On October 24, 2022 Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., "Astellas") and Taysha Gene Therapies, Inc. (NASDAQ: TSHA, CEO: RA Session II, "Taysha") reported a strategic investment to support the advancement of Taysha’s adeno-associated virus (AAV) gene therapy development programs for the treatment of Rett syndrome and GAN (Press release, Astellas, OCT 24, 2022, View Source [SID1234622287]). The future options to potentially apply Astellas’ global R&D, manufacturing and commercialization capabilities in gene therapy to Taysha’s innovative AAV gene therapy development programs for genetic diseases of the central nervous system (CNS) create the opportunity for the two companies to enhance the development of novel treatment options for patients with Rett syndrome and GAN, who have serious unmet medical needs.

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Under the terms of the agreement, Astellas will invest a total of $50 million to acquire 15% of the outstanding common stock of Taysha and to receive an exclusive option to license two of Taysha’s clinical stage programs: TSHA-102 for Rett syndrome and TSHA-120 for GAN. In addition, Taysha has granted Astellas certain rights related to any potential change of control of Taysha. Definitive agreements would be executed upon Astellas’ exercise of any such option, and any change of control transaction would require approval by Taysha’s stockholders.

Taysha is engaged in the development of intrathecally-delivered AAV gene therapies for monogenic CNS diseases. As a part of this platform approach, Taysha has a promising pipeline, including TSHA-102, which is the first-and-only gene therapy in clinical development for Rett syndrome, and TSHA-120, which is in Phase 1/2 development for the treatment of GAN and awaiting regulatory feedback.

Astellas is continuing to build its capability to bring novel gene therapies to patients, following the acquisition of Audentes (now Astellas Gene Therapies, California) in January 2020 and the construction of a state-of-the-art commercial GMP manufacturing facility in North Carolina, which was opened in June of this year.

"Gene therapy is the corner stone of Astellas’ Primary Focus, Genetic Regulation*1; our goal is to bring new transformative treatment options to patients living with serious genetic diseases and limited treatment options," said Naoki Okamura, Chief Strategy Officer, at Astellas. "Taysha is an industry leader in CNS gene therapies and this partnership fits strategically with our long-term vision of expanding Astellas’ gene therapy capabilities, allowing the company to impact the lives of a broader range of patients with urgent unmet medical needs."

"We are excited to enter this strategic investment with Astellas, a premier biopharmaceutical company with global R&D, manufacturing and commercial capabilities," said RA Session II, Taysha’s Chief Executive Officer. "We believe this investment not only further validates the potential of our technology platform, but also reinforces the therapeutic and market opportunity of our two lead clinical assets."

To further strategically align Astellas and Taysha, in connection with its equity investment, Astellas will receive one Board observer seat on Taysha’s Board of Directors, enabling Taysha to leverage Astellas’ gene therapy clinical and commercial expertise as Taysha advances TSHA-120 and TSHA-102.

*1: Astellas has established a Focus Area Approach for its research and development strategy. For more information, please visit our website at View Source

About TSHA-102
TSHA-102 is a self-complementary intrathecally delivered AAV9 gene replacement therapy under development for the treatment of Rett syndrome. TSHA-102 utilizes the novel miRNA-Responsive Auto-Regulatory Element (miRARE) platform to regulate transgene expression genotypically on a cell-by-cell basis. The miRARE technology is designed to prevent toxicity associated with transgene overexpression and can be potentially utilized across other indications. TSHA-102 has received Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA) and Orphan Drug Designation from the European Commission.

About Rett Syndrome
Rett syndrome is a severe genetic neurodevelopmental disorder caused by a mutation in the X-linked MECP2 gene essential for neuronal and synaptic function in the brain. Primarily occurring in females, Rett syndrome is one of the most common genetic causes of severe intellectual disability worldwide. Patients have normal early development, with symptom onset typically beginning between 6 to 18 months of age. Rett syndrome is characterized by rapid developmental regression that leads to intellectual disabilities, loss of speech, loss of purposeful use of hands, loss of mobility, seizures, cardiac impairments and breathing issues. Currently, there are no approved therapies that treat the underlying cause of this progressive disease.

