Hot Picks: Analyst flags biotech stocks driving next wave of innovation

The biotechnology sector continues to deliver new opportunities for investors as companies push forward with therapies for pulmonary, genetic and neurological diseases. Analysts say advances in gene therapy and targeted treatments could transform patient care and drive long-term growth across the industry.

BNN Bloomberg spoke with Joseph Thome, senior research analyst for biotech at TD Cowen, about three companies he says stand out for their innovative pipelines: United Therapeutics, uniQure and Xenon Pharmaceuticals.

Key Takeaways

• United Therapeutics’ Tyvaso therapy showed strong phase-three results in idiopathic pulmonary fibrosis, positioning it for growth in a multi-billion-dollar market.
• The company’s cardiopulmonary drug portfolio continues to generate strong revenues and could expand further if IPF approval is secured.
• uniQure’s gene therapy AMT-130 slowed Huntington’s disease progression by 75 per cent over three years in early data, with a potential filing in 2026.
• The company’s Hemgenix treatment for hemophilia B remains a milestone for one-time gene therapies targeting rare diseases.
• Xenon Pharmaceuticals’ lead drug, azetukalner, is in phase-three trials for focal onset seizures, offering hope to patients unresponsive to existing drugs.

Read the full transcript below:

ANDREW: We always love talking about the biotech sector, and here with his Hot Picks is Joseph Thome, senior research analyst for biotech at TD Cowen. Joseph, always great to hear from you on this sector. Start off with your first idea, please — United Therapeutics. What are they all about?

JOSEPH: Thank you very much for having me. United Therapeutics is a biotechnology company focused on developing cardiopulmonary drugs. The company currently generates nearly US$3 billion in annual revenue. Perhaps most exciting is that one of its therapies, Tyvaso, recently produced encouraging phase-three data in treating idiopathic pulmonary fibrosis, or IPF.

The data showed that adding Tyvaso reduced the decline of forced vital capacity, or FVC, with good tolerability compared with standard-of-care agents in a placebo-controlled setting. Our physicians view the results as very encouraging and believe the drug could become a frontline agent for IPF, an area with high unmet need.

Current standard-of-care agents only slow the progression of IPF symptoms and carry a high rate of adverse events. Despite that, the market leader, Ofev, generates about US$4 billion in annual worldwide revenue. So we see IPF as an important growth driver for United Therapeutics going forward.

ANDREW: Your next idea — I think it’s pronounced uniQure — works on hemophilia and has one of the first gene therapies in that space.

JOSEPH: Yes, uniQure is a biotechnology company focused on “one-and-done” gene therapies for rare diseases. It currently has an approved agent for hemophilia B, but the next big opportunity could be in Huntington’s disease.

In September, uniQure reported data from its AMT-130 program in Huntington’s showing that a one-time treatment could slow disease progression by 75 per cent at three years versus a natural-history comparator. Our physicians see this as great news for patients. There are roughly 40,000 people in the United States with Huntington’s disease, and there are currently no approved treatments.

We expect this drug could be filed at the beginning of 2026 and made available to patients later that year.

ANDREW: You have a PhD in microbiology and immunology from Columbia. Here’s a basic question: the body has trillions of cells, each with its own DNA. How can a therapy affect DNA throughout the body?

JOSEPH: It’s a very targeted process. These therapies use what’s called an AAV, or adeno-associated virus, to deliver treatment only to a specific cell type. For example, in Huntington’s disease, the therapy targets cells in the deep brain to knock down a specific gene.

RNA acts as a guide to ensure the therapy reaches the desired target while avoiding other tissues. That gives you an effective treatment profile without compromising tolerability.

ANDREW: Of course — immunology, a famously simple subject. Finally, we’re tight for time. Xenon Pharmaceuticals — what are they all about?

JOSEPH: Xenon Pharmaceuticals is a Canadian biotech company focused on developing ion channel modulators for neurological disorders. The company’s lead drug candidate, azetukalner, will report phase-three data in patients with focal onset seizures in early 2026. The phase-two data were strong.

Azetukalner meets many of the criteria physicians are looking for: it’s once daily, doesn’t require titration, and works well alongside other anti-seizure medications. Despite the number of available treatments, about 30 per cent of patients with seizure disorders have limited options. That will be the initial target market for azetukalner. We think the phase-three trial will be successful and that the drug could have a strong commercial launch.

ANDREW: Thank you very much. I really appreciate it. I hope you’ll come back soon — we’d love to hear more about progress in gene therapy. Joseph Thome, senior research analyst for biotech at TD Cowen. Thanks again, Joseph.

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This BNN Bloomberg summary and transcript of the Oct. 16, 2025 interview with Joseph Thome are published with the assistance of AI. Original research, interview questions and added context was created by BNN Bloomberg journalists. An editor also reviewed this material before it was published to ensure its accuracy and adherence with BNN Bloomberg editorial policies and standards.