Posts in category: Drug Discovery News

Based on promising responses seen across several clinical trials of poziotinib in patients with non–small cell lung cancer and exon 20 mutations in HER2, the FDA granted the therapy fast track designation.

The investigational epidermal growth factor inhibitor poziotinib was granted fast track designation by the FDA as therapy for patients with non–small cell lung cancer (NSCLC) whose tumors harbored HER2 exon 20 mutations, according to Spectrum Pharmaceuticals who is responsible for developing the agent.¹

This designation is granted to medications in order to facilitate and expedite their development and review to become available to treat patients who might not otherwise have a viable systemic therapy option.

“There are currently no approved therapies to treat patients with HER2 exon 20 mutations and we are pleased that the FDA has granted fast track designation for poziotinib,” Joe Turgeon, President and CEO of Spectrum Pharmaceuticals, said in a press release. “Momentum is building to unlock the potential of poziotinib.”

Previously, a single-center trial (NCT03066206) conducted at MD Anderson Cancer Center showed a higher rate of confirmed responses at 43% in patients with heavily pretreated EGFR or HER2 exon 20–mutant disease who received poziotinib therapy. Responses were seen regardless of if patients were refractory or naïve to therapy with other tyrosine kinase inhibitors.²

A recent presentation at the European Society for Medical Oncology Targeted Anticancer Therapies (ESMO TAT) Virtual Congress 2021 held March 1-2, 2021, detailed the results of 2 cohorts explored in the ZENITH20 trial (NCT03318939) of poziotinib in patients with NSCLC and mutations in exon 20 in either EGFR and HER2. Cohorts 2 and 4 of the trial are specifically for the assessment of patients with HER2 exon 20 mutations and previously treated or treatment-naïve disease, respectively, with the latter still open to enrollment. The primary end point of the trial is objective response rate with secondary outcomes of disease control rate, duration of response, and progression-free survival.³

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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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Celularity Inc. (“Celularity”), a clinical-stage biotechnology company, leading the next evolution in cellular medicine with the development of off-the-shelf allogeneic therapies derived from the postpartum human placenta, announced that the company has received Fast Track Designation from the U.S. Food and Drug Administration (FDA) for its non-genetically modified cryopreserved human placental hematopoietic stem cell-derived natural killer (NK) cell therapy, CYNK-001, for the treatment of adults with recurrent glioblastoma multiforme (GBM). CYNK-001 is currently being investigated in multiple clinical trials, including a Phase 1 clinical trial for GBM.

“This designation marks a meaningful milestone for Celularity and validates the potential of our approach to change the cell therapy landscape and improve the lives of patients with GBM and beyond,” said Robert J. Hariri, M.D., Ph.D., founder, Chairperson and Chief Executive Officer of Celularity. “We believe the unique attributes of placental-derived cell therapies will allow us to not only overcome the challenges of NK cell proliferation and persistence, but also improve therapeutic availability as compared to adult bone marrow or peripheral blood approaches. We look forward to working more closely with the FDA to advance our clinical trials and, ultimately, to bringing a new potential treatment option to patients suffering from this historically challenging disease.”

About Fast Track Designation

Fast Track Designation is an FDA process designed to facilitate the development and expedite the review of investigational product candidates that are intended to treat a serious condition and have the potential to address unmet medical needs. Fast Track Designation offers early and frequent interaction with the FDA on product development and review process. It may also provide for rolling or priority review of a Biologics License Application if certain criteria are met.

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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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BioXcel Therapeutics, Inc. (“BioXcel” or the “Company”) (Nasdaq: BTAI), a clinical-stage biopharmaceutical company utilizing artificial intelligence approaches to develop transformative medicines in neuroscience and immuno-oncology, today announced that BXCL501, the Company’s investigational, proprietary, orally dissolving thin film formulation of dexmedetomidine (“Dex”), has been granted Breakthrough Therapy designation from the U.S. Food and Drug Administration (“FDA”) for the acute treatment of agitation associated with dementia. The Breakthrough Therapy designation is intended to expedite the development and review of certain product candidates designed to treat serious or life-threatening diseases or conditions, and the designation includes increased interaction and guidance from the FDA. 

“Managing dementia related agitation, specifically in elderly patients, represents a significant challenge for physicians and caregivers, as there are currently no FDA-approved therapies and off-label drugs come with black box warnings,” stated Vimal Mehta, Chief Executive Officer of BioXcel. “The FDA’s decision to grant Breakthrough Therapy designation further underscores the significant unmet need for a new treatment for this underserved patient population, as well as highlights BXCL501’s potential in becoming the first therapeutic option, if approved, to address this debilitating medical condition. We look forward to working closely with the FDA to advance BXCL501 into a pivotal dementia program, in hopes of quickly bringing this therapy to the millions of patients across treatment settings that lack alternative options.”

