On May 5, 2021 IVERIC bio, Inc. (Nasdaq: ISEE) reported financial and operating results for the quarter ended March 31, 2021 and provided a general business update (Press release, Ophthotech, MAY 5, 2021, View Source [SID1234579255]).

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"Iveric Bio is entering an important period as we remain focused on the execution of our ongoing Zimura GATHER2 clinical trial, which is our second Phase 3 clinical trial for Zimura for the treatment of geographic atrophy secondary to age-related macular degeneration. We are committed to completing recruitment for the GATHER2 trial in the third quarter of this year," stated Glenn P. Sblendorio, Chief Executive Officer of Iveric Bio. "To date, both the recruitment and retention of patients in GATHER2 have exceeded our expectations. We are on track for initial, topline data from GATHER2 to be available approximately one year after the recruitment of the last patient in the GATHER2 clinical trial, plus the time needed for database closure and analysis of the initial, topline data."

"A key goal of ours is to expand and advance our footprint in multiple stages and types of AMD," stated Pravin U. Dugel, M.D., President of Iveric Bio. "We are excited by the opportunity to potentially expand the reach of Zimura beyond GA and to continue the development of IC-500, our HtrA1 inhibitor, which we expect could be complementary to Zimura in treating AMD patients. We remain committed to developing safe and effective therapeutic and gene therapy treatment options for retinal diseases with significant unmet medical needs."

Therapeutics Programs Targeting Geographic Atrophy Secondary to Age-Related Macular Degeneration

Zimura (avacincaptad pegol): Complement C5 Inhibitor

Enrollment and retention for GATHER2, the Company’s pivotal clinical trial of Zimura in development for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD), are progressing well with enrollment on target. In March 2021, the Company announced it accelerated the timeline for when it expects to complete enrollment in GATHER2 to the third quarter of 2021.
The Phase 2b screening clinical trial of Zimura for the treatment of autosomal recessive Stargardt disease, referred to as the STAR trial, is ongoing with the goal of enrolling approximately 120 patients.
IC-500: HtrA1 (high temperature requirement A serine peptidase 1 protein) Inhibitor

During the first quarter of 2021, the Company revised its development plans for IC-500 to include plans to investigate multiple dosing schedules for this product candidate. In April 2021, the Company commenced its first preclinical tolerability study for IC-500 and is currently planning additional preclinical studies, including pharmacokinetic and target engagement studies. Formulation optimization and other manufacturing activities are also ongoing. The Company expects to submit an IND to the FDA for IC-500 in GA secondary to AMD in the second half of 2022.
Iveric Bio to Host Dry AMD Virtual Symposium for Investors/Analysts

The Company will host a dry AMD Virtual Symposium for investors and analysts on Friday, June 18, 2021 from 10:00am – 12:00pm Eastern Time. The event will include presentations and discussions with retinal specialists and key opinion leaders on the dry AMD landscape, Zimura pivotal program in GA and highlights from the Company’s IC-500 program in AMD. The event will be accessible via webcast on the Iveric Bio website at www.ivericbio.com. For more information, please contact Kathy Galante at [email protected].

Gene Therapy Programs in Orphan Inherited Retinal Diseases (IRDs)

IC-200: BEST1-Related IRDs
The Company is completing a preclinical efficacy and toxicology study for IC-200, in the naturally occurring canine model of Best disease. Published data have demonstrated long-term rescue in this model following a single sub-retinal injection. The Company is on track to release the recently manufactured cGMP batch of IC-200 in preparation for the planned IND filing and plans to move IC-200 into the clinic, in a Phase 1/2 trial in the second half of 2021.
IC-100: Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa (RHO-adRP).
The Company continues to evaluate the results of preclinical toxicology studies for IC-100. In the Company’s preclinical efficacy and toxicology study in a naturally occurring canine model of RHO-adRP, efficacy was demonstrated at all three doses tested. The Company also tested the same three doses in a GLP toxicology study in non-human primates. Ocular inflammation on clinical exam was observed in the high dose group in canines and to varying degrees at different dosing levels tested in non-human primates. Due to the different findings in the two different species, and the Company’s high commitment to the safety of its patients, the Company is planning to discuss with regulators the design of its planned first-in-human clinical trial for IC-100 prior to submitting an IND. The Company now believes that IC-100 will likely be delayed from entering into a Phase 1/2 clinical trial this year.
Minigene Programs
The Company, in its minigene collaboration with the University of Massachusetts Medical School, has identified a lead construct for its Leber Congenital Amaurosis Type 10 (LCA10) program and is currently considering development plans for this program. The Company expects to obtain additional results from its Stargardt Disease (ABCA4) program in the second quarter of 2021, and expects to obtain preliminary results from its USH2A-related inherited retinal diseases program in the second half of 2021.
The Company announced today the formation of its Gene Therapy Inherited Retina Disease Scientific Advisory Committee that will work closely with senior management as the Company advances its gene therapy inherited retinal disease programs. The members of the advisory committee include:

