Replies to post #642 on Biotech Ideas

[gfp927z]

Orchard Therapeutics - >>> A lifesaving therapy for children with a rare disease is now the world’s most expensive drug, raising questions about access


CNN

by Brenda Goodman

March 20, 2024


https://www.yahoo.com/lifestyle/fda-approved-lifesaving-therapy-children-194412723.html


A new gene therapy for the fatal genetic disorder metachromatic leukodystrophy, or MLD, will carry a wholesale price of $4.25 million, its manufacturer announced Wednesday, making it the world’s most expensive medicine.

Lenmeldy was approved by the US Food and Drug Administration on Monday and is the first therapy for the rare and devastating disease, which typically kills affected children before they turn 7. About 40 children are born with MLD in the US each year.

The wholesale cost isn’t usually what patients pay, but it’s a cost that’s considered and shouldered by public and private health insurance plans, including state Medicaid plans, which cover roughly 4 out of every 10 children in the United States.

Manufacturers of gene therapies say the big prices reflect big benefits — the chance to be free of a disabling or even fatal disease — and they point out that they need to be able to recoup the steep costs of development, testing and manufacturing their products.

Health policy experts say that as the list of gene and cell therapies with eye-popping prices grows, it may strain the ability of states and other insurers to cover their costs, and ultimately limit patient access if plans begin to exclude these therapies as a class from coverage.

Dr. Bobby Gaspar, the co-founder and CEO of Orchard Therapeutics, the company that makes Lenmeldy, said the treatment is “paradigm-shifting medicine and has the potential to stop or slow the progression of this devastating childhood disease with a single treatment.”

“We are committed to enabling broad, expedient and sustainable access to this important therapy for eligible patients with early-onset MLD in the U.S.,” Gaspar said in a statement.

Price of hope

Lenmeldy takes stem cells from someone with MLD and uses a harmless virus to insert working copies of a faulty gene. The repaired cells are then infused back to the patient, where they begin to produce an enzyme that’s lacking in children who have the disease. Some of the cells eventually migrate to the bone marrow, where they continue to live and make new cells that also make the enzyme, providing a long-lasting benefit to patients.

The first patients treated with Lenmeldy have now been followed for more than 12 years, and researchers continue to find gene-modified cells making the missing enzyme, said Orchard Therapeutics’ Gaspar.

“We can’t say at the moment that this will last a lifetime, but what we can say is that there is a longterm durable effect,” Gaspar said.

MLD is an inherited disorder, and children born with it lack an enzyme needed break down fatty substances called sulfatides. The build-up of these fatty materials eventually becomes toxic to nerves, leading to the progressive loss of movement and thinking.

Babies with MLD develop normally for a time and but then typically begin to lose the ability to walk and talk around age 2. The disease advances rapidly, causing children to deteriorate into a vegetative state.

“We are over the moon regarding what this means for other families,” said Kendra Riley, 41, of Phoenix, who has two children with MLD.

For their family, the FDA’s action this week was bittersweet.

Riley’s 5-year-old daughter, Olivia, is in hospice after being diagnosed with MLD as a toddler.

Riley says the first clues that something was wrong came when Olivia was around the age of 2. She began to have trouble walking, and her head started to regularly tilt to the side when she would watch TV.

“We thought we just needed some physical therapy,” Riley said.

“Then the irises of her eyes started vibrating. That’s when we knew something else was going on.”

By the time doctors diagnosed Olivia with MLD, it was too late for the gene therapy to help. But knowing that the condition is inherited, they were able to get their younger daughter, Keira, tested and diagnosed.

Keira, now 4, was the 32nd child in the world to get the therapy, which is most effective before children show symptoms.

The treatment wasn’t yet available in the United States, so the family crowdsourced donations to temporarily relocate to Italy in 2020 to get the therapy. It cost them about $500,000 to live abroad and pay for Keira’s medical care, even though the company provided the gene therapy for free.

“Having this FDA-approved therapy means that if a child does get diagnosed before symptom onset, they have a chance at normal life,” Riley said.

“She is doing amazing. Zero symptoms,” she said of Keira. “You would never know.”

‘Exciting’ drug development

The Boston nonprofit Institute for Clinical and Economic Review, or ICER, which evaluates the cost effectiveness of new drugs, published a final report on Lenmeldy last fall and estimated that the cost of the therapy would match its expected benefits to patients if it was priced between $2.3 million and $3.9 million.

