Kite Conf Call - My notes

Kite Conf Call - My notes

It’s really very impressive. They have prepared for launch in a very, very professional manner and they sound ready to go. They have a large pipeline of other indications and other products. Very worth reading.


David Chang - We’re extremely encouraged by the recent advisory committee meeting and it’s unanimous support for the risk-benefit of Novartis’ CAR-T therapy. We believe it was a major milestone for the field of cell therapy and provided clarity on how the FDA will view this innovative therapy as it enters the commercial space. We have just past the midpoint of FDA review of our BLA.

To date, the FDA has completed:

its mid-cycle review,
the inspection of our commercial manufacturing facility, and
the inspections of our clinical sites.

The FDA has informed us that they will not need to schedule an advisory committee meeting for axi-cel. We continue to work closely with them as their review moves towards the final phase.

There are also some important updates to the axi-cel program as we look beyond our first potential indications. In July, we submitted of our Marketing Authorization Application in Europe. As you may recall, axi-cel is being developed in Europe under Priority Medicines with Prime Designation. Our Marketing Authorization Application has been granted an accelerated assessment which shortens the review cycle time by 60 days. As we recently announced, we have also initiated the axi-cel clinical program in the EU having successfully treated the first patient. In the next few months, we expect to open additional clinical sites across the EU in order to expand our ongoing clinical studies. We believe this growing, firsthand experience with axi-cel in key European lymphoma and transplant institutions will be critical for the future of axi-cel in this market.

Clinical supply of axi-cel in Europe currently comes from frozen peripheral mononuclear cells at our central facility in El Segundo. We’re evaluating manufacturing options in Europe.

Throughout the remainder of this year and into 2018, we look to expand potential indications of axi-cel with the ZUMA-5 clinical trials in non-Hodgkin’s lymphoma as well as to plan ZUMA-7 clinical study in second line DLBCL. Last week we dosed the first patient in ZUMA-5. This study will involve patients with follicular lymphoma. We plan to enroll approximately 50 patients in this Phase II study.

We’re also moving KTE-C19 forward with ZUMA-3 in adult ALL, ZUMA-4 in pediatric ALL and ZUMA-8 in chronic lymphocytic leukemia. As well as our ongoing efforts in the U.S. and EU, our joint venture in China and our partnership in Japan, are aimed at ensuring that axi-cel will be available for patients globally.

Looking beyond axi-cel, KITE-585 our anti-BCMA CAR program for multiple myeloma has the potential to become the next significant product candidate for Kite. As we announced this morning, we have filed our investigational new drug application or IND with FDA for KITE-585. This underscores our commitment to accelerate development of KITE-585 and we’ll continue to leverage the experience we have gained from the axi-cel program both in clinical study design and execution and product manufacturing to advance KITE-585. Now I’d like to turn the call over to Shawn.

Shawn Tomasello - I’d like to cover the buildout and launch readiness preparations for Europe and the U.S. launch strategy and full operational readiness. The headline, yes, we’re ready.

So let’s start with Europe, with the announcement of our recent MAA filing and potential product approval, launch is now officially on the radar. Many of the critical components needed to unlock the value of axi-cel in Europe are in place. In 2016, Kite made the decision to commercialize axi-cel on our own in Europe and capitalizing on the footprint of our brilliant group of researchers at Kite EU in Amsterdam, we’re aggressively building out our operational infrastructure in Europe.

Our head of Europe, Chris Nowers, is leading the buildout of a stellar team of commercial, medical affairs and other cross functional teams in London. He has hired a General Manager in Germany and expects to announce soon the hiring of General Managers in additional key EU markets. We’re fully engaged with healthcare authorities and opinion leaders in the EU and are executing early marketing preparation and commercialization activities. Our clinical teams are working closely with EU investigators to ensure they understand Kite’s CAR-T technology as well as adverse event medical management.

Lastly, we also have the not-so-secret weapons of two highly experienced and deeply engaged board members: Ian Clark, most recently CEO of Genentech, and Franz Humer, previously Chairman and CEO of Roche. A year ago, the question we heard in Europe was, Kite who? Today, we hear, how can we work with Kite?

Coming soon is a potential approval of axi-cel in the U.S.

Yes, we will be launch-ready by September, to ensure that we’re prepared for an approval at any time. Our commercial headquarters and field-based teams are fully staffed, trained and ready for launch. Awareness of CAR-T therapy which is critical component for adoption, more than doubled from a year ago. We know from research that those health care providers who are aware of CAR-T therapy are more likely to refer or prescribe CAR-T therapy. Speed, service, reliability and ease-of-use will drive success.

