BIOFEST INVEST APPLICANT

Qana Therapeutics Inc.

Company Contact:

Legal Entity Type: C-Corp

Company Type: Biotechnology

Company Stage: Development

No. of Employees: 2

Desired Financial Amount: Company is seeking $7mm over the next 12 months to file an IND and initiate a first-in-human study in adrenocortical carcinoma

Background

Company Background

The Lipoprotein Drug Delivery Research Lab of Drs. Raut and Lacko at the University of North Texas Health Science Center (UNTHSC) in partnership with Dr. Alan Remaley at the National Heart Lung Blood Institute (NHLBI) in Bethesda, MD developed a proprietary drug delivery platform (US Patent: 20140045950A1) consisting of a nanoparticle formed from identical subunits of a synthetic amphipathic Apo-A1 mimetic peptide (5A) conjugated to myristic acid (Myr), a saturated fatty acid. Remaley showed that the 5A peptide competently mimics Apo-A1 for the purpose of binding to the HDL receptor, SR-B1 (gene: SCARB1.) Lacko and others have demonstrated the potential utility of targeted drug delivery to SR-B1 expressing cells using this platform. This intellectual property was licensed into a newly formed company, Qana Therapeutics, in 2018. Since that time the company has developed a translatable and scalable production process for this drug delivery platform — which it calls Safina.

Management

Jim Graham, Founder and President. Jim is the Director of Investments at RBP Catalyst Fund, a seed investment fund focused on building successful spinouts on scientific advances from leading mid-continent academic institutions. Jim has twenty years of experience in life sciences finance, commercialization and entrepreneurship. He started his career in the West Coast office of Alex.Brown and worked at Biogen before moving to the Boston office of McKinsey in the Pharmaceutical and Medical Products practice serving global pharmaceutical, biotechnology and medical device clients. Jim moved to Texas in 2009 and has been a principal at Santé Ventures, an early stage life sciences venture capital firm, and an operating partner with a large Texas-based family office. Jim earned a B.S. in Biological Sciences with Honors from Stanford University, an S.M. in Biomedical Sciences from the Harvard-MIT Division of Health Sciences and Technology and an MBA from MIT Sloan. He holds the Chartered Financial Analyst (CFA) designation. Christian Boada, Senior Scientist Dr. Boada Sandoval received a B.A. in biosciences and Ph.D. in biotechnology from Tecnológico de Monterrey. He bolstered this sound academic foundation by carrying out research at Houston Methodist Research Institute and by working as a postdoctoral research associate at Duke University. Dr. Boada Sandoval then held the role of postdoctoral research associate, where he developed lipid nanoparticles for the delivery of RNA in cardiovascular disease. Dr. Boada Sandoval’s research has resulted in 15 peer-reviewed journal articles (3 of them first-authored) in leading journals, including Circulation Research.

Board of directors

Jim Graham (see above.) Laura Rutledge (advisor) Laura is the Founder and Executive Director of the Rutledge Cancer Foundation, a nonprofit organization whose mission is to ease the impact of cancer on the lives of teens and young adults, raise survival rates and find a cure for sarcomas and other solid tumor cancers. RCF funds patient programs that address unmet emotional and physical needs, works as a catalyst to increase awareness and early detection, and supports research for less toxic, personalized cancer therapies. Laura’s drive and ability to problem-solve, bring the right people and resources together and dream big, has been the catalyst for $1,400,000 in grants being awarded for cutting-edge research and over $950,000 in programs and care for teens and young adults with cancer. Dr. Greg Aune (advisor) Dr. Gregory J. Aune is the Greehey Distinguished University Chair for Cancer Survivorship in Children, the Stephanie Edlund Distinguished Professor of Pediatric Cancer Research, and a tenured associate professor of pediatrics at the University of Texas Health Science Center San Antonio. His experience in pediatric cancer spans over 29 years and encompasses his own patient experiences, research in experimental therapeutics, clinical care of pediatric oncology patients and childhood cancer advocacy. His interest in pediatric oncology began at age 16 when he was diagnosed with Hodgkin’s lymphoma. While fortunate to survive, the experiences he encountered as a patient initiated a path towards a research and clinical career aimed at developing less toxic chemotherapy regimens. His experience as a long-term survivor included open-heart surgery at age 35 to replace his aortic valve and bypass three blocked coronary arteries that were damaged by his teenage cancer therapies. This life-changing event initiated his research interest in cardiac disease. Greg’s training to become a successful physician scientist and pediatric oncologist has included time spent at some of the most well-respected oncology institutions in the United States, including M.D. Anderson Cancer Center, the National Cancer Institute and Johns Hopkins Hospital. In the laboratory setting, Dr. Aune has a wide range of experience in the tumor biology and experimental therapeutics. Most recently, he has applied this knowledge to both the in vitro and in vivo studies of the cardiovascular system from the level of the cardiac fibroblast to global cardiac function. His laboratory has successfully developed a novel pediatric mouse model of anthracycline-induced cardiac toxicity.

