Q3 2022 Acasti Pharma Inc Earnings Call
[music].
Yeah.
Speaker 1: Ladies and gentlemen, and welcome to the Acosta pharma third quarter fiscal 2022 business update.
Good day, ladies and gentlemen, and welcome to the third quarter of fiscal 2022 business update conference call.
Speaker 1: At this time, all participants have been placed on a listen-only mode, and the floor will be open for questions and comments after the...
At this time, all participants have been placed on a listen only mode and the floor will be open for questions and comments after the presentation.
Speaker 1: It is now my pleasure to turn the floor over to your host, David Waltman with Investor Relation.
It is now my pleasure to turn the floor over to your host David Waldman with Investor Relations.
Sir the floor is yours.
Speaker 2: Thank you, Holly, and good afternoon, everyone. I'd like to welcome you to Acosta Pharma's fiscal 2022 third quarter conference call. On the call with us this afternoon are Jan De Alvis, president and CEO , Brian Ford, chief financial officer, Dr. Pierre Lemieux, chief operating officer, Canada, and chief scientific officer, Dr. George Cattai, chief operating officer, US, and Prashant Kohli, vice president of commercial operations.
Thank you Holly and good afternoon, everyone I'd like to welcome you to our coffee farmers fiscal 2023rd quarter conference call on the call with US. This afternoon are Jan <unk>, President and CEO , Brian <unk>, Chief Financial Officer, Dr. Pierre Lemieux, Chief Operating Officer, Canada, and Chief Scientific Officer.
Sir Dr. George <unk>, Chief operating Officer U S and Prashant Coley, Vice President of commercial operations. Following our prepared remarks there'll be a Q&A session should any questions remaining after the call. Please contact crescendo communications at 2126711020, I'd also like to remind everyone that statements on this conference.
Speaker 2: Following our prepared remarks, there will be a Q&A session. Should any questions remain after the call, please contact Crescendo Communications at 212-671-1020. I'd also like to remind everyone that statements on this conference call that are not statements of historical or current fact constitute forward-looking information within the meaning of the Canadian securities laws and forward-looking statements within the meaning of the U.S. Private Securities Litigation Reform Act of 1995 and the security of the United States of America.
They are not statements of historical or current facts constitute forward looking information within the meaning of the Canadian securities laws and forward looking statements within the meaning of the U S. Private Securities Litigation Reform Act of 1095, and the Securities Exchange Act of 1934, such forward looking statements involve known and unknown risks uncertainties and other unknown factors that could.
Speaker 2: such forward-looking statements involve known and unknown risks, uncertainties, and other unknown factors that could cause the actual results of a cost data to be materially different from historical results or for many future results expressed or implied by such forward-looking statements.
Cause the actual results of our cost seem to be materially different from historical results or from.
Any future results expressed or implied by such forward looking statements. In addition to statements, which explicitly describe such risks and uncertainties readers and listeners are urged to consider statements label with the terms believes belief expects intends anticipates potential should may will plan continued targeted or other similar expressions to be uncertain and forward looking.
Speaker 2: In addition to statements which explicitly describe such risks and uncertainties, listeners are urged to consider statements labeled with the terms believes, believes, expects, intends, anticipates potential.
Speaker 2: continued, targeted, or other similar expressions be uncertain and forward-looking. Listeners are cautioned not to place undue reliance on these forward-looking statements, which speak only as a matter of time.
Speaker 2: forward-looking statements during this conference call may include but are not limited to the success and timing of regular
Speaker 2: of the PK bridging study for GTX 104 and Acosta's other preclinical and clinical trials, regulatory requirements or developments, changes to clinical trial designs and regulatory pathways, legislative, regulatory, political and economic development, Acosta's projected cash position, operating runway and the effects of COVID-19 on clinical programs.
Speaker 2: forward looking statements contained in this conference call are expressly qualified in their entirety by this cautionary statement. The cautionary note regarding forward looking information section contained in Acosta's latest quarterly report on Form Thank You, which is available on EDGAR at www.scc.gov, on CDAR at www.cdar.com, and on the investor section of Acosta's website at www.acostafarma.com. All forward looking statements in this conference call are expressly qualified in their entirety section.
Forward looking statements contained in this conference call are expressly qualified in their entirety by this cautionary statement. The cautionary note regarding forward looking information section contained in our costs. These latest quarterly report on Form 10-Q , which is available on Edgar at Www SEC Gov on SEDAR at Www, SEDAR Dot com and on the investors section of the <unk>.
Web site at Www Dot <unk> Dot com all forward looking statements in this conference call are made as of the date of this conference call capacity does not undertake to update any such forward looking statements, whether as a result of new information future events or otherwise except as required by law forward. Looking statements contained herein are also subject to general subject generally to assumptions and risks and.
Speaker 2: ACOSI does not undertake to update any such forward-looking statements, whether as a result of new information, future events, or otherwise, except as required by law. The forward-looking statements contained herein are also subject generally to assumptions and risks and uncertainties that are described from time to time in ACOSI's public filings on Securities and Exchange Commission and Canadian Securities.