Elias Traboulsi, MD, MEd
Head of the Department of Pediatric Ophthalmology
Director of the Center for Genetic Eye Diseases
Cole Eye Institute
Professor of Ophthalmology, Cleveland Clinic Lerner College of Medicine,
Cleveland Clinic
Andreas K. Lauer, MD
Chair, Department of Ophthalmology, Casey Eye Institute
Professor of Ophthalmology, School of Medicine
Bart P. Leroy, MD, PhD
Head, Department of Ophthalmology, Ghent University Hospital
Senior Staff Member, Center for Medical Genetics Ghent, Ghent University Hospital
Professor of Ophthalmology & Ophthalmic Genetics, Ghent University
Director of the Retinal Degenerations Clinic Children’s Hospital of Philadelphia
Mark Pennesi, MD, PhD
Division Chief, Ophthalmic Genetics
Associate Professor in Ophthalmology, Oregon Health & Science University
Eleonora Lad, MD, PhD
Director of Grading, Duke Reading Center
Associate Professor of Ophthalmology, Duke University Medical Center
Board of Directors and Management

Today the Company announced the promotions of Pravin U. Dugel, MD, to President, and Kathy Galante to Senior Vice President, Investor Relations, both effective as of May 1.
In April 2021, the Company announced that David R. Guyer, MD, was stepping down from the Iveric Bio Board of Directors after 14 years, effective following Iveric Bio’s 2021 Annual Stockholder Meeting scheduled to be held on May 19, 2021.
First Quarter Financial Results and 2021 Cash Guidance

As of March 31, 2021, the Company had $180.2 million in cash, cash equivalents and available for sale securities.
The Company estimates its year-end 2021 cash, cash equivalents and available for sale securities to range between $125 and $135 million. The Company also estimates that its cash, cash equivalents and available for sale securities will be sufficient to fund its planned capital expenditure requirements and operating expenses, excluding any potential approval or sales milestones payable to Archemix Corp. or any commercialization expenses for Zimura, into 2024. These estimates are based on the Company’s current business plan, including the continuation of its ongoing clinical development programs for Zimura, the progression of its IC-100 and IC-200 programs into the clinic, and the advancement of its IC-500 development program. These estimates also assume that the Company will enroll approximately 400 patients in the GATHER2 trial. These estimates do not reflect any additional expenditures related to potentially studying Zimura in other indications or resulting from the potential in-licensing or acquisition of additional product candidates or technologies or commencement of new sponsored research programs, and any associated development the Company may pursue.
2021 Q1 Financial Highlights

R&D Expenses: Research and development expenses were $18.5 million for the quarter ended March 31, 2021, compared to $13.8 million for the same period in 2020. Research and development expenses increased primarily due to the initiation of our GATHER2 trial and commencement of patient enrollment and increased manufacturing activities for Zimura, increased manufacturing and preclinical development activities associated with the Company’s IC-100 and IC-200 gene therapy programs and the progression of its IC-500 development program.
G&A Expenses: General and administrative expenses were $8.3 million for the quarter ended March 31, 2021, compared to $5.0 million for the same period in 2020. General and administration expenses increased primarily due to legal costs associated with ongoing litigation.
Income Tax Benefit: For the quarter ended March 31, 2021, the Company recorded no income tax benefit. An income tax benefit of $3.3 million was recognized in the quarter ended March 31, 2020 to reflect a favorable settlement of a state corporate income tax audit.
Net Loss: The Company reported a net loss for the quarter ended March 31, 2021 of $26.8 million, or ($0.29) per diluted share, compared to a net loss of $15.1 million, or $(0.28) per diluted share, for the same period in 2020.
Conference Call/Web Cast Information

Iveric Bio will host a conference call/webcast to discuss the Company’s financial and operating results and provide a business update. The call is scheduled for May 5, 2021 at 8:00 a.m. Eastern Time. To participate in this conference call, dial 888-317-6003 (USA) or 412-317-6061 (International), passcode 5841649. A live, listen-only audio webcast of the conference call can be accessed on the Investors section of the Iveric Bio website at www.ivericbio.com. A replay will be available approximately two hours following the live call for two weeks. The replay number is 877-344-7529 (USA) or 412-317-0088, passcode 10153477.

