Does Novartis delay in probing data manipulation claims spell trouble?

FiercePharma | August 23, 2019

Why did Novartis wait about two months before launching an internal probe into the alleged data manipulation surrounding gene therapy Zolgensma? Thats what officials at the FDA want to know. After an on-site inspection at AveXis control testing lab in Irvine, California, which appears to have been prompted by the firms communication of the problem to the FDA, inspectors issued a Form 483 on Aug. 2. The report noticed a delay from when the initial Zolgensma data allegation was flagged by a scientist on Mar. 14 to when a formal internal probe was opened on May 15. Two months seems like a lot, given that an FDA decision was looming, unnamed FDA officials toldThe Wall Street Journal. What happened during those two months? By Novartis’ account, before it started the full-on investigation in May, it “conducted an independent investigation with the support of external counsel to determine the extent of data discrepancies and whether they could be explained,” a company spokesman said. The reason why it took so long was because the preliminary probe “consisted of an extensive review of thousands of hand-written and electronic records concerning the life history of individual mice and a comparison of those records to entries on the hundreds of spreadsheets on which their data was recorded,” he said.

Spotlight

At its core, the pharmaceutical industry is about improving the quality of people’s lives. But today’s life-saving drugs are often small, high-value items which are easily stolen or diverted. Nearly impossible to authenticate by the end user, they have become an attractive target for counterfeiters and incidents of counterfeit drugs are on the rise. But counterfeit drugs are only one visible symptom of a deeper issue.

Spotlight

At its core, the pharmaceutical industry is about improving the quality of people’s lives. But today’s life-saving drugs are often small, high-value items which are easily stolen or diverted. Nearly impossible to authenticate by the end user, they have become an attractive target for counterfeiters and incidents of counterfeit drugs are on the rise. But counterfeit drugs are only one visible symptom of a deeper issue.

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PHARMACY MARKET

Pfizer Completes Acquisition of Biohaven Pharmaceuticals

Pfizer and Biohaven Pharmaceuticals | October 04, 2022

Pfizer Inc. announced the completion of its acquisition of Biohaven Pharmaceutical Holding Company Ltd., the maker of NURTEC® ODT an innovative migraine therapy approved for both acute treatment and prevention of episodic migraine in adults. The acquisition brings to Pfizer a portfolio of promising calcitonin gene-related peptide receptor antagonists including Rimegepant Approved in the United States under the trade name NURTEC® ODT, in adults for both the acute treatment of migraine with or without aura and the preventive treatment of episodic migraine Approved in the European Union under the trade name VYDURA® for both the acute treatment of migraine with or without aura in adults and the preventive treatment of episodic migraine in adults who have at least 4 migraine attacks per month Zavegepant New Drug Application for intranasal spray for the acute treatment of migraine under U.S. Food and Drug Administration review, with a Prescription Drug User Fee Act goal date in 1Q 2023 "We are proud to build on Pfizer’s legacy of delivering breakthrough medicines for patients living with complex pain disorders. The success of NURTEC® ODT coupled with Biohaven’s CGRP pipeline will strengthen Pfizer’s innovative Internal Medicine pipeline through 2030, and beyond. Combined with Pfizer’s global reach, this acquisition increases our potential to bring new treatment options to patients with migraine – a disease which affects over 1 billion people worldwide. Aamir Malik, Executive Vice President, Chief Business Innovation Officer, Pfizer Pfizer acquired all of the outstanding shares of Biohaven not already owned by Pfizer for $148.50 per share in cash, for a total transaction consideration of approximately $11.6 billion. As a result of the acquisition, Biohaven became a wholly-owned subsidiary of Pfizer. Effective immediately prior to the closing of the acquisition, Biohaven completed the spin-off of Biohaven Ltd. distributing Biohaven Ltd.’s shares to Biohaven’s shareholders. Biohaven Ltd., a new company that retained Biohaven’s non-CGRP development stage pipeline compounds, holds the Kv7 ion channel activators, glutamate modulation, and myostatin inhibition platforms, preclinical product candidates, and certain corporate infrastructure assets excluded from the Pfizer acquisition. Pfizer, a Biohaven shareholder, received a pro rata portion of Biohaven Ltd.’s shares in the distribution and owns approximately 3% of Biohaven Ltd. Biohaven Ltd. will continue to trade on the New York Stock Exchange under the ticker “BHVN”. About Migraine Worldwide, more than one billion people suffer from migraine, which predominately affects women.i Findings from the 2019 Global Burden of Disease study indicate that migraine is one of the worlds leading causes of disability.ii Migraine is characterized by debilitating attacks lasting four to 72 hours with multiple symptoms, including pulsating headaches of moderate to severe pain intensity that can be associated with nausea or vomiting, and/or sensitivity to sound and sensitivity to light. About Rimegepant Rimegepant targets a key component of migraine by reversibly blocking CGRP receptors, thereby inhibiting the biologic cascade that results in a migraine attack. Rimegepant was approved by the U.S. Food and Drug Administration (FDA) under the trade name Nurtec® ODT for the acute treatment of migraine in adults in February 2020 and for the preventive treatment of episodic migraine in adults in May 2021. Nurtec® ODT is taken orally as needed, up to once daily for acute treatment, and every other day for preventive treatment. The maximum dose in a 24 hour period is 75 mg. About Zavegepant Zavegepant is a third generation, high affinity, selective and structurally unique, small molecule CGRP receptor antagonist from the NOJECTION® Migraine Platform and the only CGRP receptor antagonist in clinical development with both intranasal and oral formulations. The FDA has accepted for review a New Drug Application (NDA) for zavegepant nasal spray, with a PDUFA date in 1Q 2023. About Pfizer: Breakthroughs That Change Patients’ Lives At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 170 years, we have worked to make a difference for all who rely on us.

