On August 8, 2024 Arcus Biosciences, Inc. (NYSE:RCUS), a clinical-stage, global biopharmaceutical company focused on developing differentiated molecules and combination therapies for people with cancer, reported financial results for the second quarter ended June 30, 2024, and provided a pipeline update on its clinical-stage investigational molecules – targeting TIGIT, the adenosine axis (CD73 and A2a/A2b receptors), HIF-2a, AXL and PD-1 – across multiple common cancers (Press release, Arcus Biosciences, AUG 8, 2024, View Source [SID1234645586]).

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"Our upcoming presentation of efficacy and safety data for casdatifan will demonstrate that it has the potential to be the best-in-class HIF-2a inhibitor," said Terry Rosen, Ph.D., chief executive officer of Arcus. "We are pursuing a broad development program in both first- and second-line settings, as well as differentiated combinations, to maximize the opportunity for casdatifan in ccRCC. Meanwhile, the accumulating data continue to enhance our confidence that our Fc-silent anti-TIGIT antibody, domvanalimab, has the potential for an improved safety profile over that of Fc-enabled antibodies, particularly when combined with chemotherapy, which may also result in an efficacy advantage for domvanalimab. With STAR-221 enrollment completed, we are looking forward to our first Phase 3 data readout."
Corporate Updates:
•In July 2024, Taiho Pharmaceutical (Taiho) exercised its option for quemliclustat, an investigational small molecule CD73 inhibitor, in Japan and certain other territories in Asia (excluding mainland China). As a result of this option exercise, Taiho will operationalize the Phase 3 PRISM-1 study evaluating quemliclustat in pancreatic cancer in Japan, and Arcus will receive an opt-in payment and is eligible to receive near-term milestone payments.
Pipeline Highlights:
Casdatifan (HIF-2a inhibitor)
•Multiple expansion cohorts evaluating casdatifan in clear cell renal cell carcinoma (ccRCC) are underway, with several data presentations expected in the next 18 months. Each cohort is enrolling approximately 30 patients.
◦ARC-20: Phase 1/1b study evaluating casdatifan as a monotherapy and in combination with other agents:
▪100 mg daily expansion cohort in 2L+ ccRCC: ORR data are expected to be presented in the fourth quarter of 2024.
▪50 mg and 150 mg expansion cohorts in 2L+ ccRCC: Enrollment has been completed for both cohorts and data are expected to be presented in 2025.
▪An expansion cohort evaluating casdatifan in combination with cabozantinib in 2L+ ccRCC is also enrolling.
•Following FDA feedback later this year, Arcus plans to initiate its first Phase 3 study, PEAK-1, evaluating casdatifan in combination with cabozantinib versus cabozantinib monotherapy in patients with metastatic ccRCC who have previously received anti-PD-1 therapy, in the first half of 2025.

