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Black Diamond Therapeutics Reports Second Quarter 2023 Financial Results and Provides Corporate Update

On August 11, 2023 Black Diamond Therapeutics, Inc. (Nasdaq: BDTX), a clinical-stage precision oncology company developing MasterKey therapies that target families of oncogenic mutations in patients with genetically defined cancers, reported financial results for the second quarter ended June 30, 2023, and provided a corporate update (Press release, Black Diamond Therapeutics, AUG 11, 2023, View Source [SID1234634271]).

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"The second quarter of 2023 marked a crucial period of clinical and operational execution for Black Diamond, and I am incredibly pleased with our initial dose escalation data demonstrating a favorable tolerability profile and clinical proof of activity for BDTX-1535 in NSCLC patients harboring acquired resistance and intrinsic driver EGFR mutations. With these data in hand, we believe we are well positioned for rapid advancement of this novel MasterKey inhibitor for NSCLC patients and are looking forward to meeting with FDA later this year to discuss our dose optimization strategy that may enable a potential accelerated approval pathway as well as the opportunity to study BDTX-1535 in newly diagnosed NSCLC patients with intrinsic driver mutations," said David Epstein, Ph.D., President and Chief Executive Officer of Black Diamond Therapeutics. "Our recently completed underwritten public offering provides us with a strong cash position and extended runway to execute on the robust set of upcoming milestones for BDTX-1535 and our broader pipeline, all with the goal of bringing our MasterKey therapies to patients in need."
Recent Developments & Upcoming Milestones:

BDTX-1535:

•In June 2023, Black Diamond announced initial clinical data from the dose escalation portion of the Phase 1 clinical study of BDTX-1535, an epidermal growth factor receptor (EGFR) MasterKey inhibitor, demonstrating a favorable tolerability profile and clinical proof of activity in non-small cell lung cancer (NSCLC) patients harboring both acquired resistance and intrinsic driver EGFR mutations. Key highlights from the readout include:

◦Confirmed radiographic partial response (PR) by RECIST 1.1 achieved across predicted therapeutic doses in 5 of 12 NSCLC patients in a subgroup, who had measurable disease at study start, and underwent post baseline tumor assessment by RECIST 1.1. One additional patient demonstrated unconfirmed PR, while the remaining six patients had stable disease.
◦Confirmed PRs were observed in NSCLC patients with a wide range of EGFR mutations including classical and intrinsic driver mutations and acquired C797S resistance mutation, as well as complex mutations that include a combination of classical, intrinsic, and acquired resistance mutations. Two NSCLC patients demonstrated radiographic improvement in the peripheral disease and central nervous system (CNS) metastases.
◦BDTX-1535 was generally well tolerated by NSCLC and glioblastoma multiforme (GBM) patients and the overall safety profile was consistent with the EGFR tyrosine kinase inhibitor (TKI) class of drugs. The most common drug-related adverse events were mild to moderate rash, diarrhea, stomatitis, paronychia, nausea and fatigue. No patients experienced dose limiting toxicity at 15-200 mg once-daily (QD) doses. One of 15 patients treated at the 300 mg QD dose experienced dose limiting diarrhea and 5 of 12 patients at the 400 mg QD dose experienced dose limiting toxicity (diarrhea, 2 patients; rash, stomatitis, fatigue and decreased appetite, 1 patient each). No unexpected safety signal was identified during dose escalation.
•In July 2023, Black Diamond initiated expansion cohorts to assess overall response rate (ORR) by RECIST 1.1 in NSCLC patients with EGFR acquired resistance mutations after progression on a third generation EGFR TKI and intrinsic driver mutations after progression on an EGFR TKI.
•Black Diamond anticipates the following key milestones for BDTX-1535:
◦Presentation of the full BDTX-1535 dose escalation data in NSCLC at a medical conference in the fourth quarter of 2023.
◦Meeting with the U.S. Food and Drug Administration (FDA) in the fourth quarter of 2023 to align on dosing strategy to enable a potential accelerated approval pathway in NSCLC.
◦Initiation of an expansion cohort in newly diagnosed NSCLC patients with intrinsic driver mutations after discussion with the FDA.
◦Clinical update on BDTX-1535 Phase 1 dose escalation data in recurrent GBM patients in the fourth quarter of 2023.

