On August 10, 2021 Cumberland Pharmaceuticals Inc. (NASDAQ: CPIX), a specialty pharmaceutical company, reported a company update and second quarter 2021 financial results (Press release, Cumberland Pharmaceuticals, AUG 10, 2021, View Source [SID1234586242]). Net revenues from continuing operations during the quarter were $9.1 million and totaled $19.6 million for the first half of 2021. The company also recorded an additional $500,000 in revenue during the second quarter and $1 million year to date, associated with divested product rights for two brands it is no longer distributing.

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!

While there was a slight decline in revenues during the second quarter 2021 compared to the prior year period, net revenues for the first half of 2021 were up 9.3% compared to the same period in 2020. The Company also posted year-to-date net income of $1.4 million during the first half of 2021, compared to a loss of $2 million during the prior year period.

The Company’s financial position included $89 million in total assets, with $26 million in cash, $41 million of total liabilities, and $48 million of shareholders’ equity at the end of the quarter.

"Cumberland continued to face headwinds due to the pandemic, but we are fortunate to have a diversified product portfolio that helped us counter the negative effects on our business," said A.J. Kazimi, Chief Executive Officer of Cumberland Pharmaceuticals. "We have adjusted our strategies and reinvented the way we operate in order to support our customers and the patients who can benefit from the delivery of our medicines."

RECENT COMPANY DEVELOPMENTS:

ESG Report

In July 2021, Cumberland released its second annual Sustainability Report (the "ESG Report"), which details the Company’s activities pertaining to environmental, social and governance ("ESG") matters. After issuing an inaugural ESG report last year, the Company remains committed to sustainability and to maintaining transparency of its corporate operations. As the largest biopharmaceutical company founded and headquartered in the Mid-South, Cumberland holds itself to the highest standards of ethical practices and understands the importance of recognizing and addressing the Company’s impact on its constituents, the community and the environment.

The ESG Report notes that in 2020, Cumberland provided nearly 2.5 million patient doses of its products, safely disposed of over 4,000 pounds of expired and damaged products and had no product recalls. The Company also had no brands that were listed on the U.S. Food and Drug Administration’s ("FDA") MedWatch Safety Alerts for Human Medical Products, no brand issues that were identified by the FDA from their Adverse Event Reporting System and no clinical trials that were terminated due to failure to practice good clinical standards.

The ESG Report also highlights several initiatives Cumberland implemented as part of its commitment to delivering high-quality pharmaceutical products to improve patient care. For example, the Company continued a program to serialize all commercial products sold in the United States, allowing it to track every unit distributed, which helps to prevent counterfeit drugs from entering the market under the Cumberland brand. In addition, through its coupon program, Cumberland covers up to 90% of patient prescription costs for the Company’s gastrointestinal products.

The ESG Report also highlights Cumberland’s investment in its employees through continuing education programs, employee development initiatives and employee recognition awards. Cumberland’s workforce is 46% women – and 18% of the Company’s employees are minorities.

Vibativ Case Studies

In June 2021, Cumberland released a series of case reports describing the effectiveness of Vibativ (telavancin) in treating secondary bacterial infections in COVID-19 patients – particularly those with other significant health problems, such as obesity, diabetes and heart disease. Cumberland’s Vibativ product has been used across the country to help COVID-19 patients who develop secondary bacterial infections in their lungs. Vibativ is a patented, FDA-approved injectable anti-infective for the treatment of certain serious bacterial infections including hospital-acquired and ventilator-associated bacterial pneumonia that can result from COVID-19, flu and other infections.

The Company compiled a dossier of patient case studies from across the country outlining several real-world instances where Vibativ effectively and safely treated the hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia resulting from MSSA and MRSA infections that developed in patients hospitalized with COVID-19.

Hyponatremia Publication in Support of Vaprisol

In late 2020, the Health Outcome Predictive Evaluation (HOPE) COVID-19 Registry Analysis, an international study of over 4,000 patients, found that patients hospitalized with COVID-19 had a high risk of developing hyponatremia. These COVID-19 patients also had a higher incidence of mortality due to their hyponatremia. The study results support the use of an intravenous vaptan to treat hyponatremia in critically ill patients afflicted with COVID-19.

Hyponatremia, an imbalance of serum sodium to body water, is the most common electrolyte disorder among hospitalized patients. Cumberland’s Vaprisol product is one of two branded prescription products indicated for the treatment of hyponatremia, and the only intravenously administered branded treatment. Vaprisol has a proven day one response to help normalize serum sodium levels in hyponatremic patients and move them out of the ICU as efficiently as possible.