We anticipate that our market-leading average manufacturing turnaround time of 17 days, combined with our Kite Connect service model will result in a positive, prescriber and patient experience. The controlled launch strategy is about maximizing the health care practitioner experience, while focusing on patient safety. Following FDA approval we expect to have at least 10 of the top centers in the US ready for final site activation. Beyond these initial centers, we’re actively working with over 30 additional sites, bringing them to our site readiness process. A process that will continue until we reach our ultimate target of 70 to 90 academic centers. The sites are fact accredited and many are creating dedicated cell therapy units within their respective institutions. As a result, they are prepared to treat patients, setting the stage for success with cell therapy in the months and years to come.

Kite Connect is a combination of people, systems and processes that holistically support a positive health care provider and patient caregiver experience with axi-cel. To prepare for execution at launch, we’re conducting end-to-end rehearsal dry runs at each site. These activities enable us and our centers to be confident in the logistics of our workflow processes surrounding axi-cel therapy. Reimbursement support for patients will be a component of Kite connect. Our market access teams have been laying the foundation for coverage and reimbursement, meeting multiple times with all of the national payers and the majority of regional payers. The teams have also held multiple meetings with CMS, centers for Medicare and Medicaid services and have also met with all 12 MACs or Medicare Administrative Contractors.

Our meetings with all payers have been consistently positive and productive and we’re confident in payer coverage of axi-cel. So let’s think about what happens following the day of approval. On day one, we will notify the centers so they can bring their teams together for final trainings on specifics around axi-cel by Kite commercial and medical teams. We expect centers will reach out to patients they’ve identified as candidate for axi-cel and we will be fully prepared to process and deliver the cell orders. In summary, our teams are excited and fully prepared to bring axi-cel to patients that have been waiting.



One of the things I have learned from this board beyond reading the conference call is listening to them. I don’t get to listen to every conference call for every company but with the up and comers like KITE I try to listen to them all because the tone of the call is usually evident. It’s one of the reasons I bought a large stake in UBNT and even more when it dropped to the mid 40’s not all that long ago (not to mention you had been beating the drum for a while). If you listened to the spring CC of UBNT there was an air of overt confidence that didn’t come through in just words on paper. The words were good but the tone of the call was even better.

Saul, I’m not sure if you listened or just read KITE’s CC but the excitement was palpable from these guys. You got the sense they were very ready for launch and that insurance coverage was not going to be as big an issue as many have been concerned about (and rightly so). The biggest things that stood out beyond that were European expansion and 585. They are ready for Europe expansion. Just as they are ready here on the word “GO” from the FDA. 585 has them very excited and I personally cannot wait to see some early results of the trials. I hope their confidence translates into results. I personally didn’t buy more after the call but would have if I didn’t already have a full position.

However, a word of caution to anyone investing in medical companies, especially ones on the verge of groundbreaking therapies (this could apply to any company but I find it more true in these types of investments). Sometimes the optimism you hear is the medical optimism. This doesn’t always translate into financial bliss I have found. It can be very exciting to talk about your new awesome product that could potentially cure children of cancer who have no hope and all the while the financials are a mess. So I encourage all here to not only read but listen. Not only listen, but read, and then make your own analysis of the data in front of you.

Long KITE and UBNT


I am trying to get some sort of handle on what the addressable market is for axi-cel upon approval for DLBCL.

Here are the new cases for all NHL per year.

DLBCL makes up around 30% of all cases.…

Based on the number of NHL deaths per year of around 20,000 people, 30% gives us about 6,000 from DLBCL. Who knows what the price will be. If it is $200k, that is $1.2B in revenues annually.

Along with the treatment working relatively well, this assumes they are ramping up quickly enough and the call sounded as if they are confident in that.

The first link showed over 650k people living with NHL in 2014. However, I believe the treatment will only be for the refractory and relapsed segment of the cohort so it is probably better to use the number of deaths assuming those patients are in the approved cohort.

None of the above includes Europe either.

What would be the market cap for such a company with many other qualified candidates in the pipeline for different cancers?

Take care,

Page 25 of the investor presentation answers the question of addressable patients.
Still unclear are price, real world efficacy and cost to produce.


Hi Saul,
Thanks for the notes. IMO the biggest risk to immediate approval is the process to modify blood and deliver the therapy has a hiccup so management confidence is encouraging to me on that front. It will be VERY interesting after approval (if KITE is still an independent company) to see how the business works out. The actual margins and cash flow could amaze or disappoint. I remain hopeful.