•Arcus is in advanced stages of planning with a clinical collaboration partner to evaluate casdatifan in a potential first-in-class combination regimen for first-line metastatic ccRCC.
Domvanalimab (Fc-silent anti-TIGIT antibody) plus Zimberelimab (anti-PD-1 antibody)
Domvanalimab-Zimberelimab Updates:
•Updated data presented at the ASCO (Free ASCO Whitepaper) Annual Meeting from Arm A1 of the Phase 2 EDGE-Gastric study showed 12.9 months median progression-free survival (PFS) for domvanalimab plus zimberelimab and chemotherapy in first-line upper GI adenocarcinomas, which exceeded historical benchmarks for anti-PD-1 plus chemotherapy.
◦The EDGE-Gastric study is evaluating the same regimen in the same setting as the STAR-221 Phase 3 study.
•STAR-221, a Phase 3 study evaluating domvanalimab plus zimberelimab and chemotherapy in PD-L1 all-comer first-line metastatic upper GI adenocarcinomas, completed enrollment in June.
•STAR-121, a Phase 3 study evaluating domvanalimab plus zimberelimab and chemotherapy in PD-L1 all-comer first-line metastatic non-small cell lung cancer (NSCLC), is expected to complete enrollment in 2024.
Upcoming Domvanalimab-Zimberelimab Milestones:
•Overall survival (OS) and PFS data from previously enrolled patients in Part 1 of the Phase 3 ARC-10 study, evaluating domvanalimab plus zimberelimab versus zimberelimab versus chemotherapy in first-line PD-L1-high NSCLC, are expected to be presented by the end of 2024.
•OS data from the Phase 2 EDGE-Gastric study, evaluating domvanalimab plus zimberelimab and chemotherapy in upper GI adenocarcinomas, are expected to be presented in 2025.
CD73-Adenosine Axis: Etrumadenant (A2a/A2b receptor antagonist) and Quemliclustat (small-molecule CD73 inhibitor)
Etrumadenant
•Cohort B data from ARC-9, a randomized Phase 1b/2 study evaluating etrumadenant plus zimberelimab, FOLFOX chemotherapy and bevacizumab (EZFB) versus regorafenib in third-line metastatic colorectal cancer (mCRC), were presented at ASCO (Free ASCO Whitepaper) in June.
◦Results showed 19.7 months median OS for the EZFB arm, and EZFB significantly reduced the risk of death by 63% and risk of disease progression by 73% compared to regorafenib. This is the longest median OS reported in third-line mCRC to date in a randomized trial.
◦Biomarker data from this study are expected to be presented at a scientific conference in the second half of 2024.
•Based on these encouraging results, Arcus and Gilead are determining next steps for the development of etrumadenant in mCRC.
Quemliclustat
•Initiation of a Phase 3 trial, PRISM-1, of quemliclustat combined with gemcitabine/nab-paclitaxel versus gemcitabine/nab-paclitaxel in pancreatic cancer is expected to begin by early 2025.
•Taiho exercised its option for an exclusive license to quemliclustat in Japan and certain territories in Asia and will operationalize PRISM-1 in Japan.
Early Clinical Programs
•Dose escalation for AB801, a potent and highly selective small-molecule AXL inhibitor, continues. Arcus anticipates advancing this molecule into expansion cohorts in NSCLC in early 2025.
Financial Results for Second Quarter 2024:
•Cash, Cash Equivalents and Marketable Securities were $1.0 billion as of June 30, 2024, compared to $866 million as of December 31, 2023. The increase during the period is primarily due to the receipt of $320 million in cash from Gilead for their January 2024 equity investment, partially offset by the use of cash in research and development activities. We believe our cash, cash equivalents and marketable securities on-hand will be sufficient to fund operations into 2027. Cash, cash equivalents and marketable securities are expected to be between $885 million and $925 million at the end of 2024.

•Revenues were $39 million for the second quarter 2024, compared to $29 million for the same period in 2023. In the second quarter 2024, Arcus recognized $28 million in license and development services revenue related to the advancement of programs, as well as $11 million in other collaboration revenue primarily related to Gilead’s ongoing rights to access Arcus’s research and development pipeline in accordance with the Gilead collaboration agreement.
•Research and Development (R&D) Expenses were $115 million for the second quarter 2024, compared to $84 million for the same period in 2023. The net increase of $31 million was primarily driven by higher clinical trial and headcount-related costs associated with our late-stage development program activities. Non-cash stock-based compensation expense was $10 million for the second quarter 2024, compared to $9 million for the same period in 2023. For the second quarter 2024 and 2023, Arcus recognized gross reimbursements of $40 million and $44 million, respectively, for shared expenses from its collaborations, primarily the Gilead collaboration. R&D expense by quarter may fluctuate due to the timing of clinical manufacturing and standard-of-care therapeutic purchases with a corresponding impact on reimbursements.
•General and Administrative (G&A) Expenses were $30 million for the second quarter 2024, compared to $28 million for the same period in 2023. The increase was primarily driven by higher headcount and costs incurred to obtain the Third Gilead Agreement Amendment. Non-cash stock-based compensation expense was $10 million for the second quarter 2024, compared to $9 million for the same period in 2023.
•Net Loss was $93 million for the second quarter 2024, compared to $75 million for the same period in 2023.

Conference Call Information:
Arcus will host a conference call and webcast today, August 8, at 2:00 PM PT / 5:00 PM ET to discuss its second-quarter 2024 financial results and pipeline updates. To access the call, please dial (404) 975-4839 (local) or (833) 470-1428 (toll-free), using Access Code: 287576. To access the live webcast and accompanying slide presentation, please visit the "Investors & Media" section of the Arcus Biosciences website at www.arcusbio.com. A replay of the webcast will be available following the live event.

