Q2 2022 Lumos Pharma Inc Earnings Call

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Good afternoon and welcome to Lumos Pharma's second quarter 2022 results and clinical update conference call. Currently, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session and instructions will follow at that time. As a reminder, this conference call is being recorded. I will now turn the call over to Lisa Miller, Senior Director of Investor Relations.

Thank you, operator. Before we proceed with the call, I would like to remind everyone that certain statements made during this call are forward-looking statements under U.S. federal securities laws. These statements are subject to risks and uncertainties that could cause actual results to differ materially from historical experience or present expectations. Additional information concerning factors that could cause actual results to differ is contained in our periodic reports filed with the SEC.

The forward-looking statements made during this call speak only as of the date thereof, and the company undertakes no obligation to update or revise the forward-looking statements. Information presented on this call is contained in the press release we issued this afternoon and in our Form 8K, which may be accessed from the investors page of the company's website.

Speaking on today's call will be Rick Hawkins, CEO and Chairman, John McHugh, our President and Chief Scientific Officer, Dr. David B. Karp, our Chief Medical Officer, and Lori Lally, our Chief Financial Officer. I will now turn the call over to Rick.

Thank you Lisa and good afternoon everyone and thank you for joining us on today's call. So after the market closed today, we issued a press release announcing our financial results for the 2022 second quarter and detailing our progress advancing our clinical program evaluating room 201 for the treatment of idiopathic or moderate pediatric growth hormone deficiency or PGHD.

On today's call, I'll provide a brief update on LUM201 clinical trials and other developments before turning it over to Laurie Lawley for a review of our financial results.

then John and David will join us for a Q&A session.

I'm pleased to report that the enrollment trends we highlighted on our last call for our oral growth 210 and 212 trials have continued and are able to confirm our intention to announce interim data from both trials in the fourth quarter of this year.

Additionally, we expect a primary outcome data readout for both trials in the second half of 2023, which we expect to include data in 80 subjects from the oral growth 210 trial and up to 24 subjects anticipated from our oral growth 212 trial.

Our interim analysis from the ORAGROW210 trial will provide an early look at the safety and annualized height velocity or AHV data at three dose levels of LUM201 compared to a standard dose of recombinant human growth hormone in 40 patients at six months on therapy.

And as we approach interim data readout, I want to take a moment to explain what we'll be looking for in the data.

As most of you know, OraGrow 210 is a global multi-site, blinded, active control clinical study evaluating oral Loom 201 in approximately 80 subjects.

Diagnosed with idiopathic or moderate PGHD.

The primary clinical outcome is annualized height velocity at six months on LUM201 for subjects selected by our predictive enrichment marker or PEM strategy compared to the control arm of subjects treated with recombinant growth hormone.

So the data signal we will be looking for will be the annualized height velocity in these PEM positive subjects versus the recombinant growth hormone control arm, not historic comparisons to the height velocity in other trials that typically enroll more severely growth hormone deficient subjects.

It's also worth noting that oral growth 210, like all of the six month phase two trials in the growth hormone space is not powered to show non-inferiority.

The oral growth 210 trial is different from all prior PGHD registration studies.

This study is enriched to include only PEM-positive, idiopathic, or moderate PGHD subjects.

Our trial does not include individuals with more severe organic PGHD.

Now this is important because it is well known that more severe PGHD subjects respond better to recombinant growth hormone than to moderate PGHD subjects.

Therefore, the annualized high-velocity values we would expect in the more moderate idiopathic patient population enrolled in our trial will be lower in all treatment groups compared to, for example, prior long-acting growth hormone studies.

You will recall that the overall goals for the ORG-ROPE-210 trial are, one,

to identify the optimal dose of LUM201 from three dose levels, that is 0.8, 1.6, and 3.2 makes per kg a day.

to be used in a phase three registration trial, and two, for the annualized height velocity to be numerically comparable to the recombinant growth hormone control arm in this trial.

Three, to validate our PEM enrichment strategy. And finally, four, to confirm safety and tolerability of Lume 201.

And as we begin to plan for a single phase three trial required for approval, we expect that its design will mirror prior pivotal trials for growth hormone deficiency therapeutics conducted by our peers.

We therefore anticipate enrolling approximately 150 to 200 subjects in our pivotal trial, randomized 2 to 1, LUM201, to a control arm of daily injectable recombinant growth hormone.

The subjects will remain on therapy for 12 months, at which point height, velocity, and safety data from both cohorts will be obtained.