On May 5, 2021 Evotec SE (Frankfurt Stock Exchange: EVT, MDAX/TecDAX, ISIN: DE0005664809) reported that the Company, together with Bristol-Myers Squibb Company (NYSE: BMY), has launched beLAB1407, a new $ 20 m academic BRIDGE to identify and advance novel and breakthrough drug discovery opportunities across therapeutic areas from the UK’s top-tier academic institutions (Press release, Evotec, MAY 5, 2021, View Source;announcements/press-releases/p/evotec-launches-belab1407-to-accelerate-translational-research-from-the-uks-academic-life-science-ecosystem-in-collaboration-with-bristol-myers-squibb-6064 [SID1234579254]). Through a unique combination of Evotec’s drug discovery and development platforms and early-stage therapeutic concepts from the Universities of Birmingham, Edinburgh, Nottingham, and Dundee, beLAB1407 offers a unique route to the advancement of first-in-class therapeutics and the creation of spin-out companies.

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Evotec’s BRIDGE (Biomedical Research, Innovation & Development Generation Efficiency) collaborations provide an integrated fund and award framework to validate exciting academic projects in collaborations with Pharma and biotech with the goal to form new companies. Since implementing the first academic BRIDGE ‘LAB282’ in Oxford in November 2016, Evotec has continued to evolve similar collaborations with a variety of academic, Pharma and venture capital partners across Europe and North America.

With beLAB1407 representing the continued refinement of the approach, Evotec links one of Europe’s foremost academic clusters of excellence in the life sciences with Bristol Myers Squibb as a major biopharmaceutical partner dedicated to advancing innovation in therapeutics from academia to patient benefit. The beLAB1407 collaboration builds on the longstanding and successful relationship between Evotec and Bristol Myers Squibb in drug discovery across several therapeutic areas.

Dr Werner Lanthaler, Chief Executive Officer of Evotec, said: "We are thrilled to launch beLAB1407 together with our partners at Bristol Myers Squibb with whom we’ve worked on a variety of projects over a period of many years. beLAB1407 provides researchers from the member institutions with a unique way to fast-track their projects, to validate them on our industrial-grade platform and have partnering options including company formations readily available to them."

Dr Thomas Hanke, Head of Academic Partnerships at Evotec, added: "We are delighted to see the BRIDGE concept gaining further traction in the UK and we very much look forward to working closely with our academic partners in the Midlands and Scotland as well as our colleagues at BMS to identify and accelerate the next generation of first-in-class therapeutics across all modalities and therapeutic areas."

Dr Rupert Vessey, Executive Vice President and President, Research and Early Development at Bristol Myers Squibb, commented: "This collaboration builds on our important connection to leading European universities. With beLAB1407, we are supporting U.K.-based universities that are exploring many interesting lines of scientific research and discovery. That research combined with Evotec’s proprietary data platforms has the potential to identify new and novel therapies for areas of unmet medical need."

The name beLAB1407 refers to the distance between Land’s End in the far southwest of Great Britain to its north-easternmost point near the village of John o’ Groats in Scotland, which – if travelled by bike – adds up to 1,407 kilometres. To learn more about beLAB1407, please visit www.belab1407.org.

On May 5, 2021 vTv Therapeutics Inc. (Nasdaq:VTVT) reported financial results for the first quarter ended March 31, 2021, and provided an update on the progress of its clinical programs (Press release, vTv Therapeutics, MAY 5, 2021, View Source [SID1234579253]).

