• CENNA Biosciences, Inc. is a privately-held biopharmaceutical company in La Jolla CA, focused on the discovery and development of novel drugs for the prevention and treatment of Alzheimer’s disease
• Company was founded in 2006 to translate over a decade’s academic research at UC San Diego by the Founders, Nazneen Dewji, Ph.D. and S. J. Singer, Ph.D. Founders became independent of UC San Diego in 2011
• Disease-modifying therapies for Alzheimer’s disease are urgently needed. There is a huge and growing market and patient population worldwide for Alzheimer’s disease and there is no disease-modifying drug available. No existing technologies under development have been shown to delay disease progression or provide significant patient benefit
• CENNA has a novel and different proprietary patented technology that arrests the underlying cause of the disease and that addresses previous failures by others
• Preclinical studies in progress with IND filing planned for 2018. Funding to date by Founders and $16M in NIH and other grants
• Seeking a $5M investment to complete studies on drug candidate P8 to IND and Phase 1 Clinical trials
Mission: To Develop Novel Therapeutics for Alzheimer’s Disease
Alzheimer’s disease (AD) is a progressive neurodegenerative disorder that affects one in ten people over the age of 65, one in two by age 90. It is the most common form of dementia and results in memory loss and a decline in mental function over time, eventually resulting in death. CENNA’s proprietary technology addresses the underlying cause of the disease, the inappropriate deposition of the toxic species, Aß, in the brains of patients with AD. The Company has developed a novel druggable target and has discovered several lead compounds that are active in vivo and in AD patient-derived induced stem cells. The intellectual property that forms the basis of CENNA’s approach is covered by seven issued U.S. Patents and seven issued foreign patents in Australia, China, select European countries, Japan, Macao and Korea. CENNA has additional pending patent applications in the US, Canada, Hong Kong and India. CENNA’s research to date has been funded by non-diluting NIH, other grants and by the Founders.
Markets: The AD market is very large and is growing rapidly. Patients with AD live for about eight years after diagnosis, although the disease can last up to 20 years. The incidence of AD grows exponentially with age, doubling every five to six years. AD remains a challenge in management, with nearly eight million sufferers in the seven major markets of the world. No disease-modifying drug is currently available that can stop the progression of the disease. Current therapies include two classes of drugs that offer mild symptomatic relief for a very short time. Thus, at present, therapeutic options are limited, and at best provide modest symptomatic relief for a short period of time with marginal cost-benefit. Disease modifying approaches to the treatment of AD, which aim to reduce the rate of disease progression, with prevention and/or reversal of pathology, are critically needed.
The current market for symptomatic AD therapeutics is now estimated at $5.8 billion per year and the introduction of disease-modifying drugs to the market will cause it to more than double to $14.5 billion by 2020 (Decision Resources, 2011). CENNA aims to be one of the first companies to develop disease-modifying drugs to arrest or slow disease progression for AD sufferers. Such a contribution may increase the market size.
The Science: The basic pathology of the disease includes the production and accumulation of Aß. CENNA’s candidate drugs reduce the production of Aß. They are small peptides derived from Presenilin-1 which is part of the gamma-secretase complex, or small molecules that function by the same mechanism as the peptides. CENNA’s drug candidates inhibit the production of Aß by specifically binding APP at a different site from the ß- and gamma-secretase cleavage sites and arresting its processing to Aß (Dewji, N.N. et al (2015) PLoS ONE 10(4): e0122451). Importantly, these peptide or small molecule-induced reductions of total Aß and Aß40 and 42 do not target, modify or inhibit either ß- or gamma-secretase activities. Our lead peptide and small molecule candidates, inhibit the production of Aß in vitro, in a Tg mouse model of AD and in patient-derived stem cells by over 50%, are stable, and can be delivered to the brain.
