Instant Report
FDF

Date: 01-May-2020

US FDA Grants Fast Track Status To Erytechís Eryaspase To Treat Metastatic Pancreatic Cancer

Erytech Pharma, a clinical-stage biopharmaceutical company developing innovative therapies by encapsulating therapeutic drug substances inside red blood cells, announced that the US Food and Drug Administration (FDA) has granted eryaspase Fast Track Designation for the development of a second-line treatment of patients with metastatic pancreatic cancer.

“This is yet another significant milestone and meaningful validation of our technology as we continue our TRYbeCA-1 phase 3 trial evaluating eryaspase in second-line metastatic pancreatic cancer,” said Gil Beyen, CEO of Erytech. “We believe that the FDA’s Fast Track designation for eryapase underscores its potential to address this high unmet medical need.”

Erytech’s lead product candidate, eryaspase, is being evaluated in a phase 3 trial (TRYbeCA-1) in second-line metastatic pancreatic cancer in 11 countries in Europe and the United States. More than 75% of the approximately 500 patients to be enrolled in the trial have been randomized. An interim superiority analysis, to be conducted by an independent data monitoring committee (IDMC) when two-thirds of the events have occurred, is currently expected to take place around year-end 2020 and the final analysis in the second half of 2021.

In a previous phase 2b trial, eryaspase demonstrated significant improvement in both overall survival (OS) and progression-free survival (PFS) with a Hazard Ratio (HR) of 0.60 and 0.59 respectively. Overall, eryaspase was well tolerated and showed a safety profile comparable to that of standard chemotherapy.

Fast Track is a program designed to facilitate the expedited development and review of a new drug, alone or in combination with other drugs, to treat serious or life-threatening conditions for which there is a demonstration of the potential to address an unmet medical need. The purpose is to advance new drugs earlier for patients who need them.

Pancreatic cancer is a disease in which malignant (cancer) cells are found in the tissues of the pancreas. Every year, there are approximately 150,000 new cases of pancreatic cancer diagnosed in Europe and the United States. Advanced pancreatic cancer is a particularly aggressive cancer, with a five-year survival rate of less than 10%. It is currently the fourth leading cause of cancer death in Europe and the United States and is projected to rise to the second leading cause by 2030. Limited therapeutic options are currently available for this indication, thereby reinforcing the need to develop new therapeutic strategies and rational drug combinations with the aim of improving overall patient outcomes and quality of life.

TRYbeCA-1 is a randomized, controlled phase 3 clinical trial evaluating eryaspase in second-line metastatic pancreatic cancer. The trial is planned to enroll approximately 500 patients at approximately 100 clinical sites in Europe and the United States. Eligible patients are randomized 1-to-1 to receive eryaspase in combination with standard chemotherapy (gemcitabine/nab-paclitaxel or an irinotecan-based regimen) or chemotherapy alone. The primary endpoint of TRYbeCA-1 is overall survival. An interim superiority analysis will be conducted when approximately two-thirds of the events will have occurred.

Fast Track is a program designed to facilitate the expedited development and review of a new drug alone or in combination with other drugs to treat serious or life-threatening conditions for which there is a demonstration of the potential to address an unmet medical need. The purpose is to advance new drugs earlier for patients who need them. Fast Track addresses a broad range of serious conditions.

Date: 29-Jun-2020

US FDA Grants Fast Track Status To Aviptadil To Treat Respiratory Distress In COVID-19

NeuroRx, in partnership with Relief Therapeutics announced that the US Food and Drug Administration (FDA) awarded Fast Track Designation to NeuroRx for the investigation of RLF-100 (Aviptadil) for the treatment of acute lung injury/acute respiratory distress syndrome associated with COVID-19. RLF-100 is a synthetic form of human Vasoactive Intestinal Peptide (VIP) which reduces inflammation in the lungs and protects the alveolar type II cells that are believed to be an entry route for the SARS-CoV-2 to invade the lungs.

As part of NeuroRx’s enrollment in the Fast Track program, the FDA has requested NeuroRx to submit a publicly-available expanded access policy, so that physicians may request RLF-100 for their patients who are being treated in hospitals not participating in the ongoing phase 2/3 clinical trials.