Experts agreed that even such hefty price tag would be worth its results. Children with the disease who aren’t treated usually die within five years of their diagnosis; the oldest patient to have the therapy has now been followed for more than 12 years and appears to be developing normally.

“It’s taking a child who would have had a miserable short life and likely giving them a normal life. And that’s worth a lot of money,” said Dr. David Rind, the chief medical officer for ICER. “This is one of the more exciting drugs that we’ve looked at.”

Still, the Lenmeldy’s price came in hundreds of thousands of dollars higher that even Rind expected.

“I think when you get into numbers this big that people don’t necessarily pay attention when it gets a little bigger, but I do think this price is too high,” Rind said.

ICER gave a range of $2.29 million to $3.94 million — that doesn’t mean that the top of the range is the right price, said Rind “despite the desire by manufacturers to think of it that way.”

“Going $310,000 above the very top of that range is actually a lot of money,” he said.

The last product to top the charts on a per-treatment basis was another gene therapy called Hemgenix, which was approved in 2022 to treat a blood clotting disorder called hemophilia B. Its list price was $3.5 million for a one-time treatment.

Close behind that one is Elvevidys, which was approved in 2023 for muscular dystrophy at $3.2 million, and Skysona, approved in 2022 for a disease related to MLD called adrenoleukodystrophy, which costs $3 million for a one-time dose.

Even with a top tier price, the MLD treatment will not be a blockbuster, Rind noted, because the disease is so rare. Some gene therapies approved for rare diseases have disappeared because the companies that made them couldn’t make enough money to stay in business. Orchard Therapeutics has already struggled to market a previous gene therapy it developed, leaving patients hanging.

Edwin Park, a research professor at the McCourt School of Public Health at Georgetown University, worries about the impact to states, which share the cost of covering patients on Medicaid with the federal government.

Medicaid requires a minimum rebate from drug manufacturers, but without any competition, there probably won’t be much negotiation about the price of the therapy, Park said.

More and more states are closely following drug pipelines after being surprised by the costs of the antiviral medications that cure hepatitis C, he said.

But even paying for one or two children to get Lenmeldy a year could be a sizeable cost for some states to absorb.

“Unless states have allocated appropriately for it, and looked at the drug pipeline, they may not be prepared for what could be significant cost spikes,” Park said.

New possibilities for patients

This latest approval comes as the FDA is working to clear a backlog of cell and gene therapies that have been waiting for its attention since the pandemic. The agency has hired more staff to review the new treatments and set up a new superoffice, the Office of Therapeutic Products, to oversee their regulation.

Lenmeldy has been available in Europe since 2020, but it took four more years to bring it to the US.

Dr. Nicole Verdun, who was hired to head the new FDA initiative last year, said Lenmeldy’s approval is a sign that things are moving in the right direction.

“MLD is a devastating disease that profoundly affects the quality of life of patients and their families,” Verdun said.

“This approval represents important progress in the advancement and availability of effective treatments, including gene therapies, for rare diseases.”

Dr. Barbara Burton, who is an attending physician, of genetics, genomics and metabolism at the Ann & Robert H. Lurie Children’s Hospital of Chicago, said it was a watershed moment for doctors, too.

“For too long, my colleagues and I have consoled families at their most vulnerable times — usually following an arduous diagnostic odyssey, coping with a dire prognosis and being told there were no treatments, and then having to watch their young child slip away.

“With this approval, we are now one significant step closer to ensuring future generations of children, families and healthcare professionals no longer need to experience first-hand the terrible manifestations this disease has on untreated patients,” she said.

The approval of a treatment is already leading to new ways to catch the disease earlier.

Gaspar, of Orchard Therapeutics, noted that the company was supporting studies of newborn screening tests in the hopes of catching and treating the condition earlier, and screening is happening in some US states.

“Over 250,000 babies have been screened them as a result of these initiatives,” Gaspar said, and five babies with MLD have been identified. At least three of those babies have now been treated.

Riley said she wishes screening had been available for Olivia. The 5-year-old is stable for now, but Riley said she lives in fear that Keira could bring germs home.

