01-21-2022

Marker Therapeutics Receives FDA Orphan Drug Designation for its T Cell Therapy for Pancreatic Cancer

Marker Therapeutics, Inc.(NASDAQ:MRKR), a clinical-stage immuno-oncology company specializing in the development of next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications, today announced that the United States Food and Drug Administration (FDA) Office of Orphan Products Development has granted Orphan Drug designation to MT-601, a multi-tumor-associated antigen (MultiTAA)-specific T cell product optimized for the treatment of patients with pancreatic cancer.

“The FDA’s orphan drug designation underscores MT-601’s potential as a treatment for pancreatic cancer, a cancer typically diagnosed at an incurable advanced stage with a total overall 5-year survival rate of 10%,” said Peter L. Hoang, President & CEO of Marker Therapeutics. “Our novel therapy has shown encouraging results in an ongoing Phase 1 trial sponsored by Marker’s partners at the Baylor College of Medicine. In results reported at the 2020 American Society of Clinical Oncology (ASCO) Virtual Annual Meeting, our therapy has demonstrated the potential to safely produce durable responses in combination with chemotherapy as a first-line treatment option for patients with advanced or metastatic pancreatic adenocarcinoma. The results also revealed that epitope spreading was consistent in responders to Multi-TAA-specific T cells. Following MT-401 for the treatment of post-transplant acute myeloid leukemia (AML), MT-601 is Marker’s second novel MultiTAA-specific T cell product to receive orphan drug designation and the first in a solid tumor indication, underscoring the potential of Marker’s multi-antigen targeting T cell therapy approach in both solid tumors and blood cancers.”

Marker developed MT-601, a new product targeting six tumor-associated antigens (PRAME, NY-ESO-1, Survivin, MAGE-A4, SSX2, WT1) highly expressed in pancreatic cancer. The Company intends to initiate a Phase 1 multicenter study of MT-601 administered in combination with front-line chemotherapy to patients with locally advanced unresectable or metastatic pancreatic cancer. Marker designed the study to include an initial antigen escalation period followed by a dose escalation period and will enroll 20 – 25 patients for the study.

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Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

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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01-20-2022

Califf, experts eye legislative fix for accelerated approval program

Regulatory and industry experts agree that completing confirmatory studies quickly is essential to the success of the US Food and Drug Administration’s (FDA) accelerated approval program, with some suggesting that new legislation might be necessary to reliably get results from these trials in a timelier manner.

Robert Califf, head of clinical policy and strategy for Verily and Google Health and the Biden Administration’s nominee to be FDA commissioner, said a legislative framework is needed to stimulate confirmatory evidence generation because current incentives are not properly aligned. (RELATED: Senate committee advances Califf’s nomination as FDA commissioner, Regulatory Focus 13 January 2022)

“Unless we have teeth in this, it’s not going to work,” Califf said at the recent UCSF-Stanford Center of Excellence in Regulatory Science and Innovation (CERSI) Innovations in Regulatory Science Summit.

He also indicated that the legislators he’s met with during the confirmation process have been particularly interested in the accelerated approval program. “It was part of the conversation with almost every senator on the [Health, Education, Labor, and Pensions] HELP committee in our one-on-one meetings, and I can assure you that Congress is very eager to understand what’s going on with accelerated approval because there’s such a division of views in the public domain, even among notable academics,” Califf said.

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Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

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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01-18-2022

Adverum Biotechnologies Granted Orphan Drug Designation by FDA for Gene Therapy Candidate for the Treatment of Blue Cone Monochromacy

Adverum Biotechnologies (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to ADVM-062 (AAV.7m8-L-opsin), a novel gene therapy product candidate being developed as a potential single intravitreal (IVT) administration for blue cone monochromacy (BCM) by delivering a functional copy of the OPN1LW gene.

“We are excited to introduce ADVM-062, which builds on our ophthalmology and gene therapy expertise to address the significant unmet medical needs of patients living with blue cone monochromacy. We are pleased the FDA has recognized the patient need by granting Orphan Drug Designation and we look forward to advancing ADVM-062 towards the clinic and generating important data,” stated Laurent Fischer, M.D., president and chief executive officer of Adverum Biotechnologies.

BCM affects approximately 1 to 9 in 100,000 males, worldwide. This X-linked recessive hereditary condition is caused by mutations in either the L or the M opsin gene(s) and can manifest in loss of visual acuity, photophobia, myopia and infantile nystagmus that can persist into adulthood. Consequently, individuals with BCM have visual impairments to important aspects of daily living such as facial recognition, learning, reading, and daylight vision. Currently, there is no cure for BCM.  

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Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

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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01-14-2022

COUR Pharmaceuticals Receives FDA Fast Track Designation for CNP-104 for the Treatment of Primary Biliary Cholangitis

Cour Pharmaceuticals, a biotechnology company developing novel immune-modifying nanoparticles (CNPs) to treat immune disorders, today announced the U.S. Food and Drug Administration (FDA) granted Fast Track Designation to the Company’s investigational therapy CNP-104 for the treatment of Primary Biliary Cholangitis. CNP-104 aims to reprogram the immune system to address autoimmune causes of PBC.