They are described from time to time in economy public filings on Securities and Exchange Commission and Canadian Securities regulators, including its latest quarterly report on Form 10-Q . In addition, any forward looking statements represent a cost these views as of today and should not be relied upon as representing our views as of any subsequent date while costs. He may update forward looking statements at.
Speaker 2: future, Acosta undertakes no obligation to update such statements.
Some point in the future a costly undertakes no obligation to update such statements to reflect events that occur or circumstances that exist. After the date on which they were made except as required by applicable securities law I'd now like to turn the call over to Ekati CEO of <unk>. Please go ahead Jan.
Speaker 2: occur or circumstances that exist after the date on which they were made except as required.
Speaker 2: I'd now like to turn the call over to Ocasio CEO , Jan De Alvis. Please go ahead, Jan.
Speaker 3: Hey, thank you, David. I'd really like to welcome everyone on the call today. During the fiscal third quarter, we made significant progress towards advancing our clinical pipeline of drugs to treat patients with rare and orphan diseases.
Okay. Thank you David I'd really like to welcome everyone on the call today.
During the fiscal third quarter, we made significant progress towards advancing our clinical pipeline of drugs to treat patients with rare and orphan diseases or.
Speaker 3: Our proprietary drug delivery technologies and capabilities are supporting an ever-expanding intellectual property portfolio of more than 40 issued and pending patents and a growing pipeline of drug candidates targeted to treat rare and orphan medical conditions.
Our proprietary drug delivery technologies and capabilities are supporting an ever expanding intellectual property portfolio of more than 40 issued and pending patents and a growing pipeline of drug candidates targeting to treat rare and orphan medical conditions are critical in preclinical stage drug candidates had been specifically.
Speaker 3: Our clinical and preclinical stage drug candidates have been specifically designed and formulated to enhance efficacy and safety by providing potentially faster onset of action, prolonged treatment effect, and reduced side effects, all while being more conveniently delivered, which can ultimately increase patient compliance and potentially lead to improved clinical outcomes.
Designed and formulated to enhance efficacy and safety by providing potentially faster onset of action prolonged treatment effect and reduce side effects, all while being more conveniently delivered which can ultimately increase patient compliance and potentially lead to improved clinical outcomes.
Speaker 3: Now, as previously disclosed, during the second quarter, we commenced enrollment for our pivotal pharmacokinetic or PK bridging study for GTX104, our novel aqueous formulation of pneumodepine that's being developed as an IV infusion for patients experiencing subarachnoid hemorrhage or SAH that is triggered by an aneurysm.
Now as previously disclosed during the second quarter, we commenced enrollment for our pivotal pharmacokinetic or PK bridging study for Gtx 104, our novel aqueous formulation of Nimodipine, that's being developed as an IV infusion for patients experiencing subarachnoid hemorrhage or sah.
That is triggered by an aneurysm. This peak PK study is the next required step in our proposed 505, two regulatory pathway and its being conducted in about 50 healthy subjects as a single center randomized two period crossover study. The primary objective of this study is to evaluate and <unk>.
Speaker 3: This PK study is the next required step in our proposed 505B2 regulatory pathway, and it's being conducted in about 50 healthy subjects as a single center randomized two-period crossover study.
Speaker 3: The primary objective of the study is to evaluate and compare the relative bioavailability of GTX 104 with the marketed oral nematopine capsules, which are currently the standard of care in the United States.
Compare the relative bioavailability of Gtx 104, with the marketed oral Nimodipine capsules, which are currently the standard of care in the United States. The second objective is to assess the safety and Tolerability of Gtx, one O four as compared to oral nimodipine capsules.
Speaker 3: The second objective is to assess the safety and tolerability of GTX 104 as compared to the oral nematopean caps.
Speaker 3: Now back in December , we were pleased to announce positive results for GTX 104 based on an interim analysis of the first 20 normal healthy subjects enrolled.
Now back in December we were pleased to announce positive results for Gtx one O four based on an interim analysis of the first 20 normal healthy subjects enrolled.
Speaker 3: GTX 104 met both primary endpoints for maximum concentration, or Cmax, on day one, and area under the concentration time curve, or AUC, over a 24-hour period on day three. These interim data were very encouraging and allowed us to continue the study under the current infusion protocol to its pending near-term completion.
<unk> met both primary endpoints for maximum concentration or C. Max on day, one and area under the concentration time curve or AUC over a 24 hour period on day three.
These interim data were very encouraging and allowed us to continue the study under the current infusion protocol.
Two it's pending near term completion.
Speaker 3: We believe that the tight correlation of the primary endpoint data for the first 20 patients is a strong indication that GTX104 could achieve comparable bioavailability with oral nematopine in the full study cohort of about 50 subjects.
We believe that the tight correlation of the primary endpoint data for the first 20 patients is a strong indication that gtx one O four could achieve comparable bioavailability with oral nimodipine in the full study cohort of about 50 subjects.
Speaker 3: Very importantly, and as we observed in the earlier PK study, the inter- and intra-subject variability in the interim analysis was significantly lower for GTX 104 as compared with oral nematopine.