“The common cold could kill her,” Riley said. “So every time her sister has come home with a cold from school, that could be potentially something that takes her out.”

Olivia can’t walk or talk, and she’s fed through a tube. Riley says she requires round-the-clock care. Physical and occupational therapists come to the house each week “to keep her mind moving. She does understand what’s going on.”

She said that as they have watched Olivia slip away, they’ve come to understand that her life has had profound purpose. But they say they hope that the approval of Lenmeldy means that no other families with MLD will have to lose one child to save another.

“We’ve always called Livvy ‘Keira’s guardian angel on Earth,’ ” Riley said. “She’s here for a reason.”

<<<



---

[north40000]

The FDA approved Amarin’s CVD risk-reducing drug, Vascepa, in 2019, having first approved it for triglyceride reduction in 2012. At a cost less than $60/year under my insurance plan, I began taking oral Vascepa capsules, off-label, with prescription in April 2013 at age 76(father died from 2nd heart attack at age 51 in 1959). Never obese, I have lost about 25 lbs while using 20% less LLY insulin to manage my T1-D since 2016. I now weigh the same as I did in my university years, 1955-1961.

PFE sells Vascepa in all Canadian provinces, per agreement.

AMRN’s share price, $0.90, is about even YTD. Share price was $18-25 in 2019. Shareholders will vote on a share repurchase plan in early April 2024 while waiting for revenue-driven earnings from Europe and Asia, and results from the Brave trial (Indication: Alzheimer’s ). Sarissa Capital holds ~ 9.9% of outstanding shares, and views Amarin as under-valued. Will AMRN share price out-perform share price of either LLY or NVO in 2024-2025 in % gain?

[gfp927z]

>>> Acurx Pharmaceuticals, Inc. (NASDAQ:ACXP) Q4 2023 Earnings Call Transcript


Published March 19, 2024

by INSIDER MONKEY TRANSCRIPTS


https://www.insidermonkey.com/blog/acurx-pharmaceuticals-inc-nasdaqacxp-q4-2023-earnings-call-transcript-1276747/#q-and-a-session


Acurx Pharmaceuticals, Inc. (NASDAQ:ACXP) Q4 2023 Earnings Call Transcript March 18, 2024

Operator: Greetings and welcome to the Acurx Pharmaceuticals Reports Fourth Quarter and Full Year 2023 Earnings Results and Business Update Call. At this time, all participants are in a listen only mode. A question-and-answer session will follow the formal presentation. [Operator Instructions] As a reminder, this conference is being recorded. I would now like to turn the call over to your host, Robert Shawah, Chief Financial Officer for Acurx Pharmaceuticals. Thank you. You may begin.

Robert Shawah : Thank you, Melissa. Good morning, and welcome to our call. This morning we issued a press release providing financial results and company highlights for the fourth quarter and full year 2023, which is available on our website at acurxpharma.com. Joining me today is David Luci, President and CEO of Acurx; as well as Robert DeLuccia, Executive Chairman. David will give a corporate update and outlook. After that, I’ll provide some highlights of the financials from the quarter and year end of December 31, 2023, and then turn the call back over to Dave for his closing remarks. As a reminder, during today’s call, we’ll be making certain forward-looking statements. These forward-looking statements are based on current information assumptions, estimates and projections about future events that are subject to change and involve a number of risks and uncertainties that may cause actual results to differ materially from those contained in forward-looking statements.

Investors should consider these risks and other information described in our filings made with the Securities and Exchange Commission, including our annual report on Form 10-K, which we filed on Friday, March 15, 2024. You are cautioned not to place undue reliance on these forward-looking statements and Acurx disclaims any obligations to update such statements at any time in the future. This conference call contains time sensitive information that’s accurate only as of the date of this live broadcast today, March 18, 2024. I’ll now turn the call over to Dave Luci. Dave?

David Luci : Thanks Rob. Good morning, everyone, and thanks for joining us to review our financial results for the fourth quarter of 2023 and also to hear some very exciting recent updates. Then we’d be pleased to take any questions. First, I’ll summarize some of our key activities for the fourth quarter of ‘23 or in some cases shortly thereafter. On October 2, 2023, we ended enrollment in our Phase IIb clinical trial of ibezapolstat or IBEZ, our lead antibiotic candidate for the treatment of patients with C. difficile infection or CDI. On November 2, 2023, we reported top line data from the Phase IIb clinical trial including overall results from the full Phase II study, demonstrating and ibezapolstat clinical cure rate at end 10 days oral treatment or EOT of 96%, 25 of 26 patients, which included 100% cure in Phase IIa and 94% cure in Phase IIb compared with vancomycin control arm the standard of which was 14 for 14 or a 100% at end of treatment and 94% was sustained cures.

