Q4 2020 Oncternal Therapeutics Inc Earnings Call

[music].

Greetings and welcome to the on internal Therapeutics incorporated fourth quarter 2020 financial results call.

At this time all participants are in a listen only mode.

Brief question and answer session will follow the formal presentation.

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As a reminder, this conference is being recorded.

It is now my pleasure to introduce your host Richard Vincent Chief Financial Officer of on internal Therapeutics. Thank you Sir you may begin.

Thank you Victor.

Good afternoon, everyone and thank you for joining us today.

Joining me on the call. This afternoon are our president and CEO, Dr. James Reitmeier, and our acting Chief Medical Officer Doctor Edwina, Baskin, Bae who has been working with us as a consultant for the past several months.

We welcome all of you.

Today's call includes a business update and discussion of our 2024th quarter and full year financial results, which will be followed by Q&A.

Today's press release and a replay of today's earnings call will be available on the Investor Relations section along journal's website for at least the next 30 days.

We filed our 10-K for the full year 2020 earlier today.

Please note that certain information discussed on the call today is covered under the Safe Harbor provisions of the private Securities Litigation Reform Act, we will be making forward looking statements. During this call about future events, such as our business and product development strategies and future financial and operating performance.

Formats.

Our actual results could differ materially from those stated or implied by these forward looking statements due to risks and uncertainties associated with our business.

These forward looking statements should be considered in conjunction with and are qualified by the cautionary statements contained in today's press release and our SEC filings.

Putting our form 10-K for the full year ended December 31 2020.

This conference call contains time sensitive information that is accurate only as of the date of this live broadcast March 11 2021.

We undertake no obligation to revise or update any forward looking statements to reflect events or circumstances occurring after the date of this conference call.

With that it's my pleasure to hand, the call over to our CEO, Dr. Jim Bright Meyer.

Thank you rich and good afternoon, everyone.

Internal we are committed to developing novel treatments for patients with cancer, who have critical unmet medical needs. We are advancing a robust product pipeline with clinical and preclinical product candidates that target several such cancer indications our development.

<unk> efforts focus on biological pathways implicated in cancer Genesis <unk> progression.

During the fourth quarter of 2020, we further advanced the development of <unk> our investigational.

Potentially first in class humanized monoclonal antibody that binds with high affinity to a biologically important epitope on rore, one or receptor tyrosine kinase like orphan receptor one.

We have been working on the <unk> target in several cancer indications for a number of years.

At the end of 'twenty 'twenty, two major acquisitions took place involving companies, who are developing drugs targeting RAR, one with the Merck and company acquisition of Velo Bio Inc.

And the Behringer Ingelheim acquisition of N B E therapeutics.

The V L. S. One on one antibody drug conjugate was initially developed at our terminal and spun out develop vial and it incorporates disarm Tuesday mab antibody.

These events certainly raised the general awareness of RAR, one is an interesting cancer target.

In December 2020, we provided a data update from the ongoing phase one two clinical trial of <unk> in combination with E group net at the American Society of Hematology or Ash 2020 virtual annual meeting.

The data from 15, evaluable patients with relapsed or refractory mantle cell lymphoma, or mcl showed a 47% complete response rate and 87% overall best objective response rate or <unk>, which is an improvement from the 83% O R. R.

We had previously reported at <unk> 2020.

Six patients or 40% achieved a partial response or PR and two patients had stable disease for a total best clinical benefit rate of 100%.

Additionally, median progression free survival or PFS had not yet been reached after a median follow up of over 12 months with a 95% confidence interval above 17, and a half months.

The combination of Super Tuesday on plus Ibrutinib continues to be well tolerated with no significant adverse events above those reported for ibrutinib treatment alone.

As a point of reference for these result historical data published for 370 patients with relapsed refractory mantle cell lymphoma from three clinical trials, who were treated with single agent a bruton nib showed an objective response rate of 66% and complete response rate of 20.

<unk> percent with a median PFS of 12 eight months.