Arcus Ongoing and Announced Clinical Studies:
Trial Name Arms Setting Status NCT No.
Lung Cancer
STAR-121
dom + zim + chemo vs. pembro + chemo 1L NSCLC (PD-L1 all-comers) Ongoing Registrational Phase 3 NCT05502237
PACIFIC-8
dom + durva vs. durva Unresectable Stage 3 NSCLC Ongoing Registrational Phase 3 NCT05211895
STAR-131 dom + zim + chemo; dom + zim Perioperative NSCLC Registrational Phase 3 In Planning TBD
ARC-7 zim vs. dom + zim vs. etruma + dom + zim 1L NSCLC (PD-L1 ≥ 50%) Ongoing Randomized Phase 2 NCT04262856
EDGE-Lung dom +/- zim +/- quemli +/- chemo 1L/2L NSCLC (lung cancer platform study) Ongoing Randomized Phase 2 NCT05676931
VELOCITY-Lung
dom +/- zim +/- etruma +/- sacituzumab govitecan-hziy or other combos 1L/2L NSCLC (lung cancer platform study) Ongoing Randomized Phase 2 NCT05633667
Upper Gastrointestinal Cancers
STAR-221 dom + zim + chemo vs. nivo + chemo 1L Gastric, GEJ and EAC Ongoing Registrational Phase 3 NCT05568095
EDGE-Gastric (ARC-21) dom +/- zim +/- quemli +/- chemo 1L/2L Upper GI Malignancies
Ongoing
Randomized Phase 2
NCT05329766
Colorectal Cancer
ARC-9 etruma + zim + mFOLFOX vs. SOC 2L/3L/3L+ CRC Ongoing
Randomized Phase 2
NCT04660812
Pancreatic Cancer
PRISM-1 quemli + gem/nab-pac vs. gem/nab-pac 1L PDAC Planned Phase 3 TBD
ARC-8 quemli + zim + gem/nab-pac vs. quemli + gem/nab-pac 1L, 2L PDAC Ongoing Randomized Phase 1/1b NCT04104672
Kidney Cancer
PEAK-1 cas + cabo vs. cabo Post-IO ccRCC Planned Phase 3 TBD
STELLAR-009 cas + zanza ccRCC Ongoing Phase 1b/2 NCT06191796
ARC-20 cas, cas + cabo Cancer Patients / ccRCC Ongoing Phase 1/1b NCT05536141
Other
ARC-25 AB598 Advanced Malignancies Ongoing NCT05891171
ARC-27 AB801 Advanced Malignancies Ongoing NCT06120075

cabo: cabozantinib; cas: casdatifan; dom: domvanalimab; durva: durvalumab; etruma: etrumadenant; gem/nab-pac: gemcitabine/nab-paclitaxel; nivo: nivolumab; pembro: pembrolizumab; quemli: quemliclustat; SOC: standard of care; zanza: zanzalintinib; zim: zimberelimab; ccRCC: clear cell renal cell carcinoma; CRC: colorectal cancer; EAC: esophageal adenocarcinoma; GEJ: gastroesophageal junction; GI: gastrointestinal; NSCLC: non-small cell lung cancer; PDAC: pancreatic ductal adenocarcinoma

On August 8, 2024 Arcellx, Inc. (NASDAQ: ACLX), a biotechnology company reimagining cell therapy through the development of innovative immunotherapies for patients with cancer and other incurable diseases, reported business highlights and financial results for the second quarter ended June 30, 2024 (Press release, Arcellx, AUG 8, 2024, View Source [SID1234645585]).

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"We continue to make significant strides as we accelerate our business," said Rami Elghandour, Arcellx’s Chairman and Chief Executive Officer. "We are thrilled about the momentum across our multiple myeloma program being developed in partnership with Kite. We believe anito-cel has the potential to be a best-in-class treatment option, and with the strength of our Kite partnership, we are well-positioned to change the treatment paradigm for multiple myeloma patients. We look forward to presenting data from the iMMagine-1 study by the end of this year. As iMMagine-1 continues to mature, we’re also pleased with the progress in iMMagine-3, which was initiated by our partner Kite. More broadly, we believe that our novel synthetic binder, the D-Domain, which forms the basis of our technology platform and our lead program anito-cel, can be developed in oncology and non-oncology indications. We are excited to share that the U.S. Food and Drug Administration has cleared our IND application for the development of anito-cel to treat patients with myasthenia gravis. Our progress this quarter reflects our incredible team and their dedication to advancing our mission of helping as many patients as possible by delivering on the promise of our platform."