No safety concerns were reported in either arm of the Phase IIb clinical trial and ibezapolstat was well tolerated in all patients in both the Phase IIa open-label trial and the Phase IIb vancomycin control segment. In consultation with our scientific advisors, the company determined that based on review of aggregate blinded data, the Phase IIb vancomycin control trial segment was terminated early due to success showing high observed clinical cure rates with no emerging safety concerns, clinical comparability was established. We also stated at that further data would be provided as it becomes available on secondary and exploratory endpoints for the Phase IIb trial segment, including sustained clinical cure data at 30 days after EOT and extended clinical cure data 94 days after EOT as well as comparative data on the impact on the patient’s microbiome.

On December 11 ‘23, we announced the sustained clinical cure data. These data showed that in the Phase IIb trial segment 100% or 15 out of 15 of our advantaged patients who were cured at EOT remained cured with no reinfection 30 days later, while vancomycin experienced a reinfection rate of 14.3%, 2 of 14 patients were reinfected.

On January 17, 2024, we announced positive comparative microbiology and microbiome data for the ibezapolstat in CDI patients from the Phase IIb clinical trial segment. Ibezapolstat to outperformed vancomycin showing eradication of fecal C. difficile at day three of treatment in 15 of 16 treated patients versus vancomycin, which had eradication of fecal C. difficile in just 10 of 14 treated patients. Additional data from the Phase IIb clinical trial showed ibezapolstat but not vancomycin consistently observed and allowed regrowth of key gut bacterial species believed to confer health benefits including prevention of recurrent C.difficile infection.

We anticipate that additional data from the secondary and exploratory endpoint will provide further favorable separation these two therapeutic options in our Phase III clinical trial program and ultimately in the marketplace if approved. Additional analysis, regarding other secondary and exploratory endpoints will be forthcoming as data become available. We remain particularly excited about the dual impact of ibezapolstat to treat acute C. difficile infection while appropriately managing the long-term care of each patient’s microbiome, which we believe is exceptional for antibiotic therapy. Having robust preclinical — clinical and manufacturing data to date, we submitted a formidable information package in early February to FDA along with a request for an end of Phase II meeting, which was granted by FDA on Feb 26, and is scheduled to occur in April.

We anticipate discussing our Phase III clinical trial mandate at this meeting and would anticipate documented meeting minutes from FDA sometime in the second quarter this year. We also announced that the European Medicines Agency approved their application to be designated as a small to medium sized enterprise or SME in Europe, which provides for certain benefits including fee reductions and other support from the European Medicines Agency for seeking a marketing authorization in Europe. In November, 2023, we filed an amendment to our shelf registration statement with the Securities and Exchange Commission and put up a $17 million at the market or ATM facility with Alliance Global Partners acting as sales agent to the company. Proceeds from the ATM will be used for general corporate purposes going forward, including our plan Phase III clinical trial mandate.

In October, 2023, at ID Week, Dr. Kevin Garey presented on our behalf with selective spectrum of activity data from our Phase IIa clinical trial. Many of you may recall, Dr. Gary is Professor and Chair University of Houston, College of Pharmacy and the Principal Investigator for our microbiome aspects of the ibezapolstat clinical trial program. Also at ID Week, Bob DeLuccia, our Executive Chairman, presented our new class of novel DNA pol IIIC inhibitors in our preclinical pipeline at the symposium entitled new antimicrobes in the pipeline. Now that’s a lot of activity to digest. So I’ll summarize further as to where we are today. As we speak, we’re preparing to advance ibezapolstat into Phase III clinical trials and anticipate a favorable outcome from our upcoming FDA meeting regarding readiness to proceed and also to obtain agreement on the regulatory pathway for a new drug application filing for marketing approval in the U.S. once Phase III is completed.

