Q3 2019 Earnings Call

Why are actual results may differ materially from what we project today due to a number of important factors, including the considerations described in the risk factor section of our form 20, Dusza filed with the F.C.C. on February 25th 2019.

And in other filing specially meta soulmates with the S.P.C. from time to time.

These forward looking statements represent our views only as of today and we caution you that we may not have to come in the future whether as a result of new information feature about or otherwise and now I'd like to turn the call over two Julian.

Okay.

Thanks to everyone, taking the time to join us so.

I'd come either so we are committed to finding cures for patients with blood cancers, and rare seriously middle logic diseases through the development of next generation cell therapies.

Are most advanced product candidate only do this though isn't faced with development and could offer he likes saving treatment option for patients in need of a bone marrow transplant.

Behind him to do the so is G.D.A. toward one hour expanded natural killer or encased soul developing candidate and immunotherapy with potential in both human logic malignancies solid tumors.

We have continued to make important progress across both programs.

Turning first to overdo. The so this is the first bone marrow transplant product to receive breaks through therapy designation from the F.D.A.

There's also received orphan drug designation in the U.S. and in Europe .

Despite the cure the potential of bone marrow transplant. This estimated that more than 40% of eligible patients do not receive a transplant for a number of reasons, including the inability to find in matched donor.

Additionally, bone marrow transplant has risks, including the risk of infections and other complications such as graft versus host disease.

We believe that omen Dubis l. has the potential to address these limitations and to improve treatment outcomes for patients who do not have imagined related donor.

Or international Multicentric random I I was on me do Purcel is designed to evaluate the safety and efficacy.

<unk> compared to standard umbilical cord blood impatience with high risk middle logic malignancies.

We expect to complete enrollment by the end of this year, enabling a top line data read out in the first half of 2020.

Upon positive data, we anticipate submitting the B.L.A. for only to be selling the U.S. and the second half of 2020 with potential F.D.A. approval in launching 2021.

We will use a common technical document for our regulatory filings, which enables us to streamline the work required to file a marketing authorization application or M.A. <unk> in the U.

We're also advance in key activities required to bring home would do besides the patients following potential F.D.A. approval work is ongoing to build the manufacturer infrastructure in both at lunch and our own facility.

Help ensure that we will have sufficient and reliable commercial supply.

We are also working to develop comprehensive hospital services and patient assistance programs designed to bring home and do the cell to patients.

Additionally, we are evaluating on would do this out and then investigator sponsored phase one to study impatience with severe a plastic anemia, a rare in life threatening blood disorder.

Earlier this year, we reported initial data from the first cohort of three patients at the transplantation and solar therapy being or the T.C.T. meeting.

The data showed that all three patients successfully underwent a bone marrow transplant consisting of comedy <unk>, plus a haploidentical stem cell graft.

Patients are now being then rolled into the second cohort, which is designed to evaluate on we do this l. as a standalone graft.

Turning to G.D.A. tool one we're encouraged by the early data generated in our phase one investigator sponsored study in patients with non Hodgkin's lymphoma.

Multiple my one.

Emboldened by the multiple complete responses reported earlier this year, we are developing it cryopreserved formulation to enable a multi centered multidose phase one to clinical study.

Patients with non Hodgkin lymphoma next year.

Both almond Dubis L. and G.D.A. tool one were created using our proprietary Nam technology platform, which is designed to enhance the number in functionality allogenic don't herself.

This represents an important advance because preserving therapeutic functionality has been a key limitation to prior approaches.

Next month, we will present data on G.D.A. to a one and on our Nam platform at the annual meeting of the American Society for him apology or ash.

Dr. Veronica Bocca, Nova will provide updated results from G.D.A. tool one study during an oral session on Monday December 9th.

That same day, we will also report new pretty clinical data that reinforce our understanding of the mechanism of action underline hour nano technology platform.

Last summer, we appointed Tracy loaded.

As Chief Scientific Officer. She has previously work at Blue Rock Ciros unsanitary Genzyme and has been instrumental in advancing several developing programs through regulatory approval in the area of transplant and auto immune.

Crazy obtained a Phd enemy analogy and pathology and completed it post doctoral fellowship at the Beth Israel Deaconess Medical Center in the Department of Human Psychology and college.

We're experiencing transplant biology, and Immunooncology research further enhances the team and she's already making an important impact on our research strategy.

Now turn off the called <unk>.

Our Chief Medical Officer, and Tracy to review, our Ash abstracts in more detail or neat.

Thank you Julianne and good afternoon to everyone joining the call today.

I'm pleased to summarize the abstract for G.D.A. to align and provide an update on what you can expect to see at the last meeting next month.

Taking a step back natural killer or in case out have potluck antitumor property.

Increasingly being recognized as a potentially transformative approach to immunotherapy.

The tumor killing activity of in case out is greatly enhanced by antibodies that recognize tumor cell, which trigger anybody dependent cellular cytotoxicity or 80 C.C.

Binding them, an antibiotic to tell market for destruction by activated encased out.

However, a key limitation to the therapeutic utility of N.K. fills in the past has been a bill is below the inability to generate sufficient numbers of highly functional though in culture.

We are using our Nam technology potentially overcome.

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This first in human study is designed to assess the safety of three increasing doses of G.D.A. to align impatience with non hodgkin lymphoma, and multiple myeloma as well as to determine the recommended faith to do.

