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Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company specializing in developing cell and gene therapies for life-threatening diseases. The company focuses on advancing therapies for rare genetic disorders and ophthalmic diseases with high unmet medical needs. Abeona's offerings include autologous cell-based gene therapies and adeno-associated virus (AAV)-based gene therapies utilizing proprietary AIM™ capsid technology.
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Autologous Cell-Based Gene Therapy - Develops prademagene zamikeracel (pz-cel) for recessive dystrophic epidermolysis bullosa (RDEB), a rare genetic skin disorder. Pz-cel has received regulatory designations from the FDA and EMA.
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AAV-Based Gene Therapy - Designs therapies for ophthalmic diseases using AIM™ capsids, which improve tropism profiles for treating devastating diseases. AIM™ capsids are licensed from the University of North Carolina and developed internally.
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Gene Therapy Manufacturing - Operates commercial-scale manufacturing facilities to produce cell and gene therapies, supporting clinical and potential commercial needs.
- Given the discussion around the $1.5 million pricing floor and exploring upside pricing for pz-cel, how does management plan to balance capturing value with ensuring payer comfort in a competitive gene therapy landscape?
- With current manufacturing capacity at 10 patient runs per month and expected demand exceeding supply, what specific steps and timeline does management foresee for scaling up production while mitigating potential hiring and engineering challenges?
- Since the resubmission primarily focused on additional CMC data—specifically around identity testing and Stargardt validations—what contingency plans does management have if the FDA raises new regulatory concerns or requests additional data during review?
- Considering the initial launch strategy of onboarding 5 EB centers across the U.S., how will management ensure rapid and consistent multi-disciplinary onboarding to avoid delays in patient treatment, especially given the complexity of coordinating across different specialties?
- With the favorable Medicare DRG 018 assignment and product-specific ICD-10-PCS coding in place, how does management intend to translate these benefits into negotiated pricing with commercial payers, particularly when maintaining a flat price for initial and subsequent treatments?
Notable M&A activity and strategic investments in the past 3 years.
| Company | Year | Details |
|---|---|---|
Abeona Therapeutics LLC | 2015 | Completed on May 15, 2015, the acquisition involved a merger where PlasmaTech issued 3,979,761 common shares (valued at approximately $31.76M) plus up to $9M in contingent milestones, totaling around $38.25M; this deal strategically provided access to gene therapy assets, including lead programs for Sanfilippo syndrome, aligning with PlasmaTech’s ambition to lead in cell and gene therapies for rare diseases. |
Recent press releases and 8-K filings for ABEO.
- FDA Approval: The FDA has approved ZEVASKYN (prademagene zamikeracel), the first autologous cell-based gene therapy for treating wounds in both adult and pediatric patients with RDEB.
- Clinical Evidence: Approval is supported by the pivotal Phase III VIITAL study and earlier clinical studies demonstrating significant wound healing and pain reduction after a single surgical application.
- Commercial Launch: ZEVASKYN is set to launch in Q3 2025 via Qualified Treatment Centers, with initial manufacturing capacity starting at approximately 4 treatments per month, expected to ramp up to 6 and eventually reach 10 monthly treatments.
- Patient Support & Market Access: Implementation of outcomes-based agreements and the launch of dedicated patient support platforms including ZEVASKYN.com and Abeona Assist will facilitate market access and address reimbursement challenges.
- FDA Review & Launch Update: Pending decision for pz-cel in RDEB with a PDUFA target date of April 29, 2025 and plans to begin patient treatments in Q3 2025 .
- Financial Performance: Full year 2024 results show a net loss of $63.7 million with cash resources of $98.1 million as of December 31, 2024, funding operations into 2026 .
- Manufacturing Readiness: The Cleveland cGMP facility is being prepped to support 4 treatments per month, with plans to ramp up to 10 monthly treatments by early 2026 .
- Commercialization Strategy: Submission preparations include onboarding treatment centers, payer engagement, and expansion of manufacturing capacity to support future commercialization efforts .
- Market Opportunity: Targeting approximately 750 RDEB patients via onboarding 5 treatment centers, creating around 1,500 treatment opportunities with a potential cumulative revenue of over $2 billion in the U.S. .