About TSHA-120
TSHA-120, an intrathecally dosed AAV9 gene replacement therapy delivering the gene gigaxonin for the treatment of GAN, is currently being evaluated in an ongoing Phase 1/2 clinical trial. TSHA-120 has received Orphan Drug and Rare Pediatric Disease designations from FDA and Orphan Drug Designation from the European Commission.

About Giant Axonal Neuropathy (GAN)
GAN is rare inherited genetic disorder that is a progressive neurodegenerative disease that affects both the central and peripheral nervous systems. The disease is caused by loss-of-function mutations in the gene coding for gigaxonin, which results in dysregulation of intermediate filament turnover, an important structural component of the cell. Children with GAN present before the age of five with symptoms including unsteady gait, frequent falls, and motor weakness. Currently, there are no approved treatments for GAN, which results in death for patients in their late teens or early twenties.

On October 24, 2022 Alpine Immune Sciences, Inc. (NASDAQ: ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for autoimmune and inflammatory diseases, reported that the Company has voluntarily terminated enrollment in both clinical studies involving davoceticept (ALPN-202), an investigational CD28 costimulator and dual checkpoint inhibitor, including the NEON-1 study of davoceticept as monotherapy and the NEON-2 study of davoceticept in combination with pembrolizumab (Press release, Alpine Immune Sciences, OCT 24, 2022, View Source [SID1234622286]). Following these decisions, the Company plans to focus its development resources primarily on ALPN-303, a potentially best-in-class dual BAFF/APRIL B cell cytokine inhibitor in development for multiple autoantibody-related inflammatory diseases, as well as acazicolcept (ALPN-101), a potentially first-in-class dual CD28/ICOS inhibitor in development for systemic lupus erythematosus (SLE) in collaboration with AbbVie.

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The decision to terminate enrollment in the davoceticept studies was made in the interest of patient safety after the Company was notified of a second death in the NEON-2 study, attributed to cardiogenic shock. The participant, who had metastatic colorectal cancer previously treated with colectomy and multiple prior systemic chemotherapies, had received a single dose each of davoceticept and pembrolizumab. NEON-2 had previously been subject to a partial clinical hold due to a death attributed to cardiogenic shock. The Company is conducting an ongoing, comprehensive assessment of all NEON study participants.

"Patient safety remains our highest priority," said Mitchell H. Gold, M.D., Executive Chairman and Chief Executive Officer of Alpine. "We have determined it is in the best interest of all patients to terminate enrollment in the davoceticept studies and we will continue to work with the U.S. Food and Drug Administration, Merck, the study Safety Monitoring Committee, and the study investigators to further understand this important safety issue. Davoceticept has shown encouraging signs of clinical activity and it is unfortunate we have not yet been able to identify a safe dose regimen for the combination with pembrolizumab. We will now prioritize the bulk of our development resources towards advancing our lead wholly-owned program ALPN-303 in multiple autoimmune and inflammatory indications, as well as acazicolcept in SLE in collaboration with AbbVie."

About Davoceticept and the NEON Studies

Davoceticept (ALPN-202) is a first-in-class, conditional CD28 costimulator and dual checkpoint inhibitor intended for the treatment of cancer. Preclinical studies of davoceticept have successfully demonstrated superior efficacy in tumor models compared to checkpoint inhibition alone. In phase 1 studies, davoceticept has demonstrated encouraging clinical activity, especially in renal cell carcinoma, as monotherapy and in combination with pembrolizumab.

NEON-1 (NCT04186637) is a phase 1 monotherapy dose escalation and expansion study in adults with advanced malignancies. NEON-2 (NCT04920383) is a phase 1 dose escalation and expansion combination study of davoceticept (ALPN-202) and pembrolizumab.

