dm+d

Unassigned

New Medicines

Recessive dystrophic epidermolysis bullosa (RDEB)

Information

New molecular entity
Abeona Therapeutics
Abeona Therapeutics

Development and Regulatory status

None
None
Phase III Clinical Trials
Yes
Yes
Jul 21Top-line results from VIITAL study in mid-2022, and company will follow with a BLA filing in the US. No EU/UK plans described [6].
Nov 20According to Abeona Q2 and Q3 2020 reports, process development at their GMP manufacturing facility in Cleveland, Ohio is ongoing to enable production of the retrovirus used for EB-101 manufactured at the facility, allowing for increased control of the supply chain and product quality, as well as reduced costs. Abeona is targeting potential commercial launch of EB-101 in late-2022 [5].
Nov 20EB101 has orphan drug status in the US and EU, plus rare pediatric disease, breakthrough therapy and regenerative medicine advanced therapy (RMAT) status in the US [3].

Category

An autologous, ex-vivo cell therapy, involving creation of a graft, using the patients keratinocytes, which are genetically engineered through the insertion of correct copies of the COL7A1 gene to express the missing type VII collagen protein
Population studies are complicated by the fact that this is not a uniform disease but a wide number of diseases of varying severity. It is likely that many patients with the more mild forms of EB remain undiagnosed. It is thought to affect 1 in 17,000 births. Around 5,000 people are affected in the UK [1]. RDEB is caused by mutations in the COL7A1 gene, which disrupt the production of type VII collagen and impairs its ability to help connect the epidermis to the dermis [2].
Recessive dystrophic epidermolysis bullosa (RDEB)
Topical

Trial or other data

Aug 20A second patient was treated in the PIII VIITAL study after enrolment resumed in June following a pause due to the COVID-19 pandemic. Additional patients are expected to be treated in the coming weeks, with completion of enrolment in the VIITAL study expected in early-2021 [5].
Jan 20PIII VIITAL trial to evaluate safety and efficacy of surgical application of EB-101 (autologous, gene-corrected keratinocyte sheets) as a treatment of RDEB starts (NCT04227106). The FDA had temporarily issued a clinical hold between Sep and Dec 2019. As per the letter, the FDA did not approve the planned VIITAL trial, until the company submits additional data points on transport stability of EB101 to clinical sites. The primary endpoint is the proportion of wounds with greater than 50% healing at three months, comparing treated with untreated wound sites on the same patient. Secondary endpoints include patient global impression of change from baseline in pain as well as other patient reported outcomes assessing pain during dressing changes, pain impact and physical function. The open-label, controlled study intends to enrol 15 patients aged six years and older in the US (no UK sites). Collection of primary outcome data is expected to complete Sep 20 [2,4].
Oct 19Positive long term efficacy and safety data from the PI/II trial (NCT01263379) was released by the Abeona Therapeutics. The data showed that EB 101 was associated with long-term molecular expression of type VII collagen protein, which plays an important role in anchoring the dermal and epidermal layers of the skin [2].
Jan 18Abeola announces results from the PI/II trial (NCT01263379). The trial met the primary endpoints for safety and efficacy, where wound healing after EB-101 administration was compared to control untreated wounds from a supporting natural history study that evaluated 128 patients and approximately 1500 chronic and recurring RDEB wounds. Secondary endpoints included expression of collagen C7 and restoration of anchoring fibrils at three and six-months post-administration. Clinical data demonstrated that EB-101 treated wounds were significantly healed >50% for more than two years post-administration. The data included wound healing, defined as >50% closure after EB-101 administration, was observed in 100% (42/42 treated wounds, n=7 subjects) at 3 months; 90% (38/42 treated wounds, n=7 subjects) at 6 months; 83% (20/24 treated wounds, n=4 subjects) at 12 months; 88% (21/24 treated wounds, n=4 subjects) at 24 months; 100% (6/6 treated wounds, n=1 subject) at 36 months post-administration. Collagen VII (C7) expression: C7 and morphologically normal NC2 reactive anchoring fibrils were observed as early as 1 month in EB-101 treated wounds and have remained for at least two years post-administration. Importantly, data from a supportive natural history study of approximately 1,500 wounds from 128 patients with RDEB, established by Stanford and EBCare Registry, were also presented to the FDA. Notably, 13 RDEB patients with a total of 15 chronic wounds were treated with an allograft product, including Apligraf® and Dermagraft®. Of these wounds treated with allografts, only 7% (1/15 treated wounds) remained healed after 12 weeks, and 0% (0/15 treated wounds) remained healed after 24 weeks [2].
Oct 10PI/II trial to evaluate the safety and efficacy of EB 101, determining the effect of type VII collagen gene-corrected grafts on wound healing efficacy in adolescent (≥ aged 13 years) patients with RDEB starts (NCT01263379). Ten patients will be enrolled in the US (none UK). Primary outcomes are percentage surface area of wound healing and investigator assessment of graft [Time Frame: 3, 6 and 12 months post grafting]; collection of these data should complete Dec 23 [4].