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]).

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"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

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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").
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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:

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SPY001 – a highly potent and selective anti-α4β7 monoclonal antibody engineered with half-life extension technology and formulated for high concentration, convenient dosing.
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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.
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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.
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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

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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.

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

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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]):

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

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]).

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"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.

On August 10, 2023 OmniAb, Inc. (NASDAQ: OABI) reported financial results for the three and six months ended June 30, 2023, and provided operating and partner program updates (Press release, OmniAb, AUG 10, 2023, View Source [SID1234634244]).

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"Our business continued to grow and perform well during the second quarter with advancements in business development and progress across partner programs. The addition of four new partners during the quarter, including Merck & Co. and Neurocrine Biosciences, highlights the relevance and robustness of our technology platform. Existing partners made progress with two new programs entering the clinic and the total number of active programs increased to 305," said Matt Foehr, Chief Executive Officer of OmniAb, Inc.

"Our ion channel business continues to identify and explore high-value targets and one of the GSK programs achieved a key milestone in its discovery process. We look forward to providing updates related to our current partners as well as to securing additional partnerships," he added.

Second Quarter 2023 Financial Results

Revenue for the second quarter of 2023 was $6.9 million, compared with $7.2 million for the same period in 2022. Milestone revenue was higher due to progress with the batoclimab program. Service revenue was lower primarily related to the completion of work on certain ion channel programs and a one-time adjustment related to the extension of one of our programs with GSK, partially offset by the recognition of a portion of a research progression milestone achieved in the quarter.

Research and development expense was $14.1 million for the second quarter of 2023, compared with $11.5 million for the same period in 2022, with the increase primarily due to higher personnel and facility costs. General and administrative expense was $8.7 million for the second quarter of 2023, compared with $5.0 million for the same period in 2022, with the increase primarily due to higher personnel costs and expenses related to being an independent publicly traded company.

Net loss for the second quarter of 2023 was $14.7 million, or $0.15 per share, compared with a net loss of $10.3 million, or $0.12 per share, for the same period in 2022.

Year-to-Date Financial Results

Revenue for the six months ended June 30, 2023 was $23.9 million, compared with $16.8 million for the same period in 2022. Milestone revenue was higher, primarily due to the recognition of a $10.0 million milestone payment related to the first commercial sale of TECVAYLI (teclistamab) in the European Union and progress with the batoclimab program. Service revenue was lower primarily related to the completion of work on certain ion channel programs and a one-time adjustment related to the extension of one of our programs with GSK, partially offset by the recognition of a portion of a research progression milestone achieved in the quarter.

Research and development expense was $27.9 million for the six months ended June 30, 2023, compared with $22.3 million for the same period in 2022, with the increase primarily due to higher personnel and facility costs. General and administrative expense was $16.9 million for the six months ended June 30, 2023, compared with $9.1 million for the same period in 2022, with the increase primarily due to higher personnel costs and expenses related to being an independent publicly traded company.

Net loss for the six months ended June 30, 2023 was $20.8 million, or $0.21 per share, compared with a net loss of $16.6 million, or $0.20 per share, for the same period in 2022.

As of June 30, 2023, OmniAb had cash, cash equivalents and short-term investments of $103.1 million. The Company continues to expect its cash, cash equivalents and short-term investments balance at year-end 2023 to be slightly higher than year-end 2022. Current cash, cash equivalents and short-term investments, along with the cash OmniAb generates from operations, are expected to be sufficient to fund operations for the foreseeable future.

On November 1, 2022, OmniAb completed a spin-off from Ligand Pharmaceuticals Incorporated (NASDAQ: LGND), resulting in OmniAb becoming an independent publicly traded company. Financial results prior to November 1, 2022, are presented on a carve-out basis derived from Ligand’s historical accounting records, as if OmniAb were an independent company.

Second Quarter 2023 and Recent Business Highlights

During the second quarter of 2023, OmniAb entered into four new license agreements including with Merck & Co., Inc., Neurocrine Biosciences Inc., Stanford University and Seattle Children’s Hospital. These additions bring the total active partner count to 74.

Programs by OmniAb’s partners continued to progress during the quarter, including two entering the clinic. Immunovant’s IMVT-1402, a next-generation FcRn antagonist, and Gloria’s GLS-012 (anti-LAG-3), initiated Phase 1 clinical trials in China. In addition, the Biologics License Application for batoclimab was accepted by the National Medical Products Administration of China (NMPA). Batoclimab continues to progress in clinical trials in other geographies. As of June 30, 2023, the Company’s partners had a total of 305 active programs. There are 29 active programs that have advanced into the clinical stage or later, including three approved drugs, one under regulatory review and 25 in various stages of clinical development.

Second quarter 2023 and recent partner highlights include the following:

Batoclimab

OmniAb earned milestone revenue related to the advancement of batoclimab into pivotal studies in two additional indications: thyroid eye disease and chronic inflammatory demyelinating polyneuropathy. These indications are in addition to the ongoing Phase 3 study in generalized myasthenia gravis (gMG).
Harbour BioMed announced the NMPA accepted the Biologics License Application of batoclimab for the treatment of gMG.
HanAll Biopharma announced it is progressing toward initiation of a Phase 3 clinical study in Japan later this year with batoclimab in gMG.
IMVT-1402

Immunovant announced it received Investigational New Drug clearance from the U.S. Food and Drug Administration (FDA) for IMVT-1402, a subcutaneously-administered FcRn inhibitor, and initiated a Phase 1 clinical trial in healthy volunteers. This trial will evaluate the safety, tolerability and pharmacodynamics of IMVT-1402. Initial data are expected later this year.
GLS-012

Gloria Pharmaceuticals initiated a Phase 1/2 study in China to investigate the safety, tolerability and preliminary efficacy of GLS-012 (anti-LAG-3) monotherapy and in combination with GLS-010 in subjects with advanced solid tumors after progression on standard treatment.
CSX-1004

Cessation Therapeutics announced that the FDA has authorized the company to initiate a clinical trial in the U.S. for CSX-1004, a monoclonal antibody designed specifically to prevent fentanyl overdose. Cessation announced plans to begin trials with the first cohort of subjects in August of 2023.
APVO436

Aptevo Therapeutics announced that its bispecific acute myeloid leukemia (AML) drug candidate APVO436, in combination with emerging standard-of-care venetoclax and azacitidine, achieved positive duration of remission results in its Phase 1b dose-escalation trial. Aptevo intends to conduct two Phase 2 clinical trials to include relapsed/refractory AML patients beginning in the second half of 2023 and frontline patients beginning in the first half of 2024.
GSK Program-1 (Ion Channels and Transporters)

OmniAb achieved a research progression milestone for small molecule inhibitors of a genetically-validated target relevant to neurological diseases in collaboration with GSK. Upon this achievement, OmniAb was entitled to a progression milestone payment of $2.0 million.
In May 2023, OmniAb highlighted some of its new technology at the 19th Annual PEGS Boston Conference and Expo, including a presentation on its OmniDeep platform. OmniDeep is a suite of in silico tools for therapeutic discovery and optimization that are woven throughout OmniAb’s various technologies and capabilities. OmniDeep facilitates rapid identification of candidates with the right affinity, specificity and developability profiles to make drug development more effective and efficient.

Conference Call and Webcast

OmniAb management will host a conference call with slides today beginning at 4:30 p.m. Eastern time to discuss this announcement and answer questions. To participate via telephone, please dial (888) 396-8049 using the conference ID 10758721. Slides, as well as the live and replay webcast of the call, are available at View Source