2018 turned out to be superb for the pharma and biotech sector so far as FDA approvals were concerned. The regulatory agency gave nod to 59 novel drugs, well above 46 drugs approved in 2017. Immuno-oncology was once again the key focus area in 2018, be it in terms of scientific conferences, clinical trial collaborations, acquisitions or FDA approvals.
The recently announced $74 billion mega-merger of two cancer giants, Bristol-Myers Squibb Co BMY and Celgene Corporation CELG, and GlaxoSmithKline's GSK and Eli Lilly And Co's LLY proposed acquisitions of smaller cancer biotech, TESARO Inc TSRO and Loxo Oncology Inc LOXO, respectively, clearly reflect the rising interest in this category.
While 2018 was a year focused on immuno-oncology, here we take a look at four therapeutic areas that we think will garner a lot of interest in 2019. These areas are marked by extensive research, innovative technologies, and the promise of transforming the way diseases are treated.
Immuno-Oncology
The basic concept of cancer immunotherapy or immuno-oncology is to utilize certain parts of the immune system to fight the disease. This can be done by stimulating the immune system to attack cancer cells or by introducing immune system components into the body. While some immuno-oncology treatments have the potential to be effective as monotherapies, others may be more effective when combined with other treatments.
Different types of immunotherapies include monoclonal antibodies/mAbs (man-made versions of immune system proteins which can be designed to attack a very specific part of a cancer cell), immune checkpoint inhibitors (recognize and attack cancer cells), vaccines (help prevent or treat cancer) and others that boost the overall immune system.
Some encouraging results, demonstrating partial and complete responses in late-stage cancer patients, and the FDA approval of two CAR-T therapies in 2017, Novartis' Kymriah and Gilead's Yescarta, have raised hopes for cancer patients and increased interest in this segment. The scope for immunotherapy is growing especially with the use of multiple treatments in combination with each other. It is aimed toward developing targeted therapies or precision medicines.
With immuno-oncology set to change the treatment paradigm for cancer, this area represents huge commercial potential.
Almost all big drugmakers have formed collaborations with smaller biotechs with innovative immuno-oncology candidates in 2018 and the trend is expected to continue in 2019.
Alzheimer's Disease
Alzheimer's disease is an area where many companies have tried their luck but with minimal success. Companies that are currently working on Alzheimer's disease treatments include players like Biogen, Lilly, Novartis, Eisai and Amgen.
Biogen's aducanumab is one of the most important candidates in the company's pipeline and is currently in a couple of late-stage studies. Biogen is also working on elenbecestat (E2609), a BACE inhibitor in a phase III program and BAN2401, a humanized beta amyloid antibody in phase II development. All three candidates are being developed in collaboration with Eisai. Earlier this month, Biogen announced deals with C4 Therapeutics and Skyhawk Therapeutics to develop potential therapies for AD, Parkinson's disease and other neurological diseases.
Lilly has flortaucipir in its pipeline, which is being developed for AD. Last month, it announced a collaboration agreement with Swiss biotech AC Immune to jointly develop morphomer tau aggregation inhibitors, which have the potential to treat Alzheimer's disease (AD) and other neurodegenerative diseases. Meanwhile, Novartis and Amgen are collaborating for BACE1 inhibitor CNP520, which has fast track status in the United Status. Roche is also collaborating with AC Immune for the development of Alzheimer's disease treatments.
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. As a result, several companies continue to invest heavily in developing Alzheimer's disease treatments. 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.
Medical Marijuana
Another segment of the pharma and biotech industry that will remain 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 industry attracted massive interest from investors in 2018 with the FDA approving the first plant-derived cannabinoid medicine, GW Pharma's epidiolex in June.
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.
Other than GW Pharma, key players in this area include INSYS Therapeutics, Corbus Pharmaceuticals Holdings and Zynerba Pharmaceuticals. Tilray was the first cannabis company to go public on a major U.S. exchange in 2018.
Epidiolex oral solution is approved to treat rare, severe forms of epilepsy, Dravet syndrome and Lennox-Gastaut syndrome. Epidiolex represents blockbuster potential. GW Pharma has several new formulations of CBD in development including improved liquid formulations, a solid dose form and an intravenous formulation.
Specialty pharma company, INSYS, uses its proprietary spray technology and capabilities for the development of pharmaceutical cannabinoids for a wide range of indications including treating opioid overdose, anaphylaxis, epilepsy, infantile spasms, anorexia in cancer patients and other disease areas with significant unmet need.
NASH
The market for non-alcoholic steatohepatitis or NASH has potential. A chronic liver disease, NASH is caused by excessive fat accumulation in the liver, which, in turn, is known as steatosis. NASH is known to affect up to 15 million people in the United States and could lead to inflammation, hepatocellular injury, progressive fibrosis and cirrhosis. By 2025, the worldwide market for NASH treatments is estimated to reach a value of $35 billion.
In spite of its large patient population and potential life-threatening effects, at present, there is no FDA-approved drug to treat NASH. Given the prospects of the NASH market, quite a few companies are developing candidates for the same and a FDA approved drug is expected to enter the market in 2020.
Key players in the NASH market are Intercept Pharmaceuticals, Gilead, Allergan and Genfit SA. In fact, combination therapies are also being investigated to treat NASH. Gilead is investigating ASK-1 inhibitor selonsertib in combination with its other NASH assets, GS-0976 and GS-9674. In October last year, Pfizer formed a collaboration agreement with Novartis to research on combination therapies comprising their early-stage investigational candidates for NASH.
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