Empowerment Through Knowledge

Shares of Gilead Sciences ticked up Friday morning after the company’s latest earnings report exceeded Wall Street’s expectations.

The results were, in part, tied to growing sales from Gilead’s cell therapy business, which consists of the marketed cancer drugs Yescarta and Tecartus. Together, sales from the two drugs totaled $398 million in the third quarter, a nearly 80% increase from the same three-month period a year prior.

Gilead’s work in cell therapy, catalyzed by the $12 billion acquisition of Kite Pharma in 2017, hasn’t always sat well with investors. Early sales from Yescarta were slower than some had hoped, and Gilead ultimately acknowledged that some assets from the Kite deal were overvalued.

But in recent months, the company’s cell therapy business has ballooned. Third quarter sales of Tecartus were up 72% year over year, reaching $81 million, while those for Yescarta rose 81% to $317 million. Gilead cited the approval of Yescarta as a “second-line” therapy for a type of hard-to-treat lymphoma, which happened in April, as a main reason for the uptick.

“This is what one could call pipeline in a pill,” said Bristol’s Chief Medical Officer Samit Hirawat.  

Sotyktu will not carry a black box warning, the US Food and Drug Administration’s strongest communication of potential risks. Analysts were closely watching the safety language in the drug’s label since such warnings have hampered other promising autoimmune drugs. 

The label is “close to the best case scenario,” Citi analyst Andrew Baum wrote in a note to clients. Shares of Ventyx Bioscience, a biotech company pursuing TYK2 drugs, soared as much as 67.14%. 

Bristol will try to unseat Amgen Inc.’s Otezla, a top-selling psoriasis pill that Sotyktu bested in clinical trials. Shares of Amgen fell as much as 4.3%. Convincing health insurers to cover Sotyktu will take time, said Bristol Chief Commercialization Officer Chris Boerner.

When she found out the cancer had spread to her lungs, chest bone and lymph nodes, she was given one year to live.

David spent the following six months undergoing chemotherapy, and had a mastectomy in April 2018. This was followed by 15 cycles of radiotherapy which cleared her of cancer.

However, the cancer returned in October 2019 when scans showed multiple lesions throughout David’s body.

David then decided to take part in a clinical trial where she was given experimental medicine combined with immunotherapy drug Atezolizumab, which she has injected every three weeks.

After two years on the trial, the mother-of-two has been declared cancer-free once again.

Dozens of gene therapies for a range of devastating illnesses are on their way. These single-dose drugs, tailored to each patient, can potentially deliver a lifetime of benefits.

But that’s reflected in their prices, which are likely to increase pressure on already stretched budgets. To make it easier for government payers to digest Zynteglo, Bluebird plans to spread out the cost over five years, with payments contingent on its success.

As a one-time therapy, Zynteglo could save governments money in the long run by cutting the need for expensive ongoing care. Treating one beta thalassemia patient today can cost as much as €60,000 a year, says Aurelio Maggio, a blood-disease specialist at the Palermo center. That’s €3 million over five decades. With multiple wonder drugs for other conditions set to reach the market soon, the upfront bill could take a heavy toll on Italy’s finances. The price tag for the therapy is twice the $900,000 that SVB Leerink analyst Mani Foroohar expected. Given the large number of patients in the country with the ailment, “the stakes are much higher,” he says.
 

It bears mentioning that computational biology is objectively important. We’re talking about life itself: human DNA; the food we eat; infectious diseases; the evolution of species, and so on. "Biology is the only technology that can directly address fundamental problems facing the world like planetary and human health," says Arvind Gupta, Managing director & Founder of IndieBio and partner at SOSV. "These are world scale problems looking for technological solutions that will be developed in the next 20 years and those that do stand to create trillions of dollars of value". Rather than manufacturing tools for us to use, like cars or software, we’re now beginning to manufacture life itself.

Jennifer Doudna, co-inventor of CRISPR and co-Founder of Mammoth Biosciences(an NFX portfolio company) told us, "Scientists have spent centuries carefully studying how living things work. We have now entered into a new era of biology where it is possible to move beyond observation and towards rewriting the underlying code of living things, creating countless opportunities to improve the world we live in, from diagnosing and treating human disease to restoring the environment around us."

Further, something that has become clearer to us at NFX in the last three years: computational biology touches every industry. There are at least 90 companies worth over $20BN that are eyeing the CompBio space: agriculture; industrial; pharma; energy companies; plus all the big tech companies, like AWS, Google, and Microsoft. (Microsoft, for example, DNA that it hopes can replace cumbersome tape drives).

All of these industries are looking deeper at computational biology, trying to see how it is going to impact them.

Despite this extraordinary case study it is still very early days for the treatment, with the current clinical trial due to run until at least 2023. After that, a Phase 3 trial will need to broaden the volume of patients treated to verify any positive results,. So, realistically a broad clinical application could be up to a decade away ... And that's assuming everything goes right.

An early form of adoptive immunotherapy, called CAR-T therapy, exhibited severe side effects across many of its clinical trials, including some deaths. The therapy also displayed some impressively positive response rates, promising at the very least an extra possibility for patients where pre-existing treatments have failed.

Last year, the first immunotherapy of this kind was approved for use by the FDA. The treatment's approval was undeniably a milestone for this new kind of therapy, but alongside the approval came a striking price tag. Kymriah, for young patients with a type of blood and bone marrow cancer, was initially costed at nearly half a million US dollars per treatment.

Thank you again for your weekly Big Picture video which continues to provide steady guidance through uncertain times.  You have often referred to opportunities in the biotech sector and, in the last summary, referred to opportunities in the development of anti-cancer treatments.  Are there any specific companies that one could follow?

An immunotherapy treatment — one that boosts the immune system — has improved survival in people newly diagnosed with the most common form of lung cancer (advanced non–small-cell lung cancer), according to an open-access study published in the New England Journal of Medicine.

The study results were presented last Monday, April 16, at the annual American Association for Cancer Research conference in Chicago.

Cutting the risk of dying in half. The new study, led by thoracic medical oncologist Leena Gandhi, MD, PhD, associate professor of medicine and director of the thoracic medical oncology program at NYU’s Perlmutter Cancer Center, shows that treating lung cancer by a combination of immunotherapy with Merck’s Keytruda (aka pembrolizumab) and chemotherapy is more effective than chemotherapy alone, according to a statement by NYU Langone Health.

Injecting minute amounts of two immune-stimulating agents directly into solid tumors in mice can eliminate all traces of cancer in the animals, including distant, untreated metastases, according to a study by researchers at the Stanford University School of Medicine.

The approach works for many different types of cancers, including those that arise spontaneously, the study found.

The researchers believe the local application of very small amounts of the agents could serve as a rapid and relatively inexpensive cancer therapy that is unlikely to cause the adverse side effects often seen with bodywide immune stimulation.

“When we use these two agents together, we see the elimination of tumors all over the body,” said Ronald Levy, MD, professor of oncology. “This approach bypasses the need to identify tumor-specific immune targets and doesn’t require wholesale activation of the immune system or customization of a patient’s immune cells.”

One agent is currently already approved for use in humans; the other has been tested for human use in several unrelated clinical trials. A clinical trial was launched in January to test the effect of the treatment in patients with lymphoma.