On May 2, 2024 Alpha-9 Oncology (Alpha-9), a clinical stage company developing radiopharmaceuticals for the treatment of cancer patients worldwide, reported that the first participant has been dosed in the Phase 1 study evaluating 68Ga-A9-3202, a radiodiagnostic targeting melanocortin 1 receptor (MC1R) for the imaging of locally advanced or metastatic melanoma (Press release, Alpha9 Oncology, MAY 2, 2024, View Source [SID1234642574]). MC1R expression is known to be elevated in malignant melanoma and other types of skin cancers.

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The Phase 1 multi-centre study is evaluating the tumour uptake and normal tissue distribution of 68Ga-A9-3202 in participants with locally advanced or metastatic melanoma (ACTRN12624000085572). This new agent, in conjunction with current standard CT or PET/CT imaging, will be used in the future to identify patients suitable to receive a novel radiotherapeutic agent targeting MC1R also developed by Alpha-9.

"This agent is the first step in our effort to develop radiopharmaceuticals targeting MC1R for the treatment of patients with advanced or metastatic melanoma," commented Dr. Ovid Trifan, Chief Medical Officer at Alpha-9. "We’re excited to advance our portfolio of molecules further into the clinic."

On May 2, 2024 ImmunityBio, Inc. (NASDAQ: IBRX) reported that it has signed an exclusive global arrangement with the Serum Institute of India, the world’s largest manufacturer of vaccines by number of doses produced, to supply ImmunityBio with Bacillus Calmette-Guerin (BCG) (Press release, ImmunityBio, MAY 2, 2024, View Source [SID1234642589]). The agreement covers the manufacturing of standard BCG (sBCG) that is currently approved for use outside the U.S., as well as a next-generation recombinant BCG (iBCG) undergoing testing, intended for use in combination with ImmunityBio’s ANKTIVA (nogapendekin alfa inbakicept-pmln) for currently approved and potential future indications, subject to regulatory approvals.

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"We are pleased to partner with the Serum Institute of India so that the power of its large-scale, world-class, GMP manufacturing capacity can be used to address the issue of BCG shortage, which affects thousands of bladder cancer patients annually," said Patrick Soon-Shiong, M.D., Executive Chairman and Global Chief Scientific and Medical Officer at ImmunityBio. "We are especially proud to be partnering with Dr. Cyrus Poonawalla, the Institute’s Chairman and founder and someone who has made such a positive impact on global health."

The arrangement will result in additional supplies of the current standard sBCG immediately for trials. At the same time, the two companies will work to accelerate the ongoing Phase 2 clinical trials of iBCG currently being conducted in Europe which has so far demonstrated safety advantageous over standard BCG as well as enhanced immunogenicity in driving both CD8+ and CD4 T cells.

The collaboration between SII and ImmunityBio comes on the heels of the FDA’s approval of ANKTIVA for the treatment of non-muscle invasive bladder cancer with carcinoma in situ (CIS). Increasing the available supply of BCG is intended to address shortages for the combination therapy with ANKTIVA.

"The collaboration between Serum Institute of India and ImmunityBio will undoubtedly transform the way we approach cancer treatment. It will improve global access to BCG and at the same time—the unique therapy is the key to achieve a complete solution for bladder cancer. We are truly excited to witness the incredible impact this collaboration will have on improving patient outcomes and saving countless lives," said Mr. Adar C. Poonawalla, CEO, Serum Institute of India.

Originally used as a tuberculosis vaccine, BCG administered via intravesical instillation (delivery to the bladder via a catheter) has been the standard of care for patients with non-muscle invasive bladder cancer since 1977. BCG is a benign bacterium that induces an immune response in the bladder in proximity to the cancer cells, leading to clearance of the cancer in many patients.

BCG is one of the most widely used vaccines worldwide and has been given to more than 4 billion individuals with astonishing safety records. However, because BCG is a biologic drug that uses benign bacteria it is more complicated to make than many other types of drugs. SII is the largest manufacturer of BCG vaccine across the world, while Merck & Co. based in New Jersey currently is the only manufacturer of BCG (TICE BCG) in the U.S.

