12032020

Protagonist’s Hepcidin Mimetic Candidate PTG-300 Receives Fast Track Designation from the U.S. FDA for Development in the Treatment of Polycythemia Vera

Protagonist Therapeutics, Inc. (Nasdaq:PTGX) today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to PTG-300 in the treatment of polycythemia vera, a rare disease characterized by the excessive production of red blood cells. PTG-300 is an injectable synthetic peptide mimetic of the natural hormone hepcidin that has demonstrated the ability to dramatically decrease the requirement for phlebotomy in an ongoing Phase 2 study in polycythemia vera patients. PTG-300 has previously received orphan drug designation for the treatment of polycythemia vera from the U.S. FDA.

“Fast Track designation reflects the potential for PTG-300 to improve upon the treatments that are currently available for patients with polycythemia vera, and provides opportunities to substantially accelerate clinical development,” commented Dinesh V. Patel, Ph.D., Protagonist President and Chief Executive Officer. “We continue to enroll and treat patients in our ongoing Phase 2 study, with complete enrollment expected in mid-2021. We look forward to working closely with the FDA to discuss and finalize a pivotal program in the first half of 2021.”

The FDA Fast Track Program is designed to facilitate the development and expedite the review of new therapeutics that are intended to treat serious conditions and that demonstrate the potential to address unmet medical needs. Drugs that receive this designation benefit from more frequent interactions and meetings with the FDA and potential pathways for expedited approval.

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12022020

Acceleron Receives FDA Orphan Drug Designation for ACE-1334 for the Treatment of Systemic Sclerosis

Acceleron Pharma Inc. (Nasdaq: XLRN), a biopharmaceutical company dedicated to the discovery, development, and commercialization of TGF-beta superfamily therapeutics to treat serious and rare diseases, today announced that the United States Food and Drug Administration (FDA) has granted Orphan Drug designation to ACE-1334 for the treatment of patients with systemic sclerosis. The Company intends to initiate a Phase 1b/Phase 2 trial in patients with systemic sclerosis-associated interstitial lung disease (SSc-ILD) in 2021.

“We’re excited that ACE-1334 has received Orphan Drug designation, as it aligns with our vision to develop novel therapies for pulmonary diseases of high unmet medical need,” said Habib Dable, President and Chief Executive Officer of Acceleron. “It’s clear that 2021 will be an important year in this regard, as we seek to expand our portfolio in this important therapeutic area. In addition to advancing our Phase 3 clinical program to investigate sotatercept’s potential to reverse vascular remodeling associated with pulmonary arterial hypertension, we’re pleased to be planning for a Phase 1b/Phase 2 study to determine whether the anti-fibrotic activity ACE-1334 has demonstrated in preclinical models of fibrosis can be replicated in patients with SSc-ILD.”

Orphan designation is granted by the FDA Office of Orphan Products Development to advance the evaluation and development of safe and effective therapies for the treatment of rare diseases or conditions affecting fewer than 200,000 people in the U.S. Under the Orphan Drug Act, the FDA may provide grant funding toward clinical trial costs, tax advantages, FDA user-fee benefits, and seven years of market exclusivity in the United States following marketing approval by the FDA. The granting of an orphan designation request does not alter the standard regulatory requirements and process for obtaining marketing approval. For more information about orphan designation, please visit the FDA website.

ACE-1334 is an investigational therapy that is not approved for any use in any country.

About ACE-1334

ACE-1334 is an Acceleron-discovered, TGF-beta superfamily-based ligand trap designed to bind and inhibit TGF-beta 1 and 3 ligands but not TGF-beta 2. TGF-beta 1 and 3 are believed to be key signaling factors in the pathogenesis of fibrotic disease. ACE-1334 has shown robust anti-fibrotic activity in multiple preclinical models of fibrosis. ACE-1334 recently completed an ascending-dose Phase 1 clinical trial in healthy volunteers. The FDA has granted Fast Track designation to ACE-1334 in patients with systemic sclerosis-associated interstitial lung disease (SSc-ILD) as well as Orphan Drug designation for the treatment of systemic sclerosis.