About ALPN-303

ALPN-303 is a dual B cell cytokine antagonist being developed for multiple autoimmune and/or inflammatory diseases. Based upon an engineered TACI (transmembrane activator and CAML interactor) domain, ALPN-303 in preclinical studies shows robust inhibition of B cell activating factor/B lymphocyte stimulator (BAFF, BLyS) and a proliferation inducing ligand (APRIL). These two pleiotropic B cell cytokines play key roles in B cell development, differentiation, and survival, and together contribute to the pathogenesis of multiple autoimmune diseases like systemic lupus erythematosus (SLE) and many other autoantibody-related inflammatory diseases. By simultaneously blocking these two cytokines, ALPN-303 has the potential to improve outcomes in patients suffering from severe autoimmune and/or inflammatory diseases. Alpine plans to conduct a phase 2 proof-of-concept study in SLE and open-label basket studies in renal, hematologic, and dermatologic autoimmune diseases, with the first of these anticipated to begin in the first half of 2023.

On October 24, 2022 Enochian BioSciences (the Company) reported that significant progress toward re-prioritizing its focus on curing some of the world’s deadliest diseases (Press release, Enochian BioSciences, OCT 24, 2022, View Source [SID1234622284]). Last week, the Company announced its oncology platform was awarded a U.S. patent and has produced promising early results in studies conducted in conjunction with Dr. Ana Jewett at UCLA. The Company also awaits potentially positive results from studies on its HIV platform, which are being conducted by scientists at the Fred Hutchinson Cancer Center.

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"We continue to prioritize the efficient research, development, and commercialization of our promising oncology and HIV pipelines," said Enochian CEO Dr. Mark Dybul. "Enochian’s future is bright, and we look forward to advancing potentially curative therapies for some of the world’s deadliest diseases."

The Company also announced it filed a complaint against Serhat Gumrukcu, William Anderson Wittekind, SG & AW Holdings LLC, and Seraph Research Institute in the California Superior Court for Los Angeles County. As alleged in the complaint, the defendants engaged in a "concerted, deliberate scheme to alter, falsify, and misrepresent to [the Company] the results of multiple studies supporting its [Hepatitis B] and SARS-CoV-2/influenza pipelines." "Defendants manipulated negative results to reflect positive outcomes from various studies, and even fabricated studies out of whole cloth. Defendants’ conduct amounts to nothing short of brazen fraud, which has caused Enochian substantial harm." Through this lawsuit, the Company "intends to hold [the] Defendants responsible for their conduct and recover damages resulting" from their actions.

On October 23, 2022 Sumitovant Biopharma Ltd. ("Sumitovant"), in conjunction with parent company Sumitomo Pharma Co., Ltd. ("Sumitomo Pharma"), and Myovant Sciences ("Myovant") (NYSE: MYOV) reported that they have entered into a definitive agreement pursuant to which Sumitovant will acquire all outstanding shares of Myovant not already owned by Sumitovant for $27.00 per share in cash (Press release, Sumitovant Biopharma, OCT 23, 2022, View Source [SID1234622281]). This corresponds to a total transaction value of $1.7 billion on a fully diluted basis, and a total company value of $2.9 billion on a fully diluted basis. Sumitovant currently beneficially owns 52% of the issued and outstanding shares of Myovant as more particularly described in Sumitovant’s Schedule 13D/A filed with the U.S. Securities and Exchange Commission (the "SEC").

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The purchase price represents a premium of approximately 50% to Myovant’s closing share price on September 30, 2022, the last day of trading prior to Sumitovant’s initial non-binding proposal, and a premium of approximately 55% to the 60-day volume weighted average price of Myovant’s shares through September 30, 2022. The agreement has been approved by the boards of Sumitovant and Sumitomo Pharma and unanimously recommended by a Special Committee of the independent directors of Myovant and, acting upon such recommendation, approved by its full board of directors with the Sumitovant designated directors recusing themselves and abstaining from the deliberations and vote.

"This transaction represents an industry-leading opportunity to combine unique expertise, platforms, and resources to successfully commercialize products in Myovant’s program and to accelerate development of a robust pipeline addressing patient needs in women’s health and prostate cancer," said Myrtle Potter, CEO of Sumitovant. "We look forward to harnessing the combined strength of our talented teams to bring needed therapies to patients sooner and are confident both Myovant and its employees will benefit from the greater resources Sumitovant can provide to further support business growth and career opportunities overall."

