May 4, 2016
Executives
Dolly Vance - EVP, Corporate Affairs & General Counsel Raul Rodriguez - CEO Ryan Maynard - CFO Anne-Marie Duliege - CMO
Analysts
Anupam Rama - JPMorgan Eun Yang - Jefferies
Operator
Good afternoon, and welcome to Rigel Pharmaceutical's Earnings Conference Call for the First Quarter 2016. [Operator Instructions].
And now, I will turn this conference over to our first speaker, Dolly Vance, who is Rigel's Executive Vice President, Corporate Affairs and General Counsel.
Dolly Vance
Hello, and welcome to our quarterly earnings conference call. The financial press release for the first quarter of 2016 was issued a short while ago, and can be viewed under the news section of our website at www.rigel.com.
As a reminder, during today's call, we may make forward-looking statements regarding our financial outlook, and our regulatory and product development plans. These statements are subject to risk and uncertainties that may cause actual results to differ from those forecasted.
A description of these risks can be found in our most recent quarterly report in Form 10-Q on file with the SEC. Any forward-looking statements are made only as of today's date, and we undertake no obligation to update these forward-looking statements to reflect subsequent events or circumstances.
At this time, I would like to turn this call over to our CEO, Raul Rodriguez.
Raul Rodriguez
Thank you, Dolly and good afternoon. During our earnings call in March I spoke about the excitement at Rigel as we prepare for the top line results from the fostamatinib studies later this year.
The last few months have been very busy. In fact I'm happy to report that we have achieved our objectives for the first quarter of the year.
Both of the ITP Phase 3 studies have completed patient enrollment and are ongoing. Based on that enrollment and the time required to organize the data we expect a mid-year read out for the first of these two studies and the readout for the second study to follow shortly thereafter.
In the meantime we are making significant progress towards becoming a commercial enterprise. Our clinical and regulatory affairs teams are preparing sections of the NDA for fostamatinib in ITP.
We hope to submit the NDA application to the FDA by the end of the first quarter of 2017. In addition, we’re actively engaged in planning for the commercial launch of fostamatinib in the U.S.
This will include hiring key people to build our commercial and medical affairs capabilities in the upcoming months. Beyond ITP the next fostamatinib project is autoimmune hemolytic anemia.
During the first quarter we announced that we have initiated the Phase 2 clinical trial for this indication. We expect an initial readout of results at the end of this year.
We also announced the arrival of Anne-Marie Duliege as our Chief Medical Officer. In a very short time Anne-Marie has assumed responsibility for the completion of the fostamatinib Phase 3 studies as well as for the NDA submission.
These activities are key to Rigel's future success in launching the commercial product. I've asked Anne-Marie join us on this call to provide some insights into ITP and autoimmune hemolytic anemia and we will turn the call over to her in a few minutes.
But first I would like to ask Ryan Maynard, Rigel's CFO to report on the financial results from the first quarter.
Ryan Maynard
Thank you, Raul. For the first quarter of 2016 we reported a net loss of 17.5 million or $0.19 per share compared to a net loss of 18.2 million or $0.21 per share in the first quarter of 2015.
Revenues of 5 million this quarter were all related to the continued amortization of the upfront payment and FTE fees earned from our deal with Bristol-Myers Squibb. We reported total cost and expenses of 22.6 million in this quarter compared to 20.4 million in the first quarter of last year.
The increase in costs this quarter were primarily due to the increase in research and development costs for our Phase 3 ITP and Phase 2 autoimmune hemolytic anemia programs with fostamatinib. As of March 31, 2016 we had cash and investments of 103.6 million which we expect to be sufficient to fund our operations into the third quarter of 2017.
At this time I'd like to introduce Anne-Marie Duliege, Rigel's Chief Medical Officer joining us today remotely.
Anne-Marie Duliege
Thank you, Ryan. I would like to start out by saying how pleased I'm to be part of this great team at such an important transitional time in Rigel's history.
As Raul said we have a lot of work to do and some of it involves making sure that investors, physician and patients understand fostamatinib's potential. The physicians treating patients with ITP and autoimmune hemolytic anemia are primarily hematologist and [indiscernible].
These are small group of specialists. ITP is a rare autoimmune disease in which the patient's body destroys a platelet at a rapid rate.