The study of being conducted by Dr. Veronica backing over at the University of Minnesota.

The abstract submitted to ash, which was accepted for all presentation and could data from 20 patients seven patients with non Hodgkin lymphoma, and 13 patients with multiple myeloma.

First it's important to know the G.D. two one was generally well tolerated with no dose limiting toxicities observed.

We were also pleased to observe clinical activity at all doses evaluated which is particularly encouraging to seen in that population of heavily pre treated patients would advance to neat.

A mouse seven patients with non Hodgkin them pharma there were three complete responses into partial responses.

Duration of response was 12 month for the complete responders and five months for patients with a partial response.

One of the patience with a history of C.L.L. and richest transformation should continue to tumor shrinkage post therapy.

This is clinically very interesting because it suggests that G.D.A., two or one elicits an adaptive immune response.

Among the 13 patients with multiple myeloma, one patient had a complete response and forehead stable disease.

Next month and oral presentation. We expect to include longer term follow up for the patients already reported as well as data from additional patients who are enrolled in a valuable since the time of abstract submission.

As Julie mention these data compel us can move forward with our clinical development plans and activities are underway to enable an I.D. submission for our next study in non Hodgkin lymphoma.

Oh now turn the call over to Tracy to review, our pre clinical abstract.

Thank you will need in good afternoon, everyone.

During a poster session. It as we were good point, new pre clinical data that reinforce our understanding of the mechanism of action underlying our Nam technology platform.

Our research included transcriptome transcription factor and halfway analysis.

By next generation sequencing to elucidate happily leading to the preservation of and graph man after enviable expansion of only Dubis l. compared to C.D. 30 fours bells grown in the absolute the man.

Rodney we found that Nam down regularly gene expression path.

Involved in the proliferation in differentiation themselves and regularly gene expression happily yep Yep.

I want that highlight.

Fine.

First we saw reduced expression of Jean involved in in production of reactive oxygen and nitrogen hmm.

Which we feel that land and minimize notes that during bands interested press differentiation.

Second Nam Cosby decrease in the expression of being responsible a matrix.

Hello quote needs secretion.

We feel that simulate the Michael environment of the bone.

There, we had increased ration until armory.

Enabling no to remain in a more quiet.

Them like.

Taken together, we believe that the president Nam mimic the Hyde Park environment of the ball now <unk> grappling ability of stem cells.

Data provide scientific rationale for the neighborhood <unk> outcome European and previously reported see one two clinical study alone.

We are really pleased to build in our scientific and ending of our technology platform, which we believe.

In any no.

Yeah. Continuing you then have mechanism of action for only do this out to characterize and the cat.

Reduced during the expansion profit.

And we are beginning to conduct similar mechanism of action study with G.D.A. to unwind.

With that I'll turn the call over just shy to review our financial results.

Thanks, Racy and good afternoon, everyone.

We are believe today advancements we have made so far this year, we said the foundation for multiple key microphones in 2020.

This afternoon I will review our financial results for the third quarter of 2019, who can find our in nine nine months results for the U.S.B.C. fighting.

As of September Dirtier 2019, we are total cash cash equivalent in available for so assets will 68.1 million compared to 60.7 million as on December 31st 2080.

The reminder, we computed following altering earlier this year, we to aggregate gore's precede or 40.3 million.

Research and development expensive for the third quarter of 2019 for 7.4 million compared to 5.1 million for the same period in 2018.

This increase reflects the advancement for me to be so and G.D. 821 programs.

Generally an administrative expenses were 4.6 million for the third quarter was pretty 19 compared to 2.4 million for the same period in 2018. They increase was mainly due to at one point to me the increase in activities related to commercial readiness as well as well as one of them an increase in professional services and other expenses, including the.

Increase in expenses associated with being a publicly traded company.

Finance income that was 1.7 million for the third quarter was pretty 19 compared to finance expenses net of 2.2 million for the same period in 28 in the.

The net increase was primarily due to non kish income, resulting from the reverberation of weren't offered by noncash expenses from the <unk> from the reverberation of the Israeli innovation authority World comparing gruntal ability.

And that's also the third quarter of three maintain was 10.1 million compared to a network of 9.8 million for the same period in 2018.

We continue to expect guess useful ongoing a breaking activities in 319 to range from 35 to 40 million, reflecting anticipated expenditure to advance the company's clinical programs.

With less than one quarter to go <unk>. We can tell you that we expect to be in the upper end of this range.

We anticipated already current total cash position with the border ongoing operating activity into the fourth quarter of 2020.

This cash runaway guidance is based on our current operational Glen and exclude any additional funding or business development activities. We did I will turn to go back over to Julian.

Thanks shy.

This has been an important executional year program in itself.

With all maneuver so.

We have the opportunity to bring the first F.D.A. proof cell therapy for bone marrow transplant station to patients were completing patient enrollment interface. We study.

Working on the B.L.A. filing and advancing key launch readiness activities required to reliably bring on would do so for patients.

With Judy a tool one we are at the forefront of advancing incase else as the next generation immunotherapy.

We're working on several activities to enable a multi centered study of G.D.A. tool one in not Hodgkin's lymphoma next year.

We have a strong came in place committed to progressing these programs and look forward to updating to providing updates on our progress over the coming months.

Now we will open the call for questions operator.