Very importantly, and as we observed in the earlier PK study, the inter and intra subject variability in the interim analysis was significantly lower for Gtx 104, as compared with oral nimodipine. This could be a very important differentiator for gtx, one O four as reduced variability in blood levels.
Speaker 3: This could be a very important differentiator for GTX 104, as reduced variability of blood levels should correlate with better control of hypotension, which could result in better outcomes for patients with SEH, as well as improved economics for the health care system.
Should correlate with better control of hypotension, which could result in better outcomes for patients with sch as well as improved economics for the health care system.
Speaker 3: Since we reported these interim results, the study has now advanced significantly, with 46 subjects having completed the full protocol. To date, there have been no serious adverse events observed, and only mild adverse events such as headaches were reported in both groups, and they were resolved with common over-the-counter medications.
Since we reported these interim results. The study has now advanced significantly with 46 subjects, having completed the full protocol.
To date, there have been no serious adverse events observed and only mild adverse events such as headaches.
Were reported in both groups and they were resolved with common over the counter medications.
Speaker 3: The last cohort of subjects required to complete the study have already been enrolled, and the study remains on track to report the full results in calendar Q2. Assuming the final study results are consistent with the interim results, we plan to proceed quickly to finalize the Phase III safety study design and protocol for GTX 104 with the FDA. We still expect to initiate this safety study in the second half of calendar 2022.
The last cohort of subjects required to complete this study have already been enrolled in this study remains on track to report the full results in calendar Q2.
Assuming the final study results are consistent with the interim results. We plan to proceed quickly to finalize the phase III safety study design and protocol for Gtx, One O four with the FDA, we still expect to initiate the safety study in the second half of calendar 2022.
Speaker 3: Based on its already favorable safety profile, we believe the safety study for GTX 104 to be relatively low risk. And given the expected small number of patients to be enrolled, it should be completed relatively quickly and cost effectively. Importantly, the safety study is expected to be the final step required before submitting our new drug application, or NDA, to the FDA under the 505B2 regulatory pathway.
Based on its already favorable safety profile, we believe the safety study for Gtx, one or four to be relatively low risk and given the expected small number of patients to be enrolled it should be completed relatively quickly and cost effectively importantly, the safety study is expected to be the final step required before.
Netting our new drug application or NDA to the FDA under the $505 two regulatory pathway. Our NDA if approved would be enhanced by the previously granted orphan drug designation already assigned to Gtx, one O four by the FDA, which would grant the company seven years of market exclusives.
Speaker 3: Our NDA, if approved, would be enhanced by the previously granted orphan drug designation already assigned to GTX 104 by the FDA, which would grant the company seven years of market exclusivity in the United States.
<unk> in the United States.
Speaker 3: As we have discussed previously, we believe GTX 104 addresses an important and underserved market. SAH is caused by a ruptured aneurysm, and it's a rare and life-threatening medical emergency. It's estimated to affect about 50,000 patients per year, representing an addressable market of more than $300 million in the United States alone.
As we have discussed previously we believe gtx one O four addresses an important and underserved market.
<unk> is caused by a ruptured aneurysm and it's a rare and life threatening medical emergency it's estimated to affect about 50000 patients per year, representing an addressable market of more than $300 million in the United States alone there.
Speaker 3: Nemotipine is currently only available as an oral dosage form in the US, and many of these SEH patients are unconscious or have a hard time swallowing during their hospital stay. We believe GTX 104 delivered intravenously could be a much more convenient and efficient way to deliver nemotipine, and importantly because of its better absorption profile and more consistent blood levels.
<unk> is currently only available as an oral dosage form in the U S and many of these <unk> patients are unconscious or have a hard time swallowing during their hospital stay we believe gtx one afford delivered intravenously could be a much more convenient and efficient way to to deliver nimodipine and importantly, because of its.
Better absorption profile and more consistent blood levels.
Speaker 3: GTX 104 could potentially provide physicians with a more effective tool for hypotension management.
<unk> could potentially provide physicians with a more effective tool for hypertension management.
Speaker 3: We really look forward to providing regular updates on how the GTX 104 PK study and the Phase III safety study protocol develop in the weeks and the months ahead.
We really look forward to providing regular updates on how the Gtx 100, <unk> PK study and the phase III safety study protocol develop in the weeks in the months ahead.
Speaker 3: Now, regarding our other two clinical candidates, GTX 101 and GTX 102, we continue to make steady progress moving these two drug candidates forward towards their next major milestone.
Now regarding our other two clinical candidates Gtx 101, and Gtx 102, we continue to make steady progress moving these two drug candidates forward towards their next major milestones as a reminder, gtx 102 is a novel easy to use oral mucosal spray formulation of beta method zone.
Speaker 3: As a reminder, GTX 102 is a novel, easy-to-use oral mucosal spray formulation of beta-methasone intended to improve the neurological symptoms of ataxia telangiectasia, or AT. AT is a progressive neurodegenerative genetic disease that primarily impacts children, causing severe disability for which no FDA-approved treatment currently exists.