"Myovant’s two products, ORGOVYX and MYFEMBREE have substantial potential. We believe the combination of Sumitovant and Myovant will strengthen Myovant’s product capabilities and help continue to deliver innovative therapies addressing unmet patient needs in prostate cancer and women’s health," said Hiroshi Nomura, CEO of Sumitomo Pharma. "By making Myovant a wholly owned subsidiary of Sumitovant, we believe that we will be able to accelerate implementation of management strategies that make full use of cash flow generated by ORGOVYX and MYFEMBREE for sustained growth of the Sumitomo Pharma Group."

"We are pleased to have reached an agreement with Sumitovant and Sumitomo Pharma that recognizes the remarkable success Myovant has achieved," said David Marek, CEO of Myovant. "With the expertise and resources of Sumitovant to best support Myovant, and our employees, we can do more to expand the impact of our differentiated therapies, advance our clinical programs, and work to remove barriers to access quality care for the patients we serve."

"After careful consideration and consultation with our legal and financial advisors, the Special Committee believes that this transaction provides immediate and compelling value to Myovant’s minority shareholders, as well as positioning the Company for continued growth, and is in the best interest of Myovant and its shareholders," said Mark Guinan, Chairman of the Special Committee.

Transaction Details
The transaction is anticipated to close in the first quarter of 2023, subject to customary closing conditions, including obtaining the requisite regulatory approvals and approval by Myovant shareholders holding a majority of the outstanding shares not beneficially owned by Sumitovant and its affiliates. The transaction will be financed through a combination of cash on hand and external debt financing. A financing commitment has been received from Sumitomo Mitsui Banking Corporation. The transaction is not subject to a financing condition.

Upon completion of the transaction, Myovant will become a wholly owned subsidiary of Sumitovant and Myovant’s shares will no longer be listed on the New York Stock Exchange.

Advisors
J.P. Morgan Securities LLC is serving as financial advisor and Sullivan & Cromwell LLP is serving as legal counsel to Sumitovant and Sumitomo Pharma. Goldman Sachs & Co. LLC is serving as financial advisor to the Special Committee of the Board of Directors of Myovant and Skadden, Arps, Slate, Meagher & Flom LLP is serving as legal counsel to the Special Committee.

On October 23, 2022 Mevion Medical Systems, the leading provider of compact proton therapy systems for use in radiation treatment for cancer patients, reported that it is developing the MEVION S250-FIT Proton Therapy System with HYPERSCAN Pencil Beam Scanning (PBS) (Press release, Mevion Medical Systems, OCT 23, 2022, View Source [SID1234622279]). Stanford Health Care has been selected as the first site where the system will be developed and installed.

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Proton therapy is a precise form of radiation therapy that reduces the amount of unnecessary radiation exposure to healthy tissue, which has the potential to decrease or eliminate the treatment side effects and lessen the risk of secondary malignancies.

Historically, the size, cost, and inherent complexity of proton technologies have limited the adoption of proton therapy. Mevion believes that the MEVION S250-FIT system has the potential to overcome the practical challenges of other technologies because the system is designed to be a more compact, affordable solution that can fit into an existing LINAC vault. Today, new proton therapy centers in the U.S. are almost exclusively compact single-room systems. Mevion’s compact system would continue to advance the design and accessibility of proton therapy. The MEVION S250-FIT system is currently in development and has not received regulatory clearance for clinical use.

"Mevion is dedicated to making proton therapy deployment similar to conventional radiation by simplifying room renovation requirements, enabling faster installation, and facilitating integration with other radiation therapy modalities," said Tina Yu, Ph.D., CEO and President of Mevion Medical Systems. "Through our arrangement with Stanford Health Care, Mevion is aiming to demonstrate that proton therapy can fit in an existing vault of a radiation oncology department and in turn become more accessible."

*The MEVION S250-FIT Proton Therapy System has not received regulatory clearance.