Who will be the biggest pharma players in orphan drugs?

pharmaphorum | September 06, 2019

New analysis from GlobalData has identified the predicted top 15 pharma players in 2025 for organ drugs, with Roche coming out in the lead. The research also notes that M&A deals have been the primary way in which big pharma companies secure these orphan drug designations, with very few rare disease drugs being developed in-house. “Despite the allure of the lucrative orphan drug market, this is not a viable sector for all pharma companies as it requires specialist capabilities,” GlobalData said. “In fact, most big pharma companies have inherited specialty business units through M&As.” The collective sales data from these therapies with organ drug designations reveal that Roche has generated the highest sales from orphan drugs and will maintain its leading position going forward.

Spotlight

Patrick Chene, a researcher in Oncology, discusses why he’s inspired seeing a compound move to the clinic, and the committed, multidisciplinary teams from oncology, chemical biology & biochemistry.

Spotlight

Patrick Chene, a researcher in Oncology, discusses why he’s inspired seeing a compound move to the clinic, and the committed, multidisciplinary teams from oncology, chemical biology & biochemistry.

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PHARMA TECH

Apollomics Inc., a Late-Stage Clinical Biopharmaceutical Company to be Listed on Nasdaq Through Business Combination

Apollomics, Inc. | September 15, 2022

Apollomics Inc. a late-stage clinical biopharmaceutical company, and Maxpro Capital Acquisition Corp. announced a definitive agreement for a business combination that would result in Apollomics becoming a publicly traded company on the Nasdaq Global Market. The business combination is expected to close in the first quarter of 2023 and Apollomics is expected to be listed on Nasdaq under the ticker symbol “APLM.” Apollomics’ broad pipeline of drug candidates includes late-stage clinical assets for the treatment of patients with difficult-to-treat cancers. Apollomics’ mission is to develop assets in critically important areas of unmet need. The Company’s leading drug candidates address certain subpopulations within lung cancer and leukemia. Globally, both lung cancer and leukemia affect over 2 million people annually. The Company is dedicated to discovering and developing oncology therapies of different mechanisms of action to inhibit cancer. Its diverse portfolio of innovative drug candidates for treating difficult-to-treat cancers includes precision therapy targeting tumors with specific mutations, as well as assets addressing broader cancer conditions. The Company’s pipeline of nine clinical, preclinical and discovery drug candidates has the potential to improve treatment of a number of tumor types. Upon the closing of the transaction, Apollomics will continue to be led by current Chairman and CEO, Dr. Guo-Liang Yu, Ph.D., a serial entrepreneur, and his team. Dr. Yu is a pharmaceutical researcher with more than 300 patents. “Apollomics’ announcement represents the next major milestone on our journey to provide solutions for patients with difficult-to-treat cancers,” Dr. Yu said. “We anticipate that the funds available to us from this transaction will help us accelerate development of our oncology pipeline.” Apollomics expects results from its global Phase 2 multi-cohort clinical trial of vebreltinib in NSCLC and other solid tumors with cMET dysregulation in 2023, which the Company believes may support its first New Drug Application (“NDA”) with the U.S. Food and Drug Administration (“FDA”) while generating clinical data for different indications. In addition, the Company expects to complete patient recruitment of its uproleselan Phase 3 study in China in 2023. “Our goal was to find an exciting company with a growing pipeline of innovative product candidates that could positively affect the lives of millions of people. Our team is excited to combine with Apollomics as it has met and exceeded all our key selection criteria. Together with Apollomics, Maxpro will do everything we can to support the Company’s vision of treating patients with difficult-to-treat cancers.” Moses Chen, CEO of Maxpro About Apollomics Inc. Apollomics Inc. is an innovative clinical-stage biopharmaceutical company focused on the discovery and development of oncology therapies with the potential to be combined with other treatment options to harness the immune system and target specific molecular pathways to inhibit cancer. Apollomics currently has a pipeline of nine drug candidates across multiple programs, six of which are currently in the clinical stage of development. Apollomics’ lead programs include investigating its core product, vebreltinib (APL-101), a potent, selective c-Met inhibitor for the treatment of non-small cell lung cancer and other advanced tumors with c-Met alterations, which is currently in a Phase 2 multicohort clinical trial in the United States, and developing an anti-cancer enhancer drug candidate, uproleselan (APL-106), a specific E-Selectin antagonist that has the potential to be used adjunctively with standard chemotherapy to treat acute myeloid leukemia and other hematologic cancers, which is currently in Phase 1 and Phase 3 clinical trials in China. About Maxpro Capital Acquisition Corp. Maxpro is a blank check company formed for the purposes of effecting a merger, capital share exchange, asset acquisition, share purchase, reorganization, or similar business combination with one or more businesses in the healthcare and technology industries. In October 2021, Maxpro consummated a $103.5 million initial public offering of 10.35 million units (including the underwriters’ full exercise of their over-allotment option), each unit consists of one share of Class A common stock and one redeemable warrant, each warrant entitles the holder to purchase one share of Class A common stock at a price of $11.50 per share. EF Hutton, division of Benchmark Investments LLC, served as the sole book-running manager of Maxpro’s initial public offering.

