On August 10, 2021 Olema Pharmaceuticals, Inc. ("Olema" or "Olema Oncology," Nasdaq: OLMA), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of targeted therapies for women’s cancers, reported second quarter financial results for the period ended June 30, 2021 (Press release, Olema Oncology, AUG 10, 2021, View Source [SID1234586231]).

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 made important progress in the second quarter of 2021 as we advanced the clinical development of our lead candidate, OP-1250, an investigational complete estrogen receptor (ER) antagonist (CERAN), and strengthened our corporate foundation to ensure that we have the talent and resources in place to support our future success," said Sean P. Bohen, M.D., Ph.D., President and Chief Executive Officer of Olema Oncology. "We have seen robust enrollment in the ongoing Phase 1/2 clinical trial of OP-1250 and look forward to sharing interim dose-escalation data at a medical meeting in the fourth quarter of this year."

Corporate Highlights and Anticipated Milestones

Significant progress advancing the Phase 1/2 clinical trial of OP-1250 in patients with metastatic, ER+ / HER2- breast cancer. As of June 4, 2021, 28 patients have been enrolled across five dose-escalation cohorts. OP-1250 has demonstrated oral bioavailability and a dose-proportional pharmacokinetic profile consistent with predictions from Olema nonclinical models. A maximum tolerated dose has not been identified. The Company plans to present interim safety, tolerability, pharmacokinetic and initial efficacy data at a medical meeting in the fourth quarter of 2021, pending abstract acceptance.
Advance into monotherapy dose expansion in the second half of 2021.
Initiate a Phase 1b clinical trial of OP-1250 in combination with a CDK4/6 inhibitor in the first quarter of 2022.
Second Quarter 2021 Financial Highlights

Cash, cash equivalents and marketable securities as of June 30, 2021 were $318.1 million. Olema anticipates that this cash balance will be sufficient to fund operations through the end of 2023.
Research and development (R&D) expenses were $11.9 million for the quarter ended June 30, 2021, compared to $1.9 million for the same period of the prior year. The increase in R&D expenses was primarily due to increased expenditures to advance the Phase 1/2 clinical trial of OP-1250, the increase in nonclinical development activities, higher personnel-related expenses as headcount grew to support the advancement of the clinical and nonclinical programs, and higher non-cash stock-based compensation expenses.
General and administrative (G&A) expenses were $4.6 million for the quarter ended June 30, 2021, compared to $0.5 million for the same period of the prior year. The increase in G&A expenses was primarily due to higher personnel-related expenses associated with increases in the number of G&A personnel supporting the growth of the organization, public company-related expenses and other corporate costs, and non-cash share-based compensation expenses.
Net loss for the quarter ended June 30, 2021 was $16.4 million, compared to $2.5 million for the same period of the prior year.

On August 10, 2021 aTyr Pharma, Inc. (Nasdaq: LIFE), a clinical stage biotherapeutics company, reported that the company has expanded its research collaboration with The Ohio State University (OSU) to deepen the understanding of the immune mechanisms of sarcoid granuloma formation and identify potential biomarkers of efficacy for the company’s lead therapeutic candidate, ATYR1923, which is currently in clinical development for the treatment of pulmonary sarcoidosis (Press release, aTyr Pharma, AUG 10, 2021, View Source [SID1234586253]). The research will be conducted in the laboratory of Elliott Crouser, M.D., Professor of Pulmonology, Critical Care and Sleep Medicine at OSU. Dr. Crouser specializes in sarcoidosis research and treatment and will serve as the principal investigator.

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 collaboration, which expands upon a successful pilot proof-of-concept study, will assess the effect of ATYR1923 on sarcoid granuloma formation in vitro in blood samples taken from sarcoidosis patients. The study will focus on identifying the relevant immune mechanisms triggered in granuloma formation and analyze promising biomarkers predictive of strong granuloma formation in order to assess whether they could be used as predictive biomarkers for treatment selection or treatment response to ATYR1923.

