Orphan drugs

Achilles Therapeutics Enrolls First US Patient in Ongoing Phase I/IIa Study in Advanced Non-small Cell Lung Cancer

Retrieved on: 
Thursday, July 1, 2021

CHIRON is an open-label, multi-center Phase I/IIa trial evaluating the safety, tolerability, and activity of clonal neoantigen T cell (cNeT) therapy as a single dose in adult patients with advanced metastatic non-small cell lung cancer (NSCLC).

Key Points: 
  • CHIRON is an open-label, multi-center Phase I/IIa trial evaluating the safety, tolerability, and activity of clonal neoantigen T cell (cNeT) therapy as a single dose in adult patients with advanced metastatic non-small cell lung cancer (NSCLC).
  • This first US patient was enrolled at the Moffitt Cancer Center in Tampa, FL where Dr. Benjamin Creelan is the Principal Investigator.
  • Achilles is a clinical-stage biopharmaceutical company developing precision T cell therapies targeting clonal neoantigens: protein markers unique to the individual that are expressed on the surface of every cancer cell.
  • The Company has two ongoing Phase I/IIa trials, the CHIRON trial in patients with advanced non-small cell lung cancer (NSCLC) and the THETIS trial in patients with recurrent or metastatic melanoma.

NANOBIOTIX Announces Initiation of New Clinical Study Evaluating NBTXR3 in Lung Cancer

Retrieved on: 
Wednesday, June 30, 2021

In addition to the lung cancer study described above, two phase II studies, each evaluating NBTXR3 in combination with anti-PD-1 for patients with head and neck cancer (inoperable locoregional recurrent amenable to reirradiation and recurrent metastatic with limited PD-L1 expression or refractory); one phase I study evaluating NBTXR3 in combination with chemotherapy for patients with esophageal cancer; and one phase I study evaluating NBTXR3 as a single-agent activated by RT for patients with pancreatic cancer are active and enrolling.

Key Points: 
  • In addition to the lung cancer study described above, two phase II studies, each evaluating NBTXR3 in combination with anti-PD-1 for patients with head and neck cancer (inoperable locoregional recurrent amenable to reirradiation and recurrent metastatic with limited PD-L1 expression or refractory); one phase I study evaluating NBTXR3 in combination with chemotherapy for patients with esophageal cancer; and one phase I study evaluating NBTXR3 as a single-agent activated by RT for patients with pancreatic cancer are active and enrolling.
  • As previously announced, the first patient has been injected with NBTXR3 in each of the esophageal cancer and pancreatic cancer studies.
  • The first NBTXR3 injections in the phase II head and neck cancer studies are expected in the second half of 2021.
  • The clinical collaboration between Nanobiotix and MD Anderson is a collaborator-led expansion of the NBTXR3 development pipeline across indications and therapeutic combinations.

Allogene Therapeutics Granted FDA Fast Track Designation for ALLO-605, the First TurboCAR™ T Cell Therapy, for the Treatment of Relapsed/Refractory Multiple Myeloma

Retrieved on: 
Wednesday, June 30, 2021

The FDA granted Fast Track designation based on the potential of ALLO-605 to address the unmet need for patients who have failed other standard multiple myeloma therapies.

Key Points: 
  • The FDA granted Fast Track designation based on the potential of ALLO-605 to address the unmet need for patients who have failed other standard multiple myeloma therapies.
  • These properties may also enable CAR T therapy to succeed in solid tumors and increase efficacy in hematologic malignancies.
  • In June 2021, ALLO-605 was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the potential treatment of relapsed/refractory multiple myeloma.
  • AlloCAR T and TurboCAR are trademarks of Allogene Therapeutics, Inc.
    ALLO-605 and ALLO-715 utilize TALEN gene-editing technology pioneered and owned by Cellectis.

Iovance Biotherapeutics to Host Conference Call and Webcast to Discuss Clinical Data for LN-145 in Non-Small Cell Lung Cancer on June 29, 2021 at 4:30pm ET

Retrieved on: 
Tuesday, June 29, 2021

To participate in the conference call, please dial 1-844-646-4465 (domestic) or 1-615-247-0257 (international) and reference the access code 2989712.

Key Points: 
  • To participate in the conference call, please dial 1-844-646-4465 (domestic) or 1-615-247-0257 (international) and reference the access code 2989712.
  • The live webcast can be accessed in the Investors section of the Companys website at www.iovance.com .
  • The archived webcast will also be available for one year in the Investors section at www.iovance.com .
  • In addition, the companys TIL therapy is being investigated in a registration-supporting study for the treatment of patients with locally advanced, recurrent or metastatic non-small cell lung cancer.

FDA Grants Rafael Pharmaceuticals Orphan Drug Designation for CPI-613® (Devimistat) for Treatment of Biliary Cancer

Retrieved on: 
Tuesday, June 29, 2021

CRANBURY, N.J., June 29, 2021 (GLOBE NEWSWIRE) -- Rafael Pharmaceuticals, Inc. (Rafael or the Company), a leader in the growing field of cancer metabolism-based therapeutics, announced today that the U.S. Food and Drug Administration (FDA) granted orphan drug designation for CPI-613 (devimistat) for the treatment of biliary cancer.

