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

The publication of the stable text of the European Union (EU) Medical Devices Regulation, and the In Vitro Diagnostic Medical Devices Regulation in June 2016 documents the political agreement between the three EU Institutions – the Commission, the Parliament and the Council – on the contents of the revision of the EU legislation for medical devices. Preparation as regulations means that the legislation will apply directly in the Member States, thereby minimizing the potential for differences being introduced during national adoptions. The availability of this text allows affected organizations to start considering the effects on their activities and what they will need to do in order to be compliant with the revised requirements. The text will undergo legal checking prior to formal adoption.

Spotlight

The publication of the stable text of the European Union (EU) Medical Devices Regulation, and the In Vitro Diagnostic Medical Devices Regulation in June 2016 documents the political agreement between the three EU Institutions – the Commission, the Parliament and the Council – on the contents of the revision of the EU legislation for medical devices. Preparation as regulations means that the legislation will apply directly in the Member States, thereby minimizing the potential for differences being introduced during national adoptions. The availability of this text allows affected organizations to start considering the effects on their activities and what they will need to do in order to be compliant with the revised requirements. The text will undergo legal checking prior to formal adoption.

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BUSINESS INSIGHTS

Zenas BioPharma Acquires Exclusive Worldwide Rights to Obexelimab from Xencor

Xencor, Zenas BioPharma | November 22, 2021

Xencor, Inc. a clinical-stage biopharmaceutical company developing engineered monoclonal antibodies and cytokines for the treatment of cancer and autoimmune diseases, and Zenas BioPharma (“Zenas”), a global biopharmaceutical company based in the USA and China committed to the development and delivery of immune-based therapies, today announced that Zenas has acquired from Xencor exclusive worldwide rights to develop, manufacture and commercialize the investigational antibody obexelimab. Obexelimab is a potential first-in-class bifunctional antibody that targets CD19 with its variable domain and uses Xencor's XmAb® Immune Inhibitor Fc Domain to target FcγRIIb, a receptor that inhibits the function of B-cells, which are important components in the immune system. Xencor demonstrated through early-stage clinical studies that obexelimab effectively inhibits B-cell function without depleting the cells and generates an encouraging treatment effect in patients with multiple autoimmune diseases. “Zenas is advancing a broad pipeline of differentiated drug candidates that are intended to bring best-in-class innovation to patients with underserved medical needs. Today, we are pleased to add obexelimab to our portfolio, and based on its clinical profile, we believe it is positioned as a first-in-class candidate with the potential to treat numerous autoimmune diseases.” Hua Mu, Ph.D., MD, president and chief executive officer at Zenas “Obexelimab’s highly potent and broad blockade of B-cell activation—without depleting B cells—differentiates it from other B-cell targeting therapies, and it has demonstrated disease-modifying activity in settings where B-cell inhibition is a proven strategy,” said Bassil Dahiyat, Ph.D., president and chief executive officer at Xencor. “In Zenas BioPharma, we have found a partner committed to broadly and aggressively developing therapeutics like obexelimab for patients with autoimmune diseases, enabling Xencor’s continued focus on the growing opportunities provided by our XmAb bispecific antibody and cytokine pipeline.” Under the terms of the new agreement, Zenas will issue to Xencor a warrant giving Xencor the right to acquire additional Zenas equity, such that Xencor’s total equity in Zenas would be 15% of its fully diluted capitalization following the closing of Zenas’ next round of equity financing, subject to certain requirements. Xencor previously received equity in Zenas under a separate license agreement. Xencor is also eligible to receive up to $480 million based on the achievement of certain clinical development, regulatory and commercialization milestones and is eligible to receive tiered, mid-single digit to mid-teen percent royalties upon commercialization of obexelimab, dependent on geography. Zenas will have sole responsibility for advancing the research, development, regulatory and commercial activities of obexelimab worldwide. About Xencor, Inc. Xencor is a clinical-stage biopharmaceutical company developing engineered monoclonal antibodies and cytokines for the treatment of cancer and autoimmune diseases. Currently, 22 candidates engineered with Xencor's XmAb® technology are in clinical development internally and with partners. Xencor's XmAb antibody engineering technology enables small changes to the structure of proteins resulting in new mechanisms of therapeutic action. About Zenas BioPharma Zenas BioPharma is a global biopharmaceutical company based in the USA and China committed to becoming a leader in the development and delivery of immune-based therapies for patients in the US, China and around the world. Zenas is rapidly advancing a deep pipeline of innovative therapeutics that continues to grow through our successful business development strategy. Our experienced leadership team and network of business partners drive operational excellence to deliver potentially transformative therapies to improve the lives of those facing autoimmune and rare diseases. Xencor Forward-Looking Statements Certain statements contained in this press release may constitute forward-looking statements within the meaning of applicable securities laws. Forward-looking statements include statements that are not purely statements of historical fact, and can generally be identified by our use of words such as “potential,” “can,” “will,” “plan,” “may,” “could,” “would,” “expect,” “anticipate,” “seek,” “look forward,” “believe,” “committed,” “investigational,” and similar terms, or by express or implied discussions relating to the development of obexelimab as a potential treatment for patients with autoimmune diseases; the commercial potential of obexelimab; the safety, tolerability, efficacy and pharmacokinetics of obexelimab; the quotations from Xencor’s president and chief executive officer; and other statements that are not purely statements of historical fact. Such statements are made on the basis of the current beliefs, expectations, and assumptions of the management of Xencor and are subject to significant known and unknown risks, uncertainties and other factors that may cause actual results, performance or achievements and the timing of events to be materially different from those implied by such statements, and therefore these statements should not be read as guarantees of future performance or results. Such risks include, without limitation, the risks associated with the process of discovering, developing, manufacturing and commercializing drugs that are safe and effective for use as human therapeutics and other risks described in Xencor's public securities filings. For a discussion of these and other factors, please refer to Xencor's annual report on Form 10-K for the year ended December 31, 2020 as well as Xencor's subsequent filings with the Securities and Exchange Commission. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. This caution is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, as amended to date. All forward-looking statements are qualified in their entirety by this cautionary statement and Xencor undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof, except as required by law.