Intended to improve the neurological symptoms of ataxia telangiectasia or a T. A T is a progressive neurodegenerative genetic disease that primarily impacts children, causing severe disability for which no FDA approved treatment currently exist.
Speaker 3: Based on an independent study conducted in Italy with an oral liquid form of beta-methasone, we believe GTX 102's novel concentrated beta-methasone oral mucosal spray formulation has the potential to improve the symptoms of AT and simplify drug administration.
Just on an independent study conducted in Italy, with an oral liquid form of beta mechanism. We believe gtx 102 as novel concentrated data and that the zone oral mucosal spray formulation.
Has the potential to improve the symptoms of <unk> and simplified drug administration.
We recently selected a CRO partner to manage our PK bridging study for Gtx 102. This study will be fully randomized open label three arm crossover study designed to evaluate the comparative bioavailability pharmacokinetics and safety of Gtx 102 administered as a.
Speaker 3: We recently selected a CRO partner to manage our PK bridging study for GTX 102. This study will be fully randomized, open-label, three-arm crossover study designed to evaluate the comparative bioavailability, pharmacokinetics, and safety of GTX 102 administered as a concentrated oral mucosal spray, and we will compare it to beta-methazone administered as an intramuscular injection.
Traded oral mucosal spray and we will compare it to beta method zone administered as an intramuscular injection for filing purposes in the U S and Canada.
Speaker 3: for filing purposes in the US and Canada and to an oral solution of beta methazone for filing purposes in Europe .
And to an oral solution a data methisazone for filing purposes in Europe .
Speaker 3: This study will be conducted in healthy male and female volunteers. We expect this PK bridging study to be initiated on schedule in calendar Q2 and to report out before the end of 2022.
This study will be conducted in healthy male and female volunteers. We expect this PK bridging study to be initiated on schedule in calendar Q2 and to report out before the end of 2022.
Speaker 3: If this study meets its primary end points, we expect to initiate our Phase III safety and efficacy study in AT patients in the first half of calendar 2023.
If this study meets its primary endpoint, we expect to initiate our phase III safety and efficacy study in 80 patients in the first half of calendar 2023.
Speaker 3: Now turning to GTX 101, we're developing this drug candidate to treat patients with postherpetic neuralgia, or PHN, which is persistent and often debilitating neuropathic pain caused by nerve damage from the varicella zoster virus, the virus that causes both chicken pocks and shingles.
Now turning to Gtx 101, we're developing this drug candidate to treat patients with post herpetic neuralgia, or PHN, which is persistent and often debilitating neuropathic pain caused by nerve damage from the various sellers zoster virus the virus that causes both chicken pox and <unk>.
Shingles.
Speaker 3: PHN can persist for months and even years after a shingles infection clears up. And some patients end up requiring opioids to relieve their severe pain.
PHN can persist for months and even years after a shingles infection clears up and some patients end up requiring opioids to reis to relieve their severe pain.
Speaker 3: Our GTX 101 is a novel bio-adhesive, film-forming topical spray formulation of bupivacaine that we believe could provide significant benefits over the current standard of care, which most often consists of oral gabapentin.
Our Gtx 101 is a novel bio adhesive film forming topical spray formulation of bupivacaine that we believe could provide significant benefits over the current standard of care, which most most often consists of oral Gaba penton.
Speaker 3: and prescription lidocaine patches. And refractory cases are often prescribed opioids to address persistent pain.
And prescription lidocaine patches and refractory cases are often prescribed opioids to address persistent pain.
Speaker 3: As you know, opioid abuse has continued to proliferate, and gabapentin and the prescription lidocaine patches are suboptimal for many reasons. According to third-party primary research commissioned by ACASTE, approximately 40% of patients using lidocaine patches experience insufficient pain relief. Lidocaine patches are also difficult to use. They fall off. They look unsightly and can cause skin sensitivity and irritation. For more information, visit ACASTE.com
As you know opioid abuse has continued to proliferate and gabapentin and the prescription lidocaine patches are suboptimal for many reasons. According to third party primary research commissioned by a costly approximately 40% of patients using lidocaine patches experienced insufficient pain relief.
Lidocaine patches are also difficult to use they fall off they look unsightly and can cause skin sensitivity and irritation. Additionally, optimal pain relief can take up to two weeks to be achieved with the lidocaine patch.
Speaker 3: Additionally, optimal pain relief can take up to two weeks to be achieved with the lidocaine pat.
Speaker 3: The potential benefits of GTX 101 could include the faster onset of action because of our active ingredient bupivacaine as compared to lidocaine and longer lasting pain relief as the patch can only be used for 12 hours on and then must be taken off for 12 hours.
The potential benefits of Gtx 101 could include the faster onset of action.
Because of our active ingredient booth bupivacaine as compared to lidocaine and longer lasting pain relief as the patch can only be used for 12 hours on and then must be taken off for 12 hours.
Speaker 3: This regimen can lead to breakthrough pain, while we expect that regular use of GTX 101 will provide continuous around-the-clock pain relief. Consequently, we believe our GTX 101 topical spray could be a very effective, non-narcotic, non-addictive alternative for PHN patients that could also provide greater ease of use and convenience.