BDTX-4933:
•BDTX-4933 is designed as a brain-penetrant, oral MasterKey inhibitor of oncogenic BRAF Class I, II and III and RAS mutations, while also avoiding paradoxical activation.
•In April 2023, Black Diamond presented a poster at the 2023 American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, outlining its approach to characterizing RAF, RAS and MAPK pathways in addition to the design and preclinical development of BDTX-4933. Based on preclinical data, BDTX-4933 has a potential best-in-class profile to treat cancer patients harboring oncogenic BRAF Class I, II, III and RAS mutations, with or without brain disease.
•Black Diamond initiated a Phase 1 clinical trial for BDTX-4933 in select indications for patients harboring all-class BRAF or RAS mutations in the second quarter of 2023.
BDTX-4876:

•BDTX-4876 is a development candidate FGFR 2/3 MasterKey inhibitor, selective against FGFR 2 and 3 alterations, while sparing FGFR 1 and 4.
•Black Diamond is evaluating strategic alternatives for BDTX-4876 as it deepens focus on its two clinical-stage assets.
Discovery-Stage Pipeline and MAP Drug Discovery Engine:
•Black Diamond continues to leverage its Mutation-Allostery-Pharmacology (MAP) drug discovery engine to advance its discovery-stage pipeline to bring therapies to underserved patients and expects to progress another undisclosed program in solid tumors to development candidate nomination in 2023.

Corporate:

•In June 2023, Black Diamond announced the promotion of Melanie Morrison to Chief Development Officer.
•In July 2023, Black Diamond closed an underwritten public offering (Follow-on Offering) of 15,000,000 shares of its common stock at a public offering price of $5.00 per share for gross proceeds of approximately $75.0 million, before deducting underwriting discounts and commissions and other offering expenses.
Financial Highlights
•Cash Position: Black Diamond ended the second quarter of 2023 with approximately $89.5 million in cash, cash equivalents, and investments compared to $122.8 million as of December 31, 2022. Net cash used in operations was $14.4 million for the second quarter of 2023 compared to $18.1 million for the second quarter of 2022.
•Research and Development Expenses: Research and development (R&D) expenses were $13.2 million for the second quarter of 2023, compared to $16.2 million for the same period in 2022. The decrease in R&D expenses was primarily due to reduced clinical trial activities stemming from the discontinuation of the development of BDTX-189 to focus on advancement of the Company’s pipeline programs, BDTX-1535 and BDTX-4933.
•General and Administrative Expenses: General and administrative (G&A) expenses were $6.9 million for the second quarter of 2023, compared to $7.0 million for the same period in 2022. The decrease in G&A expenses was primarily due to a decrease in legal and other professional fees.
•Net Loss: Net loss for the second quarter of 2023 was $20.0 million, as compared to $23.2 million for the same period in 2022.

Financial Guidance

•Black Diamond ended the second quarter of 2023 with approximately $89.5 million in cash, cash equivalents and investments. The Company believes that the net proceeds from the Follow-on Offering, together with its existing cash, cash equivalents and investments, will enable the Company to fund its operating expenses and capital expenditure requirements into the first half of 2025.

Aeglea BioTherapeutics Reports Second Quarter 2023 Financial Results

On August 11, 2023 Aeglea BioTherapeutics, Inc. ("Aeglea" or the "Company") (NASDAQ:AGLE), a biotechnology company advancing a pipeline of antibody therapeutics with the potential to transform the treatment of inflammatory bowel disease ("IBD"), reported second quarter 2023 financial results and provided program and corporate updates (Press release, Aeglea BioTherapeutics, AUG 11, 2023, View Source [SID1234634270]).

"With the acquisition of Spyre Therapeutics and concurrent financing, we are in a privileged position to create meaningful new medicines for patients with IBD and build an industry-leading development organization," said Cameron Turtle, DPhil, Chief Operating Officer of Aeglea. "Our pipeline of differentiated and potentially best-in-class IBD programs, including 47 and TL1A, combined with a strategy to investigate therapeutic combinations and precision medicine approaches, offers the possibility to transform the treatment of this chronic and debilitating disease."

"In parallel, we have made significant progress streamlining the organization with the sale of pegzilarginase and pivoting our operations and strategy around the new IBD assets," said Jonathan Alspaugh, President and Chief Financial Officer of Aeglea. "We are working to rapidly advance our co-lead product candidates with an expectation of initiating clinical studies for both SPY001 and SPY002 in 2024."

