IInvesting in biotech companies is both challenging and incredibly exciting. Watching a company go through the regulatory approval process from an early-stage clinical trial biotech to an industry juggernaut can be exhilarating.

Although positive results are not guaranteed in clinical testslet’s take a look at two promising companies at different stages of development. Medication Master Plan (NASDAQ: BPMC) has a promising drug candidate in phase 1/2 clinical trialswhile Novocure (NASDAQ: NVCR) is looking to expand the labeling of its Optune device to broader markets. If the results continue to be positive, the two healthcare companies could enjoy a multi-billion dollar windfall.

Medication Master Plan

After a few sluggish years, including a 37% drop in its share price since the start of the year, Blueprint Medicines now seems ready to move forward. Its main focus has been mastocytosis, a disease in which certain types of cells release substances that can cause signs and symptoms similar to those of an allergic reaction – and lead to severe inflammation and organ damage.

Ayvakit, its approved mastocytosis drug, is growing rapidly. The therapy generated $52.9 million in revenue in fiscal 2021, its first full year on the market, and Blueprint forecasts $115 million to $130 million in revenue from Ayvakit this year. After recently gaining approval in the EU and with likely label expansion in the US, Ayvakit should help Blueprint Medicines flourish this year.

And the good news for 2022 doesn’t end there. The $4.1 billion biotech just presented data on its potential Phase 1/2 drug, BLU-945, and is looking to share more data over the next 18 months. BLU-945 is being studied for the treatment of non-small cell lung cancer (NSCLC) with mutations in a gene called EGFR – a particularly difficult disease to treat.

It could be a multi-billion dollar market also with approximately 70% of the 59,000 annual patients in the United States needing to switch to second-line therapy. Today’s frontline worker, Astra Zenecait is Tagrisso, generated $5 billion in revenue in fiscal year 2021. In Phase 1 data released earlier this month, BLU-945 decreased circulating tumor DNA – a sign promising therapeutic response – and was able to reduce tumor size. It’s still early days, but the results are quite intriguing.

Image source: Getty Images.

Blueprint’s ability to market Ayvakit and expand its label despite the pandemic has been admirable. He certainly appears to have a healthy growth trajectory for 2022 and beyond for his treatment of mastocytosis. If its pipeline continues to show promising results, Blueprint Medicines could be one of the top stories of 2022 for biotechnology investors.


As the maker of Optune, a wearable device that disrupts cancer cell growth, Novocure could also be a big winner in 2022. Although it’s already approved primarily for glioblastoma (a cancer of the brain or spinal cord ), Optune could quadruple its addressable market almost overnight. later this year when it provides an update on its Phase 3 trial for NSCLC. If the data continues to be reassuring, shareholders should be quite satisfied. An encouraging sign, an interim analysis was stopped prematurely due to strongly positive results.

Beyond the NSCLC, the future remains rather promising for Optune. Novocure has four late-stage trials with data expected in the next few years. Optune’s opportunity in the NSCLC market is estimated at 46,000 patients in the United States each year, with another 16,000 for ovarian cancer and another 38,000 for NSCLC that has spread to the brain. Phase 3 trials in these last two indications will end in 2023 with data on pancreatic cancer, a market of 43,000 patients per year, expected in 2024. In total, this represents 14 times the current 11,200 patients with glioblastoma per year who are currently eligible for Optune treatment.

This cancer fighter’s finances are also strong. With $938 million in cash at the end of 2021, there’s plenty in the bank to fuel its clinical trials, not to mention its enviable gross margin of 79%. And it even made a profit of $1.7 million last quarter based on adjusted EBITDA.

Additionally, many oncologists are already familiar with Optune. If its future trials are positive, sales could increase rapidly, as it already has a number of treating clinicians familiar with Optune. With several catalysts that would significantly expand its addressable market, Novocure could be the big winner of 2022 for both patients and shareholders.

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Patrick Bafuma owns Blueprint Medicines. The Motley Fool owns and recommends Novocure. The Motley Fool has a disclosure policy.

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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