I would like to highlight that this published PFS, 95% confidence intervals for single agent a brute nib was between eight and a half and 16 and a half months and in fact, it does not overlap with the PFS confidence interval for the firm to the map plus Bruton nib combination.

Debt, we presented at Ash 2020, which was above 17.5 months are quite encouraging finding.

Based on these interim clinical results, we are in a dialogue with the U S. F. F D. A the food and drug administration to seek guidance on a potential accelerated approval pathway of course from <unk> plus ibrutinib in patients with relapsed refractory Mcl and we'll provide further updates after we have received.

Guidance from the FDA.

Additionally, at Ash, we presented data on 56, evaluable patients with chronic lymphocytic leukemia or C. L.

<unk> 49 of whom were treated with eastern twos, a mab plus ibrutinib combination.

We reported an overall best objective response rate of 92%.

Including one patient who achieved a complete response.

In addition for patients had stable disease for a total clinical benefit rate of 100%.

Median PFS was not reached for the 19 patient with treatment naive C. L. L. After a median follow up of 16 and a half months, while patients with relapse refractory CLO at a median PFS of 29 and a half months. After a median follow up of 17 months.

We expect to provide additional data from the ongoing phase one two trial in patients treated with a combination of true Tuesday may have been a brute nib in both Mcl N C. L. L. In the second quarter of 2021.

Please note that the FDA granted the company orphan drug designations for should we choose a map for treatment of mcl.

And for treatment of CLO.

Separately patient enrollment continues in the phase <unk> investigator sponsored study of <unk> plus paclitaxel in patients with her two negative breast cancer being run at UC San Diego.

Previously presented results showed an objective response rate of 57% with no new or accentuated adverse events compared to the known safety profile of Paclitaxel alone.

We expect additional clinical data from this trial to be available in the second quarter of 2021.

We have also been making progress to accelerate on terminals investigational <unk> targeting car T cell therapy program.

In January we announced that the company has entered into a research and development collaboration with the Karolinska Institute in Stockholm, Sweden designed to advance novel <unk> targeting car T and car NK cell therapies from the laboratory towards the clinic.

In addition, we announced an agreement with <unk> technology to manufacture lengthy viral vectors to support and accelerate on terminals investigational <unk> targeting car T cell therapy program.

These are these agreements are in addition to our existing collaboration with UC San Diego under a grant from the California Institute for regenerative medicine, as well as a partnership with Shanghai pharma.

We are tremendously excited by the potential of cell therapies targeting bar, one which may allow the selective targeting of tumor cells that express far one while relatively sparing healthy tissues.

Our goal is to initiate a first in human study of <unk> targeting car T cell therapy in China in the second half of 2021.

We also advanced the development of Teekay to 16, our investigational potentially first in class targeted small molecule inhibitor of the <unk> 26 transformation specific or ETS family Avianca proteins.

In the fourth quarter of 2020, we provided a clinical data update for the ongoing phase one two clinical trial of Teekay to 16 for patients with relapsed refractory Ewing sarcoma.

The data were presented at the connective tissue oncology Society meeting <unk>, which was held virtually in 2020.

Patients entering the trial had previously been treated with a median of three and as many as eight prior lines of systemic therapy.

The <unk> presentation included interim data for 23 Evaluable patients treated at the recommended phase two dose as of the October 16, 2020 efficacy cutoff date.

Two of the 23 patients or 9% treated at the randomized phase two dose achieved a complete response, which included one surgical xian.

Both patients achieving their see ours remain on treatment with no evidence of disease at over one and a half years on study and over eight months on the study.

The best objective response rate was 9%.

Eight additional patients treated at the randomized phase recommended phase two dose had stable disease for a disease control rate that is C. On a partial response or S. T a 43%.

The Teekay to 16 treatment was generally well tolerated with dose limiting toxicity of manageable mileposts mile of suppression and no obvious off target toxicity.

