INBRX-101

INBRX-101 is an Fc-fusion protein-based therapeutic candidate comprising a modified recombinant version of human alpha-1 antitrypsin, or AAT, that we are developing for the treatment of patients with alpha-1 antitrypsin deficiency, or AATD. AATD is a genetically defined rare respiratory disease characterized by progressive destruction of lung tissue that has an FDA approved diagnostic. According to the Alpha-1 Foundation, this disease affects roughly 100,000 people in the United States and approximately the same number of people in Europe. The current standard of care for patients with AATD has been unchanged for decades and relies on weekly infusions of plasma derived AAT, or pdAAT, therapeutics. According to the American Journal of Respiratory and Critical Care Medicine, there are currently approximately 10,000 AATD patients worldwide receiving plasma-derived augmentation therapies, and according to Transparency Market Research, the worldwide market for AATD treatment is expected to grow from $1.2 billion in 2016 to $2.9 billion in 2025. AAT has proven difficult to develop recombinantly, often displaying loss of activity and experiencing accelerated degradation. INBRX-101 is designed to offer superior clinical activity to pdAAT by providing sustained enhanced plasma concentration with a less frequent, monthly dosing regimen. The IND for INBRX-101 became effective in November 2018, and we expect to initiate a Phase 1 dose escalation clinical trial in July 2019. In May 2019, we entered into an option agreement, as amended by the First Amendment to the Option Agreement, dated August 19, 2019, or the Chiesi Option Agreement, with Chiesi, pursuant to which we granted Chiesi an exclusive option to obtain an exclusive license to develop and commercialize INBRX-101 outside of the United States and Canada following completion of the Phase 1 trial. We expect to announce preliminary functional pharmacokinetic data from the single dose escalation portion of this trial in the first half of 2020 and full Phase 1 data, including the multi-dose cohorts, in the second half of 2020.