Product / Service

disease area / application

Cancer, Adrenocortical carcinoma

product / Service

Adrenocortical carcinoma (ACC) is a rare endocrine malignancy with a poor prognosis. The majority of ACC patients have metastasis at the time of diagnosis, resulting in a 5-year survival rate of less than 35%. Complete surgical resection remains the standard of care today. Other commonly used pharmacological interventions include the adrenotoxic drug mitotane (o,p′-DDD), which is administered either alone or in combination with other cytotoxic chemotherapy, such as etoposide, doxorubicin, and platinum agents. Because this method of treatment has a relatively low response rate and carries significant systemic toxicity, better treatment methods are critically needed for more effective targeting and inhibition of ACC. An ACC cell line (H295R) from a patient presenting with elevated plasma steroid hormones shows a high level of SR-B1 expression and takes up HDL mimetic cargo both in vitro and in vivo. SR-B1 (SCARB1) is a scavenger receptor typically found on cell surface of adrenal, liver and gonadal tissues. Our platform selectively delivers drug payloads to cells that express SR-B1, making ACC a good first target. In addition, elevated SR-B1 expression has been reported or is suspected in numerous cancer types, including: bile duct cancer, bladder cancer, breast cancer, cervical cancer, colorectal cancer, esophageal cancer, gastrointestinal cancer, liver cancer, nasopharyngeal cancer, ovarian cancer, pancreatic cancer, prostate cancer, adrenocortical carcinoma, lung adenocarcinoma, renal cell carcinoma, leukemia, lymphoma, neuroblastoma, and sarcoma. We plan to systematically investigate these tumors through in vivo and in vitro studies to determine which of these tumor types might also be amenable to an SR-B1 targeted approach. For ACC and subsquent cancer indications, we beleive our solution will provide benefit to patients by concentrating drug payloads to cells expressing the SR-B1 receptor while avoiding sensitive tissues in the heart, brain and other organs.

technology / ip

This novel strategy employs an Apo-A1 mimetic peptide (5A), conjugated to a lipid molecule (myristic acid) that self-assembles into micellar nanoparticle structures (Myr5A NP). These Myr5A NPs can encapsulate a variety of therapeutic payloads and deliver them to cancer cells and tumors via the cell surface receptor SR-B1. Key benefits of this approach include: (1) Targeted delivery preferentially to cell types that express the scavenger receptor class B type 1 (SR-B1) cell surface receptor. (2) Non-endosomal SR-B1 mediated transport of nanoparticle payload into the cytoplasm of the cell. (3) Favorable pharmacokinetic attributes, including long circulating half-life and the ability to avoid mononuclear phagocytic system (MPS) and (4) Potential for scalable and cost-effective manufacturing, given the self-assembling, simple nature (single excipient) of the nanoparticle vehicle component. This NP system mimics the structure and function of plasma high-density lipoproteins (HDL). Previous studies have shown that the addition of empty HDL mimetic NP may be a promising vehicle for drug delivery.

distribution channels

We will most likely seek to license or partner our lead formulation with a partner prior to marketing. However, in general, an ACC therapeutic would be distributed through the existing specialty pharma / IV infused supply chain. We would work with medical payers to negotiate pricing based on the patient benefit delivered.

market size

Adrenocortical carcinoma is an ultra-orphan pediatric oncology indication with fewer than 1,000 cases per year. With industry-standard share and pricing assumptions, we estimate a reasonable market for ACC to be $250mm per year. This indication case load is growing in line with general population growth. If we are able to establish the clinical utility of SR-B1 targeting in ACC, we believe that we will be able to branch into other indications, such as: – Triple negative breast cancer: $1.3B – Clear cell renal cell carcinoma: $800mm – Ovarian cancer: $4.0B

competition

Corcept Therapeutics is testing Relacorilant, a small molecule antagonist of the glucocorticoid receptor (GR), in ACC patients who produce excess cortisol. This treatment is in combination with pembrolizumab, a PD-1 inhibitor. This program employs a different mechanism from ours and is unlikely to have single-agent efficacy. No other clinical programs are explicitly targeting SR-B1 as a delivery pathway for cancer. EVOQ Therapeutics is using an HDL-mimetic delivery vehicle, but they are focused on presenting tolerogenic motifs to the immune system for diseases like multiple sclerosis and rheumatoid arthritis. EVOQ has inked partnerships with Amgen and Gilead worth over $800mm, including future milestones.

Financials

Desired financial amount

Company is seeking $7mm over the next 12 months to file an IND and initiate a first-in-human study in adrenocortical carcinoma

previous funding

The company has raised $600K to date from friends and family through a series of convertible notes. The company has received one research grant of just under $60,000 to further development.

current financials

The company’s available cash as of Jan 2023 is slightly more than $200,000. The company’s core burn rate is $14,000 per month for payroll, rent, and reagents and supplies. In addition to that, the company is paying for new patent drafting and prosecution. We also have bids for contract manufacturing services pending future funding.

financial use

(1) CMC to develop and produce cGMP material for IND-enabling work and FIH clinical study (2) Conduct preclinical testing required for IND, including: toxicity, PK/PD, dose finding and reprotox. (3) Initiate FIH clinical study with a small number of leading US insitutions focused on ACC

revenue

We do not have any product sales to date. We plan to work with traditional medical payers to negotiate pricing for this drug. The revenue model is a product sales model

exit strategy

We would plan to exit the company at the conclusion of Phase 2B for the lead program. At this stage, the core technology will have been de-risked. A larger partner with a developed sales force would likely lead Phase 3 development and commercial launch. If a trade sale is not available at a reasonable valuation, the company could theoretically market a drug in an ultra-orphan indication such as ACC. As an ultra-orphan oncology indication, ACC is eligible for Priority Review Vouchers (PRVs) to the sponsor for novel agent approvals. These PRVs are valued at roughly $100mm, presenting another possible route to revenue generation.

Pitch Video

Login

Welcome back! Login below