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Sunovion, Sumitomo Dainippon Pharma and Otsuka Enter Worldwide Development and Commercialization Collaboration

On September 30, 2021 Sunovion Pharmaceuticals Inc. (Sunovion),its parent company Sumitomo Dainippon Pharma Co., Ltd. (Sumitomo Dainippon Pharma) and Otsuka Pharmaceutical Co., Ltd. (Otsuka) reported that the companies have entered into a worldwide license agreement for the joint development and commercialization of four compounds: ulotaront (SEP-363856), non-racemic ratio of amisulpride enantiomers (SEP-4199), SEP-378614 and SEP-380135 (Press release, Sunovion, SEP 30, 2021, View Source [SID1234590587]).

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Leveraging their complementary therapeutic area expertise and capabilities, the companies expect to fully explore the medical potential of the compounds in the collaboration, and accelerate development timelines, to bring forward important treatments for people living with neuropsychiatric health conditions worldwide. Otsuka’s subsidiary, Otsuka Pharmaceutical Development & Commercialization, Inc., will jointly lead, together with Sunovion, the effort to advance the research and development program worldwide, as well as plan for future commercial activities.

"Sunovion, with our parent company Sumitomo Dainippon Pharma, is proud to collaborate with Otsuka in a shared mission to contribute towards improved lives and better health globally," said Antony Loebel, M.D., President and Chief Executive Officer of Sunovion. "Otsuka’s recognition of the significant value of these assets reflects the innovative discovery and development efforts at Sunovion over the past decade, as well as our neuropsychiatry commercialization expertise and capabilities. We look forward to working with Otsuka colleagues as we advance novel compounds to treat patients with serious neuropsychiatric conditions."

"We are pleased to have signed this agreement with Otsuka, which has wide global reach and significant neuropsychiatry expertise. We will work together to more rapidly and reliably develop and commercialize valuable pharmaceuticals for patients around the world with the expectation that these new medications will grow," said Hiroshi Nomura, President and Chief Executive Officer of Sumitomo Dainippon Pharma. "Sumitomo Dainippon Pharma aims to achieve sustained growth through global collaboration in anticipation of future changes in the business environment. This collaboration is a major step forward in this initiative."

"Otsuka has been committed to providing new antipsychotics that contribute to patients worldwide in the field of neuropsychiatry by leveraging internal capabilities and external collaborations, starting with the launch of antipsychotics in the U.S. in 2002," said Makoto Inoue, President and Representative Director of Otsuka. "We are advancing in new areas such as the development of drugs to treat agitation associated with dementia of the Alzheimer’s type and the deployment of the world’s first digital medicine. Through this agreement, we are confident the companies will be able to deliver even more value to patients through the experience and networks that we have cultivated over many years worldwide."

This collaboration recognizes that there is a great need for novel treatments in the area of neuropsychiatric medicine development. The companies are focused on working together on solutions to address these areas of unmet medical need by advancing four promising compounds―ulotaront (SEP-363856), SEP-4199, SEP-378614 and SEP-380135―that address serious neuropsychiatric disorders. The goal of the co-development programs is to contribute to changing the course of serious medical conditions and provide new treatment options to patients and healthcare providers globally.

Upon the completion of the agreement, in addition to an upfront payment of USD 270 million, Sunovion is eligible for development milestone payments of up to USD 620 million for the four compounds and relevant sales milestone payments. Sunovion and Otsuka will share profits from the four compounds, as well as all expenses for clinical studies, applications for approval, and commercialization in each country. Additional details regarding terms of the agreement are not being disclosed.

About Sunovion Compounds

The four clinical-stage assets―ulotaront (SEP-363856), non-racemic ratio of amisulpride enantiomers (SEP-4199), SEP-378614 and SEP-380135―included within the collaboration span early- to late-stage development. The compounds represent a scientifically unique approach to treating symptoms that are not adequately addressed by current therapeutic options and/or for which existing treatments have an unsatisfactory safety and tolerability profile.

Ulotaront (SEP-363856), in Phase 3, is a trace amine-associated receptor 1 (TAAR1) agonist with 5-HT1A agonist activity that is under investigation for the treatment of schizophrenia with additional indications under consideration. Ulotaront was granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA) for the treatment of schizophrenia. Ulotaront is the first TAAR1 agonist to enter into Phase 3 clinical studies in adults and adolescents (13 to 17 years) with schizophrenia.

