Q1 2020 Earnings Call
I used to clinical trial, he be 613, and the financial and operating results from the first quarter of 2020.
At this time, all participants on to listen only mode.
After the speaker presentation, there will be a question and answer session to ask a question. During this session you would need to press star one on your telephone.
If you require any further assistance please press star zero.
Now I'd like to turn the call over to John LIBOR, the U.S. be CFO of in Terror. Please go ahead.
[music], Thank you and welcome to the call.
Joining me on today's call or Adam for the our CEO Schwartz, our president of R&D, and Arthur search or our CMO.
A press release announcing the interim data from the first 50% to patients enrolled in the phase two clinical trial would you be 613 and in tears financial and operating results for the quarter ended March 31st 2020 was issued earlier today.
For those of you are not yet seen it it's available on the Investor section on our website Www Dot terabyte dotcom.
On our call. This morning, we'll share with your business update and review our financial results, which will be followed by question and answer session.
Before we begin our prepared remarks I'd like to remind you that first tickets. We made during this call about the company's future results of operations and financial position business strategies and plans and objectives for future operations are considered forward looking statements within the meaning of the federal Securities laws.
Our forward looking statements are based upon current expectations that involve risks changes in circumstances.
Assumptions and uncertainties.
In particular, our analysis of the interim data from the phase two clinical trial would you be six protein is ongoing and our conclusions regarding the Kato are subject to change based on what do you learn coming weeks.
Furthermore, developments related to the covert 19 pandemic continue to evolve and the extent tourist dependent nickel impact us in the future will depend upon the duration a magnitude of such impact and on numerous factors that the company may not be able to accurately predict.
These risks are described more fully RCC filings are available on the Fccs Edgar system and our website, we encourage all investors to read or 60 fives.
The information we provide on this conference calls from not only as of today and we undertake no obligation to update any forward looking statements. We make on this call on account of new information future events or otherwise finally, please be advised for today's call is being recorded and webcast I'll now turn the call over to Adam Brooks.
Thank you John.
And thanks, everyone for joining the call. This morning earlier today, we announced the interim three month results from the phase two clinical trial, BB 613, and osteoporosis patients along with our results for the first quarter of Twentytwenty.
Those three month P. One M.P. biomarker results for the first 50% of the patients reported this morning showed meaningful and statistically significant increases in PD one MP.
With our highest <unk> 6131, 0.5 milligram dose of one month.
We also saw dose response at one month for people one empty and believe that these data may reinforce the biologic activity of the be six one free.
Well, we are currently conducting for the analyses and evaluations of the interim data sets. We believed that we have not yet reached or maximal dose.
In addition, we believe that the upcoming bone mineral density or B.M.D. data at six months.
Well be very informative an important for further clinical development in advancing a final dose into phase three study, which we have targeted for late 2021 or twentytwenty too.
We continued to be strong believers in this program given the totality the data we have generated to date.
Particularly with the highest 1.5 times.
And the significant unmet market need for an oral therapy that rebuilds dawn.
If our preliminary observations Stan and we see meaningful B.M.D. data in the coming months from this interim patient population. We plan to study at least one higher density be 613.
The favorable safety profile BB 613 would allow for dosing flexibility with the higher dose.
What we're still working through the details we believe we can shift our resources to focus on amending the protocol for this phase two trial to obtain biomarker in BMD data with higher does the D. V 613 within our current plan spend for Twentytwenty.
For the details will be provided we continue our review I'd such data in the coming month.
For the patients in the interim data set just reported.
We plan to collect their six month, the M.D. data to determine if there's a meaningful increase in BMD for the patients receiving the 1.5 dose or other trends.
We now expect to report the six month phone marker in BMD data in the third quarter Twentytwenty.
As a reminder increases with BMD are typically associated with fracture reduction.
And are now unaccepted employing for regulatory approval worldwide.
We also continue to believe that there's a clear unmet need in osteoporosis due to the fact that only a small percentage of patients with those studies are actually treated due to cost convenience and compliance challenges.
For example, we recently completed the global market Research study, indicating that there is strong preference for physicians and patients for an oral alternative to the currently marketed injectable P.T. each product.
If we are able to generate strong relative increases in the bone mineral density endpoint as compared to other products.
We believe there may be a substantial market do you need for he be 613.
Subject to the successful outcome of any potential future clinical trials.
As a reminder, at our last update on March 26, 2020, we announced we had enrolled 98 patients.
And as of today, we have 102 patients and were it not for cobot 19 related delays.
We're on track to achieve the objectives that we had set in 2019 to enroll and complete the phase two clinical trial would be 613 and twentytwenty.
