16 May Cortexyme: Addressing Unmet Needs in the Alzheimer’s Market
The stocks of Cortexyme, Inc (NASDAQ: CRTX), a clinical stage biopharmaceutical company engaged in developing novel therapeutics for Alzheimer’ disease, surged by almost 70% on its debut on the stock market on May 9. Its market value jumped to $750 million by end of trading breaking a spate of poor debut sessions by its peers such as Axcella Health (NASDAQ: AXLA) and Trevi Therpaeutics (NASDAQ: TRVI)
The company priced its IPO of over 4.4 million shares at $17 per share, granting underwriters a 30-day option to purchase up to an additional 661,800 shares at the initial public offering price, the IPO closes on May 13, 2019, subject to conditions.
Addressing Unmet Needs in the Alzheimer’s Market
Alzheimer’s is the sixth leading cause of death in the U.S, a disease that causes irreversible degeneration of the brain cells and affects a person’s cognitive and behavioural abilities such as impaired judgment, deteriorating memory and personality changes with the gradual loss of motor abilities and speech. It is primarily caused by accumulation of two types of proteins called amyloid and tau in the brain.
According to some compelling statistics made available by Alzheimer’s Association over 5.8 million Americans are affected by this disorder and the numbers will most likely reach 14 million by 2050, with a new diagnosis being made every 65 seconds. The healthcare cost of alzheimer’s and other dementia related ailments will be over $290 billion in 2019, these numbers are likely to escalate further to over $1.1 trillion by 2050.
A report by transparency market research predicts that the global market for alzheimer’s will reach US$6.4 billion by 2025, growing at a CAGR of 7.5%, from US$3.6 billion in 2017. The rise in ageing population, increased awareness about the benefits of combined therapy, a favorable regulatory and government support environment are some of the factors that will contribute to the growth of the market. The economic burden of the disease on not only the patients but also caregivers will act as an impediment for the market. The prohibitive cost of expensive treatment especially in developing nations is another obstacle for growth.
Alzheimer’s is one illness, where innumerable attempts that have been made to find a cure with no success. According to a report released by Pharmaceutical Research and Manufacturers of America (PhRMA) from 1998 to 2017 there have been about 146 failed shots at developing drugs for alzheimer’s disease. However, recent advancements in technology and the discovery of new molecules and combination therapies are offering renewed hope to those suffering from this illness.
Limitations of Conventional Alzheimer’s Treatment
The current treatment for alzheimer’s is primarily focused on symptomatic relief, and does not alter the state of the disease as such. The biggest challenge however, is that most of the drugs cannot cross the blood brain barrier, resulting in only a very low dosage of the actual administered drug to reaching the intended target.
Research in the field has led to the development of options such as intranasal, intravitreal administration and use of polymeric carriers based nanotechnology. However, the treatment is currently limited to two classes of drugs known as “cognitive enhancers”. The first are inhibitors of the enzyme acetylcholinesterase, which are available in generic form and not very expensive, but have several side-effects such as insomnia, increased risk for bleeding, and disrupted heart rhythm.
Memantine is the other class of drugs that enhance the brain’s sensitivity to amino acid neurotransmitter glutamate, which helps in memory formation. It is also responsible for the apoptosis, or programmed cell death. They are used in combination with a cholinesterase inhibitor and have minimal side-effects such as agitation, confusion, headaches and constipation.
However, both of them are severely limited in their ability to provide a significant improvement in the cognitive functioning of those suffering from the disease.
The company was founded on the basis of the work by Dr.Stephen Dominy, its co-founder and chief scientific officer, who discovered pathogenic bacteria in the brain of those afflicted with alzheimer’s. This led to the development of the company’s small molecule program aimed at preventing the progression of the disease.
It initiated a Phase 2/3 clinical trial named (GingipAIN Inhibitor for Treatment of alzheimer’s disease) “GAIN” in April 2019, to test the efficacy of its new investigational medicine COR388, targeting P. gingivalis bacteria. The company seeks to enroll at least 500 participants in the U.S for the program and is currently recruiting patients for the study. COR388 is a first-in-class, orally administered virulence factor inhibitor that targets P. gingivalis gingipains.
The study is based on scientific evidence that porphyromonas gingivalis, or P. gingivalis, hitherto associated with chronic periodontal disease, can cause alzheimer’s. COR388 has the potential to slow the progression of AD by destroying the toxic proteins, or gingipains, released by P. gingivalis. It is the first-of-its kind trial that is testing a new paradigm in the treatment of alzheimer’s, as till date amyloid plaques were the center of any study associated with creating a solution for alzheimer’s. It enjoys composition of matter patent protection until 2035, with more patents pending for approval.
In pre-clinical studies, COR388 has demonstrated the ability to reduce the bacterial load of a P. gingivalis infection, block the production of amyloid beta, reduce neuroinflammation, and protect neurons in the hippocampus, a part of the brain that mediates memory.
The company enjoys the backing of prominent venture capital firms such as Sequoia (NASDAQ: SEQUX), Vulcan capital, Verily, Epic and Breakout ventures to name a few along with big pharmaceutical players such as Pfizer (NYSE: PEE) and Takeda (NYSE: TAK)
It has cash, cash equivalents & short term investments: $59M (excludes $5.7M in long term investments) as per its Q1, 2019 financial results.
We believe that the company is poised to make great strides in the alzheimer’s treatment market; by using a novel approach to treating the disease and breaking away from amyliod plaques based trials, which haven’t been too successful in creating any significant therapeutic alternatives in several years. This is one therapeutic area that has seen repeated failures of clinical trials, with recent disappointments including Biogen, Eisai and Roche stopping their clinical trials mid-ways.
While the outcomes of clinical trials are mostly unpredictable, the approach adopted by Cortexyme and its preclinical data, shows the potential of achieving a breakthrough in the field of alzheimer’s therapeutics. The positive reception to its IPO and its successful debut on the stock market only prove to reinforce our confidence in the company’s potential.
Cortexyme, a clinical stage pharmaceutical company, is adopting a path breaking, novel disease-modifying therapeutic approach for treating the underlying cause of alzheimer’s disease and other degenerative diseases. Its lead candidate COR388 is a first-in-class, orally administered virulence factor inhibitor that targets P. gingivalis gingipains, a specific, infectious pathogen found in the brain of alzheimer’s patients and known to cause neurodegeneration and neuroinflammation in animal models. The company’s lead investigational medicine, COR388, is currently undergoing a phase 2/3 GAIN Trial in patients with mild to moderate Alzheimer’s.
The company which was founded in 2012 is headquartered at South San Francisco, California.
Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any position within the next 72 hours.
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