Non-racemic amisulpride (SEP-4199) is in Phase 3 clinical development for the treatment of major depressive episodes associated with bipolar I disorder (bipolar depression). Sunovion discovered that the pharmacology of amisulpride is enantiomer-specific and increasing the ratio of R-amisulpride to S-amisulpride increases the potency for serotonin 5-HT7 receptors relative to dopamine D2 receptors. SEP-4199 was designed with an 85:15 ratio of R-amisulpride to S-amisulpride to increase levels of serotonin 5-HT7 activity intended to enhance antidepressant efficacy and produce reduced levels of D2 receptor occupancy appropriate for the treatment of bipolar depression. In September 2021 Sunovion initiated a global clinical Phase 3 study, which is a randomized, double-blind, placebo-controlled, parallel-group, fixed-dosed study for the treatment of bipolar I depression in the U.S. Japan will join this global clinical Phase 3 study.

SEP-378614 and SEP-380135 are in Phase 1 development and can be viewed on the Sunovion pipeline here.

Sunovion discovered ulotaront, SEP-378614, and SEP-380135 in collaboration with PsychoGenics based in part on a mechanism-independent approach using the in vivo phenotypic SmartCube platform and associated artificial intelligence algorithms.

Sunovion has a robust portfolio of clinical and pre-clinical compounds, not included in this agreement, which the company continues to advance for some of the most prevalent, challenging, and underserved neuropsychiatric conditions.

About Neuropsychiatric Disorders

Neuropsychiatric disorders are among the most complex and difficult to treat. Disorders of the brain are often associated with significant and disabling effects on patients, impacting their loved ones and society more broadly. Nearly one in six people worldwide live with a neurological disorder,1 29 million people worldwide are living with bipolar disorder,2 and 20 million people worldwide are living with schizophrenia.3

Isarna Therapeutics Appoints Claus Schalper as CEO

On September 30, 2021 Isarna Therapeutics reported the appointment of Claus Schalper as Chief Executive Officer. Mr. Schalper joins Isarna with over 20 years of experience as an executive and serial entrepreneur in the life science and biotech industries (Press release, Isarna Therapeutics, SEP 30, 2021, View Source [SID1234590586]). His appointment expands Isarna’s management team, which includes Prof. Marion R. Munk as Chief Medical Officer, Dr. René Rückert as Chief Operating Officer and Chris Huiskamp as Chief Financial Officer. The leadership team will be focused on developing Isarna’s lead product ISTH0036 as a promising therapeutic candidate for diseases of the eye.

"Isarna has reached an exciting stage of development for its lead program, ISTH0036, which has broad applicability in a range of ophthalmology indications," said Claus Schalper, CEO of Isarna Therapeutics. "I value the opportunity to work together with Marion, René and Chris as we move ISTH0036 toward the next clinical trial to further evaluate its potential to benefit patients with retinal diseases that continue to have a high level of unmet medical need."

Claus Schalper, MBA, joins Isarna from Pieris Pharmaceuticals, a company that he co-founded in 2001 and where he held the position of Chief Financial Officer among other roles, playing an important part in the company’s evolution into a US-NASDAQ listed biotechnology company. Mr. Schalper also co-founded XL-protein GmbH and led the company to profitability by executing a series of collaborations with pharma and biotech companies. Mr. Schalper began his career with Arthur Andersen and subsequently served as CEO for several companies in the technology industry. He holds a Master of Business Administration from the University of Bamberg, Germany.

"Claus brings a broad range of leadership and corporate development experience to Isarna. With the management team now in place, the company is well-positioned to implement its product development strategy and reach the next value inflection points for ISTH0036 in ophthalmology," said Matthias Kromayer, Managing Partner and member of the Executive Board at MIG Capital, Isarna’s lead investor.

Prof. Marion R. Munk, MD, PhD, FEBO, joined Isarna as Chief Medical Officer in 2019. She brings over 10 years of clinical expertise in retina, uveitis and age-related macular degeneration research and is a board-certified ophthalmologist currently serving as attending retina specialist and Managing Director of the Bern Photographic Reading Center at the University Hospital Bern, Switzerland. She previously worked at the Feinberg School of Medicine, Chicago, Illinois and the AKH Vienna, Austria, and serves as a consultant for many key players in the ophthalmology drug and device development space. Prof. Munk holds a bachelor’s degree in theoretical physics as well as a MD and PhD in ophthalmology and clinical neuroscience from the Medical University, Vienna.