"We look forward to working with aTyr on this important initiative to expand the current understanding of the underlying mechanisms involved in pulmonary sarcoidosis, particularly the formation of granulomas. This work has the potential to identify promising biomarkers that may be used to predict treatment response, including to ATYR1923. Current treatment options for pulmonary sarcoidosis are limited, and the ability to determine a patient population that may benefit from a potential treatment such as ATYR1923 presents the opportunity to take a much-needed step forward in managing this disease," said Dr. Crouser.

"We are very pleased to expand this research collaboration with OSU and Dr. Crouser. This collaboration will build upon the successful findings from research conducted with Dr. Crouser that were recently accepted to be presented at the upcoming European Respiratory Society International Congress in September, which demonstrate the ability of a splice variant of histidyl-tRNA synthetase, the active portion of ATYR1923, to disrupt sarcoid granuloma formation in vitro — a hallmark of this debilitating disease," said Sanjay Shukla, M.D., M.S., President and Chief Executive Officer of aTyr. "The research generated from this collaboration may help direct us to biomarkers indicative of a population that may be sensitive to treatment with ATYR1923, which could lead to improved patient outcomes."

Sarcoidosis is an inflammatory disease characterized by the formulation of granulomas, clumps of inflammatory cells, in one or more organs of the body. Sarcoidosis in the lungs is called pulmonary sarcoidosis and occurs in more than 90% of all sarcoidosis patients. Approximately 150,000 to 200,000 Americans live with pulmonary sarcoidosis and the prognosis ranges from benign and self-limiting to chronic, debilitating disease, permanent loss of lung function and death. Current treatment options include corticosteroids and other immunosuppressive therapies, which have limited efficacy and are associated with serious side-effects when used long-term that many patients cannot tolerate.

Dr. Crouser received his medical degree from the Medical College of Ohio at Toledo, OH. He completed an internship, residency and fellowship at The Ohio State University Wexner Medical Center in Columbus, OH. He is board certified in Internal Medicine with subspecialty certifications in Pulmonary Disease and Critical Care. He is a leader in sarcoidosis research and treatment and currently serves as the Chair of the Foundation for Sarcoidosis Research’s Scientific Advisory Board. In 25 years, his laboratory has contributed to the publication of more than 100 peer-reviewed manuscripts, including the first clinical practice guidelines for sarcoidosis, which were endorsed by the American Thoracic Society in 2020. Ohio State’s Sarcoidosis Specialty Clinic was named a Center of Excellence in 2020 by the World Association of Sarcoidosis and Other Granulomatous Disorders.

About ATYR1923

aTyr is developing ATYR1923 as a potential therapeutic for patients with severe inflammatory lung diseases. ATYR1923, a fusion protein comprised of the immuno-modulatory domain of histidyl-tRNA synthetase fused to the FC region of a human antibody, is a selective modulator of neuropilin-2 that downregulates the innate and adaptive immune response in inflammatory disease states. aTyr has completed enrollment in a proof-of-concept Phase 1b/2a trial evaluating ATYR1923 in patients with pulmonary sarcoidosis. This Phase 1b/2a study is a multi-ascending dose, placebo-controlled, first-in-patient study of ATYR1923 that has been designed to evaluate the safety, tolerability, steroid sparing effect, immunogenicity and pharmacokinetic profile of multiple doses of ATYR1923. Proof-of-mechanism for ATYR1923 was established in a Phase 2 clinical trial in COVID-19 patients with severe respiratory complications, which demonstrated that ATYR1923 reduced inflammatory cytokine levels in patients consistent with preclinical models, including cytokines that are implicated in sarcoidosis and other forms of interstitial lung disease.

On August 10, 2021 VERAXA Biotech GmbH, Heidelberg, and Indivumed GmbH, Hamburg, reported their agreement to jointly discover modulatory and functional antibodies against several targets that are relevant for colorectal cancer (Press release, Indivumed, AUG 10, 2021, View Source [SID1234586270]). The targets have been identified by Indivumed’s discovery solution platform nRavel, only two months after its launch. The antibody development and commercialization will be performed by Indivumed’s subsidiary, Ix Therapeutics.

