Upcoming market catalysts in Q3 2022

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Upcoming market catalysts in Q3 2022

Upcoming catalysts in the third quarter of 2022 include top-line results from the phase IIb FREEDOM study of an autologous cell therapy known as Xowna for the treatment of angina, the phase III PaTHway study of TransCon PTH for the treatment of hypoparathyroidism in Japanese patients, and the phase III VIITAL study of EB-101 for the treatment of epidermolysis bullosa.

Caladrius Biosciences is developing Xowna, a cell therapy comprised of patient-derived autologous CD34+ cells, for the treatment of people who experience chronic angina despite not having visibly blocked arteries. Previous results from a phase IIa study showed highly statistically significant improvement in coronary flow reserve correlating with symptom relief for patients with coronary microvascular dysfunction after a single intracoronary injection of Xowna. In October 2020, the company initiated the pivotal double-blind, placebo-controlled phase IIb FREEDOM trial evaluating the safety and efficacy of Xowna in patients with coronary microvascular dysfunction and without obstructive coronary artery disease. However, patient enrollment in the study was recently suspended, and the trial only enrolled

approximately one third of the target number of patients owing to supply chain issues associated with COVID-19. Caladrius hopes that an interim analysis of the data collected so far and follow-up of the enrolled population will yield enough evidence to facilitate further investigations. The data from the analysis is expected to provide an indication of the magnitude of the Xowna effect size on the clinical endpoints likely to be required by the FDA in a future pivotal study. Results from the interim analysis are expected in August 2022. However, the company will need to find a way to overcome the supply and recruitment issues for d evelopment to be successful.

TransCon PTH is an extended release self-injectable prodrug of parathyroid hormone under development by Ascendis Pharma as a once-daily hormone replacement therapy. The company initiated the single-arm, multicentre phase III PaTHway Japan trial in August 2021 to evaluate the safety, tolerability and efficacy of daily subcutaneous injections of TransCon PTH in Japanese adults with hypoparathyroidism. Early phase III data from PaTHway studies in the USA and Europe have demonstrated positive safety and efficacy results, with

TransCon PTH-treated patients demonstrating statistically significant improvement compared to control on the primary composite endpoint of normal serum calcium levels, as well as all key secondary endpoints. Current hypoparathyroidism treatment approaches are limited to calcium supplements and active vitamin D or analogues. The only approved parathyroid hormone replacement product is Takeda's Natpara, which was approved by the FDA in 2015, but recalled in September 2019 due to the potential for rubber particulate formation. Natpara is not approved in Japan, and results from Ascendis' Japanese study could support TransCon PTH as a replacement in the market. Top-line results from the study are expected in the third quarter of 2022.

Abeona Therapeutics is developing EB-101, an ex vivo retrovirus gene therapy, for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). The therapy has shown fairly encouraging efficacy data to date, as a previous phase I/IIa trial demonstrated wound healing of 50% from baseline in 69% of wounds at 3 years and 93% at 4 years, with pain relief in 76% of treated wounds with 50% healing. Data on long-term rates of healing in more patients are needed in order to fully support efficacy for potential regulatory submissions. There are currently no approved therapies for epidermolysis bullosa, although there are numerous competitors in phase III development. EB-101's closest competitor is Castle Creek Biosciences' FCX-007, a gene-modified autologous fibroblast that encodes collagen VII (COL7) using a lentiviral vector. FCX-007 has also shown positive clinical results, although not as strong or robust as EB-101, with fewer patients and wounds. Krystal Biotech's B-VEC therapy is another close competitor in phase III development, with positive trial results disproving a previously held theory that EB-101 had the advantage in healing large wounds. Top-line results from Abeona's pivotal phase III VIITAL study in 15 patients with RDEB are anticipated in the third quarter of 2022, and will provide a clearer view of the potential advantages or disadvantages of EB-101 compared with its competitors.

Sarina Harper

Informa, New York, NY, USA.

e-mail: Sarina.Harper@



Competing interests The author declares no competing interests.

Nature Reviews | Drug Discovery

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volume 21 | July 2022 | 485

Credit: sesame/DigitalVision Vectors

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