The cost of developing new drugs is insane - for every drug that makes it to market, pharmaceutical companies spend nearly $2.8 billion just testing drugs that may never see the light of day.
So, what if there was a way to improve the drug discovery process?
For years, the pharma industry has been exploring ways to improve drug discovery with sophisticated machine learning (ML) technology and now Vyant Bio Inc (NASDAQ:VYNT) has a drug discovery engine, powered by its human biology and powerful ML-driven AnalytiX platform.
The Cherry Hill, New Jersey-based biotechnology company uses its human cell-based microBrain and computer modeling platform, AnalytiX, to identify compounds to target neurodevelopmental diseases like Rett Syndrome and CDKL5 Deficiency Disorder, and Parkinson’s disease, a progressive central nervous system (CNS) degenerative disorder that affects movement and cognitive function.
Vyant has completed a large microBrain-based screening of AI-generated novel compounds for Rett Syndrome, a rare genetic neurological disorder that affects the way the brain develops in children, causing a progressive loss of motor skills and speech, with Atomwise Inc, its joint venture partner.
“This work has yielded ‘hit’ expansion across two unique biological targets and several novel compounds,” Vyant Bio CEO Jay Roberts told Proactive in an interview.
A healthcare-tech visionary, Roberts has previously spearheaded companies like Cancer Genetics Inc and he understands the inner workings of the pharma industry from building software systems to using Big Data analytics to transform healthcare. He also specializes in corporate finance, strategy, capital markets, and M&A.
The AnalytiX advantage
Roberts is passionate about redefining drug discovery and believes Vyant Bio has an edge when it comes to discovery, early biologic target identification, and pre-clinical development work.
“The use of human-derived cells to create disease models in our labs allows us to understand a drug’s effect on human biology from the beginning of discovery. We use induced pluripotent stem cells (iPSC), a technology that uses patient-derived skin or blood cells that are first regressed to a stem cell state, and then are chemically induced to become brain cells that form three-dimensional (3D) organoids,” said Roberts.
These organoids enable Vyant Bio to study how cells interact in an organ and how drugs impact human biology. The engineered human brain models hold the key to successful early pre-clinical work when it comes to brain-related neurodegenerative or neurologic developmental diseases.
“We are able to perform high throughput screening on the microBrain and can compare how the compounds affect healthy cells or how they affect diseased cells,” said Roberts.
The Vyant Bio CEO explained that the company’s researchers are able to “digitally observe” the changes to these cells over time.
“We generate very large image files, and our machine learning analytics platform analyzes the digital images and translates them into data form. That allows us to rank order the compounds that demonstrate cell function recovery from the disease state,” said Roberts. “In other words, we are using machine learning to interrogate biology.”
De-risking drug discovery
In a nutshell, Vyant’s powerful AnalytiX platform, at the intersection of human biology and machine learning, quickly shifts through and analyzes functional data to identify the most promising drug candidates, targets, and biomarkers.
According to Roberts, regulators are good at ensuring that drug candidates approved for patient clinical trials don’t endanger patients. But they are less focused on the effectiveness of the drug.
“In the traditional drug development process, 62% of drugs that go into Phase II clinical trials fail because of their lack of effectiveness,” pointed out Roberts. “By understanding how drugs affect human biology before clinical trials, we can dramatically reduce that percentage over time. We are de-risking decision-making. We believe our platforms can meaningfully assess the effective impact that potential drugs have on human cells.”
Strong pipeline
Vyant Bio recently told shareholders that the company has entered 2022 focused on cost efficiency and effectively bringing a repurposed drug candidate into clinical trials in the first half of 2023.
“Our first two novel drug candidates are expected to enter clinical trials in the second half of 2023,” said Roberts. The company’s first program is with its repurposed drug VYNT 0126.
“Clinical trials will indicate whether our measured impact on brain-wave activity in our organoid models offers some benefit to patients,” the Vyant CEO added. “We then plan to use our platform to accelerate the discovery of other CNS monogenetic diseases such as different forms of familial Parkinson’s disease.”
Last year, Vyant Bio completed its Rett and CDKL5 Deficiency microBrain disease models for high-throughput screening to support its own R&D programs.
“This has allowed us to complete several rounds of screening on AI-generated novel compounds, based on novel scaffolds, that show dose-dependent biomarker response in our models,” said Roberts. “These results are at the core of leading us to the identification of specific compounds to take forward into the clinic, which we plan to announce later in 2022 once our intellectual property/patent work is complete and final selections are made of our lead candidates.”
Valuable partnerships
While redirecting Vyant Bio from a services company to a much more valuable drug-discovery platform company, Roberts has employed new technologies and partnerships. He has leveraged the relationships he has built in the industry over the last 25 years to forge key partnerships.
The company is well-funded with around $20 million in cash and three well-developed strategic partnerships.
“We pursued key partnerships with companies that employ AI-based therapeutic creation and prediction in three areas: novel small-molecule design, proteome-based target identification, and novel biologics (protein) design,” said Roberts. “All three arrangements have meaningfully accelerated our focused central nervous system (CNS) therapeutic discovery efforts.”
In addition to completing a large microBrain-based screening of AI-generated novel compounds for Rett, with JV partner Atomwise, Vyant has a collaboration agreement with Cyclica Inc to screen compounds using Cyclica’s proteome-wide, AI-enabled discovery platform to identify new treatments for CDKL5, which is characterized by seizures that begin in infancy, followed by delays in many aspects of development.
As part of its third partnership, Roberts said that along with Ordaōs Bio, it is designing AI-generated small protein therapeutics against novel CNS targets and will assess their disease-modifying actions in patient-derived organoids.
High growth potential
Vyant Bio’s value lies in shortening the drug development timeline from early discovery to the investigational new drug (IND) filing stage to two-to-three years.
“This is a big improvement on the traditional timeline, which is probably five-to-seven years,” said Roberts. “Our portfolio of novel human disease models and platforms are paired with powerful data science technologies to compress traditional drug-discovery timelines, improve clinical success and create a de-risked pipeline of promising drug candidates.”
Analysts at HC Wainwright & Co have noted that Vyant Bio advances drug candidates efficiently from identification of the target to the IND stage by “harnessing state-of-the-art science, technology, data analytics, and regulatory expertise.”
The Wainwright analysts recognized Vyant Bio’s value in its ability to "de-risk drug development" by establishing “translatable biomarkers to drive discovery and de-risk clinical trials quickly and efficiently.”
Vyant Bio has also caught Wall Street’s attention with its shift from being a services company to a major drug-discovery platform company that will generate higher value.
HC Wainwright currently has a 'Buy' recommendation and a $5 price target on Vyant Bio’s stock. That represents nearly 313% upside potential with shares of Vyant Bio currently trading at around $1.21 on the tech-dominated Nasdaq.
Contact the author Uttara Choudhury at uttara@proactiveinvestors.com
Follow her on Twitter: @UttaraProactive