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"The vTv team continued to build on the clinical success of our type 1 diabetes program during the quarter by obtaining Breakthrough Therapy designation from the FDA for TTP399 and by initiating a phase 1 study to understand TTP399’s impact on ketoacidosis. The FDA’s grant of Breakthrough is an acknowledgment of the significant unmet need for patients with type 1 diabetes, and the encouraging clinical results we have generated to date," said Steve Holcombe, president and CEO, vTv Therapeutics. "We look forward to working closely with the FDA to optimize the development pathway for TTP399 to bring this potential treatment to patients with type 1 diabetes as quickly as possible."

Recent Achievements and Outlook

Type 1 Diabetes

Breakthrough Therapy Designation. As announced in April, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation for TTP399 as an adjunctive therapy to insulin for the treatment of type 1 diabetes. Breakthrough Therapy designation is intended to expedite the development and review of a drug candidate that is planned for the treatment of a serious or life-threatening disease where initial clinical evidence indicates that the drug may demonstrate a substantial improvement over existing therapies on one or more clinically significant endpoints.

Mechanistic Study of Ketoacidosis with TTP399. The Company began dosing patients in a mechanistic study of TTP399 in people with type 1 diabetes to determine the impact of TTP399 on ketone body formation during a period of acute insulin withdrawal. vTv expects to report topline results from the mechanistic study in the third quarter of 2021.

Pivotal Study Planning. The Company is planning to initiate the first of two pivotal, placebo-controlled, six-month clinical trials of TTP399 in subjects with type 1 diabetes in the fourth quarter of 2021. The current study designs will be shared with the FDA for its input and advice as part of the collaborative process enabled by the Breakthrough Therapy designation.
Psoriasis

Multiple Ascending Dose Study with HPP737. The Company began dosing healthy subjects in a phase 1 multiple ascending dose study to assess the safety, tolerability, and pharmacokinetic profile of HPP737, an oral PDE4 inhibitor. This phase 1 study is expected to report topline results in the third quarter and will inform dose selection for a planned phase 2 study in psoriasis.
License Partner Updates

Reneo Pharmaceuticals Completes Initial Public Offering. We congratulate our licensee, Reneo Pharmaceuticals, Inc. (Nasdaq: RPHM), for successfully completing an initial public offering in April. The Company is party to a license agreement with Reneo for its lead asset REN001, which Reneo is currently developing in three rare genetic diseases: primary mitochondrial myopathies, long-chain fatty acid oxidation disorders, and glycogen storage disease type V (McArdle disease).
First Quarter 2021 Financial Results

Cash Position: The Company’s cash position as of March 31, 2021, was $8.4 million compared to $5.7 million as of December 31, 2020.

Revenue: Revenue for the first quarter of 2021 was $1.0 million and was $6.4 million for the fourth quarter of 2020. The revenue for the first quarter was non-cash and related to the recognition of revenue pertaining to the Huadong license agreement. The revenue for the fourth quarter was primarily attributable to the upfront consideration, consisting of cash and an equity interest, received in connection with the Company’s license agreement with Anteris Bio.

R&D Expenses: Research and development expenses were $3.1 million and $2.5 million for the three months ended March 31, 2021 and December 31, 2020, respectively. This increase of $0.6 million was driven primarily by the increase in spending for our study of HPP737 in psoriasis.

G&A Expenses: General and administrative expenses were consistent between periods at $2.2 million and $2.0 million for the three months ended March 31, 2021 and December 31, 2020, respectively.

Net (Loss)/Income Before Non-Controlling Interest: Net loss before non-controlling interest was $5.9 million for the first quarter of 2021 compared to net income of $1.6 million for the fourth quarter of 2020.

Net (Loss)/Income Per Share: Diluted net loss per share was ($0.08) for the three months ended March 31, 2021 compared to a diluted net income per share of $0.02 for the three months ended December 31, 2020, based on weighted-average diluted shares of 56.5 million and 74.4 million for the three-month periods ended March 31, 2021 and December 31, 2020, respectively. Non-GAAP net loss per fully exchanged share was ($0.09) for the three months ended March 31, 2021 compared to a net income per fully exchanged share of $0.02 at December 31, 2020, based on non-GAAP fully exchanged weighted-average shares of 79.6 million and 74.4 million for the three months ended March 31, 2021 and December 31, 2020, respectively.