Recent Business Progress

•
The Company earned a $68 million clinical milestone payment from Kite Pharma, Inc., a Gilead Company, for iMMagine-1 enrollment.

•
The Company submitted an abstract to present data for the iMMagine-1 study at the 66th ASH (Free ASH Whitepaper) Annual Meeting and Exposition.

•
Kite has initiated the global Phase 3 trial, iMMagine-3. This trial will evaluate anito-cel in patients exposed to an immunomodulatory
(IMiD) drug and an anti-CD38 monoclonal antibody.

•
The U.S. Food and Drug Administration (FDA) cleared an Investigational New Drug (IND) application for anito-cel, Arcellx’s BCMA CAR-T therapy, for myasthenia gravis, a chronic autoimmune disease. Arcellx wholly owns and is solely developing this program.

Second Quarter 2024 Financial Highlights

Cash, cash equivalents, and marketable securities:

As of June 30, 2024, Arcellx had cash, cash equivalents, and marketable securities of $646.8 million. Arcellx anticipates that its cash, cash equivalents, and marketable securities will fund its operations into 2027.

Collaboration revenue:

Collaboration revenue were $27.4 million and $14.3 million for the quarters ended June 30, 2024 and 2023, respectively, an increase of $13.1 million. This increase was primarily driven by an increase in estimated transaction price from the expansion to the license and collaboration agreement with Kite.

R&D expenses:

Research and development expenses were $41.0 million and $28.3 million for the quarters ended June 30, 2024 and 2023, respectively, an increase of $12.7 million. This increase was primarily driven by increased costs relating to other preclinical pipeline programs and increased personnel costs, which include non-cash stock-based compensation expense.

G&A expenses:

General and administrative expenses were $21.4 million and $15.5 million for the quarters ended June 30, 2024 and 2023, respectively, an increase of $5.9 million. This increase was primarily driven by increased personnel costs, which include non-cash stock-based compensation expense.

Net income or loss:

Net losses were $27.2 million and $23.9 million for the quarters ended June 30, 2024 and 2023, respectively.

On August 8, 2024 Aptose Biosciences Inc. ("Aptose" or the "Company") (NASDAQ: APTO, TSX: APS), a clinical-stage precision oncology company developing highly differentiated oral targeted agents to treat hematologic malignancies, reported financial results for the three months ended June 30, 2024, and provided a corporate update (Press release, Aptose Biosciences, AUG 8, 2024, View Source [SID1234645584]).

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"We are pleased that our triplet protocol of tuspetinib with venetoclax and azacitidine (TUS+VEN+AZA) has been allowed to proceed at the 40 mg dose of tuspetinib, a dose that as a single agent and in doublet therapy has been shown to be safe and active," said William G. Rice, Ph.D., Chairman, President and Chief Executive Officer of Aptose. "We – along with our board and outside scientific advisors – strongly believe tuspetinib is an ideal drug for frontline triplet therapy and we remain committed to securing financing to pursue its development for the newly diagnosed AML patient population in desperate need of an improved frontline therapy."