Reasons for Previous Failures by Others: All phase III drug development programs to date in the Aß space have failed to meet their clinical endpoints. There are reasons for these failures. Most such studies directly targeted the catalytic activities of the enzymes that cleave Aß from its precursor, APP. These enzymes, ß- and gamma-secretase, are known to hydrolyze as many as 50-60 other substrates besides APP, many with critical cellular functions. Thus, while inhibiting or modulating the activities of these enzymes successfully reduced Aß, they also inhibited as many as 60 other reactions in the cell, producing many detrimental off-target effects. CENNA’s approach is completely different and does not target the secretases. Another reason for failure is that these drug development programs were administered too late in the disease progression, when the damage to the brain had already occurred. New therapeutic approaches that can inhibit total Aß production early without targeting the activities of the ß- or the gamma-secretase are therefore urgently needed. CENNA’s technology does just that.
Capital Efficient Business Model: CENNA Biosciences, Inc. plans to build value in its technology platform towards a license or exit through strategic partnerships with leading pharmaceutical companies. CENNA’s novel technology has yielded several peptide and small molecule drug candidates with the ability to inhibit the production of Aß in vitro, in a transgenic mouse model of AD and in AD patient-derived induced stem cells. These are at various stages of pre-clinical development.
Summary of transformative impact of CENNA’s Technology: CENNA has a novel technology that does not target the secretases, which has yielded several peptide and small molecule drug candidates with the ability to inhibit the production of Aß in vitro, a transgenic mouse model of AD in AD patient-derived induced stem cells. Importantly, these peptide-induced reductions of total Aß and Aß40 and 42, do not modify or inhibit either ß- or gamma-secretase activities. CENNA’s technology is also the earliest in terms of intervention, as it stops the Aß from being produced, as opposed to dealing with the effects of Aß once it has accumulated. CENNA’s technology would therefore stop the disease in its tracks.
It is anticipated that CENNA’s therapeutics will have a significant impact in both treatment and prevention modes and be useful throughout the course of disease. An effective disease-modifying therapeutic that slows progression or prevents AD will have significant commercial and societal benefits related to the quality of life for an aging population. It is estimated that the revenue potential for disease-modifying agents in Alzheimer’s disease is >$15 billion per year, worldwide.
CENNA’s drug is also expected to have significant societal benefits. Safe and effective disease-modifying therapeutics will reduce the burden of AD on caregivers and the level of financial resources required to care for these patients. Patients diagnosed with mild cognitive impairment (MCI) or AD will have a treatment, which will slow or halt the course of their disease. By the year 2025, when the post-war baby-boomers reach 65 years of age, it is estimated that the number of people suffering from AD will double, with further increases to 14 million by 2050. A new drug, which merely delays the onset of AD for five years between 2010 and 2050, would yield a societal benefit of $4 trillion per year.
Milestones, Funding and Exit: Key milestones over the next 12 months are to complete the pre-clinical development of lead candidate P8 and prepare a preclinical scientific package that will include the necessary experimentation in animals to satisfy the preclinical requirements for filing an IND. Clinical safety and efficacy will be established up to the filing of a Phase IIA NDA with the FDA. Further clinical testing, product license manufacturing, distribution and sales will be accomplished by means of a joint venture with an appropriate industry partner. CENNA is seeking a Pre-Series A round of $5M to fund operations for the next 12-24 months in order to advance its development programs, hire new staff, and continue commercialization activities. The primary activities are to develop CENNA’s existing technology to complete the pre-clinical development of P8 and to file an IND. CENNA can offer an exclusive license to its technology with a commitment to advance the science through the right partnership.
The Exit Strategy is for CENNA to be acquired after successful completion of Clinical Phase IIA. It is anticipated that a combination of joint ventures, out-licensing and government funding in the form of non- diluting NIH SBIR grants will provide the necessary finances for CENNA until it can be acquired by a strategic partner at the end of clinical Phase IIA.
Summary: Why is this a good investment?
• Disruptive, novel approach
• Technology platform (multiple shots on goal)
• Data in transgenic animals and patient-derived stem cells
• An investment of $5M could lead to a mega-million dollar strategic deal and a high-level return to investors