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PHARMA TECH

Sirona Biochem Signs International Partnership Agreement with Wanbang Biopharmaceuticals

Sirona Biochem Corp. | November 28, 2022

Sirona Biochem Corp. announces that, subsequent to the LOI, Sirona and Wanbang Biopharmaceuticals have signed an expanded, international partnership agreement to collaborate on licencing Sirona’s SGLT2 inhibitor, TFC-039, as a pharmaceutical treatment in both animal and human health. The agreement adds human health to the partnership as a result of new licencing opportunities currently in due diligence. Wanbang and Sirona initially signed a licensing agreement for TFC-039, whereby Wanbang obtained the rights to develop the compound as a diabetes treatment in China and Sirona retained the global rights. Sirona has since been in discussions with animal health companies to advance TFC-039 as a treatment for diabetes and chronic kidney disease in companion animals. SGLT2 inhibitors provide an opportunity to treat inflicted animals with an oral medication as opposed to the traditional method of daily insulin injections. More recently, Sirona has entered into due diligence with a large pharmaceutical company with a regional interest in developing the compound for human diabetes. Together, Sirona and Wanbang share extensive knowledge and scientific results of TFC-039. Partnering will significantly increase the speed to third-party partnerships and commercialization. The shared data spans over 12 years of research and development, and includes in vitro and in vivo preclinical work, multiple clinical studies, advanced manufacturing process development and the ability to commercially manufacture TFC-039. “We have a long-standing relationship with Wanbang and are excited to combine our two companies’ expertise to license TFC-039. Wanbang has invested millions of dollars into the clinical stage research and development of the manufacturing processes for TFC-039. These pieces of data are critical to large organizations and will greatly increase the opportunities to move forward. The probability of a successful licensing agreement has been made much stronger by leveraging our alliance with Wanbang. We have had a successful year building Sirona’s pipeline, with positive movement on all projects and we’re looking forward to continuing this success with our SGLT2 inhibitor as well as our antiviral and anti-aging projects in 2023.” Dr. Howard Verrico, CEO About Wanbang Biopharmaceuticals and Fosun Pharmaceuticals Wanbang Biopharmaceuticals develops, manufactures, and markets drugs with indications for chronic disease treatment, antibiotics, and other endocrine diseases in China. Founded in 1981, the company is headquartered in Xuzhou, China, and is a subsidiary of Shanghai Fosun Pharmaceutical Group. Fosun is a leader in the pharmaceutical industry and is regarded as one of the top five domestic pharmaceutical companies in China. About Sirona Biochem Corp. Sirona Biochem is a cosmetic ingredient and drug discovery company with a proprietary platform technology. Sirona specializes in stabilizing carbohydrate molecules with the goal of improving efficacy and safety. New compounds are patented for maximum revenue potential. Sirona’s compounds are licensed to leading companies around the world in return for licensing fees, milestone fees and ongoing royalty payments. Sirona’s laboratory, TFChem, is located in France and is the recipient of multiple French national scientific awards and European Union and French government grants.

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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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