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!

VERAXA Biotech GmbH is a pioneer in microfluidic technology for the screening of antibodies with modulatory function on complex signal proteins like GPCRs or ion channels. The high-throughput screening platform allows for the testing of millions of fully natural IgGs from humans and mice for therapeutic effects, rather than just for binding. Results are obtained in only a fraction of the time required by other technologies. The company offers customized screening services for users worldwide. Besides carrying out service projects, VERAXA is currently also establishing its own proprietary pipeline of therapeutic antibody candidates for further joint development with pharma partners.

Indivumed has set up a leading multi-omics database with extensive medical experience, bioinformatics expertise, and AI-integrated advanced analytics for a most effective medical diagnostics and predictive therapeutic use. Just recently, the company launched its AI-based platform nRavel, which together with Indivumed’s IndivuType multi-omics database will derive more precise insights into and much better predictors for certain pathologies and diseases with high medical needs.

"The high quality of data and the artificial intelligence tools that Indivumed utilizes means that we can be more confident in the validity of the targets," said Dr. Christoph Antz, CEO of Veraxa. "With our technology, we can move rapidly into the development of functional antibodies that have the potential to serve as new therapeutics."

"Having thousands of patient cases complete with comprehensive multi-omics and clinical data coupled with the advanced AI capabilities of nRavel gives us the ability to derive novel insights in just a short period of time," said Prof. Dr. Hartmut Juhl, Founder and CEO of Indivumed. "As we continue to uncover additional targets, having a partner like Veraxa who can develop functional antibodies in an accelerated manner is critical to our ability to discover new possibilities for cancer treatment in the shortest-possible time and fight this disease."

On August 10, 2021 Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, reported financial results for the second quarter ended June 30, 2021, and highlighted recent progress and upcoming milestones for its pipeline programs (Press release, Scholar Rock, AUG 10, 2021, View Source [SID1234586232]).

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!

"Scholar Rock has had significant momentum in the first half of the year with further demonstration of apitegromab’s transformative potential to improve motor function for patients with SMA, as well as continued progression of dose escalation in the DRAGON trial evaluating SRK-181’s potential to overcome resistance to checkpoint inhibitors in solid tumors," said Nagesh Mahanthappa, Ph.D., Interim CEO of Scholar Rock. "I look forward to working closely with this exceptional team as we continue to advance our research and clinical programs to further elucidate the potential of our scientific platform and help make a difference in the lives of patients suffering from serious diseases."

Company Updates and Upcoming Milestones

Apitegromab is a selective inhibitor of myostatin activation being developed as the potential first muscle-directed therapy for the treatment of spinal muscular atrophy (SMA).