The data, we announced today with a planned interim step in advance of the third quarter completion of enrollment and her subsequent plan to report the final bond market data for all of the 160 patients.
Plan to have been involved in this study and the six month bone mineral density data.
As we evaluate the DMD another data from this study we believe we'll have a better understanding what additional dosing levels, we need in order to initiate a potential phase three studies.
Our assessment of the data that we shared with you today and the potential proceed to higher doses includes feedback from leading outside clinicians.
I have designed and conducted osteoporosis clinical trials.
Operationally, we will be carefully monitoring or spending with the goal of continuing to extend our cash runway I prioritizing various activities.
But the planned reduction in spending our current cash is sufficient to support our planned operations into the second quarter 2021 and.
And we believe this timeline, maybe extend it doesnt necessary depending on the outcomes of the upcoming B.M.D. data released from the third quarter of 2020.
We also continued to entertain interest in our underlying technology platform from potential collaborators and partners.
We've continued to make good progress on the be 612 for Hypo Parathyroid December.
Our previously published data, we confirm that world P.T., because effectively deliver again, the blood stream and activate the p. teach dependent biological pathways that are inadequately activated than patients what type of parathyroid does from.
Philip will talk a bit more about de CIX one to later in the call.
In addition to advancing your internal P.T.H. programs. We also continue to support preclinical work on the first molecule cover buyer collaborations with Amgen.
We're pleased with the progress we've made and are working with Amgen to move the research and development program forward in accordance with Amgen's project plans and objectives.
I'd like to now turn the call over to Dr. Arts and tour, our CMO to discuss the phase two trial would be 613 arc.
Thanks, Adam.
The phase two clinical trial is a dose ranging placebo controlled trial and female patients with osteoporosis or low BMD and is being conducted for leading medical centers in Israel.
Patients were randomized to receive either a placebo or one of three doses of Hebei 613, <unk> 0.5, milligram 1.0 milligram and 1.5 milligram.
We trust these doses based on our prior pharmacokinetic data showing the maximum concentration of P.T.H. and blood.
Max and area under the concentration time curve you soon afterward, <unk> 613, and believe these doses would encompass potential optimal dose equivalent to a 20 microgram injection for China.
Total enrollment was targeted.
160 patients with approximately 40 patients in each arm.
As a reminder, this trial was designed to evaluate the effects you'd be 613 on multiple biochemical markers, including he wanted to.
Hey, bone formation marker.
CTX the bone resorption, Mark for three and six months as well as a standard set of safety assessments.
We're also evaluating the impact of T V 613 on bone mineral density.
Indeed after six months.
He wanted and piece German concentration is an indicator of the rate of new <unk> formation.
In CTX CIRM concentration as an indicator of the rate of old bone resorption by us to your class to sell to remove all gone.
He T.H. and analogs are OSU anabolic because they stimulate bone formation as measured by few wanted see much more than they increase bone resorption, which is measured by CTX.
It is the relative difference between bone formation and bone resorption that determines the MD increases and the balance of modeling and remodeling is an important factor in our consideration for our next steps with this program.
Other endpoints, including the study include additional markers of bone formation and bone resorption in a variety of other measures three and six months. The limited interim analysis of changes in few weren't in tea and C. Checks are the first 50% of patients at three months was part of a trial design and.
Perspective, we plan.
Finally demographics from this trial are consistent with other previously reported Charleston literature.
The ability of our investigators to follow their patients has been excellent 72 of the 80 patients randomized completing month three visits even where the coated 19 restrictions and there have been no serious.
Product safety related adverse events or drops in the trial.
Overall, our safety profile appears to be favorable and would support higher doses.
On the data available today.
Our analysis of the data as reported in today's press release indicate that the P. One M.P. biomarker data after one month of treatment with E. B 613 was quite favorable with the 1.5 milligram dose.
And was not associated with an increase in CTX.
We also saw a dose response at this time point, but it was clear that we had not yet achieved the maximally effective dose.
The biomarker data for both P. One MP and CTX collectively as well as placebo response.
Three months clearly require additional analyses of all available data by experts over the coming weeks.
And the third quarter of 2020, we intend to analyze the six month DMD data.
As it becomes available.
These data will inform our continued clinical development plans for you'd be 613, and our planned phase three study design, which we have targeted for 2021 or 2022.
I'll now turn the call over to Dr. Phillip Schwartz, our president of R&D share some updates with you on Hebei 612, and our Amgen program.
Thank you very much ARQ <unk> good morning, everyone.
