Gene Therapy Technology Companies

Gene therapy is getting major attention again this month with the recently announced deal between Bristol-Myers Squibb and uniQure and upcoming top-line data from Celladon’s Phase 2 CUPID 2 study in heart failure.

We believe this renewed attention to the space could be translated into growth opportunities for companies in the field as well as a potential boost for three Buy-rated names in our universe: Applied Genetic Technologies (ticker: AGTC), GenVec ( GNVC ) and Oxford Biomedica ( OXBDF ).

Earlier in April, Buy-rated Bristol-Myers ( BMY ) and uniQure ( QURE ) announced they entered an agreement under which Bristol-Myers will have access to uniQure’s gene-therapy technology platform in order to develop up to 10 new candidate therapies against targets playing a role in cardiovascular diseases. An additional catalyst is expected in late April with top-line data from Celladon’s ( CLDN ) Phase 2 CUPID 2 study using adeno-associated virus (AAV)-based Mydicar in advanced heart failure. This will follow the positive clinical outcomes reported from the Phase 1 and 2 CUPID 1 study which achieved its safety and efficacy endpoints and also showed long-term benefits in patients treated with single high-dose Mydicar.

We believe these two events could contribute significantly to the gene-therapy space, as they each serve as external and clinical validations for use of the approach, even in the context of a complicated and multifactorial disease such as heart failure. We highlight three gene-therapy companies under our coverage that could benefit from the increased visibility for the space.

Applied Genetic is developing AAV-based gene-therapy candidates focused mostly on ophthalmic indications. We expect the company to “come out of its cocoon” this year as it prepares to initiate two Phase 1 and 2 studies in X-linked retinoschisis (XLRS) and achromatopsia (ACHM) patients during third-quarter 2015 with initial data from both studies expected in the second half of 2015. The company is conducting additional preclinical work assessing routes of administration in the Alpha-1 antitrypsin (AAT) deficiency program. Given the nature of these indications and the approach used to treat them we believe answers could come fast, following a single administration of the AAV-based gene therapy. This is supported by the single-dose clinical benefits achieved in Celladon’s CUPID 1 Phase 1 and 2 study showing long-term effects with a single administration.

GenVec is developing human and non-human adenovirus-based gene therapies with its main asset developed for hearing loss and licensed to Novartis ( NVS ), currently in Phase 1. Part A of this study completed enrollment of three patients. Patients in Part A are treated with a volume of 20 microliter (mcl) and are assessed for safety. Part B, will have two to five cohorts of three patients each, will assess safety with escalating volumes of the vector and determine the volume to be administered in Part C, which will assess efficacy and will include a single cohort of about 20 patients. While Novartis uses discretion with regards to its clinical programs, we believe a safety update from this study (and potential early signs of response or gene expression) is possible later this year at a major gene-therapy medical conference.

Oxford BioMedica uses its lentiviral-based technology platform to: 1) develop gene-therapy products for use in ophthalmic indications internally; and 2) externally through its Sanofi ( SNY ) collaboration; and 3) offer manufacturing services such as its Novartis collaboration supplying the CTL019 CAR-T vector. During 2015 the company expects to present final data from the RetinoStat Phase 1 study. We believe the data will prompt Phase 2 planning and partnering interest. Oxford BioMedica will also present long-term follow-up data (three years) from the ProSavin Phase 2 study in Parkinson’s and in 2016 it expects to initiate a Phase 2 study with an improved version of ProSavin, Oxford BioMedica-102.

We expect continued and increasing interest in the gene-therapy space as a result of ongoing catalysts, as well as interest in individual promising stocks. We believe all three companies represent undervalued and promising value propositions for investors with therapeutic solutions for both rare diseases as well as unmet-medical-need indications

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