Positive Top-line Results and Additional Supportive Exploratory Analyses from the TOPAZ Trial were Presented at the Cure SMA Annual Conference. In June 2021, positive top-line data from the TOPAZ Phase 2 trial (NCT03921528) were presented by the lead principal investigator, Thomas Crawford, M.D. of Johns Hopkins Medicine. Treatment with apitegromab in conjunction with nusinersen in patients with Type 2 and 3 SMA led to meaningful motor function improvements of up to 20 points as measured by Hammersmith Functional Motor Scale Expanded (HFMSE). The majority (74%, 23/31) of non-ambulatory patients showed a clinical improvement, as defined by at least a 1-point increase in HFMSE. Further evidence that improvements in motor function may be attributed to apitegromab was supported by a post-hoc analysis that showed no correlation between change in HFMSE and duration of prior maintenance nusinersen therapy. In addition, 7/35 non-ambulatory patients also showed major functional achievements as measured by World Health Organization (WHO) Motor Development Milestones, with one patient achieving two and one patient achieving three new WHO motor milestones.
Phase 3 Trial Evaluating Apitegromab in Patients with Non-Ambulatory Type 2 and 3 Patients Anticipated to Initiate by Year-End 2021. Subject to feedback from regulatory agencies, Scholar Rock intends to conduct a randomized, double-blind, placebo-controlled Phase 3 trial to evaluate apitegromab as an add-on therapy to nusinersen or risdiplam in patients with non-ambulatory Type 2 and Type 3 SMA. In the TOPAZ Phase 2 trial, non-ambulatory patients experienced the largest increases in motor function (HFMSE scores) following treatment with apitegromab as add-on to chronic maintenance nusinersen. Patients with non-ambulatory Type 2 and Type 3 SMA are estimated to represent approximately two-thirds of the overall prevalent SMA patient population.
Apitegromab Granted Fast Track Designation by the FDA for the Treatment of Patients with Spinal Muscular Atrophy. In May 2021, the U.S. Food and Drug Administration (FDA) granted Fast Track designation for apitegromab, offering Scholar Rock eligibility to submit a rolling Biologic License Application (BLA) for apitegromab if relevant criteria are met. Fast Track designation is intended to facilitate the development and expedite the review of drugs to treat serious conditions and get new drugs to patients earlier. In addition to Fast Track designation, apitegromab had previously received Orphan Drug and Rare Pediatric Disease designations from the FDA as well as PRIME and Orphan Medicine Product designations from the European Medicines Agency for the treatment of SMA, all recognizing the unmet medical needs of patients with SMA.
Additional Potential Indications Identified for Apitegromab. Scholar Rock has identified multiple diseases for which selectively inhibiting the activation of myostatin may offer therapeutic benefit, including additional patient populations in SMA (such as Type 1 SMA and ambulatory Type 3 SMA) and potential indications outside of SMA.
SRK-181 is a selective inhibitor of latent TGFβ1 activation being developed with the aim of overcoming resistance to and increasing the number of patients who may benefit from checkpoint inhibitor therapy.

Plan to Advance to Part B Dose Expansion Portion of the DRAGON Phase 1 Proof-of-Concept Trial Mid-Year. SRK-181 is being evaluated in the two-part DRAGON trial (NCT04291079) in patients with locally advanced or metastatic solid tumors exhibiting primary resistance to anti-PD-(L)1 therapy. Part A dose escalation, which is evaluating the safety and pharmacokinetics of SRK-181 as a single-agent and in combination with anti-PD-(L)1 therapy, continues to progress to identify the recommended dose for Part B of the trial. The Company is on-track to advance to Part B dose expansion, which will consist of multiple cohorts, including urothelial carcinoma, cutaneous melanoma, non-small cell lung cancer, and other solid tumors. Each cohort will enroll up to 40 patients who have demonstrated primary resistance to anti-PD-(L)1 therapy and will be treated with SRK-181 in combination with an approved anti-PD-(L)1 therapy. An update on dose escalation and initial clinical data from Part A of the DRAGON trial is anticipated by year-end.
Second Quarter 2021 Financial Results

For the quarter ended June 30, 2021, net loss was $30.7 million or $0.84 per share compared to a net loss of $19.3 million or $0.65 per share for the quarter ended June 30, 2020.

Revenue was $4.6 million for the quarter ended June 30, 2021 compared to $3.9 million for the quarter ended June 30, 2020 and was related to the Gilead fibrosis-focused collaboration (the "Gilead Collaboration Agreement") that was executed in December 2018.
Research and development expense was $25.6 million for the quarter ended June 30, 2021 compared to $17.0 million for the quarter ended June 30, 2020. The increase year-over-year primarily reflects manufacturing costs associated with apitegromab and higher personnel and facility-related costs, partially offset by lower manufacturing costs associated with SRK-181.
General and administrative expense was $9.3 million for the quarter ended June 30, 2021 compared to $6.4 million for the quarter ended June 30, 2020. The increase year-over-year was primarily attributed to higher personnel and facility-related costs and professional fees.