Key Corporate Highlights

Tuspetinib Protocol Now Ready for Triplet Therapy Study – Aptose’s company-sponsored phase 1/2 TUS+VEN+AZA triplet study is designed to test tuspetinib in combination with standard of care dosing of azacitidine and venetoclax as frontline therapy in newly diagnosed AML patients unfit for chemotherapy. The planned study will dose VEN-naïve, FLT3i-naïve, and HMA-naïve patients, a group expected to be highly responsive to the TUS+VEN+HZA triplet regimen. Current triplet therapies containing kinase inhibitors can be limited by toxicities often requiring dose reductions of all three agents and may not be effective in the larger FLT3-unmutated AML population. The U.S. Food and Drug Administration (FDA) has allowed TUS to be administered as part of the triplet at 40 mg daily, at an initial dose shown active as a single agent in relapsed or refractory AML patients.
ASH Abstract – On July 31, 2024, Aptose submitted an abstract for presentation at the 2025 Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in December 2024. Lead author Navel Daver, MD, University of Texas MD Anderson Cancer Center, Houston, TX and research team explore the safety and efficacy results that support the upcoming combination study of TUS+VEN+AZA as a triplet drug combination frontline therapy in newly diagnosed AML patients ineligible for intensive chemotherapy, independent of FLT3 mutation status, which is an important differentiator for tuspetinib.
Nasdaq – On July 19, 2024, Aptose announced that it had received a deficiency letter (the "Deficiency Letter") from the Nasdaq Listing Qualifications Department of The Nasdaq Stock Market LLC ("Nasdaq") notifying the Company that, for the last thirty (30) consecutive business days, the closing bid price for the Company’s common shares had been below the minimum $1.00 per share required for continued listing on The Nasdaq Capital Market pursuant to Nasdaq Listing Rule 5550(a)(2) (the "Minimum Bid Price Requirement"). The Deficiency Letter has no immediate effect on the listing of the Company’s common shares, and its common shares will continue to trade on The Nasdaq Capital Market under the symbol "APTO" at this time. The Company’s common shares continue to trade on the Toronto Stock Exchange ("TSX") under the symbol "APS". The Company’s listing on the TSX is independent and will not be affected by the Nasdaq listing status.
In accordance with Nasdaq Listing Rule 5810(c)(3)(A), the Company has been given one hundred and eighty (180) calendar days, or until January 10, 2025, to regain compliance with the Minimum Bid Price Requirement. If at any time before January 10, 2025, the bid price of the Company’s common shares closes at $1.00 per share or more for a minimum of ten (10) consecutive business days, the Staff will provide written confirmation that the Company has achieved compliance. If the Company does not regain compliance with the Minimum Bid Price Requirement by January 10, 2025, the Company may be afforded a second one hundred and eighty (180) calendar day period to regain compliance. The Company intends to monitor the closing bid price of its common shares and may, if appropriate, consider available options to regain compliance with the Minimum Bid Price Requirement. However, there can be no assurance that the Company will be able to regain compliance with the Minimum Bid Price Requirement or will otherwise be in compliance with other Nasdaq Listing Rules.

Multiple Planned Value-creating Milestones Ahead

Frontline therapy triplet pilot dose initiation planned in newly diagnosed (ND) AML: 2H 2024
Triplet pilot dose escalation planned with early data in ND AML: ASH (Free ASH Whitepaper) 2024
Triplet pilot completed with CR/MRD data and dose selection: EHA (Free EHA Whitepaper) 2025
Triplet Ph2/Ph3 pivotal program planned initiation: 2H 2025

On August 8, 2024 ALX Oncology Holdings Inc., ("ALX Oncology" or "the Company") (Nasdaq: ALXO), an immuno-oncology company developing therapies that block the CD47 immune checkpoint pathway, reported financial results for the second quarter ended June 30, 2024, and provided a corporate update (Press release, ALX Oncology, AUG 8, 2024, View Source [SID1234645583]).

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"Our team continues to make significant progress in the advancement of our evorpacept development pipeline across multiple oncology indications," said Jason Lettmann, Chief Executive Officer of ALX Oncology. "Data readouts across our Phase 1 and 2 clinical trials highlight the potential of evorpacept as a disruptive therapy in combination with anti-cancer antibodies and ADCs. In particular, the recent ASPEN-06 Phase 2 clinical trial readout in patients with previously treated HER2-positive advanced gastric cancer is a significant growth milestone for the Company. We are well positioned to build on our second quarter achievements and continue to advance toward our anticipated milestones in the months ahead."