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12012020

Zymeworks Receives FDA Breakthrough Therapy Designation for HER2-Targeted Bispecific Antibody Zanidatamab in Patients with Biliary Tract Cancer

Zymeworks Inc (NYSE: ZYME), a clinical-stage biopharmaceutical company developing multifunctional biotherapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for zanidatamab in patients with previously-treated HER2 gene-amplified biliary tract cancer (BTC).

The FDA grants Breakthrough Therapy designation to new medicines that are intended to treat a serious condition and where clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint. Zanidatamab will now be eligible for Accelerated Approval, Priority Review and Rolling Review, as well as intensive FDA guidance on an efficient drug development program.

“This Breakthrough Therapy designation from the FDA, based on data generated in BTC patients treated in the initial Phase 1 trial, is recognition of the potential of zanidatamab to provide a new approach to cancer treatment,” said Diana Hausman, M.D., Chief Medical Officer at Zymeworks. “This milestone supports our strategy for accelerated approval and will help make zanidatamab available for patients as quickly as possible.”

“BTC is a rare and aggressive cancer,” said James Priour, Senior Vice President, Commercial, at Zymeworks. “Receiving this designation from the FDA is testament to the potential of zanidatamab to be the first HER2-targeting therapy approved for metastatic BTC patients.”

Earlier this year, Zymeworks initiated a global Phase 2b registration-enabling study of single agent zanidatamab in patients with previously treated HER2 gene-amplified BTC. This study, which is currently enrolling patients, is designed to support accelerated approval based on a primary endpoint of objective response rate, and secondary endpoints of duration of response and safety and may enable submission of a Biologics License Application (BLA) as early as 2022.

This Breakthrough Therapy designation was based on an ongoing clinical trial of zanidatamab in patients with locally advanced (unresectable) and/or metastatic HER2-expressing tumors including BTC. Updated clinical data for single agent zanidatamab patients with BTC has been accepted for presentation at the upcoming American Society of Clinical Oncology’s Virtual Gastrointestinal Cancers Symposium (ASCO GI) January 15-17, 2021.

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11302020

Prevail Therapeutics Receives European Commission Orphan Designation for PR006 for the Treatment of Frontotemporal Dementia

Prevail Therapeutics Inc. (Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the European Commission has granted orphan designation for PR006 for the treatment of frontotemporal dementia (FTD). PR006 is an investigational AAV9 gene therapy delivering the GRN gene and is being developed as a potential therapy for patients with frontotemporal dementia with GRN mutations (FTD-GRN).

“The European Commission’s decision to grant orphan designation for PR006 is an important step in helping to advance this potential therapeutic option for patients with frontotemporal dementia with GRN mutations,” said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. “We are excited to progress clinical development of PR006 as part of our mission to deliver a potentially disease-modifying gene therapy to these patients as quickly as possible.”

The Company expects to initiate enrollment in the Phase 1/2 PROCLAIM trial of PR006 in the fourth quarter of 2020, and it currently anticipates it will provide a biomarker and safety analysis on a subset of patients in the PROCLAIM trial in 2021.

The U.S. Food and Drug Administration has also granted Orphan Drug designation for PR006 for the treatment of FTD and Fast Track designation for FTD-GRN.

About European Commission Orphan Designation
Orphan designation is granted by the European Commission to encourage development of medicines intended to treat a seriously debilitating or life-threatening condition that affects fewer than five in 10,000 people in the European Union. Orphan designation by the European Commission provides companies with certain benefits and incentives, including protocol assistance, reduced regulatory fees and 10 years of market exclusivity following regulatory approval.

About Prevail Therapeutics
Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The Company is developing PR001 for patients with Parkinson’s disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD); PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

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11252020

PolyPid Granted Breakthrough Therapy Designation from FDA for D-PLEX₁₀₀ for the Prevention of Surgical Site Infections in Colorectal Surgery

PolyPid Ltd. (Nasdaq: PYPD), a Phase 3 clinical-stage biopharmaceutical company focused on developing targeted, locally administered and prolonged-release therapeutics using its proprietary PLEX technology, today announced that D-PLEX100 has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for the prevention of surgical site infections (SSIs) in patients undergoing elective colorectal surgery.

