Some things I am interested to hear at #BIOCEO15

Friday, 6-February-2015

The 17th annual BIO CEO conference takes place on Monday and Tuesday (February 9 & 10) at the Waldorf Astoria hotel in New York.  The program this year includes presentations from approximately 140 public and 25 private companies.  In addition, there are some great panel discussions scheduled with industry leaders from both the business and investment sides.  I plan to be there, as this conference is one of my favorites.  

 

I think a neat aspect to BIO CEO is how a lot of up-and-coming companies present at this one who do not frequently do so.  It provides a unique chance to hear updates from them and fill in any blanks you might be wondering about.  With that in mind, here are a handful of things I will be listening for at this year's conference.  It is not a comprehensive list, but simply represents various companies I follow and would like an update on.

 

AcelRx (Nasdaq: ACRX):  This company had a turbulent 2014, to say the least.  AcelRx lost their CFO, Chief of Medical Affairs, and Chief Commercial Officer all during the weeks leading up to the receipt of a CRL for their drug/device combo Zalviso.  The company is working on re-submitting the application this year, but also has announced that the CEO will soon be leaving.  In short, I'll be looking for some clarity about who exactly is running this company right now, and whether the resubmission plans are currently on track and realistic.

 

Adaptimmune (Private):  Adaptimmune is going to be one of the most highly regarded IPOs of 2015.  It is just a matter of when, so any guidance they can provide at BIO CEO as to the timing would be much appreciated.  The company uses a T-cell receptor approach (TCRs) to various solid tumor types, many of which they are partnered with GSK (NYSE: GSK) on.  In October, they posted promising initial results from a pilot trial of their NY-ESO TCR in synovial sarcoma.  This is definitely a name you want to be familiar with.  

 

Aerie Pharmaceuticals (Nasdaq: AERI):  I own this stock and am a fan of the company's management team.  The thing to listen for from them has to do with their current thinking on acquiring new assets.  Aerie raised $125 million through a convertible note offering in September, and will likely use the proceeds to add to their pipeline soon.  I'll be looking not only for clues about what kind of asset they are searching for, but also when they intend to move on this.  Aerie will be in the news soon because Rocket 1, a very important phase III study for their lead drug Rhopressa, will report in the second quarter.

 

Celladon (Nasdaq: CLDN): Celladon is going to be a fascinating stock to watch this spring, because it doesn't get any more binary than this.  Their CUPID 2 gene therapy trial for heart failure is scheduled to report in April.  I'll want to hear from them if that is still on track, and whether they have any backup plans in the event the trial turns out to be a clear failure.  They want to test whether Mydicar will improve the rate of AVF maturation for hemodialysis patients, but I would like to see them branch out into other more intuitive gene therapy indications as well.  I do not plan to play the binary event, btw.

 

GenVec (Nasdaq: GNVC):  I have been a GenVec skeptic, but admit to becoming recently intrigued after the CEO of their partner Novartis (NYSE: NVS) gave the company an interesting shout-out last week.  I'll be listening at the conference for any update they can provide on the recently started trial.  So far, we know that one patient has been treated.  It will be interesting to hear how he is doing, and whether any more have started on therapy.  My understanding is that the fourth patient will be the first one stepping up to a higher dose so we might have to wait until then for any hints of efficacy.

 

Kite Pharma (Nasdaq: KITE):  Kite's CFO made a splash at last month's JP Morgan Healthcare Conference by suggesting in a Reuters story that the company's internal models assume a $150,000 price for their CAR-T therapy.  That is much lower than what investors are expecting, and might be a reason for the stock's weakness ever since.  It is still unclear if that was an off-the-cuff comment (a.k.a. an accident), or something that was said strategically.  I recently offered five reasons why I think the latter, if true, would be a big mistake.  It would be great if Kite's management can provide some clarity on this issue at BIO CEO.

 

Sorrento Therapeutics (Nasdaq: SRNE):  Sorrento recently announced that a supposed registrational trial for their lead program Cynviloq has completed enrollment.  The data from that should be coming in late March or early April.  However, I'm much more interested in learning about the collaborations they plan to do in immuno-oncology with Patrick Soon-Shiong's NantWorks.  Companies who are working on second generation CARs are pretty far behind, so I would like to see what timelines they have for getting into the clinic.  Also, investors deserve to know more about why their CFO abruptly resigned this week.  I do own some Sorrento, but have a very short leash on this one.

 

Trillium Therapeutics (Nasdaq: TRIL):  With the name change and reverse stock split behind them, it is time for Trillium to finally make it into the clinic.  It shouldn't be too much longer of a wait.  Trillium does not often present at conferences, so it will be good to hear them confirm their progress on completing IND enabling studies, and hopefully provide a roadmap about what is in store for 2015.  Previous guidance was that they will file their IND in the second half of this year, but it would be nice if they can be more specific now that 2015 is underway.

 

UniQure (Nasdaq: QURE):  As I illustrated in a recent chart, uniQure has one of the lowest market caps of any gene therapy company even though they are the only one with an approved product.  Investors are going to want some color on the Glybera launch and to hear how payers are initially reacting to the $1.4 million price tag.  While the pricing issue is interesting, I frankly have more questions about their pipeline.  For example, there are now about a half dozen gene therapy companies who have announced hemophilia programs.  I want to hear from uniQure what they think that means down the road when/if these products make it to market.  Investors don't understand very well how two gene therapies would compete against each other.  It is an important dynamic because more therapeutic areas are likely to become just as crowded as hemophilia soon.