The continuing durability of the two complete responses in these patients who had advanced and heavily pretreated relapsed refractory Ewing sarcoma is quite encouraging along with the increased number of patients experiencing stable disease.

Recruitment in this phase one two ewing's sarcoma expansion cohort is ongoing and we expect to provide another update from this trial at Teekay $2 16 in the second quarter of 2021.

We are also.

Conducting preclinical work on other <unk>, driven tumors and expect additional data in the second quarter of 2021.

In the fourth quarter of 2020 internal significantly strengthened its balance sheet, raising an aggregate $109 million in gross proceeds from two underwritten offering. This is expected to support our operations into 2023.

I will now turn the call over to rich Vincent our CFO to review our financial results.

Okay.

Thank you Jim.

In October 2017.

<unk> awarded $18 $3 million grant to the researchers at the UC San Diego School of Medicine to advance our phase one two clinical trial evaluating from two's map in combination with Ibrutinib for the treatment of patients with B cell lymphoma, <unk> malignancies, including Mcl and CLO.

We are conducting this study in collaboration with UC, San Diego and expect to receive approximately $14 million in development milestones under research sub awards throughout the award period.

In conjunction with this award our grant revenue was $1 6 million for the fourth quarter ended December 31 2020.

And $3 4 million for the full year 2020.

Our total operating expenses for the quarter ended December 31, 2020 were $4 5 million and were $20 9 million for the full year 2020.

Research and development expenses in the fourth quarter totaled $3 million and general and administrative expenses totaled $1 5 million for.

For the full year 2020, we spent $12 5 million on R&D expenses, and $8 5 million on G&A expenses.

Net loss for the fourth quarter was $2 6 million.

Or a loss of nine cents per share basic and diluted.

For the full year 2020, our net loss was $17 2 million or <unk> 85 per share basic and diluted.

As of December 31, 2020, we had $116 7 million in cash and cash equivalents, which includes the $109 million in gross proceeds raised in the fourth quarter of 2020.

As Jim mentioned, we believe these funds will be sufficient to support our operations into 2023.

As of December 31, 2020, we had 48 8 million shares of common stock outstanding.

Now I will turn the call over to Ed weighted.

Thank you rich.

I've been working with external as a consultant for several months and I'm delighted to continue to support the team as Chief Medical Officer.

I would now like to highlight the upcoming milestones that we expect to reach over the next several months.

Expect to present, our clinical data update from our ongoing phase one two trial of <unk>.

<unk> plus ibrutinib for patients with Mcl and CLO in the second quarter of 2021.

We also expect a clinical data update to be available from the phase one investigator sponsored study.

<unk> plus paclitaxel in patients with her two negative breast cancer from the second quarter of 2021.

We further expect preclinical data and additional one expressing tumors in the second quarter of 2021.

We target treating the first patient with our war one car T cell therapy in the second half of 2021.

And that's income collaboration with Shanghai pharma.

We expect to present, our clinical data update from our ongoing phase one two trial of Teekay to 16 for patients with relapsed or refractory Ewing sarcoma in the second quarter of 2021.

And preclinical data in additional EPS driven killers.

The same timeframe.

Now I will turn the call back to our CEO, Dr. Jim Great Man.

Jim.

Thank you. Thank you very much so.

First of all the Duane on to delight to have you working with us as CMO.

Happy to have you as part of the team.

In closing on internal has a strong balance sheet and multiple potential catalysts in 2021 as well as a clear priority of deploying our financial and opposite operating resources toward promising product candidates in rare and underserved cancers.

We look forward to updating you during the remainder of 2020.

With that.

I will turn things back to Victor for the Q&A portion on this afternoon's call.

Okay.

Thank you.

We will now be having a question and answer session.

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One moment, please where we now pull for questions.

Our first question comes from Carl Byrnes with Northland Securities. Please proceed with your question.

Great. Thanks for the questions and congratulations on the progress.

Jim regarding the interactions with the FDA for potential accelerated pathway to approval for <unk> plus ibrutinib for himself is that something that you think might be possible that we'd see this year or would that be more of a 2022.