“We are pleased to have received Fast Track Designation for CNP-104, which demonstrates the FDA recognizes the importance of this potential treatment for PBC and the need for improved of clinical outcomes for patients with this degenerative autoimmune disease, which currently has no treatment to stop progression of disease,” said COUR CEO John Puisis. “Based on data from another COUR autoimmune trial with a CNP platform product, our Phase 2 PBC trial is designed to show that we can stop progression of PBC disease with improved liver function.  If successful in Phase 2, COUR would seek breakthrough therapy designation in PBC given that current therapies treat only symptoms.”

Fast Track Designation is an FDA process designed to facilitate the development and review of potential therapies that seek to fill an unmet medical need. The FDA created this designation to help get new therapies to patients earlier. A therapy that receives this designation is eligible for more frequent communication with the FDA and may be eligible for Accelerated Approval and Priority review, if relevant criteria are met. 

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Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

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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01-13-2022

FDA Grants Fast Track and Regenerative Medicine Advanced Therapy Designations to C-CAR039 for DLBCL

Cellular Biomedicine Group Inc. (CBMG or the “Company”), a biopharmaceutical company developing innovative cellular immunotherapies for the treatment of cancer, today announced that the Food and Drug Administration (FDA) granted C-CAR039, a novel autologous bi-specific CAR-T therapy targeting both CD19 and CD20 antigens, both Regenerative Medicine Advanced Therapy (RMAT) Designation and Fast Track Designation for the treatment of patients with relapsed or refractory diffuse large B cell lymphoma (r/r DLBCL).

This is great news for CBMG that the FDA has granted C-CAR039 both RMAT and fast track designations based on its potential to increase objective and complete response rates in r/r DLBCL. The clinical data based on our clinical trials in China continue to support the hypothesis that C-CAR039 is the best-in-class CAR-T asset for patients in this indication. We are working towards initiating 1b/2 trials for C-CAR039 in the US soon. And we will work closely with the FDA to seek the best path forward to deliver the drug to patients in the US and EU,” said Tony (Bizuo) Liu, Chairman and CEO.

About C-CAR039
Early clinical results of C-CAR039 from an investigator-initiated trial (IIT) conducted across multiple sites in China demonstrate exciting efficacy and favorable safety data of C-CAR039 in r/r B-cell NHL. As of April 20, 2021, a total of 34 patients received C-CAR039 cell therapies, with 28 patients evaluable for safety analyses and 27 patients evaluable for efficacy analyses. Patients’ median age was 55.5 years, and 75% had cancer of Ann Arbor stage III/IV. Patients had a median of three prior lines of therapy. Bridging therapy had been given to 17.9% of patients. The best overall response rate (ORR) was reported to be 92.6%, with a complete response rate (CRR) of 85.2%. Patients had a median time to response of 1.0 month, and at a median follow-up of 7 months, 74.1% of patients continued to be in complete remission. The 6-month estimated progression-free survival rate was 83.2% (95% CI, 69.1%-100.0%). Cytokine release syndrome (CRS) occurred in 96% of patients. 92% of CRS was of grade 1/2 and only 1 patient had grade 3 CSR. Immune effector cell-associated neurotoxicity syndrome occurred at grade 1 in 2 patients and no ≥grade 2 neurologic events reported in the study. CBMG will continue to evaluate patients with longer follow-up (ClinicalTrials.gov Identifiers: NCT04317885, NCT04655677, NCT04696432, NCT04693676).

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Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

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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01-11-2022

Oncolyze Announces FDA Orphan Drug Designation for OM-301 for the Treatment of Acute Myeloid Leukemia

Oncolyze, Inc, a biotech developing a disruptive technology for the treatment of cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted the company orphan drug designation for its pipeline compound OM-301 for the treatment of acute myeloid leukemia (AML). OM-301 is currently in pre-clinical development for AML and other cancers.

The FDA’s Office of Orphan Drug Products grants orphan status to drugs and biologics that demonstrate promise for the treatment of diseases or conditions affecting fewer than 200,000 people in the United States. According to the American Cancer Society, there are approximately 20,000 new cases of AML each year. Orphan drug designation provides Oncolyze with certain development incentives, including tax credits for qualified clinical testing, exemptions from certain FDA application fees, and potential market exclusivity, if approved. 

“We are actively fundraising to advance OM-301 into clinical trials. The orphan drug designation is an important milestone that further validates our efforts to investigate the value of OM-301 as a potentially groundbreaking therapy for patients with AML and other cancers,” said Dr. Steven Evans, Co-Founder & CEO of Oncolyze.

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Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

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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01-07-2022

FDA Grants Breakthrough Therapy Designation for Cullinan Oncology’s CLN-081 in Patients with Non-Small Cell Lung Cancer

Cullinan Oncology (Nasdaq: CGEM) (“Cullinan”), a biopharmaceutical company focused on developing a diversified pipeline of targeted therapies for cancer patients, today announced that the U.S. Food and Drug Administration (“FDA”) has granted Breakthrough Therapy Designation for CLN-081 for the treatment of patients with locally advanced or metastatic non-small cell lung cancer (“NSCLC”) harboring epidermal growth factor (“EGFR”) exon 20 insertion mutations who have previously received platinum-based systemic chemotherapy.

