On August 5, 2021 Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, reported that it will report its second quarter 2021 financial and operating results on Thursday, August 12, 2021, following the close of the U.S. financial markets (Press release, Lineage Cell Therapeutics, AUG 5, 2021, View Source [SID1234585808]). Lineage management will also host a conference call and webcast on Thursday, August 12, 2021, at 4:30 p.m. Eastern Time/1:30 p.m. Pacific Time to discuss its second quarter 2021 financial and operating results and to provide a business update.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Interested parties may access the conference call by dialing (866) 888-8633 from the U.S. and Canada and (636) 812-6629 from elsewhere outside the U.S. and Canada and should request the "Lineage Cell Therapeutics Call". A live webcast of the conference call will be available online in the Investors section of Lineage’s website. A replay of the webcast will be available on Lineage’s website for 30 days and a telephone replay will be available through August 22, 2021, by dialing (855) 859-2056 from the U.S. and Canada and (404) 537-3406 from elsewhere outside the U.S. and Canada and entering conference ID number 4876810.

On August 5, 2021 Celldex Therapeutics, Inc. (NASDAQ:CLDX) reported financial results for the second quarter ended June 30, 2021 and provided a corporate update (Press release, Celldex Therapeutics, AUG 5, 2021, View Source [SID1234585855]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Last month, Celldex reported positive data from our ongoing Phase 1b study of CDX-0159 in chronic inducible urticaria, where a single dose demonstrated a rapid, profound and durable response," said Anthony Marucci, Co-founder, President and Chief Executive Officer of Celldex Therapeutics. "These data not only spoke to the potential to bring patients suffering from urticaria an opportunity for fast, lasting and meaningful relief, but also showed that CDX-0159 safely depletes mast cells, a significant scientific and medical achievement that indicates CDX-0159’s potential to help other patients in need across a myriad of diseases with mast cell involvement."

Mr. Marucci continued, "Importantly, driven by these data, we successfully completed a $287.5 million follow-on offering which will support the expansion of the CDX-0159 program into later stage studies and additional indications, along with the continued development of our bispecific platform, which is exploring important pathways in inflammatory diseases, auto-immune disorders and oncology. We look forward to building on our successes in what promises to be an exciting second half of the year."

Recent Program Highlights

CDX-0159 – KIT Inhibitor Program

CDX-0159 is a humanized monoclonal antibody developed by Celldex that binds the KIT receptor with high specificity and potently inhibits its activity. The KIT receptor tyrosine kinase is expressed in a variety of cells, including mast cells, which mediate inflammatory responses such as hypersensitivity and allergic reactions. KIT signaling controls the differentiation, tissue recruitment, survival and activity of mast cells.

On July 9, Celldex reported interim data from the CDX-0159 single dose Phase 1b open label study, which were presented in a late-breaking poster discussion session as part of the European Academy of Allergy and Clinical Immunology (EAACI) Annual Congress 2021.

All 19 patients experienced a clinical response as assessed by provocation threshold testing; 18/19 (95%) experienced a complete response and 1/19 (5%) experienced a partial response.

Rapid onset of responses after dosing and sustained durability were observed and most patients with cold urticaria and symptomatic dermographism experienced a complete response by week 1 and by week 4, respectively. The median duration of response for patients was 77+ days (11+ weeks) for cold urticaria and 57+ days (8+ weeks) for symptomatic dermographism.

A single 3 mg/kg dose of CDX-0159 resulted in rapid, marked and durable suppression of serum tryptase and depletion of skin mast cells (87% depletion) as measured through biopsy. The kinetics of serum tryptase and skin mast cell depletion mirrored clinical activity which confirmed that serum tryptase level is a robust pharmacodynamic biomarker for assessing mast cell burden and clinical activity in inducible urticaria and potentially in other diseases with mast cell driven involvement.

CDX-0159 was generally well tolerated. The most common adverse events were hair color changes, mild infusion reactions, and transient changes in taste perception.

Celldex plans to present additional Phase 1b single dose data from the cold urticaria and symptomatic dermographism cohorts, including quality of life assessments, in the fall of 2021 and data from the cholinergic cohort in the first quarter of 2022.

Celldex continues to enroll patients in the Phase 1b multi-center randomized, double-blind, placebo-controlled study of CDX-0159 in chronic spontaneous urticaria (CSU). This study is designed to assess the safety of multiple ascending doses of CDX-0159 in up to 40 patients with CSU who remain symptomatic despite treatment with antihistamines. Treatment results from this study are planned for presentation at a scientific congress in early summer of 2022.

