Orphan drugs

OncoSec Enters into a Collaboration Agreement with Merck for a Pivotal Global Phase 3 Study, KEYNOTE-C87, of TAVO™ Combined with KEYTRUDA® for Late-Stage Metastatic Melanoma

Retrieved on: 
Tuesday, July 6, 2021
Cancer treatments, Clinical medicine, Drugs, Antineoplastic drugs, Breakthrough therapy, Orphan drugs, Cancer immunotherapy, Pembrolizumab, Melanoma, Nivolumab, Merck & Co.

Under the terms of the Agreement, Merck will provide KEYTRUDA , while OncoSec will provide the investigational drug, TAVO.

Key Points: 
  • Under the terms of the Agreement, Merck will provide KEYTRUDA , while OncoSec will provide the investigational drug, TAVO.
  • Eligible patients must have Stage III or IV unresectable, metastatic melanoma, and must be refractory to prior checkpoint therapy.
  • "This Phase 3 collaboration represents a crucial milestone for OncoSec as we advance TAVO through the clinic and toward potential approval globally, and expands upon our initial 2017 clinical collaboration and supply agreement with Merck.
  • TAVO has received both Orphan Drug and Fast-Track Designation by the U.S. Food & Drug Administration for the treatment of metastatic melanoma.

Eureka Therapeutics Announces New GPRC5D License Agreement with Sanofi to Target Multiple Myeloma

Retrieved on: 
Tuesday, July 6, 2021
Research, Genetics, Clinical trials, Biotechnology, Health, Pharmaceutical, General Health, Science, Oncology, Health care, Health sciences, Orphan drugs, Multiple myeloma, Companies, Sanofi, Breakthrough therapy, Monoclonal antibodies, Protein domains

The GPRC5D binding domain was discovered using Eurekas proprietary E-ALPHA antibody discovery platform and developed under a collaboration agreement between Eureka and MSK.

Key Points: 
  • The GPRC5D binding domain was discovered using Eurekas proprietary E-ALPHA antibody discovery platform and developed under a collaboration agreement between Eureka and MSK.
  • GPRC5D is a novel target that has emerged as a promising option for the treatment of multiple myeloma, particularly for patients who have relapsed from other therapies, said Dr. Eric L. Smith, myeloma physician-scientist and an inventor on the patents.
  • We are delighted to contribute technology for the development of next generation therapies against multiple myeloma, said Dr. Cheng Liu, President and Chief Executive Officer of Eureka Therapeutics.
  • Under the terms of the agreement, Sanofi has exclusive rights to the GPRC5D binder for non-CAR use.

Kura Oncology Announces Clinical Collaboration to Evaluate Tipifarnib in Combination with Alpelisib in Head and Neck Squamous Cell Carcinoma

Retrieved on: 
Tuesday, July 6, 2021
Drugs, Health, Orphan drugs, Chloroarenes, Imidazoles, Tipifarnib, Farnesyltransferase inhibitor, Oncology, Head and neck cancer, Ras GTPase, Squamous cell carcinoma, HRAS

Tipifarnib is Kuras farnesyl transferase inhibitor drug candidate currently in a registration-directed trial as a monotherapy in patients with HRAS mutant HNSCC.

Key Points: 
  • Tipifarnib is Kuras farnesyl transferase inhibitor drug candidate currently in a registration-directed trial as a monotherapy in patients with HRAS mutant HNSCC.
  • Combining tipifarnib with alpelisib has the potential to provide a clinically meaningful increase in anti-tumor activity compared to when inhibiting either pathway alone, said Stephen Dale, M.D., Chief Medical Officer of Kura Oncology.
  • We believe this clinical collaboration will enable us to potentially expand the use of tipifarnib to a significantly higher percentage of patients with advanced HNSCC.
  • Head and neck squamous cell carcinoma (HNSCC) is the seventh most common cancer worldwide, accounting for more than 500,000 new cases each year.

Trillium Therapeutics Announces Dosing of First Patient in Phase 1b/2 Study of TTI-622 in Combination With Azacitidine and Venetoclax in TP53-Wild Type Acute Myeloid Leukemia

Retrieved on: 
Tuesday, July 6, 2021
Chemical compounds, Organic compounds, Clinical medicine, Orphan drugs, Antineoplastic drugs, Breakthrough therapy, Benzamides, Chloroarenes, Venetoclax, Azacitidine, 7+3, Chemotherapy

Preclinical studies have shown that TTI-622 exhibits anti-tumor activity against AML cells as a monotherapy that is enhanced when combined with azacitidine or venetoclax.

Key Points: 
  • Preclinical studies have shown that TTI-622 exhibits anti-tumor activity against AML cells as a monotherapy that is enhanced when combined with azacitidine or venetoclax.
  • The dosing of this patient marks the second combination cohort that has been initiated with TTI-622, commented Dr. Ingmar Bruns, Trilliums Chief Medical Officer.
  • The combination of TTI-622 and azacitidine and venetoclax is being assessed as part of the ongoing, open-label study (NCT03530683).
  • Significant unmet medical need remains for elderly AML patients or those who are unfit for intensive chemotherapy, added Dr. Bruns.

Athenex Provides Update from FDA Type A Meeting Regarding Oral Paclitaxel Plus Encequidar for the Treatment of Metastatic Breast Cancer

Retrieved on: 
Tuesday, July 6, 2021
Carbonyl compounds, Drugs, Cancer treatments, Taxanes, Orphan drugs, Bristol-Myers Squibb, Paclitaxel, Protein-bound paclitaxel, Paclitaxel trevatide

The FDA was supportive and encouraged the Company to continue development of oral paclitaxel and encequidar for the treatment of metastatic breast cancer.