I would like to provide you with a brief update on ebay fixed level for the treatment of the orphan disease Hypoparathyroidism.
As a reminder, our goal is to treat patients acute symptom, while normalizing serum and Europe calcium levels to minimize the long term effects of supplement juice.
We believe this would be applicable to patients with different levels of disease severity.
Based on interest from several parties and the announcement of data from other competitive trials in hyperthyroidism.
We recently conducted additional work on easy six fault.
Polluting the identification formulation enhancements that we believe it provides a strong foundation to advance this program to evaluate its ability to become a first in line therapy in a market that significant unmet.
As part of this work we have focused on a treatment algorithm that would replace P.T.H. at physiological levels, Ralph each day to better control. Both the short term symptoms and the long term complications of the <unk> without the need for subcutaneous injection.
As part of this work we are evaluating the possibility that twice or three times daily dosing main mimic endogenous pulsatile P.T.H. activity and therefore result in a better PK PD profile.
If we are able to extend the overall exposure and you see for each does maybe able to increase the call seemed like effect, while also limiting the risk of hypercalcemia.
We believe that a longer tail as measured by agency may provide more controlled supplements and urinary calcium.
Our goal is to finalize those formulations and preclinical work over the next six to nine month, and then determine our next clinical development steps to advance this program to a worldwide phase three trial.
Our collaboration with Amgen for the development of an anti inflammatory age has continued and we are pleased with the progress we have made today.
We're continuing to support the collaboration and Amgen has completed several studies that have included the evaluation different formulations of their truck.
We believe our technology is performing well and that we have been able to rapidly create different formulations with different the PK PD profiles to support amgen's activity.
We also continue to focus on the development of our platform as it relates to devaluation of new Apiay.
Those are new pharmaceutical ingredients and believe that these efforts they have the potential to generate value to their additional validation of our technology platform and work through potential business development activity.
I'll now couldn't or with a call to Jon Lieber, our U.S. CFO to cover the financial results.
Thank you fill up.
Revenue in cost of revenue for the quarter ended March 31st 2020 were $42000 and were solely related to the research and development services provided to Amgen.
There was no revenue or cost of revenue in the quarter ended March 31st 2019, as the plans for our work to support the collaboration we're still in the planting seeds with Amgen.
Total operating expenses for the three months ended March 31st 2020 were 2.9 million can included $1.6 million in research and development expenses and $1.3 million in general and administrative expenses.
Research and development expense for the quarter ended March 31st 2020 consisted primarily of head count related costs external costs related to the conduct of you'd be 613 phase two clinical trial and consulting expenses and fees related to the preparation of the 90 application for 80 613.
General and administrative expense for the quarter ended March 31st 2020 was primarily made up of salary and related expenses, including share based compensation professional fees do you know insurance expense and legal fees.
Nick comprehensive loss was 2.9 million were 16 cents per ordinary share basic and diluted for the quarter ended March 31st 2020.
As a reference point, we currently have approximately 18 million primary shares outstanding and 26 million fully diluted shares outstanding.
At March 31st 2020 enter had cash and cash equivalents or 13.3 million and in our 6K that we intend to file today. After the U.S. financial markets called it was reported approximately 11 in a half million in cash cash equivalents as of May 12 2020.
Based on current operating plants, we expect our 2020 operating loss to be approximately $10 million Wes.
This is subject of course, the expected timing of product development plans, including you'd be 613 and subject to any continuing impact of covert 19 on our operations.
As a result, we currently believe our cash position will fund their operations into the second quarter of 2021.
I'll now turn the call back to add them for concluding remarks before we go to QNX.
Thanks, John we appreciate everyone's patience and support as we evaluate the results of the interim data from the he be 613 phase two clinical trial.
Our upcoming six month biomarker and B.M.D. data.
And our higher dose arms expected in the coming month.
We have been aggressively managing our financial and human resources with the goal of maintaining financial flexibility during the cold that 19 crisis.
And plan to continue to do so.
We also continue to bleed the market opportunity in need for better therapies in the convenience coral format is substantial.
This has been supported by recent market research and clinician feedback that such a world farms could be their product of choice for patients with moderate to severe osteoporosis.
We also believe our technology platform offers several benefits to potential collaborators set up expressed interest in our underlying synergistic patent protected combination of protease inhibitors and absorption enhancers to both deliver and protect macro molecules.
We remain committed to opportunistically advancing those programs, while protecting our financial resources with the goal of advancing to phase screen as soon as practicable as we select our final ducts.
Operator, please open up the line for question.
Thank you Sir.
Just to ask a question you would need to press star one on your telephone.
