Oncternal Therapeutics Inc (ONCT) 2014 Q4 法說會逐字稿

Good day, ladies and gentlemen, and welcome to the GTx fourth-quarter and fiscal year-end 2014 conference update and financial results conference call. My name is Emma and I will be your operator for today. (Operator Instructions). As a reminder, this conference is being recorded for replay purposes.

I would now like to turn the conference over to Henry Doggrell, Vice President, Chief Legal Officer and Secretary of GTx. Please proceed.

Thank you. Good morning and welcome to the GTx fourth-quarter and fiscal year-end 2014 financial results conference call. This morning we issued a press release providing financial results and Company highlights for the quarter ended December 31, 2014 and for the fiscal year 2014. The press release is available on our website at www.GTxinc.com.

Joining me on the call today are Marc Hanover, our Chief Executive Officer; Dr. Rob Wills, our newly appointed Executive Chairman of the Board; Dr. Mayzie Johnston, Vice President and Head of our Enobosarm Clinical Program, and Dr. Ramesh Narayanan, Associate Professor in the Department of Medicine and Director of the Center for Cancer Drug Discovery at the University of Tennessee Health Science Center.

As a reminder, during today's call we will be making certain forward-looking statements. These forward-looking statements are based on current information, assumptions and expectations that are subject to change and involve a number risks and uncertainties that may cause actual results to differ materially from those contained in the forward-looking statements. These risks are described in our filings made with the Securities and Exchange Commission including our quarterly report on Form 10-Q filed with the SEC for the period ending September 30, 2014, and on our annual report which will be soon filed on Form 10-K.

You are cautioned not to place undue reliance on these forward-looking statements and GTX disclaims any obligation to update such statements.

In addition, this conference call contains time-sensitive information that is accurate only as of the date of this live broadcast, March 4, 2015. GTx undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date of this conference call.

I will now turn the call over to Marc Hanover.

Thank you, Henry, and thank all of you for being on the call today. I would like to officially welcome Rob Wills to the GTx team. Rob is a veteran pharmaceutical executive who brings to us more than 35 years of industry experience in the areas of clinical and preclinical development and business development. As an active Executive Chairman, Dr. Wills will dedicate his efforts to these key priorities, our clinical development programs and strategic business development efforts.

Investors and research analysts will also interact with Rob since he and I will be primarily responsible for our external communications.

On the call today I will provide a corporate update while summarizing our key accomplishments and financial highlights. Rob will then review important upcoming milestones related to our clinical development activities.

Now I will begin with our recent accomplishments. Last quarter I outlined our redefined corporate focus to treat two populations of patients with advanced breast cancer with our selective androgen receptor modulator, enobosarm. In December 2014, we presented clinical data at the San Antonio Breast Cancer Symposium from our ongoing Phase 2 open label trial evaluating a 9 mg daily dose of enobosarm as a treatment for women with both estrogen and androgen receptor positive metastatic breast cancer who had previously responded to hormone therapy and subsequently progressed.

These results demonstrated that six of the 17 evaluable AR positive patients or 35% demonstrated clinical benefit at six months of treatment, exceeding our predefined statistical threshold requiring that at least three of the 14 AR positive patients or 21% demonstrate clinical benefit. After a median duration on study of 81 days, 41% of all patients, nine out of 22, achieved clinical benefit as best overall response. One patient with measurable disease had a 27% reduction in target lesions in the liver. Another patient remains on study with stable disease after 500 days of treatment.

Enobosarm continues to be well tolerated with the majority of adverse events being grade 1. The median time to progression for all patients was 86 days and median duration of response for those patients with clinical benefit as best overall response was 255 days. Since one patient remains on study, the trial is ongoing and we will provide updated results once the study is completed.

We have now turned our focus to initiating two new Phase 2 breast cancer clinical trials, one in the estrogen androgen receptor positive population and another in the AR positive triple negative breast cancer population.

Next, let me provide an update on our Capesaris or GTx-758, our oral estrogen receptor alpha agonist that is being developed to treat advanced prostate cancer.

GTx-758 is being tested in two daily doses, 125 mgs and 250 mgs in men with metastatic and high risk non-metastatic castrate resistant prostate cancer. The 125 mg cohort of 38 patients was fully in rolled last year and we expect the 250 mg cohort of an additional 38 patients to be fully enrolled by the end of this month.

We have reported that in the 125 mg cohort, four of the 38 men were observed to have PSA reductions of greater than or equal to 50% by day 90. The drug was well tolerated in this cohort with no reported SAEs pertaining to the drug.

In the patients receiving 250 mg of GTx-758 who have reached day 90, seven of a 25 men had PSA reductions of greater than or equal to 50%. GTx-758 has been generally well tolerated. However, one patient on the 250 mg dose did experience a venous thromboembolic event, or VTE, which has been determined as possibly drug-related. The reported VTE was subsequently resolved.

The intent of this Phase 2 is to demonstrate that the doses of GTx-758 we are currently testing confirm the mechanism of action of the drug and have an acceptable safety profile for the treatment of men with advanced castrate resistant prostate cancer. As the study nears completion, the safety profile of the doses being tested appears to be acceptable and the mechanism of action of GTx-758 which has been observed in both dosing arms, has shown that the drug works to increase sex hormone binding globulin, or SHBG, and thereby decreases serum testosterone levels.

Across both cohorts, median SHBG levels have increased from 158% to 268% of baseline resulting in decreased levels of serum free testosterone.

Also we are seeing changes in the bone turnover markers which indicate there may be improvements in bone health. Data on hot flashes suggest that treatment with GTx-758 may improve this known estrogen deficiency side effect.