"The scale and quality of vaccines that the Serum Institute manufacturers is unparalleled and we are honored to partner with Dr. Poonawalla and his leadership team on this important initiative," said Richard Adcock, President and CEO of ImmunityBio. "By providing another option for BCG, we believe more NMIBC patients will be able to benefit from this proven treatment as both a monotherapy and a combination therapy with ANKTIVA."

ImmunityBio plans to conduct clinical trials to study recombinant BCG (iBCG) and sBCG manufactured by Serum Institute in combination with ANKTIVA for the treatment of different types of bladder and other cancers. Supply of BCG is expected to be available once the protocol for the trial has been authorized by the FDA. ImmunityBio plans to submit the protocol to the FDA and to global regulatory bodies in the next 30 days.

"The opportunity to initiate a trial of an immunogenic recombinant BCG, which has already demonstrated enhanced safety compared to standard BCG in Phase 1/2 studies, is exciting. We look forward to exploring ANKTIVA in combination with BCG in non-muscle invasive bladder cancer (NMIBC) and across other tumor types. With our ability to overcome immune evasion of the tumor to BCG when BCG is given alone, and by converting a MHC- negative cold tumor to a MHC+ positive hot tumor with the combination of ANKTIVA with BCG, we will now further expand the development of our therapeutic cancer vaccine with BCG," said Dr. Soon-Shiong.

For investigators interested in participating in clinical trials or to learn more information, please email [email protected]

On May 2, 2024 Lucid Diagnostics Inc. (Nasdaq: LUCD) ("Lucid" or the "Company") a commercial-stage, cancer prevention medical diagnostics company, and majority-owned subsidiary of PAVmed Inc. (Nasdaq: PAVM), reported the peer-reviewed publication of positive data from a National Cancer Institute (NCI)-sponsored, prospective, multicenter clinical validation study assessing the performance of Lucid’s EsoGuard Esophageal DNA Test on samples collected with its EsoCheck Esophageal Cell Collection Device (Press release, Lucid Diagnostics, MAY 2, 2024, View Source [SID1234642608]). This case-control study, led by Case Western Reserve University’s Amitabh Chak, M.D., was funded through the NCI’s Barrett’s Esophagus Translational Research Network (BETRNet), and included renowned investigators from leading academic medical centers, including Case Western Reserve University, Mayo Clinic, Johns Hopkins University, Washington University in St. Louis, University of North Carolina, and Cleveland Clinic. EsoGuard demonstrated unprecedented early precancer detection compared to the gold standard of upper endoscopy (EGD). The manuscript, entitled Multicenter, Prospective Trial of Non-Endoscopic Biomarker-Driven Detection of Barrett’s Esophagus And Esophageal Adenocarcinoma, was published in the American Journal of Gastroenterology, the leading clinical journal covering gastroenterology published on behalf of the American College of Gastroenterology (ACG).

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"We owe a debt of gratitude to Dr. Chak and his BETRNet co-investigators for their contributions to this landmark study, which we believe represents a pivotal step towards our goal of eliminating the scourge of esophageal cancer," said Lishan Aklog, M.D., Lucid’s Chairman and Chief Executive Officer. "EsoGuard’s unprecedented ability to noninvasively detect early ("non-dysplastic") precancer is a remarkable scientific and technological accomplishment. Its high sensitivity and estimated negative predictive value (NPV) are hallmarks of a powerful early detection test. What was particularly striking was its 89% sensitivity in short-segment non-dysplastic precancers (SSBE), which account for at least 70% of cases in a screening population and are responsible for at least half of all future cancers. Effective esophageal cancer prevention requires high SSBE sensitivity. EsoGuard’s 89% SSBE sensitivity is in stark contrast to the 63% SSBE sensitivity documented in a recent research report on methylation markers using decades-old sponge-on-a-string (SOS) technology. We believe this stark difference reflects both the superiority of EsoGuard’s precision next generation sequencing (NGS) assay and EsoCheck’s groundbreaking, patent-protected Collect+Protect technology, which provides anatomically targeted and protected sampling, without the contamination and dilution inherent in SOS devices."