We’ve also officially started the regulatory process in Europe will add other territories to the list later this year. The Phase III trials will include U.S. and international sites to enhance overall enrollment and to support international regulatory filings for marketing approval. This will save us a lot of time and money and allow us to expand our ultimate commercial reach. To ensure Phase III clinical trial and enrollment as quickly as possible, we’re adding substantially more clinical trial sites, way above the number we used to conduct our U.S.-only Phase II trials. We’re now getting our arms around the costs and time lines, but our plan is to conduct the required two Phase III registration trials consecutively not concurrently given the size of our company and need to use our financial resources most efficiently.

The time line for conduct of our Phase III trial is not a concern since ibezapolstat will have a rolling 10 years of regulatory exclusivity in the U.S. from the FDA approval date with similar legislation in Europe, the U.K. and Japan. We will continue to seek strategic transaction for the company, including a potential partner for the further development and potential commercialization of ibezapolstat as well as a potential sale merger third-party ex U.S. territorial or licensing arrangement or our strategic transaction alongside in parallel with our preparation for Phase III clinical trials. At this time, we have no commitments from potential partners or others to provide the company with capital, but we started this initiative only recently in February after our Phase IIb data was released.

So basically, we have two formidable plans going forward, and we’re equally excited about partnering M&A and Phase III enrollment as next steps over the next 12 months. As we’ve consistently reported, ibezapolstat continues to outperform in a series of potentially life-threatening infectious disease called C. difficile that the U.S. CDC categorizes as an urgent threat and there’s a need for new classes of antibiotics for initial treatment but also has a low incidence of recurrence. Ibezapolstat also has FDA Fast Track designation for treatment of C. difficile infection. Additionally, we believe ibezapolstat, if approved, could make a favorable impact by reducing the cost burden of current C. difficile infection in our U.S. health care system, which is estimated at $4.7 billion annually.

We do believe the best is yet to come. And now back to our CFO, Rob Shawah to guide you through the highlights of our financial results for the fourth quarter and full year 2023. Rob?

Robert Shawah : Thanks, Dave. Our financial results for the fourth quarter and 12 months ended December 31, 2023, were included in our press release issued earlier this morning. The company ended the year with cash totaling $7.5 million compared to $9.1 million as of December 31, 2022. Subsequent to year-end, the company sold an additional 1,121,793 shares under its ATM financing program with gross proceeds of approximately $4.5 million. Research and development expenses for the three months ended December 31, 2023, were $1.9 million compared to $1.4 million for the three months ended December 31, 2022. The increase was due to timing of Phase IIb trial related costs and an increase in consulting costs. For the year ended December 31, 2023, research and development expenses were $6 million, which is $4.8 million for the year ended December 31, 2022.

The increase to due primarily the Phase IIb costs and an increase in consulting costs. General and administrative expenses for three months ended December 31, 2023 were $3.2 million compared to $1.8 million, where the three months ended December 31, 2022. The increase was due primarily to $800,000 increase in professional fees, a $0.1 million increase in share based compensation and a $0.3 million increase in employee compensation costs. For the year ended December 31, 2023, general and administrative expenses were $8.5 million versus $7.3 million for the year ended December 31, 2022. The amounts reflect an increase in professional fees of $0.5 million, an increase of $0.3 million in share based compensation and an increase of $0.3 million in employee compensation costs.

The company reported a net loss of $5.1 million or $0.37 per diluted share for a three months ended December 31, 2023, compared to an net loss of $3.3 million or $0.28 per diluted share for the three months ended December 31, 2022, and the net loss of $14.6 million or $1.15 per share for the year ended December 31, 2023 compared to a net loss of $12.1 million or $1.12 per diluted share for the year ended December 31, 2022, the reasons previously mentioned. The company had 14,468,229 shares outstanding as of December 31, 2023. With that, I’ll turn the call back over to Dave.

David Luci : Thanks Rob, and to all of you for joining us today. I’ll now ask Bob DeLuccia our Executive Chairman to provide his perspective given Bob manages our R&D program and when Bob is finished, operator, please open the call for questions. Bob?