On May 5, 2021 DiaMedica Therapeutics Inc. (Nasdaq: DMAC), a clinical-stage biopharmaceutical company focused on developing novel treatments for neurological disorders and kidney diseases, reported that financial results for the quarter ended March 31, 2021 (Press release, DiaMedica, MAY 5, 2021, View Source [SID1234579252]). DiaMedica will host a conference call Thursday, May 6, 2021, at 7:00 a.m. Central Time to discuss its business update and first quarter financial results.

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Clinical Developments

DM199 for the Treatment of Acute Ischemic Stroke

IND Submitted
Initiation of Phase 2/3 Trial of DM199 in AIS in Summer 2021
DiaMedica has submitted an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for an adaptive Phase 2/3 clinical trial of DM199, the Company’s recombinant form of human tissue kallikrein-1 being developed as a treatment for acute ischemic stroke (AIS) patients. The proposed trial of DM199 is for AIS patients who do not receive tissue plasminogen activator (tPA) and do not have large vessel occlusions, a group that represents up to 80% of all AIS patients. DiaMedica believes that the proposed trial has the potential to serve as a pivotal registration study of DM199 in this patient population.

The FDA has confirmed its receipt of the IND and DiaMedica expects the FDA to complete its regulatory review of the IND by mid-May. If authorized by the FDA, the Company anticipates initiating the study this summer. DiaMedica has selected a contract research organization to assist with the conduct of the study and is actively working to identify, qualify and engage clinical study sites—the expected pacing item for initiation of the study.

The clinical trial is proposed to be a double blinded, placebo controlled, randomized study of approximately 350 participants, based on a 90% powering for statistical significance on the primary endpoint of modified Rankin Scale (mRS) at day 90. Secondary endpoints will include stroke recurrence, mRS shift, NIHSS and Barthel Index, deaths, safety and tolerability measures, and biomarkers relating to KLK1. Independent of this study, the Company plans to discuss with the FDA a second study for the evaluation of the efficacy of DM199 in reducing stroke recurrence based upon the statistically significant reduction in severe recurrent strokes observed in its ReMEDy Phase 2 AIS trial.

"We are pleased with the overall progress of our AIS program," commented Rick Pauls, Chief Executive Officer of DiaMedica. "We look forward to initiation of this trial and moving closer to potentially providing stroke patients and physicians a much-needed new treatment option for AIS."

DM199 for the Treatment of Chronic Kidney Disease

Data Read-out Expected in Q2 2021 on CKD Caused by Type II Diabetes Cohort
Enrollment Continues in IgA Nephropathy and CKD in African Americans with Hypertension
DiaMedica’s Phase 2 REDUX trial is a multi-center, open-label, investigation to assess the safety and efficacy of multiple doses of DM199, administered over 90 days, in participants with chronic kidney disease (CKD) (Stage 2 or 3) targeting enrollment of approximately 90 participants in three equal Cohorts. Cohort 1 of the study is focused on non-diabetic African Americans with hypertension, a group that is at greater risk for CKD than Caucasians. Additionally, the study is designed to identify African American participants with the APOL1 gene mutation as an exploratory biomarker as these individuals have an even higher risk of developing CKD. Cohort 2 of the study is focused on participants with IgA Nephropathy (IgAN) which is found more commonly in Asians, Caucasians and people of Eastern Europe and is very rare in Blacks and people of African descent and Cohort 3 includes participants with diabetic kidney disease (DKD) which is defined by elevated urine albumin excretion and/or reduced glomerular filtration rate (GFR) – is a serious complication that occurs in 20% to 40% of all diabetics.

As of April 30, 2021, DiaMedica had enrolled a total of 70 subjects, including a fully enrolled DKD Cohort, and approximately 70% of the IgAN and 60% of the African American Cohorts. The Company has continued to experience slower than expected enrollment in the first two Cohorts of the REDUX trial due to the COVID-19 pandemic. However, with the significant declines in new COVID-19 cases and the anticipated availability and effectiveness of vaccines, the Company currently anticipates completion of Cohort 1 and Cohort 2 in the second half of 2021. Preliminary topline results from the DKD Cohort are expected to be available in the second quarter of 2021.

Financial Results

Research and development (R&D) expenses were $2.4 million for the three months ended March 31, 2021, compared with $1.4 million for the three months ended March 31, 2020, an increase of $1.0 million. The increase was due to a number of factors including a year-over-year increase in costs incurred for the REDUX Phase 2 CKD study, and costs associated with an increase in staff levels, consulting services and non-clinical testing required to support preparation for the ReMEDy2 Phase 2/3 stroke study. These increases were partially offset by a year-over-year decrease in costs incurred for the ReMEDy Phase 2 stroke study which completed during 2020.