Celldex is expanding CDX-0159 development into prurigo nodularis, a chronic skin disease characterized by the development of hard, intensely itchy (pruritic) nodules on the skin. Of note, a patient with symptomatic dermographism enrolled in the chronic inducible urticaria study also had a diagnosis of prurigo nodularis. This patient experienced both a complete response of symptomatic dermographism and notable improvement of the prurigo nodularis symptoms on study. Initiation of this study is planned for the fourth quarter of 2021.

Manufacturing activities are also progressing as planned to support the introduction of the CDX-0159 subcutaneous formulation. Celldex plans to initiate a randomized, double-blind, placebo-controlled, Phase 1 study designed to evaluate the safety of single ascending doses of the subcutaneous formulation of CDX-0159 in healthy volunteers in the third quarter of 2021.
CDX-1140 – CD40 Agonist Program

CDX-1140 is a potent CD40 human agonist antibody developed by Celldex that the Company believes has the potential to successfully balance systemic doses for good tissue and tumor penetration with an acceptable safety profile.

In the Phase 1 study of CDX-1140 in patients with recurrent, locally advanced or metastatic solid tumors and B cell lymphomas, both the monotherapy and combination with CDX-301 portions of the trial are complete. Expansion cohorts are actively recruiting including CDX-1140 with KEYTRUDA (pembrolizumab) in patients with squamous cell head and neck cancer and non small cell lung cancer who have progressed on checkpoint therapy; and CDX-1140 with standard of care chemotherapy in first line metastatic pancreatic cancer. An update from this program is expected by end of 2021.
CDX-527 – Bispecific Antibody Program

CDX-527 is the first candidate developed by Celldex from its bispecific platform and utilizes the Company’s proprietary highly active anti-PD-L1 and CD27 human antibodies to couple CD27 co-stimulation with blockade of the PD-L1/PD-1 pathway.

In June, Celldex reported initial data from the Phase 1 dose-escalation study in up to ~40 patients with advanced or metastatic solid tumors that have progressed during or after standard of care therapy to be followed by tumor-specific expansion cohorts, which were presented at the 2021 ASCO (Free ASCO Whitepaper) Annual Meeting. A good safety profile was observed along with promising pharmacodynamic and pharmacokinetic activity, which are important key hurdles for the development of bispecific antibodies. The study is designed to determine the MTD during a dose-escalation phase and to recommend a dose level for further study in the subsequent expansion phase. The expansion is designed to further evaluate the tolerability, and biologic and anti-tumor effects of selected dose level(s) of CDX-527 in specific tumor types. Enrollment to the dose escalation portion of the study has been completed and expansion cohorts are being planned; additional data is expected in 2022.
Corporate Highlights

In July, Celldex closed an underwritten public offering of common stock, including the full exercise of the underwriters’ option to purchase additional shares, for gross proceeds of $287.5 million. Celldex believes that the proceeds from this offering, together with current reserves, provide the cash runway to fund key clinical, regulatory and operational activities through 2025.
While Celldex’s clinical development programs have not been significantly, negatively impacted by COVID-19 to date, the Company continues to carefully monitor the evolving situation closely across all development programs and work to minimize potential impact/disruptions.

Second Quarter 2021 Financial Highlights and 2021 Guidance

Cash Position: Cash, cash equivalents and marketable securities as of June 30, 2021 were $164.0 million compared to $176.1 million as of March 31, 2021. The decrease was primarily driven by second quarter cash used in operating activities of $11.9 million. At June 30, 2021, Celldex had 39.6 million shares outstanding. In July 2021, the Company issued 6,845,238 shares of its common stock in an underwritten public offering of common stock resulting in net proceeds to the Company of approximately $270.0 million, after deducting underwriting fees and offering expenses.

Revenues: Total revenue was $3.5 million in the second quarter of 2021 and $4.2 million for the six months ended June 30, 2021, compared to $0.2 million and $3.0 for the comparable periods in 2020. The increase in revenue was primarily due to an increase in services performed under our contract manufacturing and research and development agreements with Rockefeller University and Gilead Sciences, partially offset by a decrease in revenue from product development and licensing agreements as a result of the $1.8 million milestone payment received from Rockefeller University in the first quarter of 2020 related to Celldex’s manufacturing and development services agreement.