Robert DeLuccia : Thanks Dave and Rob for updating our stakeholders and thanks to all for the continuing support as we advance ibezapolstat into Phase III clinical trials. It is a very exciting time and really it’s the final step to commercialize ibezapolstat for patients in need of a promising new antibiotic with a novel bactericidal mechanism of action to treat CDI, and many of you will remember that approximately 30,000 people die each year in the U.S. alone from CDI and the annual incidence is more than about 500,000 per year in the United States. But the bottom line here is that if the outcome of our Phase III trials are consistent with our Phase II experience, we’ll have an approvable new drug in our hands. The data are solid, the market is large and our manufacturing cost is low.

We do have a robust preclinical — clinical microbiome safety and CMC evidence package that we’ve submitted to the FDA and as Dave mentioned, they’ve determined it acceptable to grant us an end of Phase II meeting next month to discuss trial design, patient enrollment targets and to confirm our readiness to go forward. So to complete our Phase II international Phase III trials, which will be done sequentially as Dave mentioned and as quickly as possible, we’ve already initiated steps to advance screen potential clinical trial sites, which in addition to North America will cover sites in about 17 other countries, including several in Eastern Central, Northern and Western Europe, totaling about 100 sites, which we believe will accelerate overall enrollment.

We’ll also be including several high enrolling trial sites from the fast enrolling Summit Phase III CDI trial, which was conducted during the height of COVID-19. In addition to global scope of the program compared to Phase II and the strength of our Phase II data, the Phase III protocol will allow more flexibility for inclusion and exclusion criteria as a standard for CDI pivotal registration trials. So we anticipate faster enrollment. Then once we have results from our FDA meeting and documented meeting minutes from the FDA, our next priority will be to submit our plans to the European Medicines Agency or EMA for conducting Phase III clinical trials in Europe. So if approved, we’ll have the first new class of antibiotics approved by FDA in over 30 years with only confirmatory Phase III trials between now and market introduction.

Ibezapolstat as Dave mentioned is fast tracked by FDA, it’s fully patented and with regulatory exclusivity 10 years post-market introduction in the U.S. and similar opportunities for regulatory exclusivity in other geographies to pursue at the appropriate time. And also with recent focus on minimizing effects on the microbiome to lower recurrence of infection, we stand out from other antibiotics since we’ve shown no reinfection in Phase III patients (note - I think he meant Phase 2) who were initially cured of their infection. So in my over 50 years of experience in antibiotic development and marketing. I think, I have got a great rear view mirror and a clear vision to say that we have a winner here for patients with CDI and in general for better public health as well as for our shareholders.

Thanks for letting me comment, Dave.

Robert Shawah : Thank you, Bob. Melissa, we’re now ready to go to Q&A.

Operator: [Operator Instructions] First question is from the line of Jason McCarthy with Maxim Group.

Michael Okunewitch: This is Michael Okunewithch for Jason. I guess to start off, I’d like to see if you could give an idea of what sort of time line you are expecting between getting those meeting minutes back from the FDA and actually launching the first of those Phase III studies?

David Luci: So we think that we will be ready to enroll the first patient in for the international Phase III in the fourth quarter of this year.

Michael Okunewitch: And then, I guess, regarding the international component to the study, could you talk a little bit more about the path to actually getting those sites online? And is this something that you would look to have in place ahead of the launch? Or would you start with what do you have when you usually enroll the study like you are in these international sites as they were approved?

David Luci: We will be a little bit on both. We have a number of the sites already kind of ready to go from the international list. There won’t be any hold up from the international aspect of it. We actually have a consultant who is extremely familiar with Phase III C. diff international trials who’s been guiding us in this regard. But yes, clearly, when our manufactured product is ready, we’ll be ready to go with a large wedge at the very least of the hundred sites.

Michael Okunewitch: And then one last one from me, and I’ll hop back in the queue. With regards to securing international partnerships, do you think this is something with your existing body of data that is compelling enough that you could really progress those conversations now and get a partner or would you expect it to be more after you get that first slot of Phase III data?

David Luci: No, I would expect that the prime time for us to find, I’d say a European partner or a Japanese partner would be — when we start enrolling the first of the two Phase III trials, once we have the Phase III trial mandate completed and we’re ready to find or to file a new drug application, if we haven’t had a partnership by then, I think it would probably not occur until a year after the market introduction, because folks will want to see how well we do. I think that the time is right now. I’ll make one additional comment on the territorial partnerships to carry Michael’s question response a little bit further. As we look at strategic alternatives, there’s M&A full stop and there’s territorial licensing and co-development agreements that I have entered into in the past.