Key Points: 
  • The FDA was supportive and encouraged the Company to continue development of oral paclitaxel and encequidar for the treatment of metastatic breast cancer.
  • We continue to believe that, if approved, oral paclitaxel and encequidar has the potential to address a major unmet need in metastatic breast cancer.
  • Athenex is also developing oral paclitaxel and encequidar for the treatment of cutaneous angiosarcoma, for which it has received Orphan Drug Designation from the FDA.
  • Athenex also received Orphan Designations from the European Commission for oral paclitaxel and encequidar for the treatment of soft tissue sarcoma.

FDA Approves Expanded Indication for Merck’s KEYTRUDA® (pembrolizumab) in Locally Advanced Cutaneous Squamous Cell Carcinoma (cSCC)

Retrieved on: 
Tuesday, July 6, 2021
Oncology, FDA, Health, Other Health, Pharmaceutical, Biotechnology, Clinical medicine, Cancer, Orphan drugs, Breakthrough therapy, Cancer immunotherapy, Antineoplastic drugs, Pembrolizumab, Carcinoma, Merkel-cell carcinoma, Transitional cell carcinoma, Axitinib, Renal cell carcinoma, the Merck Access Program for KEYTRUDA, Merck’s Patient Support Program for KEYTRUDA, Merck, THE MERCK ACCESS PROGRAM FOR KEYTRUDA, MERCK’S PATIENT SUPPORT PROGRAM FOR KEYTRUDA, MERCK

KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC).

Key Points: 
  • KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC).
  • KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma.
  • KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) or locally advanced cSCC that is not curable by surgery or radiation.
  • In KEYNOTE-052, KEYTRUDA was discontinued due to adverse reactions in 11% of 370 patients with locally advanced or metastatic urothelial carcinoma.

Global Rituximab Biosimilars Market Opportunities and Strategies to 2030 - ResearchAndMarkets.com

Retrieved on: 
Tuesday, July 6, 2021
Health, General Health, Life sciences, Drugs, Articles, Biotechnology, Orphan drugs, Biosimilar, Rituximab

The "Rituximab Biosimilars Global Market Opportunities and Strategies to 2030: COVID 19 Growth and Change" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Rituximab Biosimilars Global Market Opportunities and Strategies to 2030: COVID 19 Growth and Change" report has been added to ResearchAndMarkets.com's offering.
  • The market is then expected to grow at a CAGR of 10.5% from 2025 and reach $5,723.6 million in 2030.
  • The rituximab biosimilar market is highly concentrated, with a small number of large players dominating the market.
  • The top opportunities in the rituximab biosimilar market segmented by application will arise in the non-hodgkin's lymphoma segment, which will gain $565.9 million of global annual sales by 2025.

Allarity Therapeutics Receives Acceptance & Review Notification from U.S. FDA for Pre-Market Approval Application for Dovitinib-DRP®

Retrieved on: 
Monday, July 5, 2021
Kidney cancer, Food and Drug Administration, Health, Medicine, Orphan drugs, Breakthrough therapy

On 2 April, 2021, Allarity announced the filing of the PMA application.

Key Points: 
  • On 2 April, 2021, Allarity announced the filing of the PMA application.
  • The FDAs acceptance of the Companys PMA application means that the FDA has made a threshold determination that the application is sufficiently complete to begin an in-depth review.
  • Allaritys CEO, Steve Carchedi, noted, The FDAs acceptance of our PMA filing for the Dovitinib-DRP companion diagnostic is an important milestone for our Company.
  • Allarity plans to file an NDA with the FDA for the approval of dovitinib for the treatment of renal cell carcinoma (kidney cancer) during 2021.

Labcorp Launches Companion Diagnostic for Amgen’s Newly-approved Lung Cancer Therapy

Retrieved on: 
Thursday, July 1, 2021
MEN, Medical devices, Infectious diseases, Science, Family, Genetics, Consumer, Clinical trials, FDA, Biotechnology, Health, Other Science, Research, Women, Pharmaceutical, Seniors, Oncology, KRAS, Drugs, Breakthrough therapy, Orphan drugs, Health, Cancer, LabCorp, Treatment of cancer, Lung cancer, Sotorasib

Every day, cancer patients across the country anxiously await test results that directly impact treatment decisions.

Key Points: 
  • Every day, cancer patients across the country anxiously await test results that directly impact treatment decisions.
  • The therapy and this indicated use of the test as a companion diagnostic received approval from the U.S. Food and Drug Administration (FDA) in late May 2021.
  • According to the American Cancer Society, in 2020, 10-12% of the 228,000 people diagnosed with lung cancer carry the G12C KRAS mutation.
  • During the last 25 years, Labcorp has played a significant role in launching hallmark testing options for the treatment of cancer.

Merck Provides Update on KEYTRUDA® (pembrolizumab) Indication in Third-Line Gastric Cancer in the US

Retrieved on: 
Thursday, July 1, 2021
Research, Medical Supplies, FDA, Clinical trials, Biotechnology, General Health, Pharmaceutical, Health, Science, Oncology, Clinical medicine, Cancer, Orphan drugs, Antineoplastic drugs, Cancer immunotherapy, Breakthrough therapy, Chloroarenes, Pembrolizumab, Sorafenib, Microsatellite instability, Hepatocellular carcinoma, Checkpoint inhibitor

Patients being treated with KEYTRUDA for metastatic gastric cancer in the third- or further-line setting should discuss their care with their health care provider.

Key Points: 
  • Patients being treated with KEYTRUDA for metastatic gastric cancer in the third- or further-line setting should discuss their care with their health care provider.
  • The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.
  • KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib.
  • At Merck, the potential to bring new hope to people with cancer drives our purpose and supporting accessibility to our cancer medicines is our commitment.