General and administrative (G&A) expenses were $1.2 million for the three months ended March 31, 2021, up from $1.1 million for the three months ended March 31, 2020. The increase in G&A expenses resulted primarily from increased directors and officers liability insurance, personnel and non-cash share-based compensation costs.

Balance Sheet and Cash Flow

The Company had cash, cash equivalents and marketable securities of $23.4 million, current liabilities of $1.2 million and working capital of $23.0 million as of March 31, 2021, compared to $27.5 million in cash, cash equivalents and marketable securities, $2.0 million in current liabilities and $25.9 million in working capital as of December 31, 2020. The decreases in combined cash, cash equivalents and marketable securities and in working capital are due primarily to increased clinical study costs related to the REDUX Phase 2 CKD study and costs associated with preparing for the ReMEDy2 Phase 2/3 stroke study.

Net cash used in operating activities for the three months ended March 31, 2021 was $4.3 million compared to $3.0 million for the three months ended March 31, 2020. This increase relates primarily to the increase in the net loss, partially offset by the effects of the changes in operating assets and liabilities.

Conference Call Information

DiaMedica Management will host a conference call to discuss its business update and first quarter 2021 financial results on Thursday, May 6, 2021, at 7:00 a.m. Central Time:

Interested parties may access the conference call by dialing in or listening to the simultaneous webcast. Listeners should log on to the website or dial in 15 minutes prior to the call. The webcast will remain available for play back on DiaMedica’s website, under investor relations – events and presentations, following the earnings call and for 12 months thereafter. A telephonic replay of the conference call will be available until May 13, 2021, by dialing (855) 859-2056 (US Toll Free), (404) 537-3406 (International), and entering the replay passcode: 2966016.

About DM199

DM199 is a recombinant (synthetic) form of human tissue kallikrein-1 (KLK1). KLK1 is a serine protease (protein) that plays an important role in the regulation of diverse physiological processes including blood flow, inflammation, fibrosis, oxidative stress and neurogenesis via a molecular mechanism that increases production of nitric oxide and prostaglandin. KLK1 deficiency may play a role in multiple vascular and fibrotic diseases such as chronic kidney disease, retinopathy, stroke, vascular dementia, and resistant hypertension where current treatment options are limited or ineffective. DiaMedica is the first company to have developed a recombinant form of the KLK1 protein. The KLK1 protein, produced from porcine pancreas and human urine, has been used to treat patients in Japan, China and Korea for decades. DM199 is currently being studied in patients with chronic kidney disease and patients with acute ischemic stroke.

On May 5, 2021 Lumos Pharma, Inc. (NASDAQ:LUMO), a clinical-stage biopharmaceutical company focused on therapeutics for rare diseases, reported its financial results for the first quarter ending March 31, 2021 and provided an update on clinical and corporate activities (Press release, NewLink Genetics, MAY 5, 2021, View Source [SID1234579251]).

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"The first quarter of 2021 was notable for data published further supporting the differentiated mechanism of action of LUM-201 and its potential for efficacy in patients with pediatric growth hormone deficiency identified by our Predictive Enrichment Marker strategy," commented Rick Hawkins, Chairman, CEO and President of Lumos Pharma. "In addition, final proceeds received in January from our PRV sale further strengthened our balance sheet and support our clinical and corporate strategy as we advance our OraGrowtH210 Trial in PGHD and pursue licensing opportunities to expand our pipeline."