Second Quarter 2024 Highlights and Recent Developments

Evorpacept Clinical Development Program

On July 31, ALX Oncology reported topline data from the multi-center, international ASPEN-06 Phase 2 clinical trial evaluating evorpacept in combination with trastuzumab, CYRAMZA (ramucirumab) and paclitaxel ("Evo-TRP") against trastuzumab, CYRAMZA (ramucirumab) and paclitaxel ("TRP") for the treatment of patients with HER2-positive gastric/gastroesophageal junction ("GEJ") cancer, where all patients had received an anti-HER2 agent in prior lines of therapy (NCT05002127).
Results demonstrated that evorpacept improved tumor response in patients with HER2-positive gastric/GEJ cancer, becoming the first CD47 blocker to show promising and durable response with a well-tolerated safety profile in a prospective randomized study.
The primary endpoint was confirmed overall response rate ("ORR") with key secondary endpoints being safety, median duration of response ("mDOR"), progression-free survival ("PFS") and overall survival ("OS"). Primary study objectives were to compare confirmed ORR of Evo-TRP to an assumed ORR of 30% for CYRAMZA (ramucirumab) and paclitaxel ("RP") with one-sided alpha error of 0.025, and to identify a clinically meaningful contribution of Evo to TRP in ORR (delta >10%).
Evo-TRP achieved a confirmed ORR of 40.3% compared to 26.6% for the TRP control arm and demonstrated a mDOR of 15.7 months compared to 7.6 months in the intent to treat population ("ITT") (N=127). The primary analysis of the ITT compared Evo-TRP to an assumed RP control ORR of 30% (p=0.095). When a comparison of Evo-TRP to the observed TRP control arm ORR of 26.6% was explored using a similar testing procedure, a p-value of p=0.027 was observed. Secondary endpoints of PFS and OS were immature at the time of analysis.
Evo-TRP combination showed the greatest response with an ORR of 54.8% compared to 23.1% in the TRP control arm in a pre-specified population of patients with fresh HER2-positive biopsies (n=48). In this population, Evo-TRP compared to an assumed RP control ORR of 30% yielded a p-value of p=0.030 . When Evo-TRP compared to the observed TRP ORR of 23.1% was explored using a similar testing procedure, a p-value of p=0.0038 was observed, suggesting HER2-expression strongly correlates with evorpacept efficacy and validating its mechanism of action.
In June, ALX Oncology presented the first evorpacept combination data with an antibody-drug conjugate ("ADC") from the Phase 1 ASPEN-07 clinical trial in patients with advanced urothelial cancer at the 2024 American Society of Cancer Oncology ("ASCO") Annual Meeting.
This open-label, single-arm, clinical trial of evorpacept in combination with an approved ADC, PADCEV (enfortumab vedotin), demonstrated promising activity and was generally well tolerated in patients with locally advanced or metastatic urothelial cancer (NCT05524545).
In April, ALX Oncology reported positive data from the ongoing Phase 1/2 investigator-sponsored clinical trial of evorpacept in combination with standard-of-care in patients with relapsed or refractory B-cell non-Hodgkin lymphoma (NCT05025800).
The combination achieved promising initial activity with a best ORR of 94% and a complete response rate ("CRR") of 83% in patients with indolent R/R B-NHL (compared to rituximab and lenalidomide historical CRR benchmark of 34%).
In April, ALX Oncology announced the initiation of a Phase 2 investigator-sponsored trial of neoadjuvant radiation and evorpacept in combination with KEYTRUDA (pembrolizumab) in patients with previously untreated and early-stage locally advanced, resectable, human papillomavirus-mediated oropharyngeal cancer (NCT05787639).
Conference Presentations

At the 2024 ASCO (Free ASCO Whitepaper) Annual Meeting, ALX Oncology presented the first evorpacept combination data with an ADC from the Phase 1 ASPEN-07 clinical trial in patients with locally advanced or metastatic urothelial cancer.
In the open-label, single-arm, clinical trial of evorpacept in combination with an approved ADC, PADCEV (enfortumab vedotin), demonstrated promising activity and was generally well tolerated in patients.
The Company also presented results of an investigator-sponsored, Phase 2 study of evorpacept, cetuximab and pembrolizumab in patients with refractory microsatellite stable metastatic colorectal cancer.
At the 2024 American Association of Cancer Research Annual Meeting, ALX Oncology presented two evorpacept clinical abstracts including:
Phase 1 investigator-initiated trial of evorpacept, lenalidomide and rituximab for patients with relapsed or refractory B-cell non-Hodgkin lymphoma.
Phase 1 study of azacitidine in combination with evorpacept for higher-risk myelodysplastic syndrome (MDS)
Board and Executive Appointments