Again, the LUM201 dose administered in our Phase III trial will be determined from our Phase II Orgo 210 trial results.

These details are obviously subject to the approval of the FDA and other health authorities, and we'll engage the agency in discussions about the trial design at the appropriate time.

As I mentioned earlier, our upcoming interim data readout will also include data from our oral growth 212 trial, our single site open label trial evaluating the pharmacokinetic and pharmacodynamic effects of LUM201 in up to 24 PGHD subjects at the two higher dose levels of 1.6 and 3.2 mg per kg a day.

The objective of the 2-12 trial is to confirm prior clinical data demonstrating the amplified pulsatile release of endogenous growth hormone unique to LUM201.

and the potential for this mechanism of action to increase growth hormone secretion across the entire dose response curve in the majority of PGHD patients.

The primary endpoint for this trial is six months of PKPD data, with additional height velocity data also being assessed at six months and from the extension of this trial to follow subjects to near adult height.

We also expect to announce the interim data from the oral growth 212 trial in the fourth quarter of this year.

For the interim data readout, comparability of baseline characteristics of subjects in each study will determine whether it is appropriate to pull the annualized high-velocity data from the ORGRA 2010 and 2012 datasets for combined analysis.

The primary outcome data readout for oral growth 212 is anticipated around the same time as the primary data readout for the oral growth 210 trial in the second half of 2023. And we expect this oral growth 212 dataset to include data in up to 24 subjects.

In the last quarter, we announced the initiation of our R-Growth 213 trial, or the SWITCH Study.

This is an open-label, multi-center, phase 2 study evaluating the growth effects and safety of LUM201 following 12 months of daily recombinant growth hormone in up to 20 PGHD subjects who have completed the oral growth 210 trial.

Subjects will then be administered LUM201 at a dose level of 3.2 mg per kg a day for up to 12 months.

Primary outcome for the 2013 trial is annualized height velocity. Secondary outcomes include safety and PK PD measures. This trial continues to enroll subjects from the oral growth 2010 trial.

Now before turning to other updates, I want to touch on two operational matters related to our clinical trials.

We announced last quarter the latest suspended enrollment at our Oro Road 210 clinical sites in Russia and Ukraine due to ongoing conflict.

Fighting in the region has continued, so we have decided to formally close these sites. And as a reminder, no subjects were enrolled at these sites prior to their suspension, so we do not expect these closures to impact our target data timelines.

We're adding a few sites in the United States which should allow us to maintain our original enrollment timelines.

And additionally, as many as you probably know, COVID cases continue to have arisen again, both in the US and internationally, and the disease continues to be unpredictable.

Fortunately, the world continues to adjust to the presence of COVID, and we therefore do not believe that the ongoing pandemic will alter our current clinical development plan.

We will, of course, continue to monitor the situation closely.

As our clinical trials advance toward data readouts, we're noticing significant interest in the potential of LUM201 building within the wider PGHD research community.

Attendees at both the Indoconference in June and the Magic Foundation meeting in July advised us that they are following our progress closely and are looking forward to our interim data readout.

Last quarter, we announced that renowned pediatric endocrinologist, Dr. Pasit Pituktawane, or Dr. Duke as we call him, joined our medical affairs leadership team.

Dr. Duke recently attended the Human Growth Foundation Gala and has been holding investigator meetings in both the U.S. and internationally, and he has observed this growing interest as well.

We believe that this reflects the desire among healthcare professionals for an alternative to daily or weekly injections of growth hormone to treat PGHD patients.

Feedback, I can tell you of this nature goes beyond mere buzz.

Based on discussions we're having within the broader PGHD community, we believe that the availability of an oral option to treat PGHD has the potential to expand the overall market.

Considering the hesitance of some parents have been when faced with the treatment of burden of daily or weekly injections involved with current standard of care, it's not surprising that an oral option may be more attractive.

This is especially true in the more moderate cases that LUM201 is intended to treat, or the exact patient population we are rolling in our current trials.

We look forward to working with both the healthcare professionals and the PGHD patient community to raise awareness about LUM 201 programs and the potential of this drug to be a real game changer in this space.

Now turning to the collaboration we announced last quarter with Dr. Laura Dictal and Massachusetts General to explore the potential of LUM 201 in patients with non-alcoholic fatty liver disease or NAFL.

This investigator-sponsored pilot trial is a single-site, six-month, open-label study of daily oral LUM201 in adults with NAVLD.