This regimen can lead to breakthrough pain, while we expect that regular use of Gtx 101 will provide continuous around the clock pain relief. Consequently, we believe our gtx 101 topical spray could be a very effective non narcotic non addictive alternative for ph and pay.
<unk> that could also provide greater ease of use and convenience.
Speaker 3: Now, our primary market research has shown strong physician receptivity towards GTX 101's value proposition.
Our primary market research has shown strong physician receptivity towards Gtx 100 ones value proposition with a majority indicating that they saw the potential for gtx 101 to not only replace but to potentially further grow the prescription lidocaine patch market note that in the U S.
Speaker 3: with a majority indicating that they saw the potential for GTX 101 to not only replace, but to potentially further grow the prescription lidocaine patch market. Note that in the US, lidocaine patches are only indicated for PHN.
Lidocaine patches are only indicated for PHN.
Speaker 3: but in fact are broadly prescribed and reimbursed for a range of pain indications including back pain, osteoarthritis, diabetic neuropathy, and other neuralgias.
But in fact are broadly prescribed and reimburse for a range of pain indications, including back pain osteoarthritis diabetic neuropathy and other neuralgia is.
Speaker 3: PHN is estimated to affect approximately 150,000 patients per year in the United States. And according to a third-party report commissioned by ACOSTE, the total addressable market for GTX 101 could be about 1.6 billion, consisting of about 400 million for PHN pain and about 1.2 billion for non-PHN pain.
PHN is estimated to affect approximately 150000 patients per year in the United States and according to third party or third Party report commissioned by a costly the total addressable market for Gtx 101 could be about $1 6 billion.
<unk> about of about 400 million for ph and pain and about $1 2 billion for non ph in pain.
Speaker 3: As for our near-term clinical milestones for GTX 101, an IND-enabling eight-week skin sensitivity study in mini-pigs is underway now, which we expect will report out in calendar Q2. Results from this mini-pig study will enable us to start our multiple ascending dose ranging study in humans, which is a requirement for our phase 2 study that's scheduled to start recruiting patients before the end of this year.
As for our near term clinical milestones for Gtx, 101, and I N D. Enabling eight week skin sensitivity study in mini pigs is underway now, which we expect will report out in calendar Q2.
<unk> from this mini pig study will enable us to start our multiple ascending dose ranging study in humans, which is a requirement for our phase II study that's scheduled to start recruiting patients before the end of this year.
Speaker 3: By the way, I should also mention that a previous PK study in humans showed no skin sensitivity from GTX 101.
By the way I should also mention that our previous PK study in humans showed no skin sensitivity from Gtx 101.
Speaker 3: So we're very excited about our entire orphan drug portfolio and believe it holds tremendous potential that could drive significant value for our shareholders in the months and years ahead. And as I mentioned, we're expecting to achieve multiple important milestones for each of our LEAD programs throughout 2022.
So we're very excited about our entire orphan drug portfolio and believe it holds tremendous potential that could drive significant value for our shareholders in the months and years ahead and as I mentioned, we're expecting to achieve multiple important milestones for each of our lead programs throughout 2022 with.
Speaker 3: With the exciting results we've seen thus far in our PK study for GTX 104, and the active development work now underway for GTX 102 and 101, I hope you share our enthusiasm for the outlook for our entire clinical portfolio as we continue to advance our lead drug candidates through clinical development and ultimately towards commercial launch. We look forward to reporting on further progress as it occurs, as well as at our next quarterly and annual update.
But the exciting results we've seen thus far in our PK study for <unk> four and the active development work now underway for Gtx 102, and 101 I Hope you share our enthusiasm for the outlook for our entire clinical portfolio as we continue to advance our lead drug candidates through clinical development and ultimately towards <unk>.
<unk> launch, we look forward to reporting on further progress as it occurs as well is that our next quarterly and annual update.
Speaker 3: So with those operational updates, I'll now turn the call over to Brian Ford, our CFO , to discuss our financial results for fiscal Q3. Brian ?
So with those operational updates I'll now turn the call over to Brian <unk>, our CFO to discuss our financial results for fiscal Q3, Brian .
Speaker 4: Thank you, Jan. Please note that unless otherwise indicated, all numbers that I mentioned are in US dollars.
Thank you Chad.
Unless otherwise indicated all numbers that I mentioned are in U S dollar.
Speaker 4: The loss from operating activities for the three months ended December 31st, 2021 was 4.5 million compared to a loss of 2 million for the three months ended December 31st.
The loss from operating activities.
For the three months ended December 31.
One was $4 5 billion.
Loss of $2 million.
For the three months ended December 31.
Speaker 4: The increase was due mostly to an increase in research and development expenses and an increase in general and in expenses related to increased legal, tax, accounting, and other professional fees. The increase was due mostly to an increase in financial expenses and an increase in financial
2021 the.
The increase was due.
Mostly to an increase in research and development expenses and an increase in general and admin expenses.
Legal costs.
