Carmell Therapeutics has had substantial success advancing the lead program through the FDA regulatory process, which includes a Chemical, Manufacturing and Controls (CMC) section and a proposed pivotal clinical trial protocol. There are two major steps to the manufacturing of BHA: Drug Substance (aka “active ingredient”) and Drug Product (aka “finished product”). Both have been reviewed and cleared for a pivotal (late-stage) clinical study, HEAL II, by FDA as part of IND 18789. Also, FDA granted the Company an expedited review “Fast Track” - Fast track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The process is designed to expedite the development and review of drugs which may demonstrate substantial improvement over available therapy.
In Europe, the European Medicines Agency (EMA) has designated BHA as a "Class III" medical device. The company plans to pursue a CE mark with an anticipated label as a bone void filler. Our plans are subject to discussion with the EMA.
We are currently focused on accelerated bone healing in severe open tibia fractures and other applications where there are need for healing bone fractures, bone defects or need for bone fusion. The product formulation for these applications is a combination product containing the Company’s proprietary PBM technology and β-TCP. Leveraging the resources of investors and strategic vendors, we have successfully surpassed multiple critical milestones:
- FDA Investigation New Drug ("IND") submission, for the lead product candidate, BHA, in severe open tibia fractures.
- FDA has agreed that we may begin clinical trials under the IND.
- FDA granted expedited review, or Fast Track, for the BHA program, indicating FDA alignment that BHA has the potential to address a significant unmet need.
- Production of three ICH batches (The International Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use) of BHA.
- ISO 13485 Certification (International Organization for Standardization, represents the requirements for a comprehensive quality management system for the design and manufacture of medical devices.)
- Successful Rodent ISO Safety Studies.
- Successful completion of First-In-Human (“FIH”) Multi-Center Randomized, Prospective Clinical Trial supporting our belief in the ability of BHA to achieve healing at an accelerated time point of six months on an injury that typically may take greater than nine months to fully heal.
- Three pre-clinical publications supporting the potential therapeutic benefit of PBM in multiple applications including infection prevention and skin healing and re-epithelialization after radiation burns.
- European Regulatory Authorities designated BHA as Class III medical device
While the platform technology of PBM is initially targeted to accelerate and enhance healing in bone applications, in keeping with our exclusive license agreement, we believe PBM has the ability to deliver many regenerative factors, including the important GFs directly to the area in need of enhanced tissue healing. Promising results in both animal testing and human clinical studies position the core technology for potential use in a variety of therapeutic areas.