NeuBase Therapeutics, Inc.
Q1 2017 Earnings Call Transcript

Published: 14 Feb 2017

[Operator:]

Greetings and welcome to the Ohr Pharmaceutical Company's First Quarter 2017 Earnings Call. At this time, all participants are in a listen-only-mode. A Question-and-Answer Session will follow the formal presentation. [Operator Instructions]. As a reminder, this conference is being recorded. I would now like to turn the conference over to your host, Mr. Paul Amdt, Managing Director of LifeSci Advisors. Thank you, you may begin.

[Paul Amdt:]

Thank you Melisa, good morning everyone. This morning Ohr released financial results and provided a business update for the quarter ended December 31, 2016. If you do not yet receive the release, it is available on the Investor Relations section of the company's website at www.ohrpharmaceutical.com. This call is being webcast and a replay will be available. Before we begin, I would like to remind you that some of the information contained in the news release and on this conference call contain forward-looking statements that involve risks, uncertainties and assumptions that are difficult to predict. Words of expression that reflecting optimism, satisfaction of current prospects as well as words such as belief, intend, expect, planned, anticipate, and similar variations identified forward-looking statements, but their absence does not mean that the statement is not forward-looking. Such forward-looking statements are not a guarantee of performance and the company's actual results could differ materially from those contained in such statements. Several factors that could cause or contribute to such differences include our ability to raise special funds to perform any conclude clinical trials, the financial resources available to us, our ability to negotiate and conclude a strategic partnership agreement, the future success of our scientific studies, our ability to successfully top products, rapid technological change in our market, changes in demand for our future products, legislative regulative definitive involvement and general economic conditions. These factors are described in details in the company's most recently filed annual reports in Form 10-K subsequently filed Form 10-Q and as well as additional filings with the SEC. These forward-looking statements speak only as if today's date Tuesday February 14, 2016. The company undertakes no obligation to publically update any forward-looking statements or supply new information regarding the circumstances as if the date of this release. Participating in today's conference call from the company are Dr. Jason Slakter Chief Executive Officer and Mr. Sam Backenroth Chief Financial Officer. With that, I would like to turn the call over to Dr. Slakter. Please go ahead sir.

[Jason Slakter:]

Thank you very much Paul and good morning everyone, thanks for joining us today. The focus of my discussion today is to provide a strategic update on the company and talk about the ongoing clinical trial investigating our redevelopment candidate Topical Squalamine also referred to as OR-102 for neovascular age related macular degeneration or wet AMD. On the business front, we remain in active ongoing discussions with potential pharmaceutical partners. As we have outlined before, our ongoing clinical program was designed to examine the potential of Squalamine when administered as part of a combination therapy to significantly improve visual acuity in patients with wet AMD. We began enrolling patients in the first trial in April 2016. In Parallel, with ongoing business development discussions for strategic reasons, as of today we have paused enrollment in this study. I want to sate clearly that the enrollment pause is not related to any safety issues. Currently there are more than 200 subjects enrolled in the trial. This study remains double masked and no interim efficacy or futility analysis has been performed. Per the current protocol, those subjects currently enrolled in the study continue to undergo scheduled visit as assessments as well as receive assigned study treatment. Since we began this trial, the competitive landscape for new drugs in AMD has changed. In particular for combination therapy approaches to treatment. We believe the failure to show additional visual benefits in these other combination therapy studies does not impact the promise of Squalamine combination therapy to improve vision in patients suffering from wet AMD. Given what we have seen, why do we feel this way? Recall there are several key differences between Squalamine and these other experimental agents both in terms of the mechanisms of action of the therapeutic agent, as well as the design of the clinical program. First, Squalamine is a topically applied drug that inhibits multiple targets involved in ocular angiogenesis including VEGF, PDGF and basis FGF. Second, while other combination therapy agents exert their effect in the extracellular space, Squalamine acts intracellularly to inhibit the downstream activity of a number of angiogenic receptors. Third, topical application of Squalamine on a twice-daily basis can provide for sustained biologic activity in the target tissues in the back of the eye. This is an important differentiating feature of Squalamine and may result in a better therapeutic effect compared to other drug they are given us bolus injection into the vitreous. Fourth, a key factor in driving visual outcomes with combination therapy is the need for careful patient selection. In our exploratory Phase II study, we determined in a retrospective analysis that the visual acuity benefits of Squalamine combination therapy were driven by the size of the occult CNV present at the start of treatment. This analysis allowed us to select and optimize population most likely to show benefit from Squalamine combination therapy and it is this population that was enrolled in the current trial. Finally and importantly, the visual acuity outcome data from the control group in our Phase II study was very similar to the PRN Lucentis arm of a CAT trial giving us confidence that the visual acuity benefit seen with Squalamine combination therapy were real and potentially replicable in future studies like this one. Our decision to pause enrollment was made in order to prospectively demonstrate the visual acuity benefits of Squalamine combination therapy in this selected population in a shorter timeline. This will result and are having efficacy data from the ongoing trial by the end of calendar 2017, which was almost a year earlier than we had previously planned. We believe this is the right approach to maximize shareholder value and preserve company resource. In conclusion, we remain very confident about the potential of Squalamine as a differentiated topical, multi-target, angiogenesis inhibitor to provide improved visual function to patient suffering from wet AMD. To summarize, our current plan is to pause enrollment with over 200 patients, continue those patients in a double-masked manner, continuing on protocol specified visits and treatment regimen and assess efficacy data before the end of calendar 2017, which if positive will allow us to continue on the path for regulatory approval with little or no delay in the original overall timeline. With that, I would like to handover the call to Sam for the financial results. Sam.