Essential of debt and then I have a follow up as well thanks.

Thank you for the question Carl.

As you know the FDA is is difficult to predict.

But if we can complete our discussions and negotiations with them.

Relatively quickly we might be in a position to announce a regulatory path forward during this calendar year.

Time will tell though.

Great I was thinking.

Assuming that the data.

We expect or you expect to present on the second quarter is compelling that would certainly help with those are those conversations I would imagine just and then a follow up unrelated do you have any feel and this is more of a question for Richard.

With respect.

The timing of the CERN grant recognition.

Throughout 2021.

And what's the total R&D spend may be for the year. Thanks.

Thanks Carl.

As you know we have not.

Historically, providing and don't plan to provide any specific quarterly or annual guidance on our spend.

Or on the grant revenue on an annual basis.

I would say state that.

With the $14 billion in total sub award.

<unk>.

We have about five quarters left under that sub award we expect these studies.

This study portion to be completed relative to the grant requirements by March of 2022.

Great. Thanks, a lot.

Thank you.

Our next question comes from <unk> Roy with Brookline Capital markets. Please proceed with your question.

Hi, True then bill on behalf of both from IRA just from Brooklyn.

Really appreciate the business update.

And just a couple of questions.

Now given the initial results.

With solid cancers with <unk> are you planning to further explore the drug for liquid hematologic malignancies.

Thank you. Thank you for the question <unk>.

Answer is yes, and so okay. We're one of the compelling things about <unk> is a is expressed on both hematological malignancies and solid tumors.

Diffuse large b cell lymphoma, or <unk> CL is is one on cancer that we haven't.

That we Havent started working on yet and we aren't we are interested in it and theyre doing some preclinical work.

Okay. Thank you just one more general question regarding enrollment for the.

On the ongoing trials as well as these expansion cohorts that are planned.

Could you just give a little flavor of how that is growing given the pandemic are there any day.

Difficulties our tips on like it is allocated is going well.

Yes, so so of course.

Like like like every biotech company, we we were.

You know we wanted to make sure that our clinical programs remained robust.

Despite the Covid pandemic, and I'm and I'm happy to say that they have.

In particular enrollment of Ewing sarcoma patient.

We remain quite strong and in fact enrollment during the pandemic was higher than it was prior to the pandemic.

We believe that has been driven by by these strong results debt that we were pleased to announce including the complete responses mantle cell lymphoma that enrollment has has slowed down a bit and it's easy to imagine that because patients with mcl are older.

They have comorbid diseases, and they're exactly those patients that might might be at risk of developing severe COVID-19. If they did catch it and so so.

There is a certain reluctance to travel to the medical center for Mcl patients. However, we have continued to enroll and we will be announcing results on additional patients above those that we last updated you on.

On at the Ash meeting.

Great. Thank you just one last one.

With regards to the exciting collaboration that Carolines Institute.

Just wondering if there are any more recent updates on debt trunk.

So what I can say is that that collaboration is active.

Where where we're doing work together exchanging information and materials and I think as you know the Karolinska has particular depth in the natural killer space in fact, NK cells were discovered there and they have developed.

It's very deep.

<unk> expertise with both on a T cell and NK based cellular immunotherapy.

So it is it is active and moving along we look forward to finding opportunities to keep you updated.

Our car T program over the course of the year, because we think that.

It's really one of the most important things that we're doing right now and we're emphasizing accelerating that program.

Sounds great. Thank you so much for taking my questions.

Thank you <unk>.

Thank you.

There are no further questions at this time I'd like to turn the floor back over to management for any closing remarks.

So everybody. Thank you very much for joining us on our quarterly update call today and we appreciate your attention and your interest in on terminal and we look forward to staying in touch with you over the course of this year with that we will sign off thank you.

Ladies and gentlemen. This concludes today's web conference you May now disconnect. Your lines at this time. Thank you for your participation and have a great day.