Breakthrough Therapy Designation is an FDA process designed to expedite the development and review of drugs that are intended to treat a serious or life-threatening condition so patients may have access to therapies through FDA approval as soon as possible. This designation is granted based on preliminary clinical evidence indicating that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints.

“The Breakthrough Therapy Designation in the field of anti-infective drugs is rather rare, and further supports the urgency to develop new innovative therapies to prevent SSIs,” said Amir Weisberg, PolyPid’s CEO. “It also reflects on the promising clinical data of D-PLEX100 in the prevention of SSIs in complex surgical settings, such as colorectal abdominal surgeries. We are looking forward to working closely with the FDA to make D-PLEX100 available to surgeons and patients as quickly as possible. Our ongoing Phase 3 pivotal studies in abdominal surgery – SHIELD I and SHIELD II – are on track.”

The Breakthrough Therapy Designation for D-PLEX100 is based on conclusive positive results from a Phase 2 clinical trial evaluating D-PLEX100 for the prevention of surgical site infections (SSIs) in abdominal colorectal surgery. The Phase 2 clinical trial was a prospective, multicenter, randomized, controlled two arm study in 201 patients and demonstrated that the local administration of D-PLEX100 resulted in a statistically significant decrease in SSIs of 59 percent in the Intent to Treat (ITT) population (p=0.0086), and a decrease of 69 percent in the Per Protocol population (n=179; p=0.0024), as compared to the standard of care alone.

D-PLEX100 previously received two Fast Track Designations from the FDA for the prevention of post-abdominal surgery incisional infections and for the prevention of sternal wound infections post-cardiac surgery, as well as two Qualified Infectious Disease Product designations (QIDP’s) in the same indications.

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BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

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11242020

Theralase Granted FDA Fast Track Designation for NMIBC Phase II Clinical Study

Theralase® Technologies Inc. (“Theralase” or the “Company“) (TSXV:TLT) (OTCQB:TLTFF), a clinical stage pharmaceutical company focused on the research and development of light activated Photo Dynamic Compounds (“PDC“) and their associated drug formulations used to safely and effectively destroy various cancers, bacteria and viruses announced today that the U.S. Food and Drug Administration (“FDA”) has granted Theralase® Fast Track Designation (“FTD“) for its Phase II Bacillus Calmete Guérin (“BCG“)-Unresponsive Non Muscle Invasive Bladder Cancer (“NMIBC“) Carcinoma In Situ (“CIS“) clinical study (“Study II“).

As a Fast Track designee, Theralase® will have access to early and frequent communications with the FDA to discuss Theralase’s development plans and ensure timely collection of the appropriate clinical data to support the approval process. The accelerated communication with the FDA potentially allows, TLD-1433, in combination with the TLC-3200 medical laser system (“TLC-3200“), to be the first intravesical patient-specific Ruthenium-based PDC for the treatment of patients with BCG-Unresponsive NMIBC CIS, with or without papillary Ta or T1 tumours. FTD can lead to an Accelerated Approval and Priority Review, if certain criteria are met, which the FDA has previously defined to the Company to represent approximately 20 to 25 patients enrolled and treated, who demonstrate significant safety and efficacy clinical outcomes.

Michael Jewett MD, FRCSC, FACS, Inaugural Farquharson Clinical Research Chair in Oncology, Departments of Surgery (Urology) and Surgical Oncology, Princess Margaret Cancer Centre, University Health Network (“UHN“), stated “By awarding Fast Track Designation to the photosensitizer drug TLD-1433 activated by the laser TLC-3200, currently being assessed in a Phase II clinical study for the treatment of NMIBC, the FDA has recognized Theralase®’s potential to meaningfully improve patient outcomes for this life-threatening disease. This is a significant accomplishment for the Company. This latest milestone complements the clinical development strategy to provide urologists, uro-oncologists and patients with the tools to combat BCG-Unresponsive NMIBC, safely and effectively.”