R&D Expenses: Research and development (R&D) expenses were $12.4 million in the second quarter of 2021 and $25.1 million for the six months ended June 30, 2021, compared to $9.7 million and $21.4 million for the comparable periods in 2020. The increase in R&D expenses was primarily due to an increase in clinical trial, contract research, and personnel expenses, partially offset by a decrease in rent expense.

G&A Expenses: General and administrative (G&A) expenses were $4.3 million in the second quarter of 2021 and $8.4 million for the six months ended June 30, 2021, compared to $3.5 million and $7.2 million for the comparable periods in 2020. The increase in G&A expenses was primarily due to higher personnel expenses.

Intangible Asset Impairment: The Company recorded a non-cash impairment charge of $3.5 million during the second quarter of 2020 due to the discontinuation of the CDX-3379 program.

Changes in Fair Value Remeasurement of Contingent Consideration: The loss on fair value remeasurement of contingent consideration was $0.3 million for the second quarter of 2021 and $0.7 million for the six months ended June 30, 2021, primarily due to changes in discount rates and the passage of time.

Net Loss: Net loss was $13.4 million, or ($0.34) per share, for the second quarter of 2021, and $29.9 million, or ($0.76) per share, for the six months ended June 30, 2021, compared to a net loss of $11.0 million, or ($0.50) per share, for the second quarter of 2020 and $23.7 million, or ($1.20) per share, for the six months ended June 30, 2020.

Financial Guidance: Celldex believes that the cash, cash equivalents and marketable securities at June 30, 2021, along with the approximately $270.0 million in net proceeds raised in our July 2021 underwritten public offering of common stock, are sufficient to meet estimated working capital requirements and fund planned operations through 2025.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, NJ USA.

On August 5, 2021 Caladrius Biosciences, Inc. (Nasdaq: CLBS) ("Caladrius" or the "Company"), a clinical-stage biopharmaceutical company dedicated to the development of cellular therapies designed to reverse disease, reported financial results for the three and six months ended June 30, 2021 (Press release, Caladrius Biosciences, AUG 5, 2021, View Source [SID1234585872]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The second quarter and first six months of 2021 have proven to be operationally and financially positive for Caladrius. Despite the continuing challenges presented by the COVID-19 pandemic, we continued to advance and expand our clinical pipeline while also adding a large amount of additional capital to our balance sheet," stated David J. Mazzo, Ph.D., President and Chief Executive Officer of Caladrius. "Most notably, we are seeing steady progress with site activation for our Phase 2b FREEDOM Trial of CLBS16 for the treatment of coronary microvascular dysfunction, as we continue to increase outreach activities to potential subjects in order to accelerate enrollment. In addition, a Phase 2 proof-of-concept clinical trial of CLBS201, designed to assess the safety and efficacy of CD34+ cell therapy as a treatment for patients with pre-dialysis diabetic kidney disease, is on track for a planned initiation in the second half of 2021. Lastly, even though our registration-eligible study of HONEDRA in critical limb ischemia and Buerger’s disease continues to be greatly impacted by the Japanese government-issued states of emergency tied to the COVID-19 pandemic, we have managed to treat patients and remain optimistic that the few remaining patients needed to complete enrollment will be treated by year end."

Product Development and Financing Highlights

CLBS16 for the treatment of coronary microvascular dysfunction

Caladrius reported in May 2020 the compelling results of its ESCaPE-CMD Phase 2a study of CLBS16 for the treatment of coronary microvascular dysfunction ("CMD"), a disease that continues to be underdiagnosed and potentially afflicts millions annually – a vast majority of whom are female – with no current treatment options. The Company is committed to raising awareness of this growing women’s health crisis and finding an effective treatment. To this end, we have partnered with the American Heart Association on a number of activities designed to educate people about CMD and to encourage them to discuss the condition with their physician. Caladrius recently initiated, and is currently treating patients in, a rigorous 105-subject Phase 2b clinical trial (the FREEDOM Trial) which, to our knowledge, is the first controlled regenerative medicine trial in CMD.