“We are extremely pleased that Cullinan has received breakthrough therapy designation from the FDA for CLN-081, a distinction that underscores the urgent need to bring improved targeted treatments to this patient population and further supports the differentiated clinical profile of CLN-081,” said Nadim Ahmed, Chief Executive Officer of Cullinan Oncology. “The updated data from our ongoing Phase 1/2a study in a larger number of patients have demonstrated a high response rate with durable responses and encouraging progression free survival in heavily pre-treated patients. We are also encouraged by the favorable safety profile observed thus far, and we look forward to ongoing, productive regulatory discussions with the FDA, which are further enabled with this designation.”

Breakthrough Therapy Designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).

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Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

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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12-29-2021

NRx Pharmaceuticals Files Breakthrough Therapy Designation Request for ZYESAMI® in Patients at Immediate Risk of Death from COVID-19 Despite Treatment

NRx Pharmaceuticals (Nasdaq: NRXP) announced today that it has filed a new Breakthrough Therapy Designation (BTD) request with the US Food and Drug Administration (FDA) focused on patients with Critical COVID-19 and respiratory failure who are at immediate risk of death despite treatment with remdesivir and other approved therapies.

NRx filed the request after the FDA’s request for clinical data on ZYESAMI® vs. Remdesivir in these high risk patients. Prof. David Schoenfeld, one of the world’s most widely published statisticians with unique expertise in life-threatening diseases of the lung conducted the new analysis.

ZYESAMI® (aviptadil) has demonstrated a statistically significant two-fold increased odds of survival compared to placebo across all patients and hospitals studied in a randomized trial of 196 patients.  However, in 70% of patients who were already treated with Remdesivir and continued to progress despite all approved therapies, ZYESAMI has demonstrated a highly significant four-fold increased odds of survival compared to placebo at 60 days (P=.006). Moreover, those treated with ZYESAMI after Remdesivir has failed demonstrate a 3-fold increased odds of being both alive and free of respiratory failure at both 28 and 60 days, compared to placebo (P=.03).

The FDA recently declined Emergency Use Authorization (EUA) and Breakthrough Therapy Designation (BTD) for ZYESAMI and invited a new request based on new clinical evidence that aviptadil may demonstrate a significant improvement in treatment over existing therapies. Based on the FDA’s input, NRx has narrowed its BTD request to treatment of COVID-19 respiratory failure in patients who progress despite treatment with remdesivir and other approved therapies.

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Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

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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12-29-2021

Drug development and working with specialised Rare Disease networks

There are currently an estimated 300 million people living with one or more Rare Diseases across the world. There are over 7,000 Rare Diseases that have been identified, yet only 5% have treatments.

However, despite the work still left to be done, there are now a growing number of treatments in the pipeline and 2020 proved to be a remarkable year for orphan drug approvals, with the US FDA approving more orphan designated treatments than non-orphan drugs.

In a recent webinar held by pharmaphorum, in partnership with IQVIA, a discussion was held looking at how to make academic and commercial medical research more efficient, while also looking at the opportunity of genomic data to bring benefits to patients and healthcare systems.

Dr Jeff Keefer, VP and Head of the Pediatric and Rare Disease Center of Excellence at IQVIA, was one of the three panellists present at the webinar, and he stated that what makes the space unique is the “strength and depth of the relationships that form within the Rare Disease community.”

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Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

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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01-03-2022

Celularity Receives Fast Track Designation from FDA for its NK Cell Therapy for the Treatment of AML

Celularity Inc. (Nasdaq: CELU) (“Celularity”), a clinical-stage biotechnology company developing placental-derived allogeneic cell therapies, today announced the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for its non-genetically modified cryopreserved human placental hematopoietic stem cell-derived natural killer (NK) cell therapy, CYNK-001, in development for the treatment of acute myeloid leukemia (AML).

“The majority of patients with AML continue to have poor long-term outcomes, particularly those who suffer relapse or have measurable residual disease, necessitating development of novel therapies, including CYNK-001,” said Andrew Pecora, M.D., President of Celularity. Robert Hariri, M.D., Ph.D., Founder, Chairperson and Chief Executive Officer of Celularity added, “We believe that the unique properties of our cell source, including the ability to proliferate and maintain activity, could be the key to improving response rates and durability for patients. We are pleased to receive this fast-track designation from the FDA for AML supporting continued development of our placental-derived NK cell platform. CYNK-001 previously received orphan drug designation for malignant gliomas and fast track designation for glioblastoma multiforme.”

About Fast Track Designation

Fast Track Designation is an FDA process designed to facilitate the development and expedite the review of new drugs that are intended to treat a serious condition and have the potential to address unmet medical needs. The purpose of Fast Track designation is to expedite the process of getting important new drugs to patients. The designation may offer frequent interactions with the FDA review team on the product’s development and the product may be eligible for rolling review and priority review if certain criteria are met. 

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Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

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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