Biotechs are Accelerating Drug Discovery by Repurposing Platforms: What This Means for Investors

Anyone who's spent any time doing due diligence on biotech companies knows that it takes many years and great expense to bring new drugs to market. In a recent study, Deloitte found that the average cost of developing a single new drug rose to $2.3 billion in 2022.

The firm also said the average development time from starting clinical trials to regulatory approval is now about seven years, and that doesn't include time spent on pre-clinical studies. In fact, the number of years spent on developing new drugs varies quite widely and can even take up to 15 years in some cases.

Factor in all the drugs that fail to meet their endpoints in clinical trials, and the expense and time involved in drug development rise even further. However, some up-and-coming drugmakers are capitalizing on a new trend in the space that's helping them dramatically reduce the time and expense involved in new drug development.

How drug platforms can accelerate drug discovery

Platform technologies are enabling biotech companies to develop new drug candidates much more efficiently and at reduced costs. A drug platform is basically a collection of technologies upon which multiple drugs can be developed.

Essentially, a drug platform enables companies to save multiple steps when developing new therapies and treatments. Well-designed platforms fast-track the creation process for new drug candidates by establishing a base upon which to build those new drugs. In other words, companies don't start from scratch every time they develop new candidates.

Drug platforms also enable companies to continuously improve the treatments they develop because each new candidate brings another set of data points. In many cases, new candidates developed on previously used platforms share the same or similar safety profiles, reducing some of the uncertainty involved in drug development. Here are four publicly traded biotech companies working on drug platform technologies.

Biora Therapeutics

Biora Therapeutics (BIOR) markets itself as "the biotech company that is reimagining therapeutic delivery." It's currently working on two different platforms: the NaviCap Targeted Oral Delivery Platform and the BioJet Systemic Oral Delivery Platform. NaviCap is designed to deliver treatments to the specific site of disease, like in inflammatory bowel disease, while BioJet is designed to deliver large molecules orally, replacing injections.

NaviCap is in the clinical stage, while BioJet is still in the preclinical stage. Biora's BT-600 treatment delivers a liquid formulation of the autoimmune drug tofacitinib to the large intestine in ulcerative colitis patients via the NaviCap delivery platform. Preclinical data shows that targeted delivery via BT-600 can lead to reduced drug levels in the patient's blood with tissue levels that are at least 25 times higher along the length of the colon versus the equivalent standard oral dose. 

If Biora can deliver clinical results that are similar to what has been observed preclinically, BT-600 could greatly improve outcomes for ulcerative colitis patients, especially given the known efficacy of tofacitinib, commonly sold under the brand name Xeljanz.

Genprex

Genprex (GNPX) is a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes (Disclaimer: Ari Zoldan is CEO of Quantum Media Group, LLC, and Genprex is a client of Quantum Media Group, LLC). The ONCOPREX platform is a non-viral nanoparticle delivery system used to develop cancer drugs. The platform uses plasmids that contain tumor suppressor genes that are deleted in the early development stages of cancer.

Genprex's oncology platform consists of anti-cancer TUSC2 genes inside non-viral lipid nanoparticles and then delivered via an IV. These genes bind to negatively charged cancer cells and then enter those cells, delivering the tumor suppressor genes that were deleted as the cancer developed.

Genprex's immunogene therapy REQORSA, which is optimized to work with the ONCOPREX platform, is in a Phase 1 clinical trial to treat non-small cell lung cancer (NSCLC). Genprex recently received approval from the Safety Review Committee to move from the Phase 1 to the Phase 2 expansion portion of its Acclaim-1 clinical trial. The trial is studying REQORSA in combination with Tagrisso to treat late-stage NSCLC. The REQORSA + Tagrisso combination has received Fast-Track Designation from the Food and Drug Administration.

Genprex recently announced positive preliminary data from the dose escalation portion of its Phase 1 clinical trial. The company found that REQORSA was well-tolerated and displayed encouraging evidence of efficacy in treating NSCLC. Genprex published an abstract with the complete data here.

“We are proud of the notable progress we made during the Phase 1 portion of the Acclaim-1 clinical trial, and the SRC recommendation to move into the Phase 2 expansion portion of the trial is another validation for our REQORSA development program,” said Rodney Varner, chairman, president and CEO of Genprex. “As we move into the Phase 2 expansion portion of the trial, we remain steadfast in our efforts to bring new therapies to lung cancer patients with unmet medical need.”

uniQure

UniQure (QURE) is working on a gene therapy platform that targets the liver and central nervous system. The company said its platform is modular because it enables the development of gene therapies for multiple diseases using the same set of components. According to uniQure, the disease-specific gene cassette is often the only component that must be changed to target a new disease.

The company uses an adeno-associated virus (AAV) to deliver missing genes to the patient's targeted tissues. AAV-based vectors have been used in pre-clinical research and over 100 clinical trials, demonstrating the technology's efficacy, safety, and lasting therapeutic effects.

So far, uniQure has one approved treatment for hemophilia B. The company's AMT-130 candidate for Huntington's disease is in Phase 1 and 2 clinical trials, while it has several other candidates for amyotrophic lateral sclerosis, better known as ALS, Parkinson's, Fabry disease, and others at various stages.

“AMT-130 continues to be generally well-tolerated with a manageable safety profile at both doses,” said Ricardo Dolmetsch, Ph.D., president of research and development at uniQure. “Importantly, both doses show preliminary evidence of clinical and functional benefits, including favorable trends in total motor score, total functional capacity and the composite Unified Huntington’s Disease Rating Scale compared to natural history. We plan to engage with regulators to advance this promising clinical program as we collect more data from these patients and from our European study.” 

UniQure is also working on a set of technologies called QUREDose to administer multiple doses of a gene therapy. In large animal models, the company has demonstrated the ability to deliver two consecutive doses of gene therapy. Among other benefits, UniQure believes these technologies could improve gene therapy, increase the precision of its AAV dosing, and enable patients with partial responses to treatments to be treated again.

XBiotech

XBiotech (XBIT) is developing proprietary technology and a new class of antibody therapies called True Human. These antibodies come from people who are naturally immune to certain diseases, which differs from past generations of antibody therapies.

Even therapies tagged as "fully human" are created in a laboratory using gene sequence engineering technologies. XBiotech believes its True Human antibodies could tap into the body's natural immunity because they're derived from naturally occurring antibodies.

The company's lead candidate, Xilonix, is in late-stage clinical development to treat colorectal cancer after having been fast-tracked by the FDA and given an accelerated review process in Europe. Xilonix is the first True Human monoclonal antibody that neutralizes the activity of the interleukin-1alpha (IL-1α) protein, which has been linked to the growth and spread of tumors. This protein has also been linked to muscle and weight loss, anxiety and fatigue.

The candidates currently in XBiotech's pipeline include IL-1 blocker Natrunix for rheumatoid arthritis, which could soon enter a Phase 2 clinical trial.

Investing in clinical-stage drug platforms

The traditional pharmaceutical model is rapidly becoming obsolete as expenses associated with launching a single drug balloon out of control, greatly reducing profitability in the space. As a result, many investors may be less inclined to invest in pharmaceutical companies due to the high level of uncertainty.

However, platform technologies will enable smaller companies to survive and thrive over the long term. Thus, those with working platform technologies of their own stand a much greater chance of success than smaller pharmaceutical companies without such technologies.

Ultimately, drug platforms currently in the clinical stages are setting the state for future treatments built on the same platforms. Such technologies enable these companies to develop numerous new drug candidates faster and more efficiently so that they don't lose as much money on drug candidates that won't work, paving the way for future, long-term success.

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

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