Investigator and subject response to the FREEDOM Trial has been favorable and early enrollment proceeded according to plan. However, the continued impact of the COVID-19 pandemic, including the resurgence of cases occurring in select areas throughout the United States, has contributed to a general slowing of enrollment. In addition, further work with investigators and prospective subject feedback led the Company to propose to the FDA amendments to the FREEDOM Trial protocol to enhance the breadth and speed of subject enrollment. These changes included expanding the techniques that are acceptable for diagnosing CMD. Nevertheless, given the uncertainty that persists surrounding the future impact of the COVID-19 pandemic on potential patient recruitment and the accessibility of investigator sites, the Company now projects enrollment completion for the FREEDOM Trial to occur in the third quarter of 2022 with final data (based on the 6 month assessment of all subjects) expected by the second quarter of 2023.

HONEDRA (CLBS12) for the treatment of critical limb ischemia

The Company’s open-label, registration-eligible study of SAKIGAKE-designated HONEDRA in Japan for the treatment of critical limb ischemia ("CLI") and Buerger’s disease (an orphan-sized subset of CLI) has shown strong results to date. The initial responses observed in the subjects who have reached an endpoint in this study are consistent with a positive therapeutic effect and safety profile reported by previously published clinical trials in Japan and the U.S. The study’s enrollment continues to be almost stopped by the pandemic’s impact in Japan, however, the Company is encouraged that less than a handful of patients are needed to reach study completion, the exact date of which is impossible to predict given the continuing impact of COVID-19 on clinical trials in Japan. While the final outcome of the trial will depend on all data from all subjects, the data to date is encouraging (~60% of subjects in the completed Buerger’s disease cohort have reached a positive "CLI-free" endpoint despite a natural history of such patients that predicts continuing disease progression to amputation). In the U.S., the Company was pleased to report that the U.S. Food and Drug Administration ("FDA") granted orphan designation to CLBS12 as a treatment for Buerger’s disease, however, any decisions regarding potential development in the U.S. will be made after further discussion with FDA on the requirements for registration.

CLBS201 for the treatment of diabetic kidney disease

The Company has prepared an initial development plan for the clinical study of CLBS201, a CD34+ investigational product for administration via the renal arteries to slow the deterioration, or, ideally, reverse the decline of renal function in patients with diabetic kidney disease ("DKD") who, although still pre-dialysis, exhibit rapidly progressing stage 3b disease. Progressive kidney failure is associated with attrition of the microcirculation of the kidney. Pre-clinical studies in kidney disease and injury models have demonstrated that protection or replenishment of the microcirculation results in improved kidney function. A Phase 2 proof of concept, randomized, placebo-controlled study for the stage 3b chronic kidney disease patient population is planned to initiate in the second half of 2021. The protocol, pending final central institutional review board approval, calls for a six-subject open-label treatment run-in arm in which patients will be treated sequentially, to be completed, evaluated and cleared for continuation by the study’s data safety monitoring board prior to initiating the 40-patient randomized, placebo-controlled, double blinded portion of the trial. The Company is projecting that safety data from the six-subject run-in arm will be completed by the end of the second quarter of 2022.

OLOGO for the treatment of no option refractory disabling angina

Caladrius acquired the rights to data and regulatory filings for a CD34+ cell therapy program for no option refractory disabling angina ("NORDA") that had been advanced to Phase 3 by a previous sponsor. Based on the clinical evidence from the completed studies that a single administration of OLOGO reduces mortality, improves angina, and increases exercise capacity in patients with otherwise untreatable angina, this product received Regenerative Medicine Advanced Therapy ("RMAT") designation from the FDA. Discussions with the FDA have resulted in a rejection of the Company’s efforts to reduce the FDA requirement of a 400-patient Phase 3 study for registration (including an arm of 50 standard of care patients and an arm of 150 placebo patients), despite data showing that the NORDA population is orphan in size. Because enrollment of a study of this magnitude and design is projected to take many years, if executable at all, the Company has decided not to pursue a Phase 3 program for OLOGO on its own, but will continue to seek a partner to execute the study and advance the program.

Sufficient capital to fund operations beyond multiple key data readouts anticipated in 2023

As previously disclosed, in January 2021, Caladrius raised $25.0 million in a private placement priced at-the-market under Nasdaq rules. In February 2021, the Company announced that it closed a $65.0 million capital raise through the sale of its common stock and warrants to several institutional and accredited investors in two registered direct offerings priced at-the-market under Nasdaq rules. In addition, in May 2021, the Company received $1.4 million in non-dilutive funding as an approved participant of the Technology Business Tax Certificate Transfer Program (the "Program") sponsored by the New Jersey Economic Development Authority (NJEDA). The Program enables qualifying New Jersey-based biotechnology or technology companies to sell a percentage of their New Jersey net operating losses ("NOLs") and research and development tax credits to unrelated qualifying corporations.