Accounting and other professional fees.
Speaker 4: The net loss for the three months ended December 31st, 2021 was $3.8 million, or $0.09 per share, compared to a net loss of $3.2 million, or $0.26 per share, for the three months ended December 31st, 2020.
The net loss for the three months ended December 31 2021.
<unk> 8 million or nine cents per share compared to a net loss of $3 2 million or <unk> 26, a share for.
For the three months ended December 31.
2021.
Speaker 4: The increase in net loss resulted primarily from an increase in research and development expenses related to accelerating the development of the new clinical aspects quite great.
<unk> net loss resulted primarily from an increase in.
Research and development expenses related to accelerating the development of the new clinical assets.
Speaker 4: Research and development expenses before depreciation, amortization, and stock-based compensation expenses for the three months ended December 31, 2021 totaled $2 million compared to $62 million for the three months ended December 31, 2020.
Research and development expenses before depreciation amortization.
Compensation expense for the three months.
At December 31st 2021 totaled $2 million compared to the system.
Million points of $2 million.
Three months ended December 31, 2020.
Speaker 4: The net increase was again mainly attributable to increased contract research activities as the clinical
And that increase was again, mainly attributable to increased contract research activities as the clinical.
So long.
Speaker 4: as the clinical program got underway.
As the clinical program.
Got underway.
Speaker 4: As I mentioned earlier, this increase was the result of increased legal costs to the county and another professional fee.
As I mentioned earlier. This increase was the result of increased legal costs for the child and other professional fees.
Speaker 4: cash equivalents and short-term investments totaled $40.
Cash equivalents.
And short term investments totaled.
40.
Speaker 4: $46.3 million as of December 31st.
$46 3 million.
As of December 31.
Speaker 4: 2021 compared to $26.5 million in cash and cash equivalents as of December 31st, 2020.
2000 and the.
<unk> 21, compared to $26 5 million.
Cash and cash equivalents.
As of December 31st 2020.
Speaker 4: The company believes these funding resources provide at least 21 months of operating runway based on management's current projections. With that, I'll now turn the call back over to Jay.
The company believes in funding resources provided 21 months of operating in one way.
Just on management's projection.
I'll now turn the call back over to Jeff.
Speaker 3: Thanks, Brian . That concludes our prepared remarks. Now I'd like to open the call to any questions from our shareholders. Holly, can you open it up to questions?
Thanks, Brian that concludes our prepared remarks, now I'd like to open the call to any questions from our shareholders.
Colleague can you open it up to questions.
Certainly ladies and gentlemen, the floor is now open for questions.
Speaker 1: If you have any questions or comments, please press star 1 on your phone at the end of the video.
If you have any questions or comments. Please press star one on your phone at this time.
Speaker 1: We ask that while posing your question, you please pick up your handset if listening on speakerphone to provide optimum sound quality.
We ask that while posing your question you please pick up your handset.
Speaker phone to provide optimum sound quality.
Please hold while we poll for questions.
Your first question for today is coming from Alexandra Heller. Please announce your affiliation and pose your question.
Speaker 1: Our first question for today is coming from Alexandra Heller. Please announce your affiliation then pose your...
Speaker 5: Hi, this is Alex Heller on for Leland, Grisham at Oppenheimer. Congrats on the progress made this quarter and thanks for taking my question. First off, recognizing that you will be completing the PK-14 study for 104 and moving into Phase III safety study as well as the Phase II safety study of GTS 101, what can the best R&D expenses look like in the upcoming quarter?
Okay.
Oklahoma.
On the quarter.
Thank you Michael.
Recognizing that you will be completing the PK bridging studies for all living on campus.
I'll see you. Thank you. Thank you.
Tony.
R&D expenses.
Hello.
Speaker 3: Yeah, maybe I'll turn that one over to, by the way, Alex, thanks for the question. Really appreciate it. I'll turn that over to Brian . Brian , do you want to comment on kind of the ramp of our overall expenses due to the ramp-up of the clinical program?
Yeah.
Maybe I'll turn that one over to Oh by the way Alex. Thanks for the question really appreciate it I'll turn it over to Brian .
Brian do you want to comment on on kind of the ramp of our overall expenses due to the ramp up of the clinical programs.
Speaker 4: For the next 12 months, we expect our R&D expenses to be growing moderately from roughly a million and a half per month to almost two million per month. We have some incremental expenses above that.
Sure.
For the next 12 months.
We expect R&D expenses to.
The growing moderately from roughly a $1 billion.
The almost $2 million per month.
Have somebody come out all that stuff is above the.
Right.
Speaker 4: We expect the R&D to grow again another further 12 months out. As we get into more of the phase 3 travel, we begin to invest in commercialization activities for GTX 104.
We expect.
R&D.
To grow again, another further 12 months out.
As we get into more of the reach.
So we began to invest in commercialization activities for Gtx 104.
Does that answer the question.
Speaker 3: Yeah, and I might add, Alex, that, you know, as we said, the current cash that we have on our balance sheet will allow us to completely fund the full GTX 104 program and significantly advance both the 102 and the 101 programs.