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BUSINESS INSIGHTS, PHARMACY MARKET

Innovation Pharmaceuticals Announces BT BeaMedical Technologies Receives FDA Clearance for New Surgical Laser Family

Innovation Pharmaceuticals Inc | November 29, 2022

BT BeaMedical Technologies a private company developing a next-generation laser-based platform for epilepsy and cancer surgery, has notified Innovation Pharmaceuticals that it has received U.S. Food and Drug Administration clearance for its MANTA surgical laser family. The clearance is an important milestone for BeaMed in its development of an advanced laser technology with respect to shaping energy and matching it to patient specific anatomy, a novel concept that BeaMed is proving a reality. Innovation Pharmaceuticals made a strategic investment to acquire a minority stake in BeaMed in June 2022. BeaMed is the inventor and developer of the StingRay Laser System a novel laser-based thermal ablation technology designed for treatment of previously inoperable cases of epilepsy and for improvement of outcomes and enablement of new treatment options for oncology procedures, including those treating brain, prostate, liver, breast, and lung cancers. The StingRay Laser System will utilize the MANTA laser family as its core energy generation devices coupled to new fiber optic technology, allowing a perfect match between the exact structure of tumors and epileptic focal points and the energy delivery, while protecting vital functional areas against thermal damage. The MANTA surgical laser family was cleared by the FDA including five different wavelengths and soft tissue use in a very wide set of clinical specialties, including Neurosurgery, Spinal Surgery, Urology, Thoracic Surgery, Pulmonary Surgery, Gynecology, General Surgery, ENT/Head and Neck, Plastic Surgery, Orthopedics, Arthroscopy, Ophthalmology, Radiology, and Oral Surgery. BeaMed expects to soon make requisite submissions to the FDA for clearance of its fiber optics for use in multiple medical indications. “We are excited with the speed of progress BeaMed is making towards introduction of its new platform product with the potential to help millions of patients globally suffering from epilepsy and cancer in multiple specialties. The execution of this submission and the speed of clearance speaks volumes to the commercialization strategy and the exceptional capabilities of the regulatory team at BeaMed,” Leo Ehrlich, Chief Executive Officer at Innovation Pharmaceuticals “This is an exciting and important milestone for our company. This clearance gives us two key achievements. First, U.S. market access to a wide range of potential laser energy sources, each allowing us different and unique tissue interaction, an achievement which will have wide clinical implications. The second is a very wide set of intended uses covering many key potential markets for our technology. This underscores the clear regulatory strategy of BeaMed, which goes hand in hand with our broad impact business vision of revolutionizing key treatments, including brain tumors, epilepsy, lung cancer, prostate cancer, breast cancer and more,” said Gil Shapira, co-founder of BeaMed. BeaMed was founded by Moshe Eshkol and Gil Shapira, both highly experienced specialists in surgical lasers and medical devices. BeaMed and the development of the StingRay system are also supported by a special prestigious grant from the BIRD Foundation a bi-national organization supporting Israel-US collaboration. Gil Shapira brings forward 26+ years of engineering and product development and management experience, with over 17 years in the surgical laser industry. Gil owns and manages neoLaser, a company he founded in 2012, and led to revenue growth and profitability, with a 43% CAGR on its way to more than 1200 global installations and performing 50,000+ surgeries annually in over 30 countries. Previously, Gil led product development and marketing at OmniGuide, a successful high-growth laser-based medical device spin-off out of the Massachusetts Institute of Technology. About Innovation Pharmaceuticals Innovation Pharmaceuticals Inc. is a clinical stage biopharmaceutical company developing a portfolio of innovative therapies addressing multiple areas of unmet medical need, including inflammatory diseases, cancer, and infectious diseases. The Company is also active in evaluating other potential investment opportunities that can add value and diversify its portfolio.