The platelets count in a normal healthy adult is generally above 150,000 platelets per microliter but patients with ITP can have platelet counts fall below 30,000 putting them at risk for spontaneous or traumatic [ph] hemorrhage. Patients with chronic ITP have increased morbidity as evident by their increased bleeding risk and rate of hospitalization.
In addition, the disorder bleeding risk does translate into potentially higher than average mortality rate for adults with chronic ITP. Rigel Phase 3 study is evaluating fostamatinib versus placebo in a 150 adult patients with chronic ITP across two identical studies.
The primary end point for both studies is your achievement of this stable platelet count equal or greater than 50,000 over several [indiscernible] performed from midpoint to endpoint of the study. In the current standard of care for adults with chronic ITP includes a handful of available treatment.
Steroids are typically prescribed at the first line of therapy for potentially followed by intravenous immunoglobulin, rituximab, thrombolytic agent and [indiscernible] as deemed necessary. The challenge for physicians treating patients with chronic ITP is that the therapies don't always work in all patients and that we cannot predict why.
Hence physicians and patients are seeking new alternative with different mechanisms of action that are safe, effective and convenient for chronic use. Fostamatinib mechanism of action which targets the underlying immune cascade responsible for destroying platelets offers a novel therapeutic approach with an only available agent.
If the fixed studies demonstrate that the efficacy and safety profile of fostamatinib that we anticipate and if the FDA approves fostamatinib physicians and patients will have a much needed new option. Patients with autoimmune hemolytic anemia are even less well served by current therapies and tend to be quite ill.
And those are care of the same group of hematologist and [indiscernible] as the ITP patients, this growth suffers dangerously low red blood cell counts and can be highly anemic. Our Phase 2 proof of concept study is currently recording patients.
The first cohort of this open label study will evaluate the safety and efficacy of fostamatinib in 17 patients. It will provide useful insight into our plans and designs for future clinical study.
As Raul mentioned we are working to have results from this cohort read out by the end of this year. Finally the last indication currently under clinical investigation in the fostamatinib program is IgA nephropathy.
The Phase 2 study is ongoing and recently expanded its reach into Asia. The preliminary results of the first court are expected by the end of this year.
And I will look forward to sharing more information about our clinical programs with you in the future. And now I will turn the call back to Raul.
Raul Rodriguez
Thank you, Anne-Marie and it's delightful to have you with us. As you can detected we’re very enthusiastic about the fostamatinib opportunity.
One of the questions we frequently receive is how will the drug fit into the treatment paradigm for ITP. First we have been conducting extensive market research on ITP and have a good understanding of the physician and patient experience with the multiple lines of therapy currently available, only two of which are actually approved for this indication.
Physicians and patients are concerned that patients are not achieving acceptable and stable platelet counts in the long term. This despite their efforts to manage their disorder and thus there is a need for a new therapeutic that works differently where the other currently available agents may not have.
We view fostamatinib and ITP as a key addition for refractory patients. We know that this molecule has a potent and novel mechanism of action and a very well defined safety profile.
It's entirely reasonable to imagine a scenario in which fostamatinib enters the market if and when approved by the FDA and is given to patients who are [indiscernible] experienced but have been unsuccessful or unsatisfied with those prior therapies. We think this is an attractive position for fostamatinib in this indication.
In regards to autoimmune hemolytic anemia there is reason to believe to expect that fostamatinib could play a significant role in helping patients with this disease. We based our expectations on the following facts.
First, there are no currently approved therapies for autoimmune hemolytic anemia. Existing therapies or off label, it consists of steroids, IVIG and other agents used to treat ITP.
Second there is a very limited pharmaceutical development underway in this disease area. So we know that this is a patient population that is largely under served.
If fostamatinib proves safe and efficacious in this autoimmune hemolytic anemia this treatment may play a very central role in the treatment of these diseases. Clearly, we are enthusiastic about the synergies that exist between ITP and autoimmune hemolytic anemia including their shared physician audience.
In the upcoming months we look forward to having a significant answers and insights into fostamatinib's potential in both of these indications. While our priorities and resources are largely focused on advancing fostamatinib towards commercialization we've also been working on moving our IRAK, Mer, other immune and immuno-oncology projects forward into the clinic.