Yeah, and I might add Alex that you know as we've said the current cash that we have on our balance sheet will allow us to completely fund the full gtx, one O four program and significantly advance.
Both the one or two in the 101 programs.
Speaker 5: Perfect. Yeah, that was incredibly helpful. Thank you for that. And then I guess the next question I have is, as there's no approved product for AAT, have you had any discussions on what that pivotal trial design might look like?
Perfect Yeah that was incredibly helpful. Thank you.
And then I guess the next question that somehow there's no approved product.
Have you had any discussion on what that pivotal trial design might look like.
Speaker 5: when you're looking at starting it with the FDA, and maybe when we might have clarity on that design.
In 2023, when Youre looking at starting out with the FDA and.
And maybe when we might have clarity on that why that yeah. Yeah. We've had preliminary discussions with the FDA about the trial design and.
Speaker 3: Yeah, we've had preliminary discussions with the FDA about the trial design.
Speaker 3: And we expect that it will be a safety and efficacy study. Probably in a small number of patients, I would...
And we expect that it will be a safety and efficacy study.
Probably in a small number of patients I would I would guesstimate are about 100 patients may be less.
Speaker 3: guesstimate about 100 patients, maybe less. You know, we'll have to see how long the FDA wants us to, you know, treat these patients and follow them. You know, we plan to have a meeting with the FDA once we complete the PK study to lock down the study design for the Phase III. But based on the preliminary discussions, that's what I can tell you for now.
We'll have to see how long the FDA wants us to treat these patients and follow them.
We plan to have a meeting with the FDA once we complete the PK study to Lockdown the study design for the phase III.
But based on the preliminary discussions that that's what I can tell you for now.
Okay. That's helpful. Thank you.
Speaker 5: That's all my questions. I appreciate you taking them. Yeah, sure. Thanks, Alex.
That's all my questions I appreciate you taking them.
Yeah sure. Thanks, Alex.
Speaker 1: Your next question is coming from Sahil Kazmi. Please announce your affiliation, then pose your question.
Your next question is coming from South Hill Cagny. Please announce your affiliation and pose your question.
Speaker 6: Hi, team. How's it going? Congrats on the quarter and all the progress that's gone. This is Tano Kasme from B. Riley Securities. A couple of questions from us, maybe focusing on the data that you have in hand today on the 46 patients. Could you provide a bit more granularity on what we're seeing in terms of the variability both between patients and relative to the oral formulation and how that might be both clinically relevant and also inform your thoughts with further information.
Hi team Don Congrats on the quarter.
All of the progress of Dawn this is Paul.
B Riley.
A couple of questions from us maybe focusing on the data.
And that you have in hand today, I mean 46 patients quite a bit more granularity on what we're seeing some of the variability between patients relative to the oral formulation and how that might be both clinically clinically relevant and also inform your thoughts.
Speaker 3: Yeah, thanks, Ohio. Hey, listen, thanks for the question. Really appreciate it. Appreciate your attending today. I'll speak to it generally and then turn it over to Pierre and George. But we saw a dramatic difference in variability. GTX 104 showed really tightriority across all these come directly from lower Wow,
Yeah. Thanks, a highly hey listen thanks for the question really appreciate it appreciate your attending today.
I'll I'll speak to it generally and then turn it over to Pierre Ah and George but.
But we saw a dramatic difference in variability Gtx, one O four showed really tight.
Speaker 3: you know, very little variability. So from patient or subject to subject and over time, the variability was very low compared to the oral, which it was no surprise. We'd seen this before, and this is obviously the big complaint of physicians. It makes it very difficult to control.
You know very little variability, so so from patient or subject to subject and over time. The variability was very low compared to the oral which it was no surprise we've seen this before and this is obviously the big complaint.
Physicians it makes it very difficult to control blood.
Blood pressure when you have the oral nimodipine, causing huge swings in blood pressure and that's what we saw I mean, it was just significantly more variable now I would just caution that this is based on 20 subjects.
We do have 46 completed so far but we don't have the data on those 46, we're still blinded.
Speaker 3: data on those 46 were still blinded. We should be finishing up the study in the next few weeks. And then they've got to clean up the data. It'll take some time to do that and then report out the results. But we expect to report out still on track in Q2. And I don't know, Pierre, do you want to add anything? Could I pretty much cover that or anything you want to add? Pierre, I think you might be on mute. BEEP.
We should be finishing up the study in the next few weeks.
And then they've got to clean up the data it'll take some time to to do that and then report out the results, but we expect to report out still on on track.
Speaker 3: in Q2.
In in Q2.
Speaker 3: And I don't know, Pierre, do you want to add anything? Did I pretty much cover that or anything you want to add?
And I don't know.
Do you want to add anything that I pretty much covered that or anything you want to add.
There I think you might be on mute.
Okay, we're not get George are you there.
Speaker 7: Yes, I'm here Jan. George, do you want to, anything you want to add? Yeah, Sahil, so essentially exactly as Jan said, the interim data in 20 subjects,
Yes, I'm here John .