Question first the pound key please standby, while we've compiled acuity roster.
I show up first question comes from the line of Jason Mccarthy from Maxim Group. Please go ahead.
Hi, everyone have a day on the line for Jason Thanks for taking my questions.
As you guys make progress with regard to your yeah with regard to give you 613 and he'd be six one too I just wanted to see if you are you guys planned up on a onboarding any clinical trial sites in the U.S. are you.
Hi, Dave Great question. Thanks for joining the call. Indeed, we do as we think about the phase three trial, whether it be for he be six one degree or he be six one to the benefit of course in the five OPI be two pathway in the keys to be 613 is that we can do one phase three trial based on feedback that we've already gotten from the.
FDA, we would envision that this would be a global registrational trial, we've noticed that we've been in the process of preparing for an I can be with the FDA based on all of our previous very positive conversations and ideally we'd like to be able to do a trial in the United States in Europe, and then also consider opportunities in the Pacific rim.
Given that the regulatory environment is such that you're starting to see greater global cooperation, we'd rather be able to do one trial that would be sufficient for global approval. So indeed that is part of our plan arc of course has worked in the space for a long time and we've already been starting to consult experts in that process.
Great. Thanks for the additional clarity and then with regard to the timeline for you'd be six one can be so I just want to make sure I heard this correctly, we can expect A.B.M.D. data read out in Threeq, you 20, and then on the Guy that you guys expect to initiate the phase three trial and either late.
21, or early 2022 is that correct.
That's correct, Dave that's very consistent with the guidance that we had provided previously obviously cope it had an impact initially over the last couple of months, we've actually seen Israel start to return to a more normal cadence patients are starting to return back to the hospitals the hospitals and starting to open up so that we can get some of the bone mineral done.
The data, which is collected be a DEXA scan and we anticipate the timelines will hold where we will be collecting now the six month bone mineral density data in the third quarter and that's based on the first 50% of the patient that we reported today those patients had largely come into the trial by the early part of 20 Twond.
And that takes us out into the third quarter for that we also anticipate that we wouldn't be completing enrollment, but the remaining roughly 60 patients left to come into the study, including potentially with the higher dose and then as we follow those patients we would anticipate that that data would support initiation of phase three trial potentially as early.
Lee as late 2021 or early 2022 solar plants are in place cobot had a moderate impact on enrollment as we disclose it back in March but we're starting to now come out of that patients are starting to screen and then we'll determine which doses appropriate that take further in the study.
Great. Thanks, and then we could just switch gears here briefly to you'd be six one too could you just shed some color on what parameters are currently being.
And I end up that would be needed to support continued progress into phase to be or a phase three things.
Sure happy to do so and then I'll turn over to fill up so at the top level. We had reported our phase two PK and PD data back in the fall of 2019 and that was a direct comparison against the high instances not para which is the only product that it's been approved for Hypoparathyroidism unfortunate that product is on.
Recall right now and we are in the process of determining a couple of formulation enhancement. So that we can come up with an ideal profile that may actually treat not only the symptoms, but ultimately the resulting downstream effects of the disease. Philip you want to comment a little bit more on the planning work that we've been doing and trying to identify the optimal profile that maybe.
Make this a first line treatment opportunity.
Yes, Thanks, Adam Yes, Indeed, we're looking at re formulation under the re formulation that we have is not necessarily be particularly drastic.
We believe that we can link them at the tail, which is how long of the drug glass them the blood stream.
And that's primarily responsible for the kill seemed like a theme with her thirot dth. Additionally.
Because of this longer tailed we anticipate or there's a good part a decent probability that we'll be able to reduce the it the frequency of dosing, meaning that the dosing for milder patients maybe.
The idea and the dosing for more severe patients who are who need to control urinary calcium maybe t. I'd or three times a day I just want to remind everyone in hypoparathyroidism did extremely heterogeneous disease.
That.
Activate and portrays itself very differently for different patient and therefore that the idea of having all of these different doses available and having the flexibility of changing the frequency and the doses.
It's very very compatible with the patient profile of hyperthyroidism and could be very useful than treating these patients.
Thanks.
Great. Thanks to the additional clarity guys appreciate it.
Thank you Dave Thank you.
Thank you I shouldn't or further questions in the queue at this time I like to turn the call over to Mr., Adam Gridley CEO for closing remarks.
Excellent thanks to everyone for taking the time. This morning. We appreciate you joining the call. We look forward to providing you with an update on our progress overall and on the company's progress as we move toward thanks, So much have a good day.
Ladies and gentlemen, this concludes today's conference call. Thank you for participate and you may now disconnect.
[music].