Second Quarter 2021 Financial Summary

Research and development expenses were approximately $4.3 million for the three months ended June 30, 2021, compared to $1.8 million for the three months ended June 30, 2020, representing an increase of 138%. Research and development in both periods focused on the advancement of our ischemic repair platform and related to:

Expenses associated with efforts to advance the FREEDOM Trial where the first patient was dosed in the first quarter of 2021;

Expenses associated with the planning and preparation of an IND and Phase 2 proof-of-concept protocol for CLBS201 as a treatment for diabetic kidney disease; and

Ongoing expenses for HONEDRA in critical limb ischemia and Buerger’s disease in Japan for which we continue to focus spending on patient enrollment and Japanese NDA preparation.
General and administrative expenses were approximately $2.8 million for the three months ended June 30, 2021, compared to $2.5 million for the three months ended June 30, 2020, representing an increase of 14%.

Overall, net losses were $5.7 million for the three months ended June 30, 2021, compared to net income of $6.6 million for the three months ended June 30, 2020.

Balance Sheet Highlights

As of June 30, 2021, we had cash, cash equivalents and marketable securities of approximately $106.1 million. Based on existing programs and projections, the Company remains confident that its current cash balances will fund its operations for the next several years, notably through study completion for the FREEDOM Trial, through the registration-eligible study completion for HONEDRA and through the Phase 2 proof-of-concept study for CLBS201, while still potentially providing capital to explore additional pipeline expansion opportunities.

Conference Call

Caladrius will hold a live conference call today, August 5, 2021, at 4:30 p.m. (ET) to discuss financial results, provide a business update and answer questions. To join the conference call, please refer to the dial-in information provided below. A live webcast of the call will also be available under the Investors & News section of the Caladrius website, View Source, and will be available for replay for 90 days after the conclusion of the call.

On August 5, 2021 Lumos Pharma, Inc. (NASDAQ:LUMO), a clinical-stage biopharmaceutical company focused on therapeutics for rare diseases, reported that financial results for the second quarter ending June 30, 2021 and provided an update on clinical and corporate activities (Press release, NewLink Genetics, AUG 5, 2021, View Source [SID1234585887]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The enhancement of Lumos Pharma’s leadership team and encouraging recent trends in screening and enrollment for both OraGrowtH210 and OraGrowtH212 Trials are exciting developments for the Company," commented Rick Hawkins, Chairman and CEO of Lumos Pharma. "John McKew, David Karpf, and Mark Bach each bring significant clinical development and management expertise to their new roles with the Company, strengthening our ability to execute on our clinical and corporate strategy. The majority of our trial sites are now open, and we continue to work to bring the remainder online. Additionally, as children return to school this fall and regular pediatrician visits resume, we believe these factors should lead to increased referrals and an acceleration of enrollment in our trials."

Corporate Update:

John McKew, PhD, Chief Operating Officer & Chief Scientific Officer, Promoted to President

Our Chief Operating Officer and Chief Scientific Officer, John McKew, was recently promoted to President of Lumos Pharma effective August 1, 2021 as part of a planned succession process. Dr. McKew has nearly 30 years of experience developing novel therapeutics during which he successfully advanced multiple therapies through preclinical and into clinical development, both at the NIH and in the pharmaceutical industry. Dr. McKew has served as Chief Operating Officer of Lumos Pharma since April 2020 and as Chief Scientific Officer since he joined the Company in 2016.

David B. Karpf, MD, Experienced Endocrinologist and Pharma Executive, Named Chief Medical Officer

Pharma industry veteran and academician, David B. Karpf, MD, joined Lumos Pharma as Chief Medical Officer on August 3, 2021. Dr. Karpf is an Adjunct Clinical Professor in the Division of Endocrinology at Stanford University School of Medicine with over 35 years of expertise in all aspects of clinical endocrinology. He is also an accomplished executive with 30 years of experience in the development of biopharmaceuticals and small molecular weight drugs in the areas of endocrinology and rare diseases, among others. Most recently, Dr. Karpf served as Vice President, Clinical Development for Ascendis Pharma where he was responsible for several compounds in clinical development, including TransCon GH, long-acting growth hormone for once weekly treatment of growth hormone deficiency, and TransCon PTH. Dr. Karpf has held leadership positions at several biopharmaceutical and pharma companies, including Merck where he originally gained experience with Lumos Pharma’s oral growth hormone secretagogue, LUM-201.