“We at NeuroRx are enormously appreciative of the FDA’s commitment to accelerating the development of any potential treatment for COVID-19. We hope to live up to the trust that has been placed in us by bringing a potentially life-saving treatment to patients,” said Prof. Jonathan Javitt, MD, MPH, CEO and chairman of NeuroRx.

“This milestone demonstrates the effectiveness of the FDA CoronaVirus Treatment Acceleration Program and the FDA’s achievement in rising up to address the most severe Public Health Crisis of our lifetime,” said former FDA Chief Counsel, Daniel Troy, a member of the Company’s board of directors.

RLF-100 is being tested in phase 2/3 clinical trials at major medical centers including the University of Miami, Houston Methodist Hospital, University of California-Irvine, the NYU Langone Medical Center, and the Rambam Healthcare Campus (Haifa, Israel). The multicenter clinical trial enrolls patients with Critical COVID-19 and respiratory failure in the hopes that RLF-100 can decrease mortality and improve blood oxygenation in this condition by rescuing alveolar type II cells from the SARS-CoV-2 virus.

RLF-100 (Aviptadil) is a patented formulation of Vasoactive Intestinal Polypeptide (VIP) that was developed based on Dr. Said’s original work and was originally approved for human trials by the FDA in 2001 and the European Medicines Agency in 2005. VIP is primarily concentrated in the lung and is known to protect against a variety of lung injuries.

Vasoactive Intestinal Polypeptide (VIP) was first discovered by the late Dr. Sami Said in 1970. Although first identified in the intestinal tract, VIP is now known to be produced throughout the body and to be primarily concentrated in the lungs. VIP has been shown in more than 100 peer-reviewed studies to have potent anti-inflammatory/anti-cytokine activity in animal models of respiratory distress, acute lung injury, and inflammation. Most importantly, 70% of the VIP in the body is bound to a rare cell in the lung, the Alveolar Type II cell that is critical to transmission of oxygen to the body. VIP has a 20-year history of safe use in humans in multiple human trials for sarcoidosis, pulmonary fibrosis, asthma/allergy, and pulmonary hypertension.

COVID-19-related death is primarily caused by Respiratory Failure. Before this acute phase, however, there is evidence of early viral infection of the alveolar type 2 cells. These cells are known to have angiotensin converting enzyme 2 (ACE2) receptors at high levels, which serve as the route of entry for the SARS-CoV-2 into the cells. Corona Viruses are shown to replicate in alveolar type 2 cells, but not in the more numerous type 1 cells. 2 These same type 2 alveolar cells have high concentrations of VIP receptors on their cell surfaces giving rise to the hypothesis that VIP could specifically protect these cells from injury.

Injury to the type 2 alveolar cells is an increasingly plausible mechanism of COVID-19 disease progression. (Mason 2020). These specialized cells replenish the more common type 1 cells that line the lungs. More importantly, type 2 cells manufacture surfactant that coats the lung and are essential for oxygen exchange. Other than RLF-100, no currently proposed treatments for COVID-19 specifically target these vulnerable type 2 cells.

Date: 30-Jul-2020

US FDA Grants Fast Track Status To Checkmate Pharmas CMP-001 In Combo With PD-1 Blockade To Treat Certain Types Of Metastatic/unresectable Melanoma

Checkmate Pharmaceuticals, a clinical-stage biotechnology company, announced that the US Food and Drug Administration (FDA) granted Fast Track designation to its product candidate, CMP-001, a differentiated Toll-like receptor 9 (TLR9) agonist, in combination with a programmed death receptor 1 (PD-1) blocking antibody (nivolumab or pembrolizumab) for two development programmes, including initial treatment of patients with unresectable Stage III or Stage IV melanoma to prolong the time to disease progression; and treatment of patients with unresectable or metastatic melanoma refractory to prior anti-PD-1 blockade to improve the overall tumor response rate.

The FDA previously granted Orphan Drug designation to CMP-001 for Stages IIb-IV melanoma.