Dr. Aklog concluded, "The publication of the BETRNet study is the culmination of a period of rapid expansion of EsoGuard’s clinical evidence base. It represents the second peer-reviewed, and third overall, clinical validation study demonstrating consistently excellent EsoGuard performance. Soon to be released data from a fourth such clinical validation study tells the same story. These powerful clinical validity data are supplemented by three previously published clinical utility studies demonstrating near-perfect concordance between EsoGuard results and appropriate physician referral behavior, avoiding invasive endoscopy in a large majority of patients. Although the pipeline of future EsoGuard and EsoCheck clinical studies is robust, we believe we have reached a critical threshold and now have a complete package of clinical validity and utility data to accelerate our efforts to garner widespread EsoGuard payor coverage. In particular, this peer-reviewed publication allows us to proceed with our stated plan to seek Medicare coverage. We intend to review our data with MolDX at an upcoming pre-submission meeting, and then submit a formal technical assessment ("TA") seeking EsoGuard coverage under MolDX’s final and effective foundational local coverage determination on Molecular Testing for Detection of Upper Gastrointestinal Metaplasia, Dysplasia, and Neoplasia. We continue to remain optimistic that we have a line of sight to Medicare coverage."

In summary, the BETRNet study (ClinicalTrials.gov NCT00288119) included 243 evaluable patients enrolled prospectively across six academic medical centers within the BETRNet consortium—155 control patients free of esophageal precancer (Barrett’s Esophagus or BE) or cancer, and 88 patients with conditions along the spectrum from early precancer (non-dysplastic BE) to late precancer (indeterminate for dysplasia, and low-grade or high-grade dysplastic BE) to cancer (adenocarcinoma). Study patients underwent both EsoCheck esophageal cell collection (average procedure time 2.5 minutes) and upper endoscopy (EGD) with biopsies obtained as clinically indicated. Overall patient tolerance and satisfaction with EsoCheck was excellent. EsoCheck samples were sent to LucidDx Labs, Inc. (Lake Forest, CA) for EsoGuard testing. EsoGuard performance was excellent when compared to EGD. EsoGuard detected 100% of the patients with cancer. Overall sensitivity and specificity were each 85%. Based on an expected BE prevalence of approximately 10% in a population with gastroesophageal reflux disease (GERD) and additional risk factors, the authors estimated EsoGuard’s negative predictive value (NPV) and positive predictive value (PPV) to be 98% and 39%, respectively. This performance is consistent with a robust early detection or screening test. Most importantly, sensitivity for short-segment precancer (SSBE), the primary target of esophageal precancer testing accounting for over half of future cancers, was 89%—unprecedented for the detection of early precancers of limited anatomic extent. The authors conclude that "EsoCheck/EsoGuard demonstrated high sensitivity and specificity in detecting BE and BE-related neoplasia [cancer]…These results enable the more wide-spread screening of at-risk patients for BE, as recommended in the most recent ACG Guideline and AGA Clinical Update."

On May 2, 2024 Amgen reported financial results for the first quarter 2024 (Press release, Amgen, MAY 2, 2024, View Source [SID1234642573]).

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"With many of our innovative products delivering strong growth and promising new medicines advancing through our pipeline, we are excited about delivering attractive long-term growth," said Robert A. Bradway, chairman and chief executive officer.