Shawn Shirazi PhD, Chief Executive Officer, Theralase®, stated, “FDA’s FTD for our lead drug candidate, TLD-1433, activated by the TLC-3200, is another important milestone for Theralase®, as it can potentially speed the development of this drug-device combination for NMIBC patients. The TLD-1433 – TLC-3200 technology represents a paradigm shift in medical technology and an advanced approach to treat NMIBC. We are excited by the progress the Company has delivered in Study II, as we continue to enroll and successfully treat patients. The Company continues to work towards launching new clinical study sites in Canada and the US with a mandate to enroll and treat all patients for their first Study II treatment in 2021”.

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BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

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11232020

BrainEver Receives FDA Orphan Drug Designation for BREN-02, (human recombinant Engrailed 1) for the Treatment of Amyotrophic Lateral Sclerosis (ALS)

BrainEver, a biotechnology company dedicated to the research and development of innovative therapies for the treatment of neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its product BREN-02, the recombinant human homeoprotein Engrailed 1 (rhEN1), for the treatment of amyotrophic lateral sclerosis (ALS).

ALS (also known as Charcot disease) is a severe neurodegenerative disease affecting motor neurons, resulting in progressive muscle weakness leading to paralysis, with a vital prognosis of approximately 2-4 years from onset. ALS is diagnosed in approximately 16,000 people each year in Europe and North America.

“In ALS, motor neurons degenerate and die, leading to progressive muscle paralysis that includes respiratory muscles. In preclinical studies, we have observed that the intrathecal lumbar administration of rhEN1, in the early symptomatic stages, restores muscle function and prevents motor neuron death”, said Prof. Alain Prochiantz, neurobiologist at the Collège de France and co-founder of BrainEver:

BREN-02 is expected to be administered in clinical trials in ALS patients in the second half of 2021, subject to preclinical toxicity results and regulatory review. Preclinical studies have shown that the homeoprotein hEN1 is essential for the survival and maintenance of spinal cord alpha motor neurons that innervate muscles throughout the body.

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BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

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11202020

ExeGi Pharma Announces FDA Orphan Drug Designation for EXE-346 Live Biotherapeutic

ExeGi Pharma LLC, a company focused on developing live biotherapeutic (“LBP”) drugs and probiotics, announced today that its biologic drug candidate, EXE-346, has received an orphan drug designation from the U.S. Food and Drug Administration (FDA) for the prevention of disease relapse in pouchitis. This designation grants certain benefits to drugs and biological products intended to treat diseases which affect less than 200,000 patients in the U.S. EXE-346 is an LBP which contains a fixed-proportion of 8 strains of live probiotic bacteria which are intended for oral administration.

Pouchitis is a condition which impacts patients whom have had a surgical procedure to remove their colon. The surgery, called a pouch anastomosis, creates an intestinal “J-Pouch” with a portion of the lower intestine. While this procedure enables patients to pass stool normally, one challenge is pouchitis, a condition in which the reservoir becomes inflamed. While it’s not clear what causes pouchitis, studies suggest that an abnormal immune response to the bacterial microflora of the pouch may play a role in its development. The most common pouchitis symptoms are an increased number of bowel movements, abdominal pain, joint pain, cramps, and fever. There is currently no approved treatment for managing the symptoms associated with pouchitis or for helping patients reach remission. EXE-346 is intended to decrease this inflammation of the pouch and prevent active disease flare-ups. Antibiotics such as Ciprofloxacin are commonly used to treat the active form of the disease, suggesting a clear connection to a dysregulated pouch microbiome.

“The J-Pouch surgery is a great option for many people that have long suffered from ulcerative colitis and other rare conditions, but pouchitis can occur in a significant portion of these patients,” commented Dr Paolo Gionchetti of the University of Bologna in Italy. “I am pleased to see that the U.S. FDA has granted this product Orphan status because these patients need new treatment options to help maintain a normal quality of life.” 

