The shares of Abeona Therapeutics Inc(NASDAQ: ABEO) more than tripled this year, generating 226.8% returns (As of December 22nd, 2017; Source: Google finance).
Major triggers for the stock includes getting an FDA for Orphan Drug Designation for ABO-201 Juvenile Batten Disease Gene Therapy Program, starting Pivotal Phase 3 clinical trial for EB-101 Gene Therapy for Patients with Epidermolysis Bullosa in early 2018 and positive trial results. The stock still promises an upside potential of over 87.4% as per seven analysts’ consensus target price of $29.71 (Source: tipranks.com).
In July, the group reported that they are starting the Pivotal Phase 3 clinical trial in early 2018 for EB-101 gene therapy for patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB) which showed promising efficacy and safety in the Phase 1/2 clinical trial. EB-101 has got the Orphan Drug and Rare Pediatric Disease Designations in the USA and Orphan Drug Designation in the EU. The group got the FDA Orphan Drug Designation for ABO-201 Juvenile Batten Disease Gene Therapy Program.
FDA guided the group to speeden their Phase 3 trial for EB-101 autologous cell therapy during August as EB-101 has showed a major efficacy in treated patients for over 2 years. The group speeded the Phase 3 clinical trial for EB-101, the leading gene therapy for patients with Recessive Dystrophic Epidermolysis Bullosa.
The group got Announces $13.85 million Grant from Sanfilippo Syndrome Foundations for Clinical Development of MPS III Gene Therapies while closed $92 Million Underwritten Public Offering and Full Exercise of Underwriters’ Option to Purchase Additional Shares.
As of the third quarter of 2017, the group’s progress includes starting enrollments at their global clinical sites for ABO-102 for MPS IIIA while reported a further data that underscored the durability and clinical benefit of the gene therapy. Their Epidermolysis Bullosa program achieved FDA Breakthrough Therapy designation, while finished Phase 1/2 clinical trial and continued to advance as they finalized the clinical protocol before starting the pivotal Phase 3 trial next year. They are optimizing their AIM™ vector platform which comprises a better tissue tropisms against the naturally occurring AAV capsids.
For the third quarter of 2017, the group reported a Cash and cash equivalents of $56.5 million, from $58.3 million as of June 30, 2017. The Total cash as of October 31, 2017 was $142.6 million. Revenues improved to $219 thousand for the third quarter of 2017, against $184 thousand in the prior corresponding period. But Loss per share rose to $0.13 for the third quarter of 2017, against loss per share of $0.08 in the comparable period in 2016 due to rising expenses.