Fast Track is a designation granted by the FDA to facilitate the development and review of a drug intended to treat a serious condition and for which available nonclinical or clinical data demonstrate the potential to address an unmet medical need. A product candidate granted Fast Track designation may be eligible for several benefits, including more frequent meetings and communications with the FDA review teams and, if relevant criteria are met, the potential for Priority Review or Rolling Review of a Biologics License Application (BLA) or a New Drug Application (NDA).

“These FDA designations for CMP-001 are testaments to the critical need for new drugs designed to treat patients with melanoma,” said Barry Labinger, CEO of Checkmate. “We look forward to continued engagement with the FDA in advancing the development of CMP-001 in combination with PD-1 blockade in melanoma and head and neck squamous cell carcinoma.”

CMP-001 comprises a virus-like particle utilizing a CpG-A oligonucleotide. It is designed to trigger the body’s innate immune system via TLR9 and infiltrate the tumor microenvironment by the subsequent induction of both innate and adaptive anti-tumor immune responses. Checkmate believes CMP-001 is the only compound utilizing a CpG-A class TLR9 agonist in clinical development.

Melanoma is a serious form of skin cancer that arises from a particular skin cell type called a melanocyte. Melanoma is a particularly dangerous form of cancer because of its ability to spread to other organs rapidly if not surgically removed at an early stage, as well as low response rates and limited durability of response when treated with commonly used chemotherapeutics. In 2020, melanoma of the skin is estimated to be the fifth most diagnosed cancer, and accounts for approximately 1% of all skin cancers in the US According to the American Cancer Society, there will be an estimated 100,350 new diagnoses and approximately 6,850 patients will die as a result of melanoma in the United States in 2020 alone

Date: 11-Mar-2021

US FDA Grants Fast Track Status To Passage Bioís Three Gene Therapy Candidates To Treat GM1 Gangliosidosis Frontotemporal Dementia With Granulin Mut

Passage Bio, a genetic medicines company, announced the US Food and Drug Administration (FDA) have granted Fast Track designation to the company’s three lead investigational gene therapies: PBGM01 for the treatment of GM1 gangliosidosis (GM1), PBFT02 for frontotemporal dementia with granulin mutations (FTD-GRN), and PBKR03 for Krabbe disease. Passage Bio plans to initiate clinical trials in GM1 in the first quarter of 2021 and in FTD-GRN and Krabbe disease in the first half of 2021.

“At Passage Bio, we are working to address some of the world’s rarest and most devastating neurological diseases that affect infants and adults,” said Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio. “The FDA’s decision to grant Fast Track designation to each of our lead gene therapy candidates highlights the urgent need for new treatments in these diseases and represents an important step towards achieving our objective of getting potentially transformative therapies to patients as quickly as possible.”

Fast Track designation facilitates the expedited development and review of a new drug that demonstrates potential to address unmet medical needs and treats a serious or life-threatening disease. Benefits of this designation include more frequent interactions with the FDA to discuss the drug’s development plan, as well as eligibility for other regulatory mechanisms intended to expedite development and review, such as priority review.

“In the last year, we have laid the groundwork for Passage Bio to boldly execute on our strategy,” said Dr. Goldsmith. “These regulatory designations are intended to accelerate the timelines in our efforts to make a meaningful difference in patients’ lives.”

PBGM01 is being studied for the treatment of GM1, a rare and often life-threatening CNS disorder that impacts patients worldwide. Passage Bio is targeting the infantile form of the disease, which is the most severe, with a rapid disease course and no current treatment options beyond support care. PBGM01 has received Orphan Drug and Rare Pediatric Disease designations from the FDA, as well as Orphan Drug designation from the European Medicines Agency (EMA). Passage Bio has activated its first clinical trial site in the United States for the global Imagine-1 study evaluating PBGM01 and is currently recruiting patients. The company plans to dose the first patient in the trial in the first quarter of 2021 and to report initial 30-day safety and biomarker data mid-year.

Passage Bio is developing PBKR03 to address the significant unmet treatment need in Krabbe disease, a rare pediatric disorder that has a devastating burden on patients and their families. The company plans to study PBKR03 as a monotherapy for the treatment of early infantile Krabbe disease and was previously granted both Orphan Drug and Rare Pediatric Disease designations for PBKR03 by the FDA.