René Rückert, MD, MBA, joined Isarna in 2018 as interim CMO and in 2019 took the role of the Chief Operating Officer. He brings extensive experience in ophthalmology drug development including many years as a leader and global manager at Bayer and Novartis where he led the global development of the current gold standard therapies in AMD and DME, Eylea and Lucentis. Dr. Rückert previously served as Clinical Vice President, Chief Medical Officer and Chief Operating Officer at a number of innovative biotech and Medtech Companies. In his role at Isarna, Dr. Rückert oversees the company’s operations and clinical development programs and supports the company with his business acumen and his global network. Dr. Rückert is a trained immunologist and board-certified for biochemistry; he received his medical degree from the Charité Berlin, Germany and an MBA from the Warwick Business School, UK.

Isarna Therapeutics has extensive expertise in antisense therapies targeting the messenger RNA (mRNA) transcript for transforming growth factor (TGF)-β, a protein that is chronically elevated in ophthalmic and fibrotic diseases and is used as escape mechanisms by tumors during immune therapy. In ophthalmic indications, fibrosis is a key driver of reduced vision and lack of long-term efficacy of current therapies, TGF-β is a key driver of fibrosis, so ISTH0036 could be the first therapy to prevent the fibrotic changes in patients with retinal pathologies. The company’s Phase 2 candidate, ISTH0036, blocks TGF-β 2, which is a major driver of severe retinal diseases such as, wet (neovascular) age-related macular degeneration (AMD) and diabetic macular edema (DME). Preclinical evidence supports a key role of TGF-β 2 in macular edema and neovascularization, supporting the development in AMD and DME as an intravitreal injection. Animal data support target engagement and therefore suppression of TGF-β 2 beyond four months after a single intravitreal injection. The company previously presented data from its Phase 1 dose-escalation trial with ISTH0036 in which the compound showed excellent safety and was well-tolerated at all dose levels.

Takeda to Commercialize Next-Generation Hunter Syndrome Therapy Through Collaboration with JCR Pharmaceuticals

On September 30, 2021 Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) ("Takeda") and JCR Pharmaceuticals Co., Ltd. (TSE:4552) ("JCR") reported a geographically-focused exclusive collaboration and license agreement to commercialize JR-141 (INN: pabinafusp alfa), an investigational, next-generation recombinant fusion protein of an antibody against the human transferrin receptor and iduronate-2-sulfatase (IDS) enzyme for the treatment of Hunter syndrome (also known as Mucopolysaccharidosis type II or MPS II) (Press release, Takeda, SEP 30, 2021, View Source [SID1234590585]). Hunter syndrome is caused by a deficiency of IDS and manifests in different forms. JR-141, applied with J-Brain Cargo, JCR’s proprietary blood-brain barrier (BBB) technology, is engineered to transport the therapeutic enzyme across the BBB to directly reach the brain and address both the somatic and neuronopathic manifestations of the disease, which can lead to progressive cognitive decline.

Under the terms of the exclusive collaboration and license agreement, Takeda will exclusively commercialize JR-141 outside of the United States, including Canada, Europe, and other regions (excluding Japan and certain other Asia-Pacific countries). JCR will receive an upfront payment for such ex-U.S. license, and is eligible to receive additional development and commercial milestones as well as tiered royalties on potential sales. The two companies will collaborate to bring this therapy to patients as quickly as possible upon completion of the global Phase 3 program, which will be conducted by JCR.

Takeda receives an option under a separate option agreement, which allows Takeda to acquire an exclusive license to commercialize JR-141 in the U.S. upon completion of the Phase 3 program.

"Takeda is committed to continuously improving the way Hunter syndrome is treated. JR-141 introduces a new way to deliver proteins across the blood-brain barrier, overcoming our current challenges to treat the underlying neuronopathic manifestations of Hunter syndrome and help maintain or improve cognitive function in these patients," said Dan Curran, M.D., Head, Rare Genetics & Hematology Therapeutic Area Unit at Takeda. "We will work closely with JCR to apply our expertise in enzyme replacement therapies with the hope of bringing this potentially transformative therapy to patients as quickly as possible."

"JCR is pleased to have reached an agreement with Takeda who is well placed to achieve our common goal of maximizing the impact of JR-141," said Shin Ashida, President, Chairman of JCR. "Our mission is to provide transformative treatment options as soon as possible to patients with lysosomal storage disorders (LSDs) with central nervous system symptoms, such as Hunter syndrome. JR-141 is the first-ever approved biopharmaceutical in Japan that penetrates the blood-brain barrier. I expect that we will be able to achieve this mission through our partnership with Takeda to deliver a new treatment option to Hunter patients around the world as swiftly as possible."