[Samuel Backenroth:]

Thank you Jason. For the first quarter, ended December 31, 2016, total operating expenses were approximately $7 million consisting of $1.7 million in general and administrative expenses, $4.9 million in research and development expenses and $0.3 million in depreciation and amortization. This compares to approximately $3.6 million in total operating expenses in the same period of 2016 consisting of $1.2 million in general and administrative expenses, $2.1 million in research and development expenses and $0.3 million in depreciation and amortization. For the first quarter 2017, we reported a net loss of approximately $7 million or $0.21 per share compared to a net loss of approximately $6.1 million or $0.20 per share in the same period last year. At December 31, we had cash and cash equivalence of approximately $13.5 million, this compares to approximately $12.5 million at September 30, 2016. For a more detailed description of the financials, I would encourage you to refer to our 10-Q which should be filed shortly this morning. That completes my review of the financials. I would like to now open up the call for questions. Operator.

[Operator:]

Thank you. At this time, we will be conducting a Question-and-Answer Session. [Operator Instructions]. Thank you, our first question comes from the line of Corey Davis with H.C. Wainwright. Please proceed with your questions.

[Corey Davis:]

Hi good morning, thanks. Could you please spend a little more time on any changes you are going to have to make to this statistical analysis plan, given kind of the change in the patient numbers, how the powering of this study will now change with only 200 patients? And based on previous Phase II, what gives you the confidence that you can hit statistical significance with this number of patients. And remind us what level of difference between the two groups would be deemed to be clinically relevant?

[Jason Slakter:]

That's a great question Corey and I think it's going to be the question that a lot of people were asking, which fundamentally is what does this study get us in terms of data. And I think the way to answer it is to realize that when you design the Phase III program as we have indicated that we were enrolling 650 patients to the original overall population in our Phase III. You obviously power it for a very high level number, such as around 90% power. That doesn't mean you can't show statistical significance with the smaller population. I think the most important thing to remember is to recall our Phase II study. In this selected population, we had 94 patients in our Phase II trial that met the inclusion criteria that we are using in this current study. In that Phase II trial, we saw a difference of 5.3 letters in favor of Squalamine combination therapy over Lucentis monotherapy. And even with just 94 patients, we had an exploratory P value of 0.33. With more than twice that number already enrolled in this current trial, we feel we are quite well enabled to confirm these Phase II results in a predefined in prospective manner.

[Corey Davis:]

So in other words, if you hit the same effect size that you did in your analysis to the Phase II where this number of patients being a little more than 200, you should easily hit statistical and clinical significance. Didn’t want to put words in your mouth, but want to make sure that that's what you are saying?

[Jason Slakter:]

Yes. That’s okay. I think it's fair to say that if we have similar sizes with more patients, we would expect that the P value is going to be below the level of that which is considered significant not as non-statistician, I would never attempt to use any words that would not be verified by our statistical team. But clearly having a P value in that same range of 0.03 or below would certainly validate the visual efficacy that we are seeing in that study.

[Corey Davis:]

Okay and last question would be, given this kind modification to your protocol. Do you need to file anything formally with the FDA, and if so, do they need to sign off on it or is this just your decision and you will inform them when the results come out?

[Jason Slakter:]

Well as we have said, the only decision that’s been made is a pause in enrollment. So that's the only decision that has been made at this point. We will be having ongoing dialogs with the FDA over the coming weeks as we move forward and certainly we will take their advice and input in any decision that we make.

[Corey Davis:]

Okay. That's all I had thanks very much.

[Jason Slakter:]

Thank you very much.

[Operator:]

Thank you. Dr. Slakter there are no other questions at this time. I would like to turn the floor back to you for final remarks.

[Jason Slakter:]

Thank you operator. In concluding this morning's call, I just want to speak not only as the CEO and one of the largest shareholder of the Ohr, but also as a practicing clinician. As such, I feel it's critical that we get the answer as to how effective Squalamine is in improving visual function above that which is achieved with current standard-of-care. We need this answer not just for the medical and scientific community and not just to maximize value for our shareholders, but also for the vast number of patients that could benefit from a topical angiogenic inhibitor like Squalamine, if our trial is successful. I want to thank all of you for joining us on the call today. And we look forward to having discussions with investors face-to-face over the coming weeks, as we are confident that this is the right move and the right plan to move forward. Thank you all and have a great day.

[Operator:]

Thank you. This concludes today's teleconference. You may disconnect your lines at this time. Thank you for your participation.