Inceptua Early Access and Sentynl Therapeutics Inc. Announce the
Launch of the Early Access Program for Nulibry® (fosdenopterin) for Pediatric Patients with Molybdenum Cofactor Deficiency (MoCD) Type A

Early Access (a business unit of the Inceptua Group), and Sentynl Therapeutics Inc. (“Sentynl”), a U.S.-based biopharmaceutical company focused on bringing innovative therapies to patients living with rare diseases today announced that they have entered into a partnership to make Sentynl’s Nulibry® available via an Early Access Program for eligible patients with molybdenum cofactor deficiency (MoCD) Type A.

MoCD Type A is a rapidly progressive autosomal recessive inborn error of metabolism resulting in toxic sulfite levels causing neurologic sequelae including seizures, difficulty feeding, severe developmental delays, and death within the first 4 years of life if left untreated. Nulibry is the first approved treatment option for MoCD Type A. Nulibry was approved by the FDA in 2021, and in September 2022 the European Commission granted marketing authorisation for Nulibry under exceptional circumstances.

Early Access Programs (also known as expanded access, early access, compassionate use, named patient supply) are a compliant route through which medicines which are either still under development or not approved in their country of intended use, can be made available for patients who either have no alternative treatment option, or have exhausted all other treatment options available in their country of residence.

“Inceptua Early Access is delighted to be supporting Sentynl with the Nulibry Early Access Program to allow appropriate patients with MoCD Type A the opportunity to access the first approved therapeutic option for this disease. MoCD Type A is a devastating disease with significant unmet need and anything we can do to support early access to this new treatment option will give hope to the families of the children affected by this condition.” said Stuart Bell, Vice President, Early Access, Inceptua Group.

Grant Castor, Senior Vice President, Commercial Strategy and Operations at Sentynl, said, “We are pleased to be partnering with Inceptua Early Access to provide a formal centralized process for handling requests where there are suspected and confirmed cases of MoCD Type A globally. A presumptive diagnosis of MoCD Type A and rapid initiation of treatment is critical for all children with this ultra-rare disease worldwide.”

Requests for access to Nulibry must be made by a Healthcare Professional (HCP). HCPs can request access for a patient by contacting Inceptua at: access@inceptua.com or calling + 44 20 3910 7670.

About Sentynl Therapeutics, Inc.

Sentynl Therapeutics is a U.S.-based biopharmaceutical company focused on bringing innovative therapies to patients living with rare diseases. The company was acquired by the Zydus Group in 2017. Sentynl’s experienced management team has previously built multiple successful pharmaceutical companies. With a focus on commercialization, Sentynl looks to source effective and highly differentiated products across a broad spectrum of therapeutic areas to address unmet needs. Sentynl is committed to the highest ethical standards and compliance with all applicable laws, regulations, and industry guidelines. For more information, visit www.sentynl.com.

About Molybdenum Cofactor Deficiency (MoCD) Type A

MoCD Type A is an autosomal recessive, inborn error of metabolism caused by mutations in the molybdenum cofactor synthesis 1 gene and characterized by a deficiency in molybdenum cofactor production, leading to a lack of molybdenum-dependent enzyme activity. The lack of activity leads to decreased sulfite oxidase activity with buildup of sulfite and secondary metabolites (such as S-sulfocysteine) in the brain, which causes irreversible neurological damage.

The most common presenting symptoms of MoCD Type A are seizures, feeding difficulties and encephalopathy. Patients with MoCD Type A who survive beyond infancy typically suffer from progressive brain damage, which presents in characteristic patterns on magnetic resonance imaging (MRI). This damage leads to severe psychomotor impairment and an inability to make coordinated movements or communicate with their environment.

About Nulibry

NULIBRY(fosdenopterin) for Injection is a substrate replacement therapy that provides an exogenous source of cPMP, which is converted to molybdopterin. Molybdopterin is then converted to molybdenum cofactor, which is needed for the activation of molybdenum-dependent enzymes, including sulfite oxidase, an enzyme that reduces levels of neurotoxic sulfites. It is the first and only FDA-approved therapy indicated to reduce the risk of mortality in patients with MoCD Type A, and clinical trials have demonstrated that patients treated with NULIBRY or rcPMP had an improvement in overall survival compared to the untreated, genotype-matched, historical control group. Full Prescribing Information can be found by clicking here.

About Inceptua Early Access

Inceptua Early Access partners with pharma and biotech companies to provide truly global early access programs, which allow patients around the world access to potentially-life saving medicines

Inceptua Early Access is one of three business units of the Inceptua Group (a specialty pharmaceutical company and a premium global service partner) along with:

Inceptua Clinical Trial Supply which supports pharma and biotech companies through global clinical trial comparator sourcing, packaging and labelling solutions.

Inceptua Pharma which commercializes and markets orphan and specialty care products.

The Inceptua Group draws on over 25 years of experience and a global infrastructure to support its clients around the world.