Key Points: 
  • CRANBURY, N.J., June 29, 2021 (GLOBE NEWSWIRE) -- Rafael Pharmaceuticals, Inc. (Rafael or the Company), a leader in the growing field of cancer metabolism-based therapeutics, announced today that the U.S. Food and Drug Administration (FDA) granted orphan drug designation for CPI-613 (devimistat) for the treatment of biliary cancer.
  • Biliary cancer appears when cancer cells form in the bile ducts or gallbladder, an essential aspect of the gastrointestinal system.
  • The orphan drug designation for devimistat showcases the importance of discovering these new treatment options.
  • Devimistat has been granted orphan drug status by the FDA for the treatment of pancreatic cancer, acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), Burkitts lymphoma, peripheral T-cell lymphomas, and soft tissue sarcoma, and now, biliary cancer.

MediWound Receives Complete Response Letter from U.S. FDA for NexoBrid Biologics License Application

Retrieved on: 
Tuesday, June 29, 2021

Food and Drug Administration(FDA) regarding the Biologics License Application (BLA) seeking approval of NexoBrid for eschar removal (debridement) in adults with deep partial-thickness and/or full-thickness thermal burns.

Key Points: 
  • Food and Drug Administration(FDA) regarding the Biologics License Application (BLA) seeking approval of NexoBrid for eschar removal (debridement) in adults with deep partial-thickness and/or full-thickness thermal burns.
  • The FDA identified issues related to the Chemistry, Manufacturing and Controls (CMC) section of the BLA and requested additional CMC information.
  • The FDA acknowledged receipt of several CMC amendments, submitted in response the CMC information requests, which were not reviewed for this action.
  • Vericel Corporation (NASDAQ: VCEL) holds an exclusive license for North American commercial rights to NexoBrid.

CicloMed Announces Initiation of Phase 1B Trial of Fosciclopirox in Newly Diagnosed and Recurrent Urothelial Cancer Patients

Retrieved on: 
Tuesday, June 29, 2021

We are excited to have initiated this Phase 1B trial of fosciclopirox in newly diagnosed and recurrent urothelial cancer patients scheduled for transurethral resection of bladder tumors, said Tammy Ham, CEO of CicloMed.

Key Points: 
  • We are excited to have initiated this Phase 1B trial of fosciclopirox in newly diagnosed and recurrent urothelial cancer patients scheduled for transurethral resection of bladder tumors, said Tammy Ham, CEO of CicloMed.
  • We believe that fosciclopirox provides strong potential to improve treatment outcomes for patients with bladder cancer.
  • In September 2020, CicloMed was awarded a Phase II Small Business Innovation Research (SBIR) grant by the National Cancer Institute (NCI), enabling the Phase 1B trial.
  • The initiation of this Phase 1B trial enables us to characterize the pharmacologic activity and mechanisms of fosciclopirox action following treatment in urothelial cancer patients, Weir added.

Cend Therapeutics’ CEND-1 Granted Fast Track Designation for Pancreatic Cancer

Retrieved on: 
Tuesday, June 29, 2021

CEND-1 was granted Orphan Drug Designation by the FDA for the treatment of pancreatic cancer in January 2019.

Key Points: 
  • CEND-1 was granted Orphan Drug Designation by the FDA for the treatment of pancreatic cancer in January 2019.
  • By awarding Fast Track Designation, the FDA has recognized CEND-1s potential to meaningfully improve outcomes for pancreatic cancer patients,said Andrew Dorr, MD, Chief Medical Officer of Cend.
  • Fast Track designation can lead to an Accelerated Approval and Priority Review if certain criteria are met.
  • Cend is also applying its technology to alter immunosuppression selectively within the tumor microenvironment to enable a patients immune system and immunotherapies to more effectively fight cancer.

Bristol Myers Squibb Receives European Commission Approval for Opdivo (nivolumab) Plus Yervoy (ipilimumab) for the Treatment of Mismatch Repair Deficient or Microsatellite Instability–High Metastatic Colorectal Cancer After Prior Chemotherapy

Retrieved on: 
Tuesday, June 29, 2021

Bristol Myers Squibb (NYSE: BMY) announced that the European Commission (EC) has approved Opdivo (nivolumab) plus Yervoy (ipilimumab) for the treatment of adult patients with mismatch repair deficient (dMMR) or microsatellite instability-high (MSI-H) metastatic colorectal cancer (mCRC) after prior fluoropyrimidine-based combination chemotherapy.

Key Points: 
  • Bristol Myers Squibb (NYSE: BMY) announced that the European Commission (EC) has approved Opdivo (nivolumab) plus Yervoy (ipilimumab) for the treatment of adult patients with mismatch repair deficient (dMMR) or microsatellite instability-high (MSI-H) metastatic colorectal cancer (mCRC) after prior fluoropyrimidine-based combination chemotherapy.
  • Because as a leader in cancer care, Bristol Myers Squibb is working to empower all people with cancer to have a better future.
  • OPDIVO (nivolumab), in combination with YERVOY (ipilimumab), is indicated for the treatment of patients with unresectable or metastatic melanoma.
  • OPDIVO (nivolumab) is indicated for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy.

Iovance Biotherapeutics Announces Clinical Data for LN-145 in Non-Small Cell Lung Cancer

Retrieved on: 
Tuesday, June 29, 2021

The initial clinical results are available in a slide presentation on the Iovance website here .

Key Points: 
  • The initial clinical results are available in a slide presentation on the Iovance website here .
  • Iovance anticipates presenting additional Cohort 3B data at a medical meeting in the second half of 2021.
  • Friedrich Graf Finckenstein, M.D., Chief Medical Officer of Iovance, stated, There remains a very significant unmet need to increase response rates and prolong survival in the second line non-small cell lung cancer treatment setting.
  • In addition, the companys TIL cell therapy is being investigated in a registration-supporting study for the treatment of patients with locally advanced, recurrent or metastatic non-small cell lung cancer (NSCLC).