Booming Abeona Plans to Hire Dozens of New Employees in Expansion
Published: Nov 21, 2017 By Mark Terry
The company, which focuses on gene therapies for rare diseases, had an underwritten public offering in October with a $92 million offering at the offering prices of $16 per share. This was offered pursuant to a shelf registration statement on June 2015 that went into effect on July 23, 2015. This new offering was made only by a written prospectus. Shares are currently trading for $14.80.
And on Nov. 9, the company announced that the first patient was enrolled in its Phase I/II clinical trial for ABO-102 MPS IIIA, a lysosomal storage disease. The patient was at the Hospital Clinico Universitario of Santiago de Compostela, Spain. As part of the launch of the Spain clinical site, the company established a local subsidiary to handle clinical trial and regulatory development activities in Europe.
ABO-102 is a gene therapy that involves delivering a corrective gene via the AAV viral vector. MPS IIIA (Sanfilippo syndrome IIIA) is a recessive inherited disease where the patients are missing an essential enzyme that breaks down the mucopolysaccharide heparin sulphate, which causes progressive damage. It results in behavioral problems, diarrhea, sleep difficulties, stiff joints, walking problems, and some phenotypic effects like full lips and heavy eyebrows.
“We are pleased to initiate enrollment at our Spain clinical site for ABO-102,” said Juan Ruiz, Abeona’s chief medical officer, in a statement. “We remain encouraged by the improvements observed in clinically relevant biomarkers post-dosing of the gene therapy in the patients in Cohort 3 and the ongoing safety profile ABO-102 demonstrates. Developing a local company presence in Spain allows us to exercise closer supervision and further advance Abeona’s gene therapy programs in Europe, as well as advance our relationships with the patient community. We are grateful to the many patient foundations and parents who have supported the research needed to advance a potential treatment for this devastating unmet medical need. We are also proud to collaborate with one of the leading clinical centers in Spain dealing with MPS IIIA patients.”
In addition to ABO-102, Abeona is developing EB-101 for recessive dystrophic epidermolysis bullosa (RDEB), ABO-101 for Sanfilippo syndrome type B (MPS IIIB), ABO-201 for juvenile Batten disease, ABO-202 for infantile Batten disease, EB-201 for epidermolysis bullosa, ABO-301 for Fanconi anemia, and ABO-302 for rare blood diseases.
The company’s expansion and hiring is expected to take place over the next six months. Timothy Miller, the company’s chief executive officer, told ABC News 5 Cleveland, “Cleveland’s been a great biotech hub. It’s biotech on the North Coast. Next-generation rare disease gene therapies and cell therapies for kids with rare genetic conditions,” are the company’s focus. “It’s very Star Trek in some respects.”
The company’s vice president of patient advocacy, Michelle Berg, told ABC News 5 Cleveland, “What we’ve heard from the families is that we brought them hope—this possibility where nothing existed before. These are rare diseases where there is nothing even in preclinical development.”