Pediatric Endocrinologist and Biopharma Executive, Mark Bach, MD, PhD, Joins Advisory Board

Mark Bach, MD, PhD, pediatric endocrinologist and seasoned pharmaceutical executive, joined Lumos Pharma’s Clinical Scientific Advisory Board on July 15, 2021. Dr. Bach is currently the Chief Medical Officer for ShouTi Inc., having recently joined from Ascendis Pharma where he served as Senior Vice President, Endocrine Medical Sciences. Dr. Bach is a pediatric endocrinologist with 30 years of clinical research and pharmaceutical development experience. Prior to Ascendis, Dr. Bach held successive leadership roles in research and development at both Johnson & Johnson and Merck. While at Merck, he conducted extensive clinical and preclinical research on growth hormone, IGF-1 and LUM-201 (MK-0677).

Clinical Update:

OraGrowtH210 Trial of LUM-201 in PGHD

As the Company announced on July 21, 2021, given the slower pace of site initiation and enrollment of the Phase 2 OraGrowtH210 Trial of LUM-201 in PGHD primarily due to the impact of COVID, the 6-month primary outcome data for OraGrowtH210 is now anticipated in the second half of 2023. The treatment period for this trial has been extended to 12 months to capture additional data for future regulatory filings and to meet FDA requirements to initiate our proposed 3-year long-term extension study, the OraGrowtH211 Trial. We do not anticipate these protocol changes, on a stand-alone basis, to extend the time to initiation of our Phase 3 clinical trial. The primary outcome for the Phase 2 OraGrowtH210 Trial continues to be the preliminary validity of our Predictive Enrichment Marker (PEM) strategy with the goal of selecting the optimal dose for a pivotal Phase 3 study in PGHD.

PK/PD OraGrowtH212 Trial of LUM-201 in PGHD Initiated Q2 2021

The OraGrowtH212 Trial was initiated in June and is currently enrolling patients. This study will evaluate the PK/PD effects of LUM-201 in PGHD patients at two dose levels, 1.6 and 3.2 mg/kg/day, to confirm prior clinical data illustrating the increased pulsatile release of endogenous growth hormone unique to LUM-201 and its potential for this mechanism of action to contribute to efficacy in PGHD. This open-label trial will be extended from six months to twelve months to capture additional PK/PD and height velocity data. The PD pulsatility assessment will continue to occur at six months on therapy as planned.

Pipeline Expansion

We have been conducting an assessment of the range of disease areas where LUM-201 would have medical utility for purposes of prioritizing our next indication and longer-term life cycle planning. These assessments reinforce our conviction that LUM-201 represents a pipeline-in-a-product and look forward to advancing the next phase of LUM-201 opportunities. In addition, with a heightened sensitivity toward value creation, we continue to evaluate select rare disease assets under consideration to add to our product portfolio.

Financial Results for the Quarter Ended June 30, 2021

Cash Position – Lumos Pharma ended the second quarter on June 30, 2021, with cash and cash equivalents totaling $107.7 million compared to $98.7 million on December 31, 2020. Cash on hand as of the end of Q2 2021 is expected to support operations through the primary outcome data readout from OraGrowtH210 Trial in the second half of 2023 and the OraGrowtH212 Trial.
R&D Expenses – Research and development expenses were $4.1 million, an increase of $1.4 million for the three months ended June 30, 2021 compared to the same period in 2020, primarily due to increases of $1.8 million in clinical trial and contract manufacturing expenses, offset by a decrease of $0.4 million in personnel-related and operating expenses for insurance, rent, supplies and depreciation.
G&A Expenses – General and administrative expenses were $4.6 million, an increase of $0.4 million for the three months ended June 30, 2021, compared to the same period in 2020, primarily due to increases of $0.6 million in personnel-related expenses, of which $0.9 million relates to severance expense recorded for the departure of our former CFO, Carl W. Langren on June 30, 2021 and $0.5 million in stock compensation expenses, of which $0.4 million relates to the accelerated vesting of all non-vested equity awards held by Mr. Langren upon his departure. These increases were offset by a $0.4 million decrease in legal and consulting expenses and a $0.3 million reduction in operating expenses for insurance, rent, supplies, and depreciation.
Net Loss – The net loss for the second quarter ended June 30, 2021 was $8.7 million compared to net loss of $5.4 million for the same period in 2020.
Lumos Pharma ended Q2 2021 with 8,357,391 shares outstanding.
Conference Call and Webcast Details

The Company has scheduled a conference call and webcast for 11:00 a.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 8050625. The replay will be available for two weeks from the date of the call.

