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AskBio and Selecta Biosciences Receive Orphan Drug Designation for MMA-101 to Treat Methylmalonic Acidemia
[November 19, 2020]

AskBio and Selecta Biosciences Receive Orphan Drug Designation for MMA-101 to Treat Methylmalonic Acidemia


RESEARCH TRIANGLE PARK, N.C. and WATERTOWN, Mass., Nov. 19, 2020 (GLOBE NEWSWIRE) -- Asklepios BioPharmaceutical, Inc. (AskBio) and Selecta Biosciences, Inc. (NASDAQ: SELB) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to MMA-101, an AAV-based gene therapy in development for the treatment of isolated methylmalonic acidemia (MMA) due to methylmalonyl-CoA mutase (MMUT) gene mutations. MMA-101 previously received rare pediatric disease designation from the FDA in October 2020.

“The orphan drug and rare pediatric disease designations for MMA-101 support the tremendous medical need that exists for patients with MMA,” said Sheila Mikhail, CEO and co-founder of AskBio. “We look forward to working with the FDA to bring a new treatment to these patients as efficiently and quickly as possible.”

“With ImmTOR in combination with AAV technology, Selecta and AskBio are seeking to address the significant unmet need in MMA. We’re pleased the FDA has further acknowledged these efforts by awarding the program orphan drug designation,” said Carsten Brunn, Ph.D., chief executive officer of Selecta Biosciences. “We are excited to continue evaluation of the ImmTOR platform to induce AAV-specific immune tolerance and potentially enable gene therapy redosing.”

MMA is a rare monogenic disorder in which the body cannot break down certain proteins and fats. This metabolic disease may lead to metabolic crisis and is associated with long-term complications, including feeding problems, developmental delays, intellectual impairment, chronic kidney disease, optic nerve atrophy, osteopenia and pancreatitis. Typically, well-managed patients have periods of relative health with intermittent metabolic decompensation events that may result in multiorgan failure, triggered by intercurrent infections or stress episodes. Symptoms of MMA usually appear in early infancy and vary from mild to life-threatening. Without treatment, this disorder can lead to coma and, in some cases, death.

AskBio and Selecta expect to initiate a Phase 1 clinical trial of MMA-101 and ImmTOR for patients with MMA in the first half of 2021.

The FDA Office of Orphan Products Development grants orphan drug designation to investigational treatments intended for rare diseases affecting fewer than 200,000 people in the U.S. The program was created to encourage the development of medicines for rare diseases, and benefits include tax credits and application fee waivers designed to offset some development costs, as well as eligibility for market exclusivity for seven years following approval.

The FDA grants rare pediatric disease designation to incentivize the development of new treatments for serious and life-threatening diseases that primarily affect children ages 18 years or younger, with fewer than 200,000 people affected in the U.S. The rare pediatric disease designation program allows sponsors who receive approval for a product to potentially qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product.

About AskBio
Founded in 2001, Asklepios BioPharmaceutical, Inc. (AskBio) is a privately held, fully integrated AAV gene therapy company dedicated to developing life-saving medicines that cure genetic diseases. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications with a current clinical pipeline that includes therapeutics for Pompe disease, Parkinson’s disease and congestive heart failure, as well as out-licensed clinical indications for hemophilia and Duchenne muscular dystrophy. AskBio’s gene therapy platform includes Pro10™, an industry-leading proprietary cell line manufacturing process, and an extensive AAV capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, UK, the company has generated hundreds of proprietary third-generation AAV capsids and promoters, 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 and chimeric and self-coplementary capsids. Learn more at www.askbio.com or follow us on LinkedIn.



About Selecta Biosciences, Inc.
Selecta Biosciences Inc. (NASDAQ: SELB) is leveraging its clinically validated ImmTOR™ platform to develop tolerogenic therapies that selectively mitigate unwanted immune responses. With a proven ability to induce tolerance to highly immunogenic proteins, ImmTOR has the potential to amplify the efficacy of biologic therapies, including redosing of life-saving gene therapies, as well as restore the body’s natural self-tolerance in autoimmune diseases. The company’s first program aimed at addressing immunogenicity to AAV gene therapies is expected to enter clinical trials in early 2021 in partnership with AskBio for the treatment of methylmalonic acidemia (MMA), a rare metabolic disorder. A wholly owned program focused on addressing IgA nephropathy driven by ImmTOR and a therapeutic enzyme is also in development among additional product candidates. Selecta recently licensed its Phase 3 clinical product candidate, SEL-212, in chronic refractory gout to Sobi. For more information, please visit www.selectabio.com.

AskBio Forward-Looking Statements
This press release contains “forward-looking statements” regarding AskBio. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “will,” “intends,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding MMA-101, including the potential timing of the Phase 1 clinical trial for patients with MMA, AskBio’s pipeline of development candidates; AskBio’s goal of developing life-saving medicines aimed at curing genetic diseases; the potential benefits of AskBio’s development candidates to patients.


These forward-looking statements involve risks and uncertainties, many of which are beyond AskBio’s control. Known risks include, among others: AskBio may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, clinical development plans and bringing its product candidates to market, due to a variety of reasons, including the ongoing COVID-19 pandemic, possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved in a timely manner, potential disagreements or other issues with our third-party collaborators and partners, and regulatory, court or agency feedback or decisions, such as feedback and decisions from the United States Food and Drug Administration or the United States Patent and Trademark Office.

Any of the foregoing risks could materially and adversely affect AskBio’s business and results of operations. You should not place undue reliance on the forward-looking statements contained in this press release. AskBio does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

Selecta Forward-Looking Statements
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 redosing of AAV gene therapy, the potential treatment applications of product candidates utilizing the ImmTOR platform in areas such as gene therapy and MMA, the company’s plans to initiate a clinical trial for a product candidate to treat MMA, the ability of the company and AskBio to develop gene therapy products using ImmTOR and AskBio’s technology, any development plans the company and AskBio have for product candidates to treat serious and life-threatening diseases and the intention to seek regulatory approval thereof, 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 rare and orphan genetic diseases, the potential of the company’s intellectual property to enable repeat administration in gene therapy product candidates and products, the ability to redose patients and the potential of ImmTOR to allow for redosing, the potential to safely redose 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, whether the company will receive any tax credits, fee waivers, or other benefits 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 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.

AskBio Contact
Robin Fastenau
Vice President, Communications
+1 984.275.2705
rfastenau@askbio.com

Selecta Contacts
For Investors:
Lee M. Stern
Solebury Trout
+1 646.378.2922
lstern@soleburytrout.com

For Media:
Meredith Sosulski, Ph.D.
LifeSci Communications, LLC
+1 929.469.3851
msosulski@lifescicomms.com


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