"We are executing towards key milestones and continue to work closely with regulatory authorities to finalize the design of the Phase 3 trial for apitegromab and to progress the DRAGON trial to Part B to evaluate SRK-181 across multiple tumor types," said Ted Myles, CFO and Head of Business Operations of Scholar Rock. "We ended the second quarter with approximately $282 million in cash and cash equivalents, which will allow us to achieve meaningful milestones as we continue to execute against our plan."

On August 10, 2021 Pulmatrix, Inc. (NASDAQ: PULM), a clinical stage biopharmaceutical company developing innovative inhaled therapies to address serious pulmonary and non-pulmonary disease using its patented iSPERSE technology, reported its second quarter 2021 financial results and provides a business update (Press release, Pulmatrix, AUG 10, 2021, View Source [SID1234586254]).

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 have made steady progress across our pipeline in the second quarter," said Ted Raad, Chief Executive Officer of Pulmatrix. "Recent toxicology data from PUR1800 suggests the potential to expand into indications that require chronic dosing. We look forward to presenting topline data from the fully enrolled, ongoing Phase 1b study of PUR1800 in Q1 2022. We are also rapidly advancing towards the clinic with PUR3100 in acute migraine. We believe that our strong cash position allows us to advance our pipeline through major data milestones into 2023."

Second Quarter and Recent Highlights:

PUR1800

Completed enrollment in ongoing Phase 1b clinical study of PUR1800 in acute exacerbations in COPD (AECOPD). Study endpoints include safety, tolerability, and exploratory biomarkers to demonstrate target engagement and anti-inflammatory effect.
PUR1800 Phase 1b top-line data is expected in Q1 2022.
Results from 6-month rat and 9-month dog toxicology results demonstrate no progression of 28-day findings, suggesting potential for chronic dosing of PUR1800 in indications beyond AECOPD including, but not limited to, steroid resistant asthma, chronic obstructive pulmonary disease (COPD) and idiopathic pulmonary fibrosis (IPF).
The Company estimates an approximate peak net revenue opportunity of $2.4B in AECOPD1, due to the significant unmet need beyond standard of care oral steroids and/or antibiotic therapy. Expansion to chronic indications should further broaden the market potential of PUR1800.
PUR3100

14-day toxicology results for PUR3100, Pulmatrix’s dry powder iSPERSE formulation of dihydroergotamine (DHE) for pulmonary delivery to treat acute migraine are expected in Q3 2021.
IND filing planned in Q4 2021, with a Phase 1/ Phase 2 clinical study start date anticipated in Q1 2022. Data from this proof-of-concept study are expected in Q4 2022.
Pulmazole

Pulmatrix notified Cipla Technologies LLC (Cipla) that it is in material breach of the Development and Commercialization Agreement (the Cipla Agreement) in May 2021. The Company continues to seek Cipla’s reaffirmation of all of its obligations under the Cipla Agreement and, in the absence of such reaffirmation, to pursue all available remedies.
1 Market research and analysis from ClearView Healthcare Partners

Financials

As of June 30, 2021, Pulmatrix had $56.9 million in cash and cash equivalents, compared to $31.7 million for the year ended December 31, 2020.

Revenue for the second quarter of 2021 was $2.2 million, compared to $3.5 million for the same period in 2020. The revenue for the second quarter of 2021 was the result of the collaboration and licensing agreements with Cipla and JJEI.

Research and development expense was $4.5 million in the second quarter of 2021 compared to $3.2 million for the same period in 2020. The increase year–over-year was primarily attributable to increased preclinical and manufacturing costs related to the PUR3100 project partially offset by decreased spend on the Pulmazole clinical trial.

General and administrative expense was $1.6 million for the second quarter of 2021 compared to $1.5 million for the same period in 2020. The increase year–over-year was primarily attributable to increase legal, patent, ad public company costs partially offset by decreased employment costs.

Net loss was $3.9 million for the second quarter of 2021 compared to a net loss of $1.2 million for the same period of 2020. The $2.7 million increase in net loss year-over-year was due to increased spend for preclinical and manufacturing expenses on the PUR3100 program and reduced revenue recognized that related to the Pulmazole program.