Key results include:
•For the first quarter, total revenues increased 22% to $7.4 billion in comparison to the first quarter of 2023. Product sales grew 22%, driven by 25% volume growth.
◦Ten products delivered at least double-digit volume growth in the first quarter, including Repatha (evolocumab), TEZSPIRE (tezepelumab-ekko), EVENITY (romosozumab-aqqg), BLINCYTO (blinatumomab), and TAVNEOS (avacopan).
◦U.S. volume grew 29% and ex-U.S. volume grew 17%.
◦Our performance included $914 million of sales from our Horizon Therapeutics (Horizon) acquisition, driven by several first-in-class, early-in-lifecycle medicines, including TEPEZZA (teprotumumab-trbw), KRYSTEXXA (pegloticase) and UPLIZNA (inebilizumab-cdon).
◦Excluding sales from Horizon, our product sales grew 6%, driven by volume growth of 9%.
•GAAP loss per share was $0.21 for the first quarter of 2024 compared with GAAP earnings per share (EPS) of $5.28 for the first quarter of 2023, driven by a mark-to-market loss on our BeiGene, Ltd. equity investment and higher operating expenses, including higher amortization expense from Horizon-acquired assets and incremental expenses from Horizon, partially offset by higher revenues.
◦GAAP operating income decreased from $1.9 billion to $1.0 billion, and GAAP operating margin decreased 19.0 percentage points to 13.9%.
•Non-GAAP EPS decreased 1% from $3.98 to $3.96, due to higher operating and interest expenses driven by the Horizon acquisition, partially offset by higher revenues.
◦Non-GAAP operating income increased from $2.8 billion to $3.1 billion, and non-GAAP operating margin decreased 5.1 percentage points to 43.2%.
•The Company generated $0.5 billion of free cash flow for the first quarter of 2024 versus $0.7 billion in the first quarter of 2023. This decrease was driven by an $800 million tax deposit, partially offset by timing of working capital items.

On May 2, 2024 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, reported first quarter 2024 financial results and provided a corporate update (Press release, Kura Oncology, MAY 2, 2024, View Source [SID1234642591]).

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"Ziftomenib continues to demonstrate a best-in-class safety and efficacy profile as well as optimal pharmaceutical properties, which we believe will enable it to become a cornerstone of therapy in acute leukemias and beyond," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "This belief is backed by increasing investigator enthusiasm, as evidenced by rapid enrollment across our ongoing ziftomenib studies, and further supported by the FDA’s decision to grant Breakthrough Therapy Designation (BTD) to ziftomenib, making it the first investigational treatment to be granted BTD for the treatment of NPM1-mutant acute myeloid leukemia (AML). In the near term, we look forward to completing enrollment in our registration-directed trial of ziftomenib in NPM1-mutant AML and working closely with FDA to bring this potentially innovative medicine to patients in urgent need of effective treatments."

Recent Highlights

Breakthrough Therapy Designation for ziftomenib in NPM1-mutant AML – Last month, the U.S. Food and Drug Administration (FDA) granted BTD to ziftomenib for the treatment of relapsed/refractory (R/R) NPM1-mutant AML. FDA granted BTD based on data from Kura’s ongoing KOMET-001 trial of ziftomenib in patients with R/R NPM1-mutant AML. BTD is awarded for a drug that treats a serious or life-threatening condition and may demonstrate substantial improvement on one or more clinically significant endpoints over available therapies.

Registration-directed trial of ziftomenib in NPM1-mutant AML nearing completion – Kura remains on track to complete enrollment of 85 patients in its KOMET-001 registration-directed trial of ziftomenib in NPM1-mutant R/R AML by mid-2024. In the Phase 1 trial, ziftomenib demonstrated a 35% CR rate and 45% overall response rate in 20 heavily pretreated patients with NPM1-mutant AML treated at the recommended Phase 2 dose. NPM1-mutant AML accounts for approximately 30% of new AML cases annually and represents a disease of significant unmet need for which no approved targeted therapy exists.

Positive preliminary combination data for ziftomenib in NPM1-mutant and KMT2A-rearranged AML – In January 2024, Kura reported preliminary data from the KOMET-007 dose-escalation trial of ziftomenib in combination with venetoclax/azacitidine or cytarabine/daunorubicin (7+3) in patients with NPM1-mutant or KMT2A-rearranged AML. As of the data cutoff on January 11, 2024, all five newly diagnosed patients treated with ziftomenib and 7+3 achieved a complete remission (CR) with full count recovery, for a CR rate of 100%. The overall response rate among the 15 R/R patients treated with ziftomenib and venetoclax/azacitidine was 53%. Continuous daily dosing of ziftomenib at 200 mg was well tolerated. No differentiation syndrome events of any grade were reported, and no dose-limiting toxicities, evidence of QTc prolongation, drug-drug interactions or additive myelosuppression were observed. As of the data cutoff, 16 of the first 20 patients remained on trial, including all 11 NPM1-mutant patients.

