu hospital, Associate, Assistant, Trial of the century, MRI, Hospital, University of Texas Southwestern Medical Center, MD, Patient, Physician, Standard of care, Laboratory developed test, Neurology, Neoplasm, Principal, Northwestern University, Nursing, Medical imaging, LM, Medicine

This first patient was seen at the UT Southwestern Medical Center, one of the largest and most respected hospitals in the nation.

Key Points: 
  • This first patient was seen at the UT Southwestern Medical Center, one of the largest and most respected hospitals in the nation.
  • The FORESEE trial is a multicenter, prospective clinical trial expected to enroll 40 patients with breast or non-small cell lung cancer (NSCLC) who have suspicious or confirmed leptomeningeal metastases (LM).
  • The goal of the FORESEE trial is to evaluate the performance of CNSide in monitoring the LM's response to treatment and to assess the impact of CNSide on treatment decisions made by physicians.
  • Standard-of-care methods to diagnose or assess the treatment response of LM (i.e., clinical evaluation, MRI and cytology) have limited sensitivity and specificity.

Aptose Reports Results for the Fourth Quarter and Full Year 2022

Retrieved on: 
Thursday, March 23, 2023
ASH, Lux, APS, Controller, ITD, TKD, G1, BCR, Associate, Accounting, Plasma, RAS, Investment, Patient, RUNX1, Clinical, LYN, TP53, DNMT3A, SYK, European Hematology Association, Research, Aplastic anemia, MRD, BCL2, PLOS One, MLL, Data, FLT3, TSX, Amylin Pharmaceuticals, FDA, Combination, RSK, Lymphoid leukemia, Absorption, BTK, Acute myeloid leukemia, Phase 1, CR, Hand, MD Anderson Cancer Center, Inflammation, Lymphoma, Phosphorylation, National university, NPM1, Clinical trial, European School, Data monitoring committee, AML, BID, EHA, ISTA Pharmaceuticals, Harvard Business School, Toxic leukoencephalopathy, G3, Gene, Society, Pharmaceutical industry, Cryptocurrency, Vaccine, Running

SAN DIEGO and TORONTO, March 23, 2023 (GLOBE NEWSWIRE) -- Aptose Biosciences Inc. (“Aptose” or the “Company”) (NASDAQ: APTO, TSX: APS), a clinical-stage precision oncology company developing highly differentiated oral kinase inhibitors to treat hematologic malignancies, today announced financial results for the fourth quarter and year ended December 31, 2022, and provided a corporate update.

Key Points: 
  • The net loss for the quarter ended December 31, 2022, was $10.0 million ($0.11 per share) compared with $24.3 million ($0.27 per share) for the quarter ended December 31, 2021.
  • Total cash and cash equivalents and investments as of December 31, 2022, were $47.0 million.
  • Aptose Appoints VP, Controller – During the fourth quarter, Aptose appointed Brooks Ensign, Vice President and Controller.
  • A summary of the results of operations for the years ended December 31, 2022 and 2021 is presented below:

Finch Therapeutics Reports Fourth Quarter and Full Year 2022 Financial Results and Provides Business Updates

Retrieved on: 
Thursday, March 23, 2023
Prejudice, Workforce, Patent, Intellectual property, CDI, University, University of Minnesota, IBD, Spectrum, Research, Doctor of Philosophy, Șaeș, Inflammatory bowel disease, Public expenditure, Clinical trial, ASD, UMN, R, FNCH, Therapy, Second Half, Clostridioides difficile infection, HBV, Pharmaceutical industry, Video game, G&A, Finch

SOMERVILLE, Mass., March 23, 2023 (GLOBE NEWSWIRE) -- Finch Therapeutics Group, Inc. (“Finch”, “Finch Therapeutics” or the “Company”) (Nasdaq: FNCH), a microbiome technology company with a portfolio of intellectual property and microbiome assets, today reported fourth quarter and full year 2022 financial results and provided business updates.