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BUSINESS INSIGHTS

NeuroBo Pharmaceuticals, Inc. and Dong-A ST Co. Ltd. Announce Strategic Collaboration

NeuroBo Pharmaceuticals, Inc. | September 16, 2022

NeuroBo Pharmaceuticals, Inc. and Dong-A ST Co., Ltd. announced that they have entered into a conditional exclusive license agreement for NeuroBo to develop and commercialize DA-1241 and DA-1726, which are currently being evaluated for the treatment of nonalcoholic steatohepatitis obesity and type 2 diabetes. DA-1241 is a novel G-Protein-Coupled Receptor 119 agonist, which promotes the release of key gut peptides GLP-1, GIP and PYY, which, in turn, play an important role in glucose metabolism, lipid metabolism and weight loss. DA-1241 is a synthetic, selective small molecule, suitable for oral administration and has been shown to be well tolerated in phase 1 studies. Further, its multimodal mechanism appears to induce strong anti-NASH effects, supported by potential best-in-class efficacy, as demonstrated in pre-clinical studies. DA-1726 is a novel oxyntomodulin analogue functioning as a glucagon-like peptide-1 receptor and glucagon receptor dual agonist. OXM is a naturally-occurring, 37-amino acid peptide hormone that is released from the gut after ingestion of a meal, activating both the GLP-1 and glucagon receptors, prompting reduced food intake as well as an increase in energy expenditure, potentially resulting in superior body weight loss compared to selective GLP-1 receptor agonists. The beneficial effects of this dual mechanism of DA-1726 on weight loss compared to selective GLP-1 activity has been demonstrated in animal models. Additionally, DA-1726 has shown the ability to improve hepatic steatosis, inflammation and fibrosis when compared to the GLP-1 agonist, semaglutide in these same models. Under the license agreement, NeuroBo will be responsible for global development, regulatory and commercial activities other than for certain Asian-Pacific geographies. Dong-A will manufacture clinical supplies and initial commercial supplies of the product at its manufacturing facility in Korea. "The acquisition of these two cardiometabolic assets marks a seismic shift for NeuroBo, providing us with a highly promising, diversified pipeline with several upcoming value inflection points in the NASH and obesity space -- areas with enormous market opportunity," stated Gil Price, M.D., President and Chief Executive Officer of NeuroBo. "Through this agreement, Dong-A, one of our largest shareholders, has reaffirmed its commitment to remain a long-term strategic partner of NeuroBo. Dong-A is dedicated to our success and we are grateful it has also committed to provide continued support to facilitate the clinical development of the licensed assets. Once the transaction has closed, which is contingent upon certain closing conditions, we will be uniquely positioned to initiate a phase 2a study of DA-1241 in NASH in the first half of 2023, with data expected in the second half of 2024. We also intend to initiate a phase 1a safety study of DA-1726 in the first half of 2023, for which data is expected in the second half of 2023. We are truly excited about the prospects of NeuroBo as we transition to a cardiometabolic company across the large and growing markets of obesity and NASH." "We are highly enthusiastic about this opportunity to accelerate development of our novel treatments in partnership with NeuroBo. Dong-A plans to continue to strengthen its R&D capability and to seek additional collaboration opportunities to establish ourselves in the US market", Min Young Kim, Chief Executive Officer of Dong-A About the Proposed Licensing Transaction Under the terms of the license agreement, Dong-A will receive an upfront payment of $22 million in Series A convertible preferred stock, which will automatically convert into common stock upon receipt of requisite stockholder approval, and will be eligible to receive commercial- and regulatory-based milestone payments, dependent upon the achievement of specific regulatory and commercial developments. Dong-A will also be entitled to single digit royalties on net sales of the two assets. Dong-A has also agreed to commit $15,000,000 toward financing the assets, subject to NeuroBo's ability to obtain additional financing under the terms of the license agreement. The license agreement has been approved by the board of directors of NeuroBo. The transaction is expected to close in the third quarter of 2022, subject to obtaining third party financing for development of the assets and other customary closing conditions. About NeuroBo Pharmaceuticals NeuroBo Pharmaceuticals, Inc., is a clinical-stage biotechnology company historically focused on therapies for neurodegenerative, infectious, and, upon closing of the license agreement, cardiometabolic diseases. Its therapeutics programs currently include ANA001, an oral niclosamide formulation, which is in Phase 2/3 clinical trials to treat patients with moderate coronavirus disease (COVID-19); NB-01 for the treatment of painful diabetic neuropathy; NB-02 for the treatment of symptoms of cognitive impairment and to modify the progression of neurodegenerative diseases associated with the malfunction of tau protein; and gemcabene currently being assessed as an acute treatment for COVID-19 in combination with ANA001. NeuroBo Pharmaceuticals, Inc. is headquartered in Boston, Massachusetts. About Dong-A Dong-A ST Co. is a leading healthcare company in South Korea with a business focus on developing, manufacturing and distributing pharmaceutical products and medical devices worldwide. Dong-A has successfully developed and marketed several products globally and continues to develop prospective clinical candidates. Dong-A also provides licensed-in and licensed-out drugs, and medical devices, including high-technology medical devices, custom-made products, and sets of artificial cardiac circuits for use in open-heart surgery. Dong-A has over 5,500 employees including 2,300 in the pharmaceutical sector. Dong-A was founded in 1932 and is headquartered in Seoul, South Korea.

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