I don’t want to get on the CAR-T

So happy to provide a Monty Python clip about a cart. Consider this an update, and perhaps a precursor, to cell and gene therapy products. It seems Novartis’ breakthrough CAR-T therapy Kymriah has a few things on its plate. Quick recap; approved in May 2017, costs $400,000 with ~$40,000 in post-care treatments, has a REMS program to ensure treatment for severe side effect, sales are under-performing, partly due to manufacturing “variability,” and UnitedHealthcare has formally asked the CMS for a National Coverage Determination. CMS convened last Wednesday on the matter in a Medicare Evidence Development & Coverage Advisory Committee (MEDCAC) meeting. FYI, that was a new acronym for us. The result? Wait for it (because you will) … the “Proposed Decision Memo Due Date” is Feb 16th of 2019. Sigh. If you really want to, you can track the progress here.

“But wait, there’s more”

Order now and we’ll ship you the Thighmaster…and if you’re not satisfied, just return it within 14 days for a full refund. Money-back guarantees are almost a necessity for TV products, but what about for medications? Certainly not the norm, there have been some value-based contracts popping up in the pharma world. There’s a very interesting Forbes article written by former Pfizer global R&D President John LaMattina that lobbies for two major trade organizations (BIO and PhRMA) to pass resolutions that money-back guarantees become a formal practice with member companies. Wow, that would Shake Weight things up. LaMattina points to Amgen’s Repatha (LDL-C lowering) and Novartis’ new gene therapy Kymriah (CAR-T therapy), priced at $475,000/patient. Keep this on your radar, few things could change the healthcare landscape like widespread adoption of value-based pricing.

Adding CAR-T to the shopping cart

How about some exciting, multi-billion dollar deals to spice up the first quarter? First, Sanofi acquired Bioverativ, a hemophilia-focused biopharmaceutical company that spun out of Biogen last February. Since losing patent protection, Sanofi has seen flagging revenue from their flagship Lantus products—which occupy the #4 and #15 spots on IQVIA’s list of Top Medicines by Invoice Spending—and they’re hoping Bioverativ can give their treatment portfolio a boost. Similarly, Celgene boosted their pipeline prospects by acquiring Juno Therapeutics, who have a promising CAR-T candidate expected to be FDA-approved in 2019. Celgene also recently bought Impact Biomedicines, all part of a strategy to preemptively address profit losses when their blood cancer drug Revlimid goes off-patent in a few years.

It’s aliiive! And it’s approved!

Last week, the FDA approved Novartis’ Kymriah—a “living drug” that works by making immune cells realize they should get rid of those pesky leukemia cells making a mess of things. We’ve been following this story since June, because it’s really cool for a couple reasons. First, it marks the first time a gene therapy has been approved for use in the US, although more CAR-T treatments are in the pipeline. Second, the treatment is designated for the most prevalent form of childhood cancer in the US—acute lymphoblastic leukemia (ALL). There is a bit of worry that the treatment is prohibitively expensive, which is what sunk the first gene therapy approved in the EU, but maybe competition will help drop prices.

FDA panel ALL in on CAR-T cancer therapy

An FDA advisory board voted unanimously for the approval of a CAR-T cancer therapy for acute lymphoblastic leukemia, or ALL. This could be the first gene therapy—where one’s own genes are altered to create “killer T-cells” to attack cancerous cells—approved in the US. CAR-T cell therapy is a one-time treatment and shows promise in its ability to knock out ALL where other cancer drugs have failed. Novartis, the drug developer, reported 83% of the patients achieved complete remission within three months and a high probability of being relapse-free at 12 months. The new treatment isn’t without safety concerns, but traditional treatments are more toxic than CAR-T, must be taken long term and eventually stop working. Take THAT, childhood cancer!

1. Gene therapy for the masses

Here’s a riddle for you. What has four wheels and costs $150K? Hint: it weighs 150 pounds. Give up? It’s the new portable device that automates CAR-T production—we had to Google it, too. The current gene therapy process isn’t what we’d call convenient. It involves: flying the patient or patient’s blood to one of only a dozen research centers globally, a room full of smart people, and dozens of hours just to process one person’s cells. As you can imagine that rings up a hefty price tag—some estimates are around $500K. With the new portable device, Prodigy, the goal is to automate this tedious procedure and take gene therapy to the patient instead of the other way around.