On August 5, 2021 Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a clinical-stage biopharmaceutical company focused on developing novel drug candidates for immuno-inflammatory diseases, reported its financial results for the second quarter of 2021 and provided a corporate update (Press release, Aclaris Therapeutics, AUG 5, 2021, View Source [SID1234585903]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We’re very pleased with the preliminary topline data from our Phase 2a trial of our "soft" topical JAK1/3 inhibitor, ATI-1777, that we announced during the quarter," said Dr. Neal Walker, President & CEO of Aclaris. "Our recent clinical trial successes with ATI-450 and ATI-1777 demonstrate the value and productivity of our proprietary KINect drug discovery platform. With our financing in June, we are well positioned to advance our clinical trial programs for ATI-450 and ATI-1777 and develop compounds from our early stage pipeline."

Research and Development Highlights:

The global COVID-19 pandemic continues to rapidly evolve and has caused and may continue to cause Aclaris to experience disruptions that could impact the timing of its research and development and regulatory activities listed below.

MK2 Inhibitor Assets
ATI-450, an investigational oral small molecule MK2 inhibitor compound:
Aclaris plans to progress ATI-450 into a Phase 2b trial in moderate to severe rheumatoid arthritis in the fourth quarter of 2021.
Aclaris also plans to progress ATI-450 into Phase 2 trials in hidradenitis suppurativa and psoriatic arthritis.
In pre-clinical studies, positive effects on MK2 inhibition have been observed for breast cancer metastasis and cancer-associated bone loss.

ATI-2231, an investigational oral MK2 inhibitor compound:
Second MK2 inhibitor generated from Aclaris’ proprietary KINect drug discovery platform and designed to have a long half-life.
Currently being explored as a potential treatment for metastatic breast cancer and pancreatic cancer as well as use in preventing bone loss in this patient population.
IND-enabling studies are underway.
"Soft" JAK Inhibitor Asset
ATI-1777, an investigational topical "soft" Janus kinase (JAK) 1/3 inhibitor compound:
ATI-1777-AD-201: A Phase 2a, multicenter, randomized, double-blind, vehicle-controlled, parallel-group clinical trial to evaluate the efficacy, safety, tolerability and pharmacokinetics of ATI-1777 in 50 subjects with moderate to severe atopic dermatitis (AD). The trial consisted of a 4-week treatment period and a 2-week follow-up period during which no treatment was given.
As announced in June 2021, the trial achieved its primary endpoint, the percent change from baseline in the modified Eczema Area and Severity Index (mEASI) score at week 4, with a high degree of statistical significance (p<0.001) (one-sided p-value), which corresponded to a 74.4% reduction in mEASI score from baseline at week 4 in subjects applying ATI-1777 compared to a 41.4% reduction in subjects applying vehicle. The preliminary topline data was based on the full analysis set (FAS), which was comprised of 48 subjects randomized and documented to have received at least one dose of trial medication.
Positive trends in favor of ATI-1777 were observed in key secondary efficacy endpoints, such as improvement in itch, percent of mEASI-50 responders, investigator’s global assessment responder analysis, and reduction in body surface area impacted by disease. In addition, the FAS analysis also showed positive trends in favor of ATI-1777 in percent of mEASI-75 responders (65.2% for ATI-1777 compared to 24.0% for vehicle) and mEASI-90 responders (30.4% for ATI-1777 compared to 20.0% for vehicle). These secondary efficacy endpoints were not powered for statistical significance.
Based on an analysis of pharmacokinetic plasma samples in the ATI-1777 arm at multiple timepoints, minimal systemic exposure was observed which supports a "soft" topical JAK inhibitor approach.
ATI-1777 was generally well tolerated. No serious adverse events were reported. The most common adverse events (AEs) (reported in ≥2 subjects in the trial) were increased blood creatinine phosphokinase levels and headache in subjects in the ATI-1777 arm and urinary tract infection (one in each of the ATI-1777 and the vehicle arm); none of these AEs in the ATI-1777 arm were determined by the clinical trial investigators to be related to ATI-1777. One treatment-related AE, application site pruritus, was reported in one subject in the ATI-1777 arm.
Aclaris plans to progress ATI-1777 into a Phase 2b trial in moderate to severe atopic dermatitis.
Preclinical Asset
ATI-2138, an investigational oral ITK/TXK/JAK3 (ITJ) inhibitor compound:
Currently being developed as a potential treatment for psoriasis and/or inflammatory bowel disease.
Submission of Investigational New Drug Application (IND) is expected in the second half of 2021.
Discovery Assets
Currently developing oral gut-restricted JAK inhibitors with limited systemic exposure as potential treatments for inflammatory bowel disease.
Identification of a lead development candidate is expected by the end of 2021.
Central nervous system (CNS) kinase inhibitor targets
Currently engaged in research to identify brain penetrant kinase inhibitor candidates and their impact on neuronal pro-inflammatory cytokine production, microglia growth and survival, and neurodegeneration.
Financial Highlights:

Liquidity and Capital Resources

As of June 30, 2021, Aclaris had aggregate cash, cash equivalents and marketable securities of $266.2 million compared to $54.1 million as of December 31, 2020. The primary factors for the change in cash, cash equivalents and marketable securities during the six months ended June 30, 2021 included:

Net proceeds of $134.9 million from a public offering in June 2021 in which Aclaris sold 8.1 million shares of common stock.

Net proceeds of $103.3 million from a public offering in January 2021 in which Aclaris sold 6.3 million shares of common stock.

Net cash used in operating activities of $24.5 million. This amount was comprised of a net loss of $46.9 million and changes in operating assets and liabilities of $5.8 million, partially offset by non-cash charges of $21.2 million for the revaluation of contingent consideration and $6.5 million for stock-based compensation.
Aclaris anticipates that its cash, cash equivalents and marketable securities as of June 30, 2021 will be sufficient to fund its operations through the end of 2024, without giving effect to any potential business development transactions or financing activities.

Financial Results

Second Quarter 2021

Net loss was $18.2 million for the second quarter of 2021 compared to $11.6 million for the second quarter of 2020.

Total revenue was $1.8 million for the second quarter of 2021 compared to $2.0 million for the second quarter of 2020.

Research and development (R&D) expenses were $7.9 million for the quarter ended June 30, 2021 compared to $6.5 million for the prior year period.

The quarter-over-quarter increase of $1.4 million was primarily the result of continued investment in the further development of Aclaris’ immuno-inflammatory drug development pipeline, including ATI-450 and ATI-2138, partially offset by lower quarter-over-quarter development costs for ATI-1777.
General and administrative (G&A) expenses were $5.9 million for the quarter ended June 30, 2021 compared to $5.6 million for the prior year period.

The quarter-over-quarter increase of $0.3 million was primarily the result of higher legal and compliance costs, partially offset by lower compensation expenses.
Revaluation of contingent consideration charges related to the Confluence acquisition was $4.8 million for the quarter ended June 30, 2021 compared to $0 for the prior year period.
Year-to-date 2021

Net loss was $46.9 million for the six months ended June 30, 2021 compared to $27.2 million for the six months ended June 30, 2020.

Total revenue was $3.6 million for the six months ended June 30, 2021 compared to $3.5 million for the six months ended June 30, 2020.

R&D expenses were $15.7 million for the six months ended June 30, 2021 compared to $14.1 million for the prior year period.

The quarter-over-quarter increase of $1.6 million was primarily the result of continued investment in the further development of Aclaris’ immuno-inflammatory drug development pipeline, including ATI-450 and ATI-2138, partially offset by lower quarter-over-quarter development costs for ATI-1777 and compensation expenses.
G&A expenses were $10.7 million for the six months ended June 30, 2021 compared to $11.8 million for the prior year period.

The quarter-over-quarter decrease of $1.1 million was primarily the result of lower compensation expenses, partially offset by higher legal and compliance costs.
Revaluation of contingent consideration charges related to the Confluence acquisition was $21.2 million for the six months ended June 30, 2021 compared to $1.8 million for the prior year period.