Key Points: 
  • SOMERVILLE, Mass., March 23, 2023 (GLOBE NEWSWIRE) -- Finch Therapeutics Group, Inc. (“Finch”, “Finch Therapeutics” or the “Company”) (Nasdaq: FNCH), a microbiome technology company with a portfolio of intellectual property and microbiome assets, today reported fourth quarter and full year 2022 financial results and provided business updates.
  • Finch reported a net loss of $27.0 million for the fourth quarter of 2022, compared to a net loss of $19.1 million for the same period in 2021.
  • R&D expenses were $16.6 million for the fourth quarter of 2022, compared to $14.8 million for the same period in 2021.
  • G&A expenses were $10.9 million for the fourth quarter of 2022, compared to $5.1 million for the same period in 2021.

Gain Therapeutics Reports Full Year 2022 Financial Results and Business Update

Retrieved on: 
Thursday, March 23, 2023
Insurance, GLP, GBA1, University, University of Helsinki, PD, Information technology, Hepatology, Clinical trial, Pharmacokinetics, G&A, GAAP, Research, Accounting, Lung, Institute for Research in Biomedicine, Patent, DSM-IV codes, Toxicology, AAT, R, Glucocerebrosidase, Therapy, Safety, IND, Alpha-1 antitrypsin deficiency, Eurostar, Disease, Cryptocurrency, Pharmaceutical industry

“We look forward to commencing the clinical program for GT-02287 with the submission of the dossier for the Phase 1 clinical trial by mid-2023.

Key Points: 
  • “We look forward to commencing the clinical program for GT-02287 with the submission of the dossier for the Phase 1 clinical trial by mid-2023.
  • General and administrative (G&A) expenses for 2022 were $9.54 million, as compared to $6.82 million in 2021.
  • Net loss as of December 31, 2022 included non-cash compensation expenses of $1.53 million as compared to 2021 non-cash compensation expenses of $1.87 million.
  • As of December 31, 2022, the Company had cash, cash equivalents and marketable securities totaling $22.1 million for the year ended December 31, 2022.

Vor Bio Reports Fourth Quarter and Full Year 2022 Financial Results and Provides Company Update

Retrieved on: 
Thursday, March 23, 2023
Haematopoiesis, GMP, Research and development, Siddhartha Mukherjee, Bone marrow, Clinical trial, Passive treatment system, Coronavirus Scientific Advisory Board, Cmax, Doctor of Philosophy, HSCT, Trial of the century, Acute myeloid leukemia, MRD, CAR, AML, Good manufacturing practice, CD33, Patient, Investment, CGMP, Transplant, Administration, Blood, AUC, Biology, TANDEM, HSC, R, Services General Administration and Coordination Department, Government of Sindh, IND, Acute promyelocytic leukemia, Satellite navigation device, Nursing, Vaccine, Dentistry, Cryptocurrency, G&A, MD, Gemtuzumab ozogamicin

In December 2022, Vor Bio announced the pricing of an underwritten offering and a private placement, with combined gross proceeds of approximately $115.8 million.

Key Points: 
  • In December 2022, Vor Bio announced the pricing of an underwritten offering and a private placement, with combined gross proceeds of approximately $115.8 million.
  • The Company intends to evaluate VCAR33ALLO in combination with trem-cel as a Treatment System, aiming at prolonged remissions or cures following transplant.
  • General & Administrative (G&A) Expenses: G&A expenses for the fourth quarter of 2022 were $7.7 million, compared to $5.6 million for the fourth quarter of 2021, and for the year ended December 31, 2022, were $28.9 million, compared to $21.5 million for the year ended December 31, 2021.
  • Net Loss: Net loss for the fourth quarter of 2022 was $23.9 million, compared to $18.3 million for the fourth quarter of 2021, and for the year ended December 31, 2022, was $92.1 million, compared to $68.9 million for the year ended December 31, 2021.