ALX Oncology strengthened the Company’s board and leadership team with the appointment of Alan Sandler, M.D., to its Board of Directors, and the addition of Allison Dillon, Ph.D., to its executive leadership team as Chief Business Officer.
Upcoming Clinical Milestones for Evorpacept’s Development Pipeline

ALX Oncology is well-positioned to achieve numerous milestones across multiple oncology indications in its evorpacept clinical development program:
Head and Neck Squamous Cell Carcinoma – Topline results from a Phase 2 randomized clinical trial of ASPEN-03 with KEYTRUDA (1H 2025)
Head and Neck Squamous Cell Carcinoma – Topline results from a Phase 2 randomized clinical trial of ASPEN-04 with KEYTRUDA and chemotherapy (1H 2025)
Gastric/GEJ Cancer – Updated results of ASPEN-06 Phase 2 clinical trial (1H 2025)
Urothelial Cancer – Updated results from a Phase 1 clinical trial of ASPEN-07 in combination with PADCEV (1H 2025)
Gastric/GEJ Cancer – Initiation of Phase 3 registrational randomized clinical trial for evorpacept (mid-2025)
Breast Cancer – Topline results from a Phase 1b I-SPY TRIAL with ENHERTU (fam-trastuzumab deruxtecan-nxki) (2H 2025)
Second Quarter 2024 Financial Results:

Cash, Cash Equivalents and Investments: Cash, cash equivalents and investments as of June 30, 2024, were $186.2 million. The Company believes its cash, cash equivalents and investments, which includes the proceeds from sales under its at-the-market ("ATM") offering in the first half of 2024 are sufficient to fund planned operations well into Q1 2026.
Research and Development ("R&D") Expenses: R&D expenses consist primarily of pre-clinical, clinical and manufacturing expenses related to the development of the Company’s current lead product candidate, evorpacept, and R&D employee-related expenses. These expenses for the three months ended June 30, 2024, were $34.7 million, compared to $29.5 million for the prior-year period. R&D expenses increased by $5.2 million during the three months ended June 30, 2024, compared to the three months ended June 30, 2023. The increase was primarily attributable to an increase of $1.7 million in preclinical costs for development of new targets, an increase of $1.2 million in personnel and related costs primarily driven by headcount growth, an increase of $1.8 million in stock-based compensation expense and an increase of $0.7 million in other research costs primarily due to absence of VAT refunds in the current quarter compared to prior year quarter.
General and Administrative ("G&A") Expenses: G&A expenses consist primarily of administrative employee-related expenses, legal and other professional fees, patent filing and maintenance fees, and insurance. These expenses for the three months ended June 30, 2024, were $6.9 million, compared to $7.3 million for the prior year period. G&A expenses decreased by $0.4 million during the three months ended June 30, 2024, compared to the three months ended June 30, 2023. The decrease was primarily attributable to a decrease of $0.8 million in stock-based compensation expense primarily due to a change in classification from the comparative periods of stock-based compensation from G&A to R&D as described above under R&D expenses, offset by an increase of $0.3 million in other G&A costs from accounting consulting and personnel costs driven by headcount growth.
Net loss: GAAP net loss was $39.4 million for the three months ended June 30, 2024, or ($0.76) per basic and diluted share, as compared to a GAAP net loss of $34.2 million for the three months ended June 30, 2023, or ($0.84) per basic and diluted share. Non-GAAP net loss was $32.1 million for the three months ended June 30, 2024, as compared to a non-GAAP net loss of $27.9 million for the three months ended June 30, 2023. A reconciliation of GAAP to non-GAAP financial results can be found at the end of this news release.

On August 8, 2024 Akebia Therapeutics, Inc. (Nasdaq: AKBA), a biopharmaceutical company with the purpose to better the lives of people impacted by kidney disease, reported financial results for the second quarter ended June 30, 2024, and recent business highlights (Press release, Akebia, AUG 8, 2024, View Source [SID1234645582]). During the quarter, Akebia made significant progress across multiple initiatives related to the commercial launch of Vafseo (vadadustat) Tablets recently approved by the U.S. Food and Drug Administration (FDA) for the treatment of anemia due to chronic kidney disease (CKD) in adults who have been receiving dialysis for at least three months.