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BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

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11192020

F2G Receives Second US FDA Breakthrough Therapy Designation for Olorofim

F2G Ltd, a UK- and Austria-based biotech company developing novel therapies for life-threatening systemic fungal infections, announced today that the US Food and Drug Administration (FDA) has granted an additional Breakthrough Therapy Designation to its lead first-in-class candidate, olorofim, for the indication of ‘Treatment of Central Nervous System (CNS) coccidioidomycosis refractory or otherwise unable to be treated with standard of care therapy’. This is the second Breakthrough Therapy Designation for olorofim; a designation was granted previously on 11 November 2019 for ‘Treatment of invasive mold infections in patients with limited or no treatment options, including aspergillosis refractory orintolerant tocurrently available therapy, and infections due to Lomentospora prolificans, Scedosporium, and Scopulariopsis species’. Olorofim (formerly F901318) is the first antifungal agent to be granted Breakthrough Therapy Designation.

Olorofim has orphan drug designation in the United States for coccidioidomycosis, an endemic fungal infection with a geographic distribution focused mainly in the desert Southwest of the United States but also including Mexico, Central America, and South America. Infection begins by inhalation and can spread to any part of the body, even in otherwise healthy individuals. Spread to the brain is particularly feared as it produces a devastating illness that cannot always be controlled with any currently available agent.

Patients with coccidioidomycosis are being studied in an ongoing open-label single-arm Phase 2b study (ClinicalTrials.gov Identifier: NCT03583164) in patients with proven invasive fungal disease (IFD) or probable invasive aspergillosis (IA) and either refractory disease, resistance, or intolerance to available agents. Olorofim has been well tolerated across more than 30 years of cumulative patient dosing days with a median therapy duration of 12 weeks. Preliminary data from this study were provided to the FDA as part of the Breakthrough Therapy Designation submission.

Breakthrough Therapy Designation is an FDA process designed to expedite the development and review of drugs that are intended to treat a serious or life-threatening condition and is granted based on preliminary clinical evidence indicating that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints.

Breakthrough Therapy Designation conveys all the features of fast track designation, more intensive FDA guidance on an efficient drug development program, an organisational commitment by FDA to involve senior managers, and eligibility for rolling review and priority review.

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11182020

Aruvant Receives Orphan Designation from European Medicines Agency for Gene Therapy ARU-1801 for Sickle Cell Disease

Aruvant, a clinical-stage biopharmaceutical company focused on developing and commercializing transformative gene therapies for the treatment of rare diseases, today announced that the European Medicines Agency (EMA) has granted orphan designation for ARU-1801, an investigational lentiviral gene therapy. The U.S. Food and Drug Administration (FDA) previously granted Orphan Drug and Rare Pediatric Disease Designations to ARU-1801 for the treatment of sickle cell disease (SCD).

“The EMA orphan designation highlights the importance of ARU-1801, our potentially curative gene therapy for individuals living with severe SCD,” said Will Chou, M.D., chief executive officer of Aruvant. “We are encouraged by the preliminary clinical data from our ongoing Phase 1/2 study that demonstrates ARU-1801, administered with only reduced intensity conditioning, can achieve durable reductions in disease burden.”

Orphan drug designation in the European Union (EU) is granted by the European Commission based on a positive opinion issued by the EMA Committee for Orphan Medicinal Products. To qualify, an investigational medicine must be intended to treat a seriously debilitating or life-threatening condition that affects fewer than five in 10,000 people in the EU, and there must be sufficient non-clinical or clinical data to suggest the investigational medicine may produce clinically relevant outcomes. EMA orphan drug designation provides companies with certain benefits and incentives, including clinical protocol assistance, differentiated evaluation procedures for Health Technology Assessments in certain countries, access to a centralized marketing authorization procedure valid in all EU member states, reduced regulatory fees and 10 years of market exclusivity upon marketing authorization.

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