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PHARMACY MARKET

Bushu Pharma's Partnership with Suzuken Group Increases Specialty Pharmaceutical Services

Bushu Pharmaceuticals Ltd. | April 28, 2021

Bushu Pharmaceuticals Ltd. has entered into a business partnership arrangement with Suzuken Co., Ltd. to provide specialty pharmaceutical producers looking to enter the Japanese market with extensive support for new product releases. In recent years, the market has seen an increase in the number of innovative specialty pharmaceuticals, such as biopharmaceuticals, regenerative medicines, and orphan drugs for rare diseases, that require strict temperature, inventory, and security control. In line with this trend, the number of pharmaceutical companies planning to bring these types of drugs to Japan has grown. These pharmaceutical manufacturers, on the other hand, often lack the necessary infrastructure, such as production facilities, delivery centers, and networks, to reach the Japanese market with minimal resources, and must therefore outsource these functions to trusted partners. Suzuken, Bushu Pharma's partner, has played an industry-leading role in Japan's pharmaceutical distribution market. Suzuken, one of the leading organizations in the industry, has a distribution network in Japan capable of supporting the international shipping of specialty pharmaceuticals. Bushu Pharma, on the other hand, has been in the contract manufacturing industry in Japan for over 20 years, holds GMP certifications from all over the world, and has a track record of exporting to over 43 countries. Bushu Pharma will establish a platform that provides one-stop holistic assistance for pharmaceutical producers seeking to penetrate the Japanese market by aligning functions and capabilities with Suzuken. About Bushu Pharmaceuticals Ltd. Bushu Pharma manufactures solid reparations, solid preparation primary packaging, injection manufacturing, and injection packaging not just for domestic pharmaceuticals but also for multinational pharmaceutical manufacturers, drawing on its expertise and understanding as a contract development and manufacturing organization (CDMO) cultivated over 20 years. Bushu Pharma has a diverse product and service offering, ranging from clinical trial supplies to commercial product development support to commercialization, as well as various production facilities and technologies. Bushu Pharma is a leading domestic contract manufacturing company in Japan that can offer optimum solutions such as technology transfer, production, packaging, and logistics from project start to project approval.

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BUSINESS INSIGHTS

Health Canada Approves Ipsen’s Sohonos™ (palovarotene capsules) as the First Approved Treatment for Fibrodysplasia Ossificans Progressiva

Ipsen | January 24, 2022

Ipsen announced the Health Canada approval of Sohonos, an oral selective retinoic-acid receptor gamma agonist indicated to reduce the formation of heterotopic ossification in adults and children aged 8 years and above for females and 10 years and above for males with fibrodysplasia ossificans progressiva.1 Sohonos is approved for the treatment of patients with FOP for both chronic use, and for flare-ups, in these patient populations. This decision marks the first approval for Sohonos worldwide. “FOP is a progressive and debilitating condition which has such a profound impact on patients, and their families. Until today, there was no approved medicine, and we are proud to bring this important new medicine to the FOP community.” Dr. Howard Mayer, Executive Vice President and Head of Research and Development, Ipsen FOP is characterized by new bone formation outside of the normal skeletal system, like in soft connective tissues, a process known as heterotopic ossification,2 which can be preceded by painful soft tissue swelling or “flare-ups.”2 Flare-up episodes are common and are a substantial contributor to the formation of new bone, however bone can also form in the absence of a flare-up. Once formed, it is irreversible and leads to loss of mobility and shortened life expectancy.2 It is an ultra-rare genetic disorder with an estimated prevalence of 1.36 per million individuals; however, the number of confirmed cases varies by country.3,4. As part of the ongoing commitment to the FOP and rare disease community, Ipsen plans to file in the US in H1 2022 and is in discussions with other regulatory authorities around the world. About the MOVE clinical program This approval is based on data from the ongoing MOVE trial, the first global multi-center Phase III trial in FOP. MOVE is an open-label, single-arm trial, evaluating the efficacy and safety of a chronic/flare-up dosing regimen of palovarotene in decreasing new annualized HO volume in patients with FOP. About Sohonos Sohonos is an oral, selective RARγ agonist developed as a treatment for people living with the debilitating ultra-rare genetic disorder, FOP. The treatment was acquired by Ipsen through the acquisition of Clementia Pharmaceuticals in April 2019. It is a member of the retinoid class of drugs that is associated with birth defects in humans. Sohonos must not be used by patients who are, or intend to become, pregnant due to the risk of teratogenicity. To minimize fetal exposure, Sohonos is to be administered only if all conditions for pregnancy prevention are met. Sohonos has been shown to cause premature physeal closure in growing children with FOP; periodic monitoring is recommended. About Ipsen Ipsen is a global, mid-sized biopharmaceutical company focused on transformative medicines in Oncology, Rare Disease and Neuroscience; it also has a well-established Consumer Healthcare business. With total sales of over €2.5bn in FY 2020, Ipsen sells more than 20 medicines in over 115 countries, with a direct commercial presence in more than 30 countries. The Company’s research and development efforts are focused on its innovative and differentiated technological platforms located in the heart of leading biotechnological and life-science hubs: Paris-Saclay, France; Oxford, U.K.; Cambridge, U.S.; Shanghai, China. Ipsen has around 5,700 colleagues worldwide and is listed in Paris and in the U.S. through a Sponsored Level I American Depositary Receipt program.

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