The trial has been rapidly prescreening patients and should begin enrollment in the near future.

Again, this pilot study will evaluate the dose of 25 makes per day of LUM 201 in 10 subjects with non-alcoholic fatty liver disease or NAFL.

and relative IGF-1 deficiency.

Prior studies evaluating growth hormone in this indication have been promising, supporting the potential for oral Loom 201 to address this condition.

And while we remain focused on our core Loom 201 program and PGHD, we are pleased to support Mass General's exploration of Loom 201's potential in this indication, a condition estimated to be prevalent in approximately 25% of adults worldwide, and which can often advance to the more serious liver diseases and result in need for a liver transplant.

As we have mentioned...

We continue to explore expansion opportunities for LUM201 and other conditions where injectable recombinant human growth hormone is a standard of care.

We believe that loop 201 is a pipeline and a product and through its unique mechanism of action may have the potential to be efficacious in indications such as Turner syndrome Prader-Willi syndrome idiopathic short stature and children born small for gestational age.

We're actively reviewing potential clinical development plans for LUM201 and several of these indications and believe that interim data from our org-led trials should clarify the direction we take.

We continue to be judicious in our pursuit of rare disease assets beyond LUM201 with careful attention to shareholder value creation. That said,

Our priority remains Loom 201 and its development.

And with that, I'm going to pass it over to Laurie for a review of our second quarter financial results. Laurie?

Thanks Rick, and good afternoon everyone. For the quarter ended on June 30, 2022, we had cash and cash equivalents totaling $79.5 million, compared to $94.8 million on December 31, 2021. We expect average cash use of approximately $8.5 to $9.5 million per quarter through the remainder of 2022. Cash on hand as of the end of our second quarter is expected to support our operations into Q2 2024.

Research and development expenses were $4.6 million for the second quarter, an increase compared to $4.1 million for the same period in 2021, primarily due to an increase of $0.3 million in personnel and stock option expense and $0.3 million in legal and consulting expenses, offset by a decrease of $0.1 million in clinical trial and contract manufacturing expenses.

General and administrative expenses were $3.7 million for the quarter ended June 30, 2022, a decrease as compared to $4.6 million for the same period in 2021, primarily due to decreases of $0.6 million in personnel related expenses, $0.4 million in stock compensation expenses, and $0.3 million in legal and other expenses, offset by an increase of $0.3 million in royalty expenses.

The increase in royalty expenses were due to fees paid to the Public Health Agency of Canada as a result of royalties earned of $0.4 million from MARC for the sale of Urbibo, the Ebola vaccination.

The net loss for the second quarter was 7.8 million compared to net loss 8.7 million for the same period in 2021. Lumos Pharma ended the second quarter with 8,377,567 shares outstanding.

I will now turn it back to Rick for closing remarks.

Thank you, Laurie. And so during our second quarter, we continued to execute on our plans for Loom 201.

Enrollment trends for our oral growth trials continues to be positive.

This is a very special time in the history of Lumos Pharma.

We're excited to be able to announce the results of our interim analyses from two Phase II studies evaluating oral Loom 201 in our fourth quarter.

The therapy potential of LUM201 continues to garner attention in both clinical and patient communities.

Our cash position is solid, sufficient to carry us into the second quarter of 2024, beyond both the interim readouts in Q4 of this year and the primary outcome data readouts for oral growth 210 and oral growth 212 in the second half of 2023. Once again, this is an exciting time for Lumos Pharma as we look forward to providing interim results by the end of this year.

Operator, we're now ready to take questions.

Thank you. We'll now begin the question and answer session.

To ask a question, you may press star then one on your touch tone phone. If you're using a speakerphone, please pick up your handset before pressing the keys. To withdraw your question, please press star then two.

At this time, we'll pause momentarily to assemble our roster.

Our first question comes from Charles Duncan from Kander Fitzgerald. Please go ahead. Yes, thank you.

Hi, good afternoon, Rick and team. Congratulations on the progress and thanks for taking our questions. Had a couple of questions about the oral growth 210 study. I guess I'm wondering

if as you characterize enrollment patterns

second quarter, can we assume that those are continuing roughly the same into the third quarter despite some of the kind of usual summer slowdown? And then you mentioned the height, not studying highly severe PGHD and really looking at moderate levels. So could you let us know what you would tell those particular patients?

even just responding to recombinant growth hormone.