George do you want to anything you want to add Yep Yep.
So essentially exactly as John said.
Interim data and blended patient 20 subjects.
Speaker 7: showed very little variability both from AUC perspective as well as from a C-max perspective. We don't expect this to be any different after we complete the analysis of 56 or so subjects. And the clinical portion of the study should be complete by mid-February this month and we will have some kind of a draft report at the end of March, early April .
Showed very little variability.
Well AUC perspective, as well as some of them are C. Max perspective, we don't expect this to be any different after we complete the analysis of 50.
56, or so subjects and the clinical portion of the study.
Should be complete by mid February this this month and the but we will have some kind of a draft report at the end of March early April .
Okay.
Thanks.
Speaker 6: And then maybe one more from us, you know, either Dan or Brian , just as it relates to, you know, the cash position today and the guided 21 months of runway, just given what we're seeing in the 104 program, how you guys were actually able to go a little bit quicker than anticipated, can you talk about how you might be able to, you know, use this capital to either focus on one of the emerging programs and how you think about the pace of development for both AC and hit?
And then maybe one more from us.
Or are there John and Brian .
To.
Today in the guided 21 months of runway given what we're seeing in the 104 program. How are you guys were actually able to go a little bit quicker than anticipated can you talk about how you might be able to.
Good capital to either focus on one of the emerging programs in how you think about the pace of development for for both H E.
Yeah.
Speaker 3: Yeah, so I'll jump in and then Brian , you can add more from a financial perspective. But yeah, we expect the current cash to complete GTX 104. We are running a little bit ahead of schedule. I would say a few weeks, maybe a month or so.
Yeah, So I'll I'll jump in and then Brian you can add more from a financial perspective, but yeah. We.
We expect our current cash to complete Gtx 104.
We're running a little bit ahead of schedule I would say a few weeks, maybe a month or so but I don't think it's going to have as a significant impact on that.
Speaker 3: But I don't think it's going to have a significant impact on the bottom line cash projection. We are moving 102 and 101 forward very aggressively. But again, all of that was in our plan.
The bottomline cash projection.
We are moving one O two and 101 forward very aggressively.
But again all of that was in our plan.
Speaker 3: So, having said that, we have a couple other assets in the preclinical pipeline that are quite interesting, and we are looking at potentially moving one of those forward into clinical development later this year. But a lot more planning work has to be done before we say too much more on that.
So having said that we have a couple of other assets in the preclinical pipeline that are quite interesting and we are looking at potentially moving one of those forward into clinical development later this year.
But a lot more planning work has to be done before we say too much more on that.
Speaker 3: But Brian , do you want to make any additional comments on the cash runway?
But Brian do you want to make any additional comments on the cash runway.
Speaker 4: No, I mean we've been tracking our cash a little better than planned from the days of the merger.
No I mean, we've been tracking our cash a little better than planned from the date of the merger.
Speaker 4: and not that that's necessarily going to continue as we start, you know, continue to build on these programs.
And.
Not that that's necessarily going to continue as we continue to build on these programs.
Speaker 4: But we continuously look at our plans and revise the forecasts as new information and new agreements and new research agreements come into play. But so far, our estimates have been pretty good.
But.
We continuously look at.
Our plans and our revised forecast is.
As new information and new agreements for new research agreements come into play.
Well so far.
Okay.
Sure.
Held up pretty well.
Speaker 3: Yeah, I would say overall we are running a bit ahead. In other words, we've managed our cache very well, so we are running a bit ahead of our projection in terms of cache runway. And I fully expect we'll continue to run these programs tightly. And let's hope the data holds up and we can move forward as planned. So I hope that.
Yeah, I would say overall, we are running a bit ahead in other words, we've we've manage our cash very well.
We are running a bit ahead of our projection in terms of cash runway.
And I fully expect we will continue to run these programs tightly and.
Let's let's hope the data holds up and we can move forward as planned.
So I.
I hope that answers your question somehow.
Speaker 6: Yes, that was extremely helpful and congrats again on the progress in this environment. Look forward to seeing the update on 104. Great, thank you.
Yes that was extremely helpful.
All right.
In this environment and look forward to seeing me up.
They don't want it.
Great. Thank you.
Okay.
Once again, if there are any questions or comments. Please press star one.
Speaker 1: Once again, if there are any questions or comments, please press star 1.
There are no questions in queue at this time.
Speaker 3: Okay, well again I want to thank everyone for joining us today. We have, as you can tell, a lot of really exciting programs underway and we look forward to continuing to provide updates in the very near future. So again, thanks and have a good day.
Okay, well again I want to thank everyone for joining us today and we have as you can tell a lot of really exciting programs underway and we look forward to continuing to provide updates in the very near future. So again. Thanks.
And have a good day.
Speaker 1: Ladies and gentlemen, this does conclude today's conference call. You may disconnect your phone lines at this time and have a wonderful day. Thank you for your...
Thank you ladies and gentlemen, this does.
Today's conference call you.
You may disconnect your phone lines at this time and have a wonderful day.
Thank you for your participation.
Yeah.
Okay.
Yeah.