Recent Program and Corporate Updates

Corporate

•
Completed the asset acquisition (the "Acquisition") of Spyre Therapeutics, Inc. ("Spyre"), a privately held biotechnology company with a pipeline of antibody therapeutics possessing the potential to transform the treatment of IBD alongside its research partner, Paragon Therapeutics, Inc. ("Paragon").
•
Raised $210.0 million in gross proceeds (before deducting approximately $12.7 million in placement fees and other offering expenses) through a sale of Series A non-voting convertible preferred stock (the "Series A Preferred Stock") in a private placement to a group of investors.

IBD Portfolio

With the Acquisition, Aeglea shifted its focus to the development of a potentially best-in-class IBD portfolio including:

•
SPY001 – a highly potent and selective anti-α4β7 monoclonal antibody engineered with half-life extension technology and formulated for high concentration, convenient dosing.
o
SPY001 is currently progressing through IND-enabling studies and is expected to enter first-in-human ("FIH") studies in the first half of 2024. Data from a healthy volunteer study are expected by the end of 2024.
•
SPY002 – a highly potent and selective anti-TL1A monoclonal antibody engineered with half-life extension technology. TL1A has emerged as one of the most promising targets in IBD and broader immunology indications.
o
We expect to begin FIH studies of the SPY002 program in the second half of 2024 with healthy volunteer data expected in the first half of 2025.

Pegzilarginase

•
Sold global rights to pegzilarginase in development for Arginase 1 Deficiency to Immedica Pharma AB ("Immedica") for $15.0 million upfront cash proceeds and up to $100.0 million of contingent milestone payments. The sale of pegzilarginase to Immedica supersedes the previous license agreement between the companies.
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Marketing Authorisation Application for pegzilarginase is under review by the European Medicines Agency.
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The milestone payments are contingent on formal reimbursement decisions by national authorities in key European markets and pegzilarginase approval by the FDA, among other events.
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Net proceeds of the sale are to be distributed to holders of contingent value rights ("CVR") pursuant to the terms of the CVR Agreement dated July 7, 2023 by and between the Company and a rights agent.

Second Quarter 2023 Financial Results

As of June 30, 2023, Aeglea had available cash and cash equivalents and restricted cash of $236.7 million, including the $210.0 million in gross proceeds from the private placement offering in June 2023.

Aeglea recognized development fee and royalty revenues of $0.7 million in the second quarter of 2023, as a result of its license and supply agreement with Immedica for the commercial rights to pegzilarginase in Europe and several countries in the Middle East. The revenues recorded in the second quarter of 2023 are related to drug supply and royalties from an early access program in France. Aeglea recognized $0.6 million for the second quarter of 2022 in development fee revenues.

Research and development expenses totaled $17.4 million for the second quarter of 2023 and $15.4 million for the second quarter of 2022. The increase was primarily related to restructuring costs net of savings and an increase in reimbursable costs under the Paragon Agreement (as described below).

General and administrative expenses totaled $12.1 million for the second quarter of 2023 and $7.7 million for the second quarter of 2022. This increase was primarily due to restructuring costs, net of savings.

Acquired in-process research and development expenses totaled $130.5 million for the second quarter of 2023 and no expenses for the second quarter of 2022. As the Spyre assets have no alternative use, the net assets acquired are expensed in the period acquired.

Change in the fair value of forward contract liability expense was $58.2 million for the second quarter of 2023 and no expenses for the second quarter of 2022. The stock consideration provided in the Acquisition was not issued until July 7, 2023. Accordingly, the Company recognized a forward contract liability to represent the contractual obligation to issue stock as of June 30, 2023. The increase in expense represents the change in fair value between June 22, 2023 (the Acquisition date) and June 30, 2023 for the redeemable Series A Preferred Stock.

Net loss totaled $217.1 million and $22.3 million for the second quarter of 2023 and 2022, respectively, which includes non-cash stock compensation expense of $1.9 million and $2.0 million for the second quarter of 2023 and 2022, respectively.

Interim Report Q2 2023

On August 10, 2023 Oncopeptides reported its interim report for Q2 2023 (Presentation, Oncopeptides, AUG 10, 2023, View Source [SID1234634922]).

Innate Pharma to Participate in Upcoming Investor Conference

On August 10, 2023 Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) ("Innate" or the "Company") reported that members of its senior management team are scheduled to participate in the following upcoming conference (Press release, Innate Pharma, AUG 10, 2023, View Source [SID1234634246]):

H.C. Wainwright Immune Cell Engager Virtual Conference
Event Date: August 17 – virtual

Rocket Pharmaceuticals Reports Second Quarter 2023 Financial Results and Highlights Recent Progress

On August 10, 2023 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, reported financial results for the quarter ending June 30, 2023, and updates from the Company’s key pipeline developments, business operations and upcoming milestones (Press release, Rocket Pharmaceuticals, AUG 10, 2023, View Source [SID1234634245]).