New Chief Financial Officer Appointment

On May 17, 2021, Cumberland appointed John Hamm as its new Senior Director Finance & Accounting and Chief Financial Officer. In this role, his responsibilities include management of all the Company’s finance and accounting activities, while he continues to oversee corporate development and legal matters.

Mr. Hamm has more than 25 years of finance and accounting experience, including 20 years in health care. He previously held the positions of Chief Operating Officer and Chief Financial Officer, Pharmacy at HealthSpring, Inc., a managed care organization now operating as Cigna-HealthSpring.

He was also the Vice President Finance at Emdeon Business Services. Emdeon Inc., a healthcare technology firm that now operates as Change Healthcare Inc., a NASDAQ listed company with over $3 billion in annual revenues.

Mr. Hamm holds a Bachelor of Science in Business Administration with a minor in Accounting from Wheeling University. He earned his Master’s in Business Administration with an emphasis in Accounting from West Virginia University. He is a Certified Management Accountant (CMA) and Certified Financial Manager (CFM).

Prior to this new appointment, Mr. Hamm served as Cumberland’s Director Corporate Development.

Paycheck Protection Program

On April 20, 2020, Cumberland received a loan from Pinnacle Bank in the aggregate amount of $2,187,140 pursuant to the Paycheck Protection Program (the "PPP") under the Federal Coronavirus Aid, Relief and Economic Security Act ("CARES Act"), which was enacted March 27, 2020. The PPP is administered by the U.S. Small Business Administration ("SBA").

Pursuant to the PPP requirements, loan funds were used to maintain payroll, continue group health care benefits, and pay for rent and utilities during the pandemic. Cumberland applied for this loan after carefully considering, with Pinnacle Bank, the eligibility criteria to participate in this program, and determining that it met those criteria. The Company evaluated and provided information on its payroll and other qualifying expenses to determine the amount of PPP funds to apply for. Due to assistance from the PPP loan, the Company did not lay off or furlough any employees as a result of the COVID-19 pandemic.

Under the terms of the PPP, certain amounts of the loan may be forgiven if they are used for qualifying expenses as described in the CARES Act. Cumberland used the PPP loan funds for such qualifying expenses. In October 2020, Cumberland submitted a request for the loan’s forgiveness and on June 11, 2021, the Company received a formal notice from the SBA that the full amount of the loan was forgiven.

RediTrex Launch

During late 2018, Cumberland completed the submission of and filed with the FDA a New Drug Application for its RediTrex methotrexate injection product. RediTrex is a new line of pre-filled syringes specifically designed for ease of handling and dosing accuracy for the subcutaneous administration of methotrexate in patients with arthritis and psoriasis.

In December 2019, the Company received FDA approval for RediTrex and began planning for the launch of the product line. Cumberland provided initial shipments of RediTrex to accounts in November 2020 and is planning to launch the product line nationally in late September 2021.

Ifetroban Phase II Studies

Cumberland is sponsoring Phase II clinical programs to evaluate its ifetroban product candidates in 1) patients with cardiomyopathy associated with Duchenne Muscular Dystrophy, a rare, fatal, genetic neuromuscular disease that results in deterioration of the skeletal, heart and lung muscles, 2) Systemic Sclerosis or scleroderma, a debilitating autoimmune disorder characterized by diffuse fibrosis of the skin and internal organs and 3) Aspirin-Exacerbated Respiratory Disease, a severe form of asthma.

In addition, the Company has completed two pilot Phase II studies involving 1) patients suffering from Hepatorenal Syndrome, a life-threatening condition involving liver and kidney failure and 2) patients with Portal Hypertension that is associated with chronic liver disease.

Additional pilot preclinical and clinical studies of ifetroban are underway, including several investigator-initiated trials.

Enrollment in these clinical studies was interrupted due to the COVID-19 pandemic. While enrollment of new patients has been limited in 2021, many of the clinical study sites have reopened and resumed screening of patients for potential enrollment into the studies. Cumberland is awaiting results from the studies underway before deciding on the best development path for the registration of ifetroban, the Company’s first new chemical entity.

FINANCIAL RESULTS:

Net Revenues: For the three months ended June 30, 2021, net revenues from ongoing operations were $9.1 million, compared to $9.6 million for the prior year period. The company also recorded an additional $500,000 in revenue during the second quarter associated with divested rights to products that the company no longer distributes.

Net revenue by product for the second quarter 2021 included $5.3 million for Kristalose, $1.8 million for Vibativ, $0.9 million for Caldolor and $0.4 million for Vaprisol.