Before I turn the call over to Rob, let me conclude with a brief update on our financial results. The financial results for the quarter and year ending December 31, 2014 are included in the press release issued earlier today. I would like to point out that we reported $49.3 million in cash and short-term investments at December 31, 2014, which includes the $42.8 million in net proceeds we raised from the financing we announced in November of last year.

Our cash should be sufficient to carry us through calendar year 2016 and allow us to see at a minimum preliminary results from each of the two new breast cancer studies that we will be initiating this year.

I will now turn the call over to Rob to discuss our ongoing clinical development priorities.

Thank you, Marc. I am very excited about joining the GTx team and I would like to begin by sharing with you a few of the things that attracted me to the Company.

First, the GTx team. Not many investors make the trek to Memphis and get to meet with the team but I can tell you that I have been very impressed with the scientists and clinicians I have met along with the key experts with whom they are collaborating. This expert list is long and distinguished. To share a few names, the team is working with renowned hormonal experts Prof. Mitch Dowsett and Dr. Stephen Johnston from the United Kingdom, and Prof. William Tilley from Australia.

In addition, GTx has secured leading clinical experts as principal investigators for both breast cancer trials. Dr. Hope Rugo from the University of California San Francisco and Dr. Beth Overmoyer from Dana-Farber.

Lastly, we are fortunate enough to be working closely with Dr. Lee Schwartzberg, a nationally recognized expert in breast cancer who resides here in Memphis.

Second, the science. I am enthusiastic about enobosarm as a treatment for breast cancer especially since it is rare to have the opportunity to develop a drug in this space that already has extensive clinical data, an acceptable safety profile to date and a valuable intellectual property position. I have been impressed with the amount and level of preclinical and clinical data that have been generated to support the rationale for pursuing both breast cancer trials.

In addition to the evidence supporting our current approach for the hormonal treatment of breast cancer, there is impressive preclinical data to support exploring other indications with GTx compounds. These compounds if successfully developed in and of themselves can create significant value for the Company above and beyond breast cancer. I will be exploring with the team the best ways to maximize the development of these opportunities capitalizing on my clinical development and business development experience.

Third, the market, which I prefer to call the patient since we don't have a market without patients. As has been well documented, there is currently no cure for metastatic breast cancer and there are over 3 million women in the United States with a history of the disease. Unfortunately approximately one-third of these women will relapse from current treatments and will require additional therapies to treat their disease. This is where enobosarm could prove to be very beneficial for patients and GTx.

With regard to triple negative breast cancer, although these patients represent 20% of the total breast cancer population, this is a devastating disease where treatment is limited to chemotherapy. Again, this presents an important unmet medical need which if enobosarm can demonstrate success, it will be a significant medical advance.

In summary, we believe enobosarm provides an excellent opportunity to introduce a new hormonal treatment for androgen receptor positive breast cancer patients.

So with this background in mind, let me discuss GTx's goals for 2015 with regard to our clinical programs. We plan to start the triple negative breast cancer study during the second quarter of this year subject to appropriate regulatory clearances.

Based on the extensive preclinical data the Company has generated and given how few therapeutic options are currently available for women with triple negative breast cancer, we have received enthusiastic support from the investigators who will be involved in the clinical trial.

And the estrogen receptor breast cancer study should be again within a month or two of the triple negative breast cancer study. It is targeted for the third quarter of this year.

With regard to Capesaris, we believe data from the ongoing study is encouraging and suggests there may be a realistic path forward for future development. Following our receipt of all of the data from the clinical trials, we will evaluate potential next steps in the clinical development of Capesaris including potentially seeking a partner or collaborative arrangement in order to fund additional clinical development.

I am truly excited to be a member of the GTx team and I am optimistic about our prospects.

Operator, we are now ready to take our first question.

(Operator Instructions). Hugo Ong, Jefferies.

Hi, guys. This is Hugo speaking in for Biren Amin. Thanks for taking my questions. Just one on the breast cancer program. Can you tell us how you are defining AR positivity in the upcoming Phase 2 trial?

Yes. This is Mayzie. Hugo, we are defining AR positivity by immunohistochemistry where we are requiring greater than 10% of the cells are staying positive for the androgen receptor and that will be centrally confirmed.

Got it. Okay. And on the previous study I think you had evaluated 9 mg of enobosarm and can you tell us if you have evaluated higher doses and whether a maximum tolerated dose has been identified?

Yes, we have studied doses up to 30 mg. We used a 30 mg dose in a thorough QT study where we saw no negative effects in that study. We have also studied up to 30 mg in our multiple ascending dose study with patients dosed for up to two weeks and again with no major toxicity. As we increase the dose we do see greater reductions in HDL, cholesterol and potential for increased liver enzymes but no notable toxicities other than that.

We have not done -- if you are asking if we have done an MTD, a dose limiting toxicity study, we have not done one.

Got it, great. And just one last question. The triple negative trial is a evaluating CBR at 16 weeks. Why 16 and not say 24 weeks as in some of the other trials?

Excellent question. We have actually discussed that with many of our collaborators and steering committee. The standard as you know is at 24 weeks so that is a secondary endpoint. But because these patients progress so rapidly and the disease is aggressive, we wanted to make sure we had patients that were going to live long enough to actually achieve an endpoint especially if we receive patients who were on third, fourth, fifth rounds of therapy.

So as our primary endpoint to continue to move from Stage I to Stage II, we decided to look at a 16-week endpoint as our primary which is consistent with other recent triple negative studies targeting androgen receptor. But again for the traditional endpoint of 24 weeks, that is our secondary endpoint.

Got it. Great. Thanks for taking my questions.

I would now like to turn the call back over to Marc Hanover, CEO, for closing remarks.

Thank you, Emma. We would like to thank you all for your interest in GTx and we look forward to updating you on our progress in the future. Thank you.

Thank you for your participation in today's conference. This concludes the presentation. You may now disconnect. Good day.