The Breakthrough Therapy designation for BXCL501 was supported by the positive topline data from the Phase 1b/2 TRANQUILITY study for the acute treatment of agitation associated with dementia, including Alzheimer’s disease. BXCL501 demonstrated statistically significant reductions in agitation measures at 2 hours post-dose with both the 30 and 60 mcg doses as measured by multiple scales. The dose dependent response observed has the potential to support the Company’s plans to evaluate BXCL501 for use across the full range of dementia care settings.

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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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BERLIN HEALS, a company developing novel life-saving treatments for heart failure, announced today that the US Food and Drug Administration (FDA) has granted a Breakthrough Device Designation for its proprietary Cardiac Microcurrent Therapy Device (“C-MIC”). The C-MIC is an implantable device that delivers a constant but minimal electrical DC-current to the heart via two electrodes. Based on results of first-in-human studies in 2019/20, the C-MIC device has shown potential to become the first near-curative heart failure therapy. Berlin Heals, which has already launched a European-wide CE-study, is now about to file an Investigational Device Exemption (IDE) – Application with the FDA for an Early Feasibility Study (EFS) to commence in the US later this year.

The FDA’s Breakthrough Devices program accelerates the development of medical devices with the potential to effectively treat or diagnose life-threatening or irreversibly debilitating diseases. Heart failure, the world’s most lethal disease, is a chronic inflammatory disorder affecting millions of patients globally. It is responsible for 16% of all deaths and has shown the largest increase amongst all fatal diseases since 2000, rising by more than 2 million to 8.9 million deaths in 2019. The C-MIC device has shown potential to be the first treatment to enable patients of cardiac failure to lead a normal and mobile life and has consequently been assigned as a breakthrough device by the FDA.

As of today, no therapy, drug, or method has been developed and made available to reverse cardiac failure. Designation of the C-MIC as a breakthrough device by the FDA allows significant acceleration in the development and review of the technology, providing patients faster access. Marko Bagaric, CEO of Berlin Heals said, “Our completely novel therapeutic approach has the potential to almost cure heart failure rather than just delaying the progression of the disease. The FDA Breakthrough Designation is a tremendous turning point in the approval process for the US, enabling us to make our device available to patients with life-threatening heart disease much faster.”

Since 2014, Berlin Heals has been developing novel and revolutionary technology to treat heart failure. The proprietary C-MIC method was developed following results of pre-clinical trials in an intensive scientific cooperation with the Medical University of Vienna which confirmed that microcurrents have an extremely positive effect on cardiac cells. The C-MIC is a small implantable device that propagates reverse remodeling of the cardiac muscle tissue by delivering a constant but minimal electrical DC current to the heart. Not only did extensive preclinical trials confirm the efficacy and safety of microcurrent application via C-MIC, but also a strong anti-inflammatory effect with significant improvement in cardiac function. After confirming the clearly beneficial effects of microcurrent in preclinical studies, Berlin Heals started first implantations in patients under study conditions.

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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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Saniona (OMX: SANION), a clinical stage biopharmaceutical company focused on rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Tesomet for the treatment of Prader-Willi syndrome (PWS). Saniona is preparing to initiate a Phase 2b study of Tesomet in PWS in the first half of this year.

Orphan drug designation is a special status granted by the FDA to medicines and biologics that are intended for the treatment of rare diseases that affect fewer than 200,000 people in the U.S. The number of patients with PWS is estimated to be between 11,000 and 34,000 in the U.S. and between 17,000 and 50,000 in Europe. Receiving orphan designation qualifies Saniona for certain development benefits, including tax credits, elimination of certain FDA license application fees, and seven years of market exclusivity in the U.S. following approval.

“There is currently no cure for Prader-Willi syndrome and no medicines approved to address the uncontrollable hunger, or hyperphagia, that characterizes this rare disease,” said Rudolf Baumgartner, M.D., Chief Medical Officer and Head of Clinical Development at Saniona. “Receiving orphan drug designation will help us advance Tesomet as expeditiously and efficiently as possible.”

Saniona previously evaluated Tesomet in a randomized, double-blind, placebo-controlled Phase 2a trial in adults and adolescents with PWS. Adult patients receiving Tesomet achieved a statistically significant reduction in hyperphagia, as well as a clinically meaningful reduction in body weight at a dose of 0.5 mg per day. A smaller study extension in an adolescent population showed that Tesomet appeared to be well tolerated at lower doses (0.125 mg/day and 0.25 mg/day) and suggested dose-dependent effects on weight and hyperphagia. 

Saniona is currently planning to initiate a Phase 2b study of Tesomet in PWS in the first half of 2021. In preparation for this study, Saniona has opened an Investigational New Drug (IND) filing with the FDA, has selected the clinical research organization (CRO) that will support the clinical trial, and is in the process of assessing and selecting clinical trial sites in the U.S. and globally. Saniona has also selected the contract manufacturer to produce Tesomet for Phase 2b and Phase 3 clinical trials, and the contract manufacturer is actively working on clinical production. Additionally, Saniona has initiated multiple partnerships with the PWS advocacy community to ensure caregiver and patient feedback is incorporated into the clinical trial process and to provide the community with education on clinical trials.

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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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