Year-to-date 2021 net revenues were $19.6 million, compared to $17.9 million during the first half of 2020. There were additional revenues of $1 million during the first six months of 2021 associated with the divested product rights.

Year-to-date 2021 net revenues by product were $8.3 million for Kristalose, $6.9 million for Vibativ, $2.5 million for Caldolor and $1.5 million for Vaprisol.

Operating Expenses: Total operating expenses for the three months ended June 30, 2021 were $10.5 million, compared to $11.2 million during the prior year period.

Earnings: Net income for the second quarter 2021 was $1.2 million, or $0.08 a share, compared to a loss of $0.9 million, or $0.06 a share for the prior year period. The adjusted loss for the second quarter was $16,000, compared to an adjusted loss of $120,000 for the prior year period.

Year-to-date net income in 2021 was $1.4 million, compared to a loss of $2 million during the first six months of 2020. Adjusted earnings for the first half of 2021 were $1 million, compared to an adjusted loss of $0.5 million during the same period in 2020.

Balance Sheet: At June 30, 2021, Cumberland had $88.9 million in total assets, including $25.7 million in cash and cash equivalents. Total liabilities were $41 million, including $14 million outstanding on the Company’s revolving line of credit, resulting in total shareholders’ equity of $48 million.

CONFERENCE CALL & WEBCAST:

A conference call and live internet webcast will be held on Tuesday, August 10, at 4:30 p.m. Eastern Time to discuss the results. To participate in the call, please dial 877-303-1298 (for U.S. callers) or 253-237-1032 (for international callers). A rebroadcast of the teleconference will be available for one week and can be accessed by dialing 855-859-2056 (for U.S. callers) or 404-537-3406 (for international callers). The Conference ID for the rebroadcast is 3985462. The live webcast and rebroadcast can be accessed via Cumberland’s website at View Source

On August 10, 2021 Akoya Biosciences, Inc. (Nasdaq: AKYA) ("Akoya"), The Spatial Biology Company, reported its financial results for the second quarter ending June 30, 2021 (Press release, Akoya Biosciences, AUG 10, 2021, View Source [SID1234586241]).

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!

Second Quarter Financial Highlights

Total revenue for the second quarter of 2021 was $13.1 million, as compared to $8.6 million in the second quarter of 2020, representing 53% growth.
Strong quarter of reagent sales, with $4.3 million of reagent revenue compared to $1.5 million in the second quarter of 2020.
Gross profit was $8.1 million in the second quarter of 2021, compared to $5.3 million in the second quarter of 2020, resulting in a gross profit margin of 62.2%, as compared to 61.6% in the prior year period.
Second Quarter Business Highlights

Continued growth in scientific publications related to our platforms: over 135 new publications in the first half of 2021, compared to 109 for all of 2020.
Announced seminal publications in Science from Johns Hopkins University describing a novel biomarker signature for response to immune checkpoint inhibitors in melanoma and a multi-institutional study in the Journal for ImmunoTherapy of Cancer highlighting the analytical robustness and clinical trial readiness of our Phenoptics platform.
Launched Advanced Biopharma Solutions (ABS) and a partnership with AstraZeneca delivering spatial biomarker-guided drug development and clinical trials services out of our Marlborough labs.
Announced the industry’s first Imaging Innovators (I2) Network to accelerate spatial phenotyping application innovation on CODEX, which is enabled by recently announced partnerships with leading microscopy providers ZEISS, Nikon, CrestOptics and Andor.
Appointed Scott Mendel to our board of directors, bringing 25 years of experience as one of the most accomplished executives in the diagnostics industry and a proven track record of transforming innovative technologies into clinical testing solutions.
"Akoya’s performance in the second quarter demonstrates the growth of spatial biology for discovery, translational and clinical research, as well as the continued adoption of our CODEX and Phenoptics solutions. Our dedicated team delivered strong financial results as well as exciting strategic initiatives, which position Akoya for continued growth and leadership in spatial biology," said Brian McKelligon, CEO of Akoya. "Our strong financial performance will continue to help us execute on our mission of delivering a revolutionary new class of spatially derived biomarkers that empower life sciences researchers to better understand disease and response to therapy."

Second Quarter Financial Results

Total revenue for the second quarter of 2021 was $13.1 million, compared to $8.6 million in the second quarter of 2020.

Product revenue was $10.7 million in the second quarter of 2021, compared to $6.2 million in the prior year period. Within product revenue, instrument revenue was $6.3 million in the second quarter of 2021, compared to $4.5 million in the second quarter of 2020. Reagent revenue was $4.3 million in the second quarter of 2021, compared to $1.5 million in the second quarter of 2020.

