Company: GRCE
Filing Date: 2025-11-13
Form Type: 10-Q
Source: 0001140361-25-041804
Chunk: 27

Company: Grace Therapeutics, Inc.
Filing Date: 2025-11-13
Form: 10-Q
Item: Item 8
Chunk 27
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liquid solution of betamethasone to reduce ataxia symptoms in patients with
A-T. This oral liquid solution is not marketed in the United States, and
therefore is not available for clinical use. Currently, betamethasone is only
available in the United States as an injectable or as a topical cream. This
license gives us the right to reference the trial’s data in our NDA filing. On
November 12, 2015, we submitted the data from the Zannolli trial to the FDA’s
Division of Neurology at a pre-Investigational New Drug (“IND”) meeting and
received guidance from the agency on the regulatory requirements to seek
approval. 

   About Ataxia Telangiectasia

   A-T is a rare genetic progressive autosomal recessive neurodegenerative disorder that affects children, with the hallmark symptoms of cerebellar ataxia and other motor dysfunction, and dilated blood vessels (telangiectasia) that occur in the sclera of the eyes. A-T is caused by mutations in the ataxia telangiectasia gene, which is responsible for modulating cellular response to stress, including breaks in the double strands of DNA.

   Children with A-T begin to experience balance and coordination problems when they begin to walk (toddler age), and ultimately become wheelchair-bound in their second decade of life. In pre-adolescence (between ages 5 and 8), patients experience oculomotor apraxia, dysarthria, and dysphagia. They also often develop compromised immune systems and are at increased risk of developing respiratory tract infections and cancer (typically lymphomas and leukemia).

   Unmet Needs

There
is no known treatment to slow disease progression, and treatments that are used
are strictly aimed at controlling the symptoms (or conditions secondary to the
disease).  There are no FDA-approved
therapeutic options currently available. Patients typically die by age 25 from
complications of lung disease or cancer. 

   Market Opportunity

   According to a third-party report we commissioned, A-T affects approximately 4,300 patients per year in the United States and has a potential total addressable market of $150 million, based on the number of treatable patients in the United States.

   Clinical Data

   In a multicenter, double-blind, randomized, placebo-controlled crossover trial conducted in Italy, Dr. Zannolli et al. studied the effect of an oral