Recent Highlights

PK/PD Data for LUM-201 in PGHD Presented by Investigator at KOL Event – Newly released data from a prior PK/PD trial evaluating LUM-201 in PGHD patients was presented in April by Dr. Fernando Cassorla at a KOL event hosted by Lumos Pharma. These data on three PGHD patients demonstrate the potential for Predictive Enrichment Markers of baseline IGF-1 levels and peak stimulated GH levels to identify patients likely to respond to LUM-201. These data further illustrate the potential for LUM-201 to augment the pulsatile secretion of GH for 24 hours, and induce a substantial increase in height velocity, over six-months of treatment in PEM-positive patients.
Poster Presented at ENDO 2021 Differentiates LUM-201 from Standard GH Secretagogues – The poster entitled, "LUM-201 Elicits Greater GH Response than Standard GH Secretagogues in Pediatric Growth Hormone Deficiency," was presented at the Endocrine Society 2021 Annual Meeting, March 20th-23rd. These data showed GH responses to single oral doses of LUM-201 were substantially higher than those elicited by standard GH secretagogues in two stimulation tests and that the difference in responses increased with higher baseline IGF-1 and higher GH stimulation test results.
Phase 2b OraGrowtH210 Trial Continues to Advance – The Phase 2b OraGrowtH210 Trial initiated in Q4 2020 continues to add clinical sites and enroll patients. Over 50% of the trial sites are currently open with additional sites to open more imminently as we advance toward our target of 40-50 sites. This trial will evaluate orally administered LUM-201 in approximately 80 patients diagnosed with PGHD. The purpose of the OraGrowtH210 Trial will be to prospectively confirm both the repeatability of our selected Predictive Enrichment Markers (PEMs) and the validity of our PEM strategy, and to identify the optimal dose of LUM-201 to be used in a Phase 3 registration trial. The Company continues to anticipate data read-out for the OraGrowtH210 Trial mid-year 2022.
Initiation of PK/PD OraGrowtH212 Trial of LUM-201 in PGHD Anticipated Q2 2021 – This study will evaluate the PK/PD effects of LUM-201 in PGHD patients at two dose levels to confirm prior clinical data illustrating the increased pulsatile release of endogenous growth hormone unique to LUM-201 and its potential for efficacy in a sizable PGHD patient population identified by Predictive Enrichment Markers (PEMs). We continue to anticipate the initiation of this trial in Q2 2021.
Final Tranche of Funds from PRV Sale Received – In January 2021, Lumos received the second and final tranche of $26.0 million from the total $60.0 million due to the Company from the PRV sale. We anticipate these funds will serve as additional capital to support the expansion of the Company’s pipeline through its business development efforts.
Financial Results for the Quarter Ended March 31, 2021

Cash Position – Lumos Pharma ended the first quarter on March 31, 2021, with cash and cash equivalents totaling $114.1 million compared to $98.7 million on December 31, 2020. The Company expects an average cash use of approximately $8.0 to $9.0 million per quarter through 2021. Cash on hand as of the end of Q1 is expected to support operations through OraGrowtH210 readout and completion of the OraGrowtH212 Trial.
R&D Expenses – Research and development expenses increased by $2.8 million for the three months ended March 31, 2021 compared to the same period in 2020 primarily due to increases of $1.6 million in personnel-related and stock compensation expenses, $1.3 million in clinical trial and contract manufacturing expenses, $0.2 million in supplies and other expenses and $0.1 million in legal expenses, offset by a decrease of $0.4 million in expensed IPR&D.
G&A Expenses – General and administrative expenses increased by $0.6 million for the three months ended March 31, 2021 as compared to the same period in 2020 primarily due to increases of $0.9 million in personnel-related and stock compensation expenses and $0.5 million in operating expenses for insurance, rent, supplies, and depreciation expenses, offset by a decrease of $0.8 million in legal and consulting.
Net Loss – The net loss for the first quarter ended March 31, 2021 was $8.6 million compared to net income of $0.3 million for the same period in 2020.
Lumos Pharma ended Q1 2021 with 8,332,193 shares outstanding.
Conference Call and Webcast Details

The Company has scheduled a conference call and webcast for 4:30 p.m. ET today to discuss its financial results and to give an update on clinical and business development activities. There will also be a question-and-answer session following management’s prepared remarks.

Access to the live conference call is available five minutes prior to the start of the call by dialing (855) 469-0612 (U.S.) or (484) 756-4268 (international). The conference call will be webcast live and a link to the webcast can be accessed through the Lumos Pharma website at View Source in the "Investors & Media" section under "Events and Presentations" or through this link: View Source To ensure a timely connection, it is recommended that users register at least 10 minutes prior to the scheduled webcast. A replay of the call will be available approximately two hours after the completion of the call and can be accessed by dialing (855) 859-2056 (U.S.) or (404) 537-3406 (international) and using the passcode 5863619. The replay will be available for two weeks from the date of the call.