David, why don't you proceed with that question? Hi Charles, both great questions. So to the first question as far as the enrollment, your assumption is correct.

There does not appear to have been any slowdown in enrollment and that is why we can really report that the enrollment is still going very, very well.

And again, it's really important to understand that...

PGHD writ large is primarily combined of two subsets. 35-40% of the kids.

have organic GHD which is indicated by an abnormality at MRI involving the pituitary usually or occasionally can result from a brain cancer and radiation therapy knocking off all the rhythm producing cells.

About two-thirds, 60-65%.

of the population has so-called idiopathic GHD, which means non-organic. Idiopathic basically means you don't know why they have GHD, although it's felt now to be probably a hypothalamic.

dysregulation because the hypothalamus controls the pituitary and that's tailor-made really for a compound with the mechanism of LUM201 which really you know binds to the growth hormone secreting or hormone receptor in the hypothalamus as well as pituitary and also suppresses the metastatin.

So by both those actions it enhances normal release of growth hormone. So as far as the question about what kind of growth we expect, when we look at the studies that have been done in the past that have evaluated this.

And we've looked at a cut of the Genesis database that we've reported previously and is published where we have kids that basically meet the requirement for our studies. And also we look at a study that was published actually in a Spanish group of 100 kids with idiopathic GHD.

So, the response to the standard dose of growth hormone really...

are aligned in both of those analyses, and it's right around 8.3 centimeters per year.

which is somewhat less than what you see in the more severe GHD kids who can grow 10, 10.5, etc. centimeters per year.

So, um,

Many, many studies over the past 30, 40 years, you know, have demonstrated that the more severe the GHT, the better response to growth hormone. So, kids that are severely GHT, you can basically wave the bottle over them, they'll grow. They grow perfectly well on much less than standard doses of growth hormone, those as though it's 0.16 to 0.18 mgs per kick per week. Whereas the standard in the trials is 0.24 and the average in the US is probably 0.28, 0.30, makes per kick per week.

And so all these studies show that if you are younger, if you have more lower growth hormone peak response to stimulus, like lesson three, which is exclusionary for our study, the lower your IGF-1, SDS, the farther away you are from mid-prandtl height, the lower your base and high velocity. All of these factors predict a greater response to growth hormone.

And so because we're really identifying the bulk of the population with idiopathic, we don't think that they have exactly the same growth potential as the more severe subjects who were studied, for example, in the height study that I ran when I was at

And so because we're really identifying the bulk of the population with idiopathic, we don't think that they have exactly the same growth potential as the more severe subjects who were studied, for example, in the height study that I ran when I was in tremendous, for example, and all the other long-acting with urban studies, which tend to favor enrollment of the more severe organic subjects over the less severe subjects. Does that answer your question?

I think it absolutely does. Very thorough and I appreciate that. And then when you look at on a blinded basis at some of the phenotypic variables that you can easily see such as age, etc. and other measures, do you feel that your subject or your vocal

sample is enrolling the patients that you really want to have in this study.

Yes, we're doing a very good job of excluding organic subjects in the trial. And that's pretty key because a subject with organic GHD is not going to respond to our PEM test because they can't.

respond, nor are they likely to respond to Loom 201. And that's part of the reason why our screen failure rate is actually lower than we had anticipated. Because if the investigators were just enrolling any kid, we expect about, you know, two-thirds of the population to be PIM.

respond to LUM201. And that's part of the reason why our screen failure rate is actually lower than we had anticipated. Because if the investigators were just enrolling any kid, we'd expect about, you know, two-thirds of the population to be Facebook search created and that would keep them positive.

And we had to be excluding one-third of the kids that are screened, but because they're really identifying kids with the diagnosis of idiopathic GHD, we're experiencing a much lower screen failure rate than we had put into the protocol.

Okay, last question quickly on the switch study. Rick mentioned that you are enrolling patients. Can you be at all quantitative or at least qualitative with regard to the percentage of patients who complete the 210 study who decide to switch over to the switch study? We can right now because it is only 30 minutes throughout.

So the sample size is really too small to comment, I think, right now.

It's fair to say that all the subjects who have completed to date have desired to continue on in LUM201.

Okay, very good. That's helpful. Thanks for taking the questions.

Next question comes from Jasmine Rahini from Piper Sandler. Please go ahead.

Hi, this is Emma on for ya. Thank you for taking our questions.

pub.

40 or 45 percent enrollment from London, Australia and New Zealand. So it's