"The second quarter of 2023 marked a period of strong forward momentum across our cardiovascular AAV and hematology LV portfolios highlighted by the BLA submission for our LAD-I program, Rocket’s first product filing. In addition, we are in advanced discussions with the FDA to finalize the components of the primary composite endpoint for the single-arm, pivotal Phase 2 Danon Disease study. In parallel, we are initiating enrollment in the trial following recent alignment with the FDA on our proposed two-patient pediatric safety run-in," said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma. "We appreciate the strong and positive collaboration with the FDA afforded by our RMAT designation, which we believe will lead to the most optimal global development path forward for the first pivotal gene therapy trial for a cardiac condition. Simultaneously, in our second cardiac program, RP-A601 for the treatment of PKP2-ACM, we are rapidly moving towards first patient treatment following IND clearance and have also received FDA Fast Track and Orphan Drug designations."

Dr. Shah continued, "Building on the BLA submission for LAD-I, we remain on track to submit the BLA for our LV-based Fanconi Anemia program in the fourth quarter. Taken together, I am very pleased with our progress across our entire pipeline of AAV and LV assets and look forward to continuing our momentum through the remainder of the year and beyond."

Key Pipeline and Operational Updates

AAV Cardiovascular Portfolio

Danon Disease

Received European designations including Priority Medicines (PRIME) and Advanced Therapy Medicinal Products (ATMP) for RP-A501. PRIME designation offers the benefits of early and enhanced support from the European Medicines Agency (EMA) and the opportunity for accelerated marketing application review. It is granted to medicines that target an unmet need for which no treatment option exists and with data showing a meaningful improvement of clinical outcomes. ATMP designation is intended for medicines that offer groundbreaking new opportunities and allows for a single evaluation and authorization procedure.
Positive clinical data from the Phase 1 trial of RP-A501 presented at the ASGCT (Free ASGCT Whitepaper) Annual Meeting. As of May 2023, all six optimally enrolled patients in the Danon Disease Phase 1 trial continued to demonstrate improvement or stabilization in ongoing follow-up of six to 36 months. Patients would have typically experienced progressive disease or death without treatment.
Initiating two-patient pediatric safety run-in for RP-A501 pivotal study; approaching final alignment with FDA on primary composite endpoint to support accelerated approval. Rocket has obtained IRB approvals and begun screening activities. Initial clinical sites are the University of California, San Diego (UCSD) and Children’s Hospital of Philadelphia.
PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)

Received FDA Fast Track and Orphan Drug designations for RP-A601. Fast Track designation facilitates the development and expedited review of medicines that treat serious conditions and fill an unmet medical need. It also enables increased communication with the FDA, offering the potential for accelerated approval and priority review if criteria are met, and permits a rolling Biologics License Application (BLA) review. Orphan Drug designation is granted to support the development of medicines for rare disorders and provides certain benefits, including market exclusivity upon regulatory approval, exemption of FDA application fees, and tax credits for qualified clinical trials.
Positive proof of concept from preclinical studies of RP-A601 presented at the ASGCT (Free ASGCT Whitepaper) Annual Meeting. Data demonstrated decreased arrhythmias and increased survival in the PKP2 knockout mouse model. Based on these data and the completion of Investigational New Drug (IND)-enabling toxicology studies, Rocket received IND clearance from the FDA for a Phase 1 study of RP-A601 that will assess the impact of RP-A601 on PKP2 myocardial protein expression, cardiac biomarkers, and clinical predictors of life-threatening ventricular arrhythmias and sudden cardiac death.
Phase 1 start-up activities for RP-A601 are well progressed following IND clearance to allow for first patient treatment. Rocket has gained IRB approval at UCSD, initiated Phase 1 study start up activities and is rapidly advancing toward first patient treatment.
LV Hematology Portfolio

Leukocyte Adhesion Deficiency-I (LAD-I)

Positive clinical data from pivotal Phase 2 trial of RP-L201 presented at the ASGCT (Free ASGCT Whitepaper) Annual Meeting. Positive, updated top-line data demonstrated 100% overall survival at 12 months post-infusion (and for entire duration of follow-up) via Kaplan Meier estimate for all nine LAD-I patients with 12 to 24 months of available follow-up. Data also showed evidence of resolution of LAD-I-related skin rash and restoration of wound repair capabilities. The safety profile was highly favorable in all patients with no RP-L201-related serious adverse events.
BLA submitted to FDA for RP-L201. Based on the positive efficacy and safety data from the pivotal study of RP-L201, Rocket submitted the BLA to the FDA, as its first product filing.
Fanconi Anemia (FA)

