AskBio Licenses Rights to Selecta Biosciences’ ImmTOR™ Immune Tolerance Platform for the Treatment of Pompe Disease
– Selecta eligible to receive upfront and milestone payments of over $240 million –
– AskBio’s AAV gene therapy combined with ImmTOR could prevent the formation of neutralizing antibodies and potentially enable re-treatment of patients with Pompe disease –
Watertown, Mass. and Research Triangle Park, N.C., December 19, 2019 – Selecta Biosciences, Inc. (NASDAQ: SELB) and Asklepios BioPharmaceutical, Inc. (AskBio), today announced that the companies have entered into a license agreement. Under the terms of the agreement, AskBio has exercised its option to exclusively license rights to develop and commercialize Selecta’s immune tolerance platform, ImmTOR, for use in adeno-associated virus (AAV) gene therapy for the treatment of Pompe disease. When used in combination with AAV gene therapy vectors, Selecta’s ImmTOR has been shown to inhibit the immune response to the vector (Nature Communications, October 2018).
Selecta and AskBio previously announced a strategic partnership in August 2019 to jointly develop, manufacture, and commercialize targeted therapeutics for next-generation AAV gene therapies in areas of high medical need. Under the terms of this new license agreement, Selecta is eligible to receive upfront and milestone payments of over $240 million plus royalties on product sales.
“We are pleased to advance our ImmTOR platform in a new partnership with AskBio for Pompe disease, a serious and progressively debilitating disease,” said Carsten Brunn, Ph.D., President and Chief Executive Officer of Selecta. “We are excited to collaborate with AskBio to potentially enable the retreatment of AAV gene therapies.”
“There is a demonstrated unmet medical need for better treatment approaches for Pompe disease, and this collaboration will enable us to effectively advance our Pompe program with the added benefit of Selecta’s ImmTOR technology,” said Sheila Mikhail, CEO and co-founder of AskBio. “The opportunity to re-treat patients holds significant promise, and we are pleased to be able to leverage our relationship with Selecta and apply the ImmTOR technology to potentially overcome the challenges associated with re-administering systemic AAV gene therapies.”
Pompe disease is a rare, progressive, debilitating condition that affects 5,000 to 10,000 people worldwide. It impacts ventilator, cardiac and skeletal muscles and can cause motor neuron dysfunction, with effects on cognition, hearing, speech and fine motor skills. Pompe disease can manifest any time between infancy and late adulthood and may have differing symptoms and severity, depending on when it develops. There is a substantial unmet medical need for more efficient and effective treatments for Pompe disease. Currently, the only approved treatment is ERT with recombinant human GAA (rhGAA), a chronically administered therapy requiring increasing doses, which can elevate both costs and treatment burden.
About Selecta Biosciences, Inc.
Selecta Biosciences, Inc. is a clinical-stage biotechnology company focused on unlocking the full potential of biologic therapies based on its immune tolerance technology (ImmTOR) platform. Selecta plans to combine ImmTOR with a range of biologic therapies for rare and serious diseases that require new treatment options due to high immunogenicity. The company’s current proprietary pipeline includes ImmTOR-powered therapeutic enzyme and gene therapy product candidates. SEL-212, the company’s lead product candidate, is being developed to treat chronic refractory gout patients and resolve their debilitating symptoms, including flares and gouty arthritis. Selecta’s proprietary gene therapy product candidates are in preclinical development for certain rare inborn errors of metabolism and incorporate ImmTOR with the goal of addressing barriers to repeat administration. Selecta is based in Watertown, Massachusetts. For more information, please visit http://selectabio.com.
Founded in 2001, Asklepios BioPharmaceutical, Inc. (AskBio) is a privately held, clinical-stage gene therapy platform company dedicated to improving the lives of children and adults with genetic disorders. AskBio’s gene therapy platform includes an industry-leading proprietary cell line manufacturing process known as Pro10™ and an extensive AAV capsid library. Based in Research Triangle Park, North Carolina, the company has generated hundreds of proprietary third-generation gene vectors, several of which have entered clinical testing. An early innovator in the space, the company holds more than 500 patents in areas such as AAV production, chimeric vectors and self-complementary DNA. AskBio maintains a portfolio of clinical programs across a range of neurodegenerative and neuromuscular indications with a current clinical pipeline that includes therapeutics for Pompe disease, limb-girdle muscular dystrophy and congestive heart failure, as well as out-licensed clinical indications for hemophilia (Chatham Therapeutics acquired by Takeda) and Duchenne muscular dystrophy (Bamboo Therapeutics acquired by Pfizer). For more information, visit https://www.askbio.com/.
About Pompe Disease
Pompe disease is an inherited lysosomal storage disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA lead to accumulation of glycogen in cells, which is believed to result in the clinical manifestations of Pompe disease. The disease can be debilitating and is characterized by severe muscle weakness that worsens over time. Pompe disease ranges from a rapidly fatal infantile form with significant impacts to heart function, to a more slowly progressive, late-onset form primarily affecting skeletal muscle. It is estimated that Pompe disease affects approximately 5,000 to 10,000 people worldwide.
Any statements in this press release about the future expectations, plans and prospects of Selecta Biosciences, Inc. (“the company”), including without limitation, statements regarding the unique proprietary technology platform of the company, and the unique proprietary platform of its partners, the potential of ImmTOR to enable re-dosing of AAV gene therapy, the potential treatment applications of product candidates utilizing the ImmTOR platform in areas such as gene therapy, the ability of the company and AskBio to develop gene therapy products using ImmTOR and AskBio’s technology, the novelty of treatment paradigms that the Company is able to develop, the potential of any therapies developed by the Company and AskBio to fulfill unmet medical needs, the company’s plan to apply its ImmTOR technology platform to a range of biologics for treatment of Pompe disease, the potential of the company’s intellectual property to enable repeat administration in gene therapy product candidates and products, the ability to re-dose patients and the potential of ImmTOR to allow for re-dosing, the potential to safely re-dose AAV, the ability to restore transgene expression, the potential of the ImmTOR technology platform generally and the company’s ability to grow its strategic partnerships, the ability of the company and AskBio to achieve developmental milestones, and other statements containing the words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “hypothesize,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would,” and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including, but not limited to, the following: the uncertainties inherent in the initiation, completion and cost of clinical trials including proof of concept trials, including the uncertain outcomes, the availability and timing of data from ongoing and future clinical trials and the results of such trials, whether preliminary results from a particular clinical trial will be predictive of the final results of that trial or whether results of early clinical trials will be indicative of the results of later clinical trials, the unproven approach of the company’s ImmTOR technology, potential delays in enrollment of patients, undesirable side effects of the company’s product candidates, its reliance on third parties to manufacture its product candidates and to conduct its clinical trials, the company’s inability to maintain its existing or future collaborations, licenses or contractual relationships, its inability to protect its proprietary technology and intellectual property, potential delays in regulatory approvals, the availability of AskBio’s funding to meet contractual obligations regarding developmental milestones, the availability of funding sufficient for its foreseeable and unforeseeable operating expenses and capital expenditure requirements, the company’s recurring losses from operations and negative cash flows from operations raise substantial doubt regarding its ability to continue as a going concern, substantial fluctuation in the price of its common stock, and other important factors discussed in the “Risk Factors” section of the company’s most recent Quarterly Report on Form 10-Q, and in other filings that the company makes with the Securities and Exchange Commission. In addition, any forward-looking statements included in this press release represent the company’s views only as of the date of its publication and should not be relied upon as representing its views as of any subsequent date. The company specifically disclaims any intention to update any forward-looking statements included in this press release.
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Joshua R. Mansbach
Lee M. Stern