JR-141 met its primary endpoint in an open-label Phase 2/3 clinical trial in Japan demonstrating significant reductions in heparan sulfate (HS) in the cerebrospinal fluid, a biomarker for assessing the drug’s effectiveness in reducing disease-causing substrate in the central nervous system, in all subjects for whom measurements were available after 52 weeks of treatment. Somatic disease control was maintained in patients who switched from standard enzyme replacement therapy (ERT). The study also demonstrated an improvement in somatic symptoms in participants who had not previously received standard ERT prior to the start of the trial. Additionally, a neurocognitive development assessment demonstrated maintenance or improvement of age-equivalent function in 21 of the 25 patients at one year. There were no reports of serious treatment-related adverse events in the trial.1

About JR-141

JR-141 is a recombinant fusion protein of an antibody against the human transferrin receptor and iduronate-2-sulfatase, the enzyme that is missing or malfunctioning in subjects with Hunter syndrome. It is expected to be effective against the neuronopathic manifestations of the disease by crossing the BBB through transferrin receptor mediated transcytosis using J-Brain Cargo, JCR’s proprietary BBB technology. Uptake into cells is mediated through the transferrin receptor and mannose-6-phosphate receptor. JCR has advanced development activities by establishing the necessary evidence from the molecular design stage to the nonclinical and clinical trial phases. In non-clinical trials, JCR has confirmed both high affinity binding of JR-141 to transferrin receptors, and passage across the BBB into neuronal cells as evidenced by electron microscopy.

In addition, JCR has confirmed that using J-Brain Cargo technology, enzymes are taken up into various brain tissues. A decrease in substrate accumulation has also been confirmed in an animal model of Hunter syndrome.2,3,4 In several clinical trials with JR-141, JCR obtained evidence of reduction of heparan sulfate concentrations in the CSF, a biomarker for assessing the drug’s effectiveness in reducing disease-causing substrate in the central nervous system, consistent with the results obtained from non-clinical studies. JCR also obtained clinical results that demonstrate positive effects of JR-141 on neurocognition.5,6,7,8

JR-141 was approved by the Ministry of Health, Labour and Welfare and marketed since May 2021 under the brand name "IZCARGO I.V. Infusion 10mg."

About Hunter Syndrome

Hunter syndrome is a severely debilitating, rare lysosomal disease caused by a deficiency of iduronate-2-sulfatase, an enzyme that is needed to break down substances in the body called glycosaminoglycans (GAGs).9 Without this enzyme, GAGs can build up, causing a range of disease-related signs and symptoms.9,10 Roughly two of every three patients with Hunter syndrome are also affected with progressive cognitive decline.11 Hunter syndrome affects 1 in 162,000 total live births, and almost exclusively males.12

Merck Announces Mutual Decision to End Bintrafusp Alfa Agreement With GSK

On September 30, 2021 Merck, a leading science and technology company, reported a mutual decision with GSK to terminate their agreement on bintrafusp alfa, effective September 30, 2021 (Press release, Merck & Co, SEP 30, 2021, View Source [SID1234590583]).

The decision is based on the clinical trial data generated to date, most notably the previously reported results from the INTR@PID Lung 037 study, which did not replicate the encouraging data observed in earlier studies. Based on the data generated during the agreement, no milestone payments were made by GSK and no future milestone obligations remain.

The INTR@PID clinical program sought to validate the potential of the novel mechanism of simultaneously blocking TGF-ß and PD-L1, with the ambition to improve outcomes for patients with difficult-to-treat cancers. Given the extent of the clinical program, Merck will deepen its scientific leadership in the field and interrogate the data leveraging the power of advanced analytics. The important insights this program has yielded about the biology of TGF-β will inform the collective understanding of this pathway.

Merck is a science-led organization dedicated to delivering transformative medicines with the goal of making a meaningful difference in the lives of people affected by cancer. Our oncology research efforts, comprised of 10 ongoing development programs, aim to leverage our synergistic portfolio in oncogenic pathways, immuno-oncology, and DNA Damage Response (DDR) to tackle challenging tumor types in gastrointestinal, genitourinary, and head and neck and thoracic cancers. Learn more at View Source

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Mustang Bio to Participate in Chardan’s Virtual 5th Annual Genetic Medicines Conference

On September 30, 2021 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, reported that Manuel Litchman, M.D., President and Chief Executive Officer, and members of the Mustang Leadership Team will participate in a fireside chat at Chardan’s Virtual 5th Annual Genetic Medicines Conference, taking place on Tuesday, October 5, 2021, at 8:00 a.m. ET (Press release, Mustang Bio, SEP 30, 2021, View Source [SID1234590581]). The company will also participate in one-on-one meetings during the conference. A live webcast of the company’s chat will be available on the Events page of the Investor Relations section of Mustang’s website, www.mustangbio.com, for approximately 30 days after the meeting.