With our share price being where it is, the territorial licensing is a bit easier to consummate because it’s all about the intrinsic value of your drug as opposed to M&A, which you can’t in my view, wholly divorce yourself of your NASDAQ share price. So it may be a more attractive option for our board of directors and but we’ll see what term sheets present themselves.

Operator: Our next question comes from the line of James Molloy with Alliance Global Partners.

James Molloy : The Phase III trials, I know that obviously a lot to go here, but can you walk through expectation which we should expect to see coming out of the end of Phase II? And then the trial designed to sort of the length of time to run is about two years start to finish. So Phase IIb, I know we we’re dealing with COVID then as well. Is that seem reasonable to run these two Phase IIIs from start to finish?

David Luci: So we’re going to address one Phase III and then the idea would be to either do a strategic transaction after that first Phase III or with positive data on the first Phase III, if it’s the same or consistent with our Phase II, get a share price rise and then raise capital for the second Phase III. So we’re targeting a year and a half from start to finish for the first Phase III, and that would have us completing the first Phase III in the second quarter of ‘26. Now I can tell you that we spent a lot of time looking at the Summit Therapeutics experience and Summit enrolled in the teeth of COVID, 750 or so patients in about two years time largely in Eastern Europe, where the behavior patterns weren’t dramatically changed apparently, like they were here in the U.S. So, that’s a public list on clinicaltrial.gov.

So we were able to see exactly the heat patterns from their enrollment. So that’s gone a long way to helping us get comfortable with fixing the enrollment issues we had during COVID with our U.S. only trial.

James Molloy : I thought I’d heard you say earlier in the call that you’re going to run the two trials at the same time obviously misheard that, you’re running back to back.

David Luci: No. Our going in plan is to do them consecutively instead of concurrently for the financial reasons that we discussed. But if we get a partnership, say a European licensing agreement and that brings in and enough money to call an audible along the way and at that point then we would certainly start the second Phase III accordingly.

Operator: [Operator Instructions] Our next question comes from the line of Ed Arce with H.C. Wainwright.

Ed Arce : So a couple from me, firstly on the end of Phase II next month and I apologize I joined late, so maybe you went over this already, but wanted to make sure to review sort of the key objectives in particular what do you not have agreement with the agency yet on that you would seek to get agreement on next month in that meeting? And then secondly, I think you made mention of a wider inclusion exclusion criteria for the Phase III relative to the prior Phase II. And so just wanted to get a little more clarity on that in particular that a view to concerns there might be around the risk that you could change the sort of design of the trial and there could be some disruption from a slightly different patient population.

David Luci: Thank you, Ed. I’ll answer the first part of your question and ask Bob to provide a response for the inclusion and exclusion criteria part of your question. So for the first part of your question, what we hope to agree on with the FDA is the overall protocol designed for our Phase III trial, including the number of patients to be enrolled the notion that we’re going to have a vancomycin control arm like we had in our Phase IIb. Largely, the trial design is 90% plus of what the trail design was for our Phase IIb. So we don’t expect a real lot of drama, but it’s a necessary step in order to kind of be allow to into Phase III, so we’re looking forward to our valuation point that we have to get through. Bob, did you want to add some comment on the inclusion and exclusion criteria?

Robert DeLuccia: I think really two things, which is standard fair for going forward in Phase III is that we’ll have additional patients beyond mild and moderate that are included in the trial. So this is not severe CDI, but a little bit more than moderate according to the IDSA criteria for patient entry into a trial in the study. So we’ve already submitted a framework to the FDA in the meeting package. It’s very straightforward. I’d even say a higher percentage that they’ve said at 90%. We’re pretty much there with the design. It’s the same design basically, it’s Phase II and very standard according to the FDA guidance from October 2022, I believe it is, as to what needs to be included in the Phase III clinical trials. So we’ll be discussing final numbers of patients, as I said before and statistical analysis plan will be finalized at that upcoming FDA meeting as well. Anything else? Does that answer your question?

Ed Arce: Yes, that’s helpful.

Operator: Thank you. Ladies and gentlemen, that concludes our question-and-answer session. And this concludes our call today. We thank you for your interest and participation. You may now disconnect your lines.

<<<



---