Positive clinical data from pivotal Phase 2 trial of RP-L102 presented at the ASGCT (Free ASGCT Whitepaper) Annual Meeting. Positive, updated top-line data showed sustained genetic correction in eight of 12 evaluable patients and comprehensive phenotypic correction in seven of 12 evaluable patients with ≥12 months of follow up as demonstrated by increased resistance to mitomycin-C (MMC) in bone marrow (BM)-derived colony forming cells and hematologic stabilization. The safety profile was highly favorable with no significant safety signals, and the treatment, administered without any cytotoxic conditioning, was well tolerated.
BLA submission for RP-L102 anticipated in Q4 2023. Based on the positive efficacy and safety data from the pivotal study of RP-L102, Rocket is on track to submit the BLA during the fourth quarter of 2023 and anticipates providing an update following FDA acceptance.
Pyruvate Kinase Deficiency (PKD)

Presented positive clinical data from Phase 1 trial of RP-L301 at the ASGCT (Free ASGCT Whitepaper) Annual Meeting. Data showed robust and sustained efficacy in both adult patients for up to 30 months. Results from the first pediatric patient indicate preliminary efficacy and favorable safety.
Received EMA Priority Medicines (PRIME) designation for RP-L301 in July. PRIME designation offers the benefits of early and enhanced support from the European Medicines Agency (EMA) and the opportunity for accelerated marketing application review. It is granted to medicines that target an unmet need for which no treatment option exists and with data showing a meaningful improvement of clinical outcomes.
Received FDA Regenerative Medicine Advanced Therapy (RMAT) designation for RP-L301. RMAT designation was granted based on robust safety and efficacy data from the ongoing Phase 1 study and its potential to cure a life-threatening disease for which no curative therapies currently exist. The designation will provide the benefits of added FDA guidance and expedited review through the program’s development. All four Rocket-sponsored programs with clinical data have received RMAT designation.
Adult and pediatric enrollment are completed in the Phase 1 study. Phase 2 pivotal trial initiation activities are anticipated in the fourth quarter of 2023.
Operational Update

Entered into data licensing agreement with Invitae to support timely access to genetic testing for patients who may have genetic forms of cardiomyopathy. The Invitae Unlock Cardiomyopathy and Arrhythmia genetic testing program evaluates causal mutations in approximately 170 genes, including LAMP2, PKP2 and BAG3, that result in Danon Disease, PKP2-ACM and BAG3-DCM, respectively. As part of the agreement, Rocket is raising awareness of this testing program and educating healthcare professionals across the U.S. on the benefits of screening for genetic forms of cardiomyopathy.
Second Quarter Financial Results

Cash position. Cash, cash equivalents and investments as of June 30, 2023, were $307.0 million.
R&D expenses. Research and development expenses were $51.4 million for the three months ended June 30, 2023, compared to $41.4 million for the three months ended June 30, 2022. The increase in R&D expenses was primarily driven by increases in compensation and benefits expense of $5.7 million due to increased R&D headcount, clinical trial expenses of $2.7 million, and non-cash stock-based compensation of $1.7 million.
G&A expenses. General and administrative expenses were $17.4 million for the three months ended June 30, 2023, compared to $12.9 million for the three months ended June 30, 2022. The increase in G&A expenses was primarily driven by increases in commercial preparation expenses which consists of commercial strategy, medical affairs, market development and pricing analysis of $1.3 million, compensation and benefits of $0.8 million due to increased G&A headcount and non-cash stock compensation expense of $1.1 million.
Net loss. Net loss was $65.7 million or $0.82 per share (basic and diluted) for the three months ended June 30, 2023, compared to $54.4 million or $0.83 (basic and diluted) for the three months ended June 30, 2022.
Shares outstanding. 80,521,415 shares of common stock were outstanding as of June 30, 2023.
Financial Guidance

Cash position. As of June 30, 2023, Rocket had cash, cash equivalents and investments of $307.0 million. Rocket expects such resources will be sufficient to fund its operations into the first half of 2025, including producing AAV cGMP batches at the Company’s Cranbury, N.J. R&D and manufacturing facility and continued development of its six clinical and/or preclinical programs.