Services and other revenue totaled $2.4 million in the second quarter of 2021, as compared to $2.4 million in the second quarter of 2020.

We also monitor instruments sold and installed based as key performance indicators for our business:
- We sold 31 instruments in second quarter of 2021; 13 CODEX, 18 Phenoptics (includes Polaris, Vectra, and Mantra).
- Instrument installed base of 618 as of June 30, 2021; CODEX 145, Phenoptics 473.
2021 Guidance

We are refining our revenue guidance for full year 2021 from at least $52.0 million to a range of $52.5 to $53.0 million. The third quarter of 2021 is expected to have revenue growth of approximately 28-30% over the prior year quarter.

Webcast and Conference Call Details

Akoya will host a conference call today, August 10, 2021, at 5:00 p.m. Eastern Time to discuss its second quarter 2021 financial results. The dial-in numbers are (833) 562-0146 for domestic callers or (661) 567-1226 for international callers, followed by Conference ID: 8057237. A live webcast of the conference call will be available on the "Investors" section of the Company’s website at View Source The webcast will be archived on the website following the completion of the call for three months.

On August 10, 2021 Rain Therapeutics Inc., (NasdaqGS: RAIN),. ("Rain"), a late-stage company developing precision oncology therapeutics, reported financial results for the second quarter and six months ended June 30, 2021, along with an update on the company’s key developments, business operations and upcoming milestones (Press release, Rain Therapeutics, AUG 10, 2021, View Source [SID1234586240]).

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!

"Rain has made strong progress in the second quarter and six months ended 2021," said Avanish Vellanki, co-founder and chief executive officer of Rain. "Patients with dedifferentiated liposarcoma are in desperate need of new therapies, and we are proud to have been able to dose the first patient in a pivotal Phase 3 trial in under 12 months from acquiring the program. As we move forward with the milademetan clinical strategy, we look to commence our second trial, MANTRA-2, in patients with MDM2-amplified solid tumors, in the second half of 2021."

Key Developments and Operational Updates

First Patient Dosed in Phase 3 MANTRA Clinical Trial of Milademetan (RAIN-32) for DD LPS
In July 2021, Rain announced that the first patient was randomized in the multicenter, open-label, Phase 3 registrational trial (MANTRA) evaluating milademetan, an oral mouse double minute 2 ("MDM2") inhibitor, for the treatment of DD LPS.
Rain anticipates data from this trial in 2023.
MDM2-Amplified Phase 2 Basket Trial (MANTRA-2) to commence shortly
Rain anticipates enrolling the first patient in its basket study of patients with MDM2-amplified advanced solid tumors (MANTRA-2) in the second half of 2021. These patients will exhibit a degree of MDM2 amplification that Rain believes is oncogenic and sensitive to MDM2 inhibition.
Rain anticipates interim data in 2H 2022.
Collaborations with Tempus and Caris Life Sciences for the Milademetan MDM2-Amplified Phase 2 Basket Trial (MANTRA-2)
In June 2021, Rain announced an agreement with Tempus, an artificial intelligence and precision medicine company, to use their comprehensive genomic profiling testing platform for the Phase 2, MANTRA-2 basket trial for milademetan. Under the terms of the agreement, Tempus will provide both centralized tumor testing and patient matching services using their Connect & TIME Trial Network.
In June 2021, Rain also announced a patient referral partnership with Caris Life Sciences ("Caris"). Under the terms of the partnership, Caris will provide patient referral services using their molecular intelligence trials platform for MANTRA-2.
Milademetan Non-Clinical Data Abstracts Submitted to Upcoming Conferences
Rain, in collaboration with several research partners, intends to present non-clinical data at upcoming conferences relating to additional clinical opportunities for milademetan in the second half of 2021. Presentations for milademetan have been submitted and accepted to the 2021 World Conference of Lung Cancer (Sept. 8-14, 2021) and the 2021 EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) virtual conference (Oct. 7-10, 2021).
Anticipated Near-term Milestones

Milademetan MDM2-Amplified Phase 2 Basket Study (MANTRA-2)
Phase 2 trial expected to commence in the second half of 2021
Rain anticipates interim data in 2H 2022
Milademetan Intimal Sarcoma Phase 2 Study
Phase 2 trial expected to commence by early 2022
Rain anticipates interim data in late 2022
Milademetan DD LPS Phase 3 Study (MANTRA)
Rain anticipates data from this trial in 2023
RAD52 Research Program
Lead candidate selection expected in 2022
Second Quarter Financial Results
For the three and six months ended June 30, 2021, Rain reported a net loss of $8.2 million and $15.0 million, respectively, as compared to a net loss of $2.6 million and $5.2 million for the same periods in 2020, respectively. Net loss per share for the three and six months ended June 30, 2021, were $0.39 and $1.23, respectively, as compared to a net loss per share of $0.78 and $1.60 for the same periods in 2020, respectively.

