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3 Pharma & Biotech Pipeline Areas to Keep an Eye on in 2020

Kinjel Shah

In 2019, the FDA granted approval to 48 novel drugs. As in the past years, several approvals were for cancer drugs. These apart, the agency's approvals included treatment options for patients living with cystic fibrosis, sickle cell disease and rare muscular disorders. Important approvals of non-cancer drugs were for Caplyta for schizophrenia, Nourianz for Parkinson’s disease patients experiencing “off” episodes, Vyondys 53 to treat some patients with Duchenne muscular dystrophy and Oxbryta for sickle cell disease, among others.

The last year clearly proved that the FDA is increasingly giving green signal to innovative new products in various therapeutic areas. Though cancer will always be a key area for R&D research, here we take a look at three non-cancer therapeutic areas that we think will garner a lot of interest in 2020. These areas are marked by extensive research, innovative technologies, and the promise of transforming the way diseases are treated.

Alzheimer’s Disease

Alzheimer’s disease is an area where many companies have tried their luck but with minimal success. Over the last several years, big players like Merck MRK, AstraZeneca AZN, Lilly LLY, Pfizer PFE and J&J JNJ have faced setbacks in their quest to find a treatment for Alzheimer’s.

While Pfizer sports a Zacks Rank #1 (Strong Buy), Lilly & J&J have a Zacks Rank of 2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Companies that are currently working on Alzheimer’s disease treatments include players like Biogen, Lilly, Novartis and Eisai.

Biogen’s aducanumab is a leading candidate for AD. In October last year, Biogen and partner Eisai announced their intention to seek U.S. approval for aducanumab, a few months after it halted two late-stage studies on the same drug, following a futility analysis. Biogen’s decision was based on positive results of a new analysis of larger dataset, which became available after the discontinuation of the studies. The candidate, if approved by the FDA, will become the first medicine to treat Alzheimer’s disease.

A group of analysts believe that the promising new data from two failed studies on aducanumab have revived hopes for approval of this almost-written off experimental Alzheimer’s therapy. However, another group believes that the FDA may not approve aducanumab, particularly with mixed outcome results across the two studies. Also, the FDA may require additional studies to be conducted to confirm the candidate’s benefits. Biogen also has another AD candidate in its portfolio, BAN2401which is in late-stage development.

Although Alzheimer’s has historically been a very challenging therapeutic area with quite a few companies stumbling in their efforts to find a treatment, the market is highly lucrative. This is because more than five million Americans are living with Alzheimer’s disease with the numbers expected to triple by 2050 (Data: Alzheimer's Association). The market has immense commercial potential and companies coming out with new treatments could rake in billions of dollars in sales. As a result, several companies continue to invest heavily in developing Alzheimer’s disease treatments.

Medical Marijuana

Another segment of the pharma and biotech industry that will be in the limelight this year is medical marijuana. Canada has become the first major economy to legalize recreational pot, with quite a number of U.S. states giving green signal to cannabis in some capacity last year.

The only FDA-approved cannabidiol drug, Epidolex (marketed by GW Pharma) has shown strong uptake in sales and new patient enrollments since its launch in November 2018 in the United States. Epidiolex was approved in Europe in September 2019.

Epidiolex oral solution is approved to treat seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome. A new drug application seeking approval of Epidiolex for a new indication — tuberous sclerosis complex — is expected to be filed in early 2020. A late-stage study on Rett Syndrome is ongoing. Epidiolex represents blockbuster potential. GW Pharma has several new formulations of CBD in development including modified oral solution, capsule and intravenous formulation

The industry is expected to grow rapidly with legislators allowing or looking to allow more legal uses of marijuana and marijuana-related products, especially in the medical and life sciences industry.

Zynerba Pharmaceuticals is developing its CBD candidate, Zygel, in pediatric patients with autism spectrum disorder. Several Canada-based companies like Tilray, Aurora Cannabis, Cronos Group and Canopy Growth Corporation are involved in cultivating, producing and commercial distribution of recreational and medical marijuana.

We note that there is huge untapped market opportunity for cannabis-based products, which can create the next booming industry. We expect legalization of marijuana at the federal level to attract several big pharma companies into development of marijuana-based drugs, which can boost sales of cannabis-based drugs. Moreover, this will likely create new treatment options for patients if the unsubstantiated claims are proved in clinical studies.

Rare Diseases

A rare disease is a condition affecting less than 200,000 people in the United States. There are around 7,000 known rare diseases. Though each such disease affects only a few people, 25-30 million Americans are living with such diseases. There are numerous types of rare diseases including infectious diseases, CNS disorders, birth defects, and cancers. Though significant progress has been made to diagnose, treat, and even prevent a variety of rare diseases, mainly rare genetic diseases, there are still many such diseases for which no treatments are available. Also, the FDA supports development of drugs for these diseases and there are incentives for drug companies to develop treatments for rare diseases, which also explain increased interest of drug companies in this field. Also, advances in technology, such as gene-editing technologies, and improvements in gene therapies, have drawn increased commercial interest to the realm of rare disorders.

Alexion ALXN, BioMarin, Sanofi SNY and Pfizer are some of the bigger companies making rare disease drugs. Meanwhile, mostly smaller companies have rare disease drugs in their pipelines, which also make them attractive buyout targets for their larger counterparts.

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