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"Since receiving FDA approval in late March, our key priority has been to execute on our launch strategy developed with a goal for Vafseo to become the standard of care in the treatment of anemia for dialysis patients," said John P. Butler, Chief Executive Officer of Akebia. "Our team is actively engaged with prescribers, and I’m extremely encouraged by the positive reception we’ve seen across the kidney community for a new choice in anemia management. Equally important, our commercial team is now in active discussions with dialysis organizations covering the vast majority of patients to contract both Auryxia (ferric citrate) and Vafseo, giving our team a unique opportunity to contract across the portfolio."

Vafseo Global Launch Activities

•In June, Akebia submitted its Transitional Drug Add-on Payment Adjustment (TDAPA) application. Akebia expects to have Healthcare Common Procedure Coding System (HCPCS) codes assigned in October 2024 and TDAPA designation by January 1, 2025.
•Akebia set the Vafseo wholesale acquisition cost (WAC) at $1,278 for a 30-day supply at the labeled starting dose, or approximately $15,500 per year. All Vafseo sales in dialysis will be under contracts that include an off-invoice discount as well as volume-based tier discounts off the WAC price.
•Akebia partner MEDICE Arzneimittel Pütter GmbH&Co.KG (Medice) launched Vafseo in Germany and Austria in June and in the Netherlands in August.
•In July, Akebia regained full rights to sell Vafseo in the U.S. and is now able to contract directly with all dialysis organizations following the execution of a royalty-based termination agreement with CSL Vifor to simplify operational execution and improve economics.

Corporate Updates

In June, Erik Ostrowski joined Akebia as Senior Vice President, Chief Financial Officer and Chief Business Officer. Mr. Ostrowski brings over 20 years of finance and biotech operating experience, with a background in investment banking, including as a director of healthcare investment banking at Leerink Partners. He brings an impressive track record of corporate development leadership and strategic transaction execution.
Akebia reported second quarter 2024 Auryxia net product revenues of $41.2 million. Akebia expects Auryxia full year 2024 net product revenues to be in line with 2023 Auryxia net product revenue levels. Akebia’s commercial organization is heavily engaged in efforts to contract Auryxia through dialysis organizations in 2025, as phosphate binders are expected to be added to the Centers for Medicare & Medicaid Services bundled payment for dialysis care in January 2025.
Financial Results
•Revenues: Total revenues were $43.6 million in the second quarter of 2024 compared to $56.4 million in the second quarter of 2023. The decrease was driven by a reduction in license, collaboration and other revenue, which included a one-time $10 million upfront payment related to our Medice license agreement in the second quarter of 2023.

▪Net product revenues were $41.2 million in the second quarter of 2024 compared to $42.2 million in the second quarter of 2023.

▪License, collaboration and other revenues were $2.4 million in the second quarter of 2024 compared to $14.1 million in the second quarter of 2023.

•Cost of Goods Sold: Cost of goods sold (COGS) was $17.0 million in the second quarter of 2024 compared to $17.3 million in the second quarter of 2023. Akebia continues to carry a non-cash intangible amortization charge of $9.0 million per quarter in COGS through the fourth quarter of 2024.

•Research & Development Expenses: Research and development expenses were $7.6 million in the second quarter of 2024 compared to $20.2 million in the second quarter of 2023. The decrease was largely due to the completion of activities related to certain clinical trials, a reduction in consulting expenses and lower headcount related costs.

•Selling, General & Administrative Expenses: Selling, general and administrative expenses were $26.9 million for the second quarter of 2024 compared to $27.0 million in the second quarter of 2023.

•Net Loss: Net loss was $8.6 million in the second quarter of 2024 compared to $11.2 million in the second quarter of 2023.

•Cash Position: Cash and cash equivalents as of June 30, 2024 were $39.5 million. Akebia expects its existing cash resources and cash from operations will be sufficient to fund its current operating plan, including the U.S. Vafseo launch, for at least two years.
Conference Call
Akebia will host a conference call on Thursday, August 8 at 8:00 a.m. Eastern Time to discuss second quarter 2024 earnings. To access the call, please dial (800) 715-9871 (USA & Canada – Toll-Free) and enter Conference ID: 4155557.