Research and development ("R&D") expenses were $5.5 million and $10.8 million for the three and six months ended June 30, 2021, respectively, as compared to $1.5 million and $3.2 million for the same periods in 2020, respectively. The increases were primarily driven by the clinical costs for Rain’s lead product candidate, milademetan, as Rain prepared to launch its Phase 3 pivotal trial in DD LPS in July 2021, as well as personnel costs. Non-cash stock-based compensation expenses included in R&D expenses were approximately $0.6 million and $0.8 million in the three and six months ended June 30, 2021, respectively, as compared to $0.1 million and $0.2 million in the same periods in 2020, respectively.

General and administrative ("G&A") expenses were $2.7 million and $4.2 million for the three and six months ended June 30, 2021, respectively, as compared to $1.1 million and $1.8 million for the same periods in 2020, respectively. The increases were primarily due to increases in various third-party G&A costs, including legal, outside consulting, as well as accounting and audit fees. Non-cash stock-based compensation expense included in G&A expenses were approximately $0.2 million in each of the three and six months ended June 30, 2021, as compared to $0.1 million and $0.2 million for the same periods in 2020, respectively.

Total non-cash stock-based compensation expenses were approximately $0.8 million and $1.0 million in the three and six months ended June 30, 2021, respectively, as compared to $0.2 million and $0.4 million for the same periods in 2020, respectively.

As of June 30, 2021, Rain had $164.6 million in cash, cash equivalents and short-term investments. This included the $121.5 million in net proceeds from Rain’s initial public offering in April 2021. Rain’s quarter-end cash position adequately provides runway through late-2024.

As of June 30, 2021, Rain had approximately 26.5 million shares of common stock outstanding.

The Company continues to expect its full year 2021 net cash used in operating activities to be approximately $50.0 million to $60.0 million and a projected year end cash balance of approximately $137.0 million to $147.0 million in cash, cash equivalents and short-term investments.

Second Quarter 2021 Results Conference Call and Webcast Details
The management of Rain Therapeutics will host a conference call and webcast for the investment community today, August 10, 2021, at 1:30 p.m. PT (4:30 p.m. ET). The conference call can be accessed by dialing 1 (800) 708-4539 (U.S. Toll Free) / 1 (847) 619-6396 (U.S. Toll). The passcode for the conference call is 50202648. A live webcast may be accessed by visiting the "Investors" section of the Rain Therapeutics’ website at www.rainthera.com. The call will be recorded and available for replay on the Company’s website for approximately 30 days after the call.

About Well-Differentiated/Dedifferentiated Liposarcoma
Liposarcoma ("LPS") is a rare cancer originating from fat cells located in the soft tissues of the body. It is a malignant cancer that can spread to other parts of the body. Well-differentiated ("WD") LPS is less aggressive and tends to present as a large painless mass found in deeper tissues. Dedifferentiated ("DD") LPS is more aggressive, arising from WD LPS, and is usually found in tissue behind the abdominal area (retroperitoneal) or the extremities. WD/DD LPS are the most frequent subtypes of LPS and share common genomic abnormalities, predominately MDM2 gene amplification. The incidence of LPS is estimated at approximately 3,000 patients annually in the U.S., of which two-thirds are of the DD and WD type, and for which there are few effective treatment options.

About MANTRA Trial
The MANTRA trial is a randomized, multicenter, open-label, Phase 3 registrational trial evaluating milademetan, an oral MDM2 inhibitor, for the treatment of DD LPS. The MANTRA trial is designed to evaluate the safety and efficacy of milademetan compared to trabectedin, a current standard of care, in patients with unresectable or metastatic DD LPS that progressed on one or more prior systemic therapies, including at least one anthracycline-based therapy. Approximately 160 patients will be randomly assigned in a 1:1 ratio to receive milademetan or trabectedin. The primary objective of the MANTRA trial is to compare progression-free survival as determined by blinded independent review between the milademetan treatment arm and trabectedin control arm, in patients with unresectable or metastatic DD LPS, with or without a well-differentiated LPS component. Overall survival, disease control rate, objective response rate, duration of response, safety and patient reported outcomes will also be evaluated.

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 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.