Dose escalation continues in KOMET-007 combination trial of ziftomenib – To date, the 400 mg dose of ziftomenib has been cleared in three of the four cohorts in the KOMET-007 trial: 1) in combination with venetoclax/azacitidine in R/R NPM1-mutant AML, 2) in combination with venetoclax/azacitidine in R/R KMT2A-rearranged AML and 3) in combination with 7+3 in newly diagnosed adverse risk NPM1-mutant AML. Enrollment at the 600 mg dose is ongoing in all three cohorts. Enrollment continues at the 400 mg dose in combination with 7+3 in newly diagnosed adverse risk KMT2A-rearranged AML.

First patients dosed in KOMET-008 combination trial of ziftomenib – In February, Kura began dosing patients in its KOMET-008 trial of ziftomenib in combination with additional standards of care, including the FLT3 inhibitor gilteritinib, FLAG-IDA or LDAC, for the treatment of R/R NPM1-mutant or KMT2A-rearranged AML. Preclinical data for ziftomenib in combination with FLT3 inhibitors demonstrate strong synergistic effects compared to either single agent alone. Roughly half of patients with R/R NPM1-mutant AML have co-occurring FLT3 mutations, and the prognosis for these patients is poor.

First patients dosed with KO-2806 and cabozantinib in renal cell carcinoma – In March, Kura announced dosing of the first patient with KO-2806, the Company’s next-generation farnesyl transferase inhibitor (FTI), in combination with cabozantinib in clear cell renal cell carcinoma, just four months after dosing the first patients with KO-2806 as a monotherapy in the FIT-001 dose-escalation trial. The Company remains on track to dose the first patient in combination with adagrasib in KRASG12C-mutated non-small cell lung cancer by the middle of this year, as dose escalation of KO-2806 as a monotherapy continues in parallel.
Financial Results

Research and development expenses for the first quarter of 2024 were $36.3 million, compared to $25.2 million for the first quarter of 2023.

General and administrative expenses for the first quarter of 2024 were $18.2 million, compared to $11.4 million for the first quarter of 2023.

Net loss for the first quarter of 2024 was $49.5 million, compared to a net loss of $34.1 million for the first quarter of 2023. This included non-cash share-based compensation expense of $8.5 million, compared to $6.8 million for the same period in 2023.

As of March 31, 2024, Kura had cash, cash equivalents and short-term investments of $527 million, compared to $424 million as of December 31, 2023. This includes net proceeds of approximately $145.8 million from the Company’s private placement in January 2024.

Based on its operating plan, management expects that cash, cash equivalents and short-term investments will fund current operations into 2027.
Forecasted Milestones

Complete enrollment of 85 patients in the KOMET-001 registration-directed trial of ziftomenib in NPM1-mutant R/R AML by mid-2024.

Identify the recommended Phase 2 dose of ziftomenib in combination with venetoclax and azacitidine by mid-2024.

Identify the recommended Phase 2 dose of ziftomenib in combination with 7+3 by mid-2024.

Initiate Phase 1b expansion study of ziftomenib in combination with standards of care, including venetoclax/azacitidine in newly diagnosed NPM1-mutant or KMT2A-rearranged AML, in the second half of 2024.

Submit an investigational new drug application for ziftomenib in a solid tumor indication and present preclinical data at a medical meeting in the second half of 2024.

Dose the first patients with KO-2806 and adagrasib in KRASG12C-mutated non-small cell lung cancer by mid-2024.

Complete enrollment of two expansion cohorts in KURRENT-HN and identify the optimal biologically active dose of tipifarnib and alpelisib by the end of 2024.

Present data from the KURRENT-HN trial of tipifarnib in combination with alpelisib in PIK3CA-dependent head and neck squamous cell carcinoma (HNSCC) in the first half of 2025.
Conference Call and Webcast

Kura’s management will host a webcast and conference call at 4:30 p.m. ET / 1:30 p.m. PT today, May 2, 2024, to discuss the financial results for the first quarter 2024 and to provide a corporate update. The live call may be accessed by dialing (888) 886-7786 for domestic callers and (416) 764-8658 for international callers and entering the conference ID: 20226736. A live webcast and archive of the call will be available online from the investor relations section of the company website at www.kuraoncology.com.