On April 26, 2024 Biodexa Pharmaceuticals PLC, (Nasdaq: BDRX), an acquisition-focused clinical stage biopharmaceutical company developing a pipeline of innovative products for the treatment of diseases with unmet medical needs, reported it entered into a definitive agreement with Rapamycin Holdings Inc. (d/b/a Emtora Biosciences) ("Emtora") for the rights to eRapa under an exclusive, worldwide license (with the ability to grant sublicenses) to develop, manufacture, commercialize and otherwise advance the clinical potential of eRapa.

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Stephen Stamp, CEO and CFO of Biodexa said, "Acquiring a Phase 3 ready asset, particularly one supported by $17 million of non-dilutive grant funding, significantly advances Biodexa’s oncology pipeline and adds numerous valuation catalysts for our stakeholders. We are delighted to be working with the Emtora team which has excelled in bringing eRapa close to the end of Phase 2 in Non-muscle Invasive Bladder Cancer and to the beginning of a Phase 3 trial in FAP, a devastating disease for which there is currently no approved pharmacological agent for altering its progression. Left untreated, it almost always leads to incredibly invasive surgery and a major deterioration in the quality of life."

Stephen Dufilho, Executive Chairman of Emtora added, "The transaction with Biodexa is the culmination of a decade-long effort to advance our potentially game-changing eRapa to a registrational Phase 3 trial – and ultimately to patients in need. Our story began in San Antonio – where eRapa was originally invented at the University of Texas and funded in part by grants from the Cancer Prevention Research Institute of Texas. We thank the scientists, clinicians, investors and biotech executives that supported our efforts to reach this important milestone. We look forward to working with the Biodexa team as we embark upon this next chapter."

About eRapa
eRapa is a proprietary oral tablet formulation of rapamycin, also known as sirolimus. Rapamycin is an mTOR (mammalian Target Of Rapamycin) inhibitor. mTOR has been shown to have a significant role in the signalling pathway that regulates cellular metabolism, growth and proliferation and is activated during tumorgenesis1. Rapamycin is approved in the US for organ rejection in renal transplantation as Rapamune(Pfizer). Through the use of nanotechnology and pH sensitive polymers, eRapa is designed to address the poor bioavailability, variable pharmacokinetics and toxicity generally associated with the currently available forms of rapamycin. eRapa is protected by a number of issued patents which extend through 2035, with other pending applications potentially providing further protection beyond 2035.

eRapa in FAP
FAP is characterized as a proliferation of polyps in the colon and/or rectum, usually occurring in mid-teens. There is no approved therapeutic option for treating FAP patients, for whom active surveillance and surgical resection of the colon and/or rectum remain the standard of care. If untreated, FAP typically leads to cancer of the colon and/or rectum. There is a significant hereditary component to FAP with a reported incidence of one in 5,000 to 10,000 in the US2 and one in 11,300 to 37,600 in Europe3. eRapa has received Orphan Designation in the US with plans to seek such designation in Europe. Importantly, mTOR has been shown to be over-expressed in FAP polyps – thereby underscoring the rationale for using a potent and safe mTOR inhibitor like eRapa to treat FAP.

Emtora is currently completing an open-label, multi-center Phase 2 study in 30 patients with confirmed FAP with the primary endpoints of safety and tolerability, and percentage change in polyp burden after six months of treatment with eRapa. The Phase 2 study was partially funded by a $3.0 million grant from CPRIT.

The results of the Phase 2 study will be presented at two leading scientific conferences in Q2 24. Following a positive end of Phase 2 meeting with the FDA and, Emtora plans to initiate a Phase 3 multi-center, double-blind, placebo-controlled study in FAP. The Phase 3 study, which is expected to be registrational, plans to recruit approximately 140 patients across thirty or more sites, with a primary endpoint being time to a progression free survival event (the occurrence of which is related to the reduction in polyp burden studied in the earlier Phase 2 trial). The study is expected to recruit over 15 months and is supported by a further non-dilutive grant of $17.0 million from CPRIT.

eRapa in Non-Muscle Invasive Bladder Cancer
Non-muscle Invasive Bladder Cancer ("NMIBC") refers to tumors found in the tissue that lines the inner surface of the bladder. The most common treatment is transurethral resection of the bladder tumor followed by intravesical Bacillus Calmette-Guerin ("BCG") with chemotherapy depending upon assessment of risk of recurrence. NMIBC is the fourth most common cancer in men with an incidence of 10.1 per 100,000 and 2.5 per 100,000 in women4.

Emtora’s ongoing multi-center, double-blind, placebo-controlled Phase 2 study in NMIBC is expected to enroll up to 166 patients with primary endpoints of safety/tolerability and relapse free survival after 12 months of treatment. The Phase 2 study, which is supported by a $3.0 million non-dilutive grant from the National Cancer Institute, part of the National Institutes of Health, is expected to read out in Q2 ’25.

Other Potential Indications for eRapa
A number of rare/orphan gastro-intestinal diseases (other than FAP) have been identified that share the strong scientific rationale (relating to mTOR inhibition) and support the potential utility of eRapa in FAP. Biodexa intends to evaluate such opportunities over the coming months, including by leveraging low-cost, investigator-sponsored trials to produce initial proof of concept data.

The License Transaction
The transaction terms include the issuance to Emtora of 378,163 of the Company’s American Depository Shares (representing 5% of the Company’s issued and outstanding ordinary shares on a fully-diluted basis (including in-the-money warrants)) at close. In addition, the Company may pay up to $41.5 million in sales milestones within the first six months of the first commercial sale of a first-approved indication of eRapa in major markets, with decreasing milestones for subsequent approvals for additional indications. The Company is also obligated to pay single digit tiered royalties on net sales of eRapa in addition to meeting Emtora’s legacy royalty obligations and paying Emtora a certain (stage-dependent) percentage of income derived from sublicensing and partnering of eRapa.

Ladenburg Thalmann & Co. Inc. acted as financial advisor to BioDexa in connection with this transaction.

The Cancer Prevention and Research Institute of Texas
To date, CPRIT has awarded $2.9 billion in grants to Texas research institutions and organizations through its academic research, prevention and product development research programs. CPRIT has recruited 237 distinguished researchers, supported the establishment, expansion or relocation of 43 companies to Texas and generated over $5.7 billion in additional public and private investment. CPRIT funding has advanced scientific and clinical knowledge and provided 7.4 million life-saving cancer prevention and early detection services reaching Texans from all 254 counties. On November 5, 2019, Texas voters overwhelmingly approved a constitutional amendment to provide an additional $3 billion to CPRIT for a total $6 billion investment in cancer research and prevention. Learn more at View Source

(Press release, Biodexa Pharmaceuticals, APR 26, 2024, View Source [SID1234673477])