Recently our researchers have given presentations on these topics at the AACR Meeting and we will soon be presenting at the Immunology Conference in Seattle later in May. One of the topics that will be presented is our work on developing a novel class of Nrf2 activators that may have therapeutic effect in inflammatory and neurodegenerative disorders.
We have identified orally bioavailable lead candidate that which has shown positive results in preclinical studies in a murine model of multiple sclerosis. In addition we are actively pursuing collaborations for select projects including fostamatinib in Europe and in Asia.
As Rigel mentioned earlier we have a good cash position. We continue to manage our resources wisely and we have enough cash to carry us for 12 months after the Phase 3 studies are reported.
This does not take into account any new collaboration's or possible financing. In fact any new deals or collaboration's or milestones from current collaborations would further extend this runway.
By the end of the year we will have pivotal information of fostamatinibs potential in ITP and these two other indications. In anticipation of the data, Rigel's focus for the remainder of 2016 includes planning and building a commercial organization for the potential loss of fostamatinib in the U.S.
and in addition we will continue to focus on growing the R&D pipeline beyond fostamatinib while monetizing select pipeline assets through attractive partnership. It's an exciting time to be part of the Rigel team and we hope you also agree.
Now let's open the call up for questions.
Operator
[Operator Instructions]. Our first question comes from Anupam Rama of JPMorgan.
Anupam Rama
Just a quick one for me, are you buying is -- what your assumptions are around the placebo response for the ITP trial and can you comment on this? Thanks.
Raul Rodriguez
Sure. I could take a shot at that Anne-Marie feel free to chime in as well.
We expect a very low placebo response rate in the Phase 3 studies and this comes from the experience that Amgen had with it's similar trial design for it in-plate [ph] product. They did a very similar design looking at multiple readouts in the second half of their study much as we're doing and they also saw a very low placebo response rate.
Operator
Our next question comes from Sami Yang [ph] of Jefferies.
Eun Yang
This is Eun. You mentioned that data from two Phase 3 studies will be made this year but based on the completion of our patient enrollment, do you expect that data read out to be about two months apart of [indiscernible] study?
Raul Rodriguez
The first study we will have in mid-year and the second study will be a couple months after that.
Eun Yang
So you know what's the last patient treatment is over, the six month the treatment is over. How soon can you clean up the data and report a top line?
Raul Rodriguez
Probably about six weeks plus or minus two.
Eun Yang
Okay. Now are you currently in discussions for Ex-U.S.
partner?
Raul Rodriguez
Ex-U.S. partnerships?
Eun Yang
Yes.
Raul Rodriguez
We’re initiating those discussions now with parties that might be interested, Europe being perhaps the first priority since we think that’s most similar to the U.S. I think we definitely will need that data before we sign any deals obviously but right now in advance of having the data we want to establish relations, good introductions to potential parties.
So we will be having those meetings very short.
Eun Yang
Okay. And then question on hemolytic anemia, so you mentioned that we’re going to be seeing 17 patient data by the end of this year and then you will start to discussion with the FDA guarding the scope of the protocol for pivotal study but from talking to KOLs is it fair to assume that the percentage of patients, the hemoglobin levels are going above 10 g/dL, would it good primary end point for this study for registration?
Raul Rodriguez
We haven't had discussion with the FDA about that being the pivotal readout in a pivotal study. So it's premature to say that that's what it will be.
Eun Yang
Sure but how about KOL?
Raul Rodriguez
We have had some discussions with KOLs and it was with their suggestion, concurrence and support that we went ahead and did the design of the Phase 2 study as we had looking at hemoglobin levels trying to get patients with hemoglobin levels above 10 and so clearly there are KOLs to support that.
Operator
[Operator Instructions]. I show no further questions in the queue at this time.
I will now turn the call over to Mr. Rodriguez.
Raul Rodriguez
Thank you. Well it's been a very exciting quarter and I think a successful quarter for Rigel and clearly very interesting times and I think a bright future for the rest of the year in terms of getting the data from these various additional studies on fostamatinib.
So we continue to appreciate your continued interest in and support for Rigel. And we look forward to keeping you up to-date on our progress.
Thank you so much.
Operator
Thank you ladies and gentlemen for attending today's conference. This concludes the program.
You may all disconnect. Good day.