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.

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 Illumina, Inc. (Nasdaq: ILMN) ("Illumina" or the "company") reported its financial results for the first quarter of fiscal year 2024, which include the consolidated financial results for GRAIL (Press release, Illumina, MAY 2, 2024, View Source [SID1234642587]).

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"The Illumina team delivered results ahead of our expectations by supporting our customers with innovative solutions that enable the next wave of progress in genomics and multiomics," said Jacob Thaysen, Chief Executive Officer. "Our customers are leveraging the NovaSeq X to drive increased sequencing activity, even in a persistently challenging global macroeconomic environment. Additionally, our team’s strong execution and continued focus on operational excellence enabled us to deliver year-over-year improvement in both gross margin and non-GAAP operating margin."

First quarter consolidated results
GAAP Non-GAAP (a)
Dollars in millions, except per share amounts
Q1 2024
Q1 2023
Q1 2024
Q1 2023
Revenue $ 1,076 $ 1,087 $ 1,076 $ 1,087
Gross margin 62.0 % 60.3 % 66.5 % 64.7 %
Research and development ("R&D") expense $ 339 $ 341 $ 335 $ 339
Selling, general and administrative ("SG&A") expense $ 439 $ 378 $ 349 $ 343
Operating (loss) profit $ (111) $ (64) $ 33 $ 21
Operating margin (10.3) % (5.7) % 3.1 % 1.9 %
Tax provision (benefit) $ 17 $ (81) $ 13 $ 5
Tax rate (15.3) % 103.9 % 46.4 % 27.3 %
Net (loss) income $ (126) $ 3 $ 14 $ 13
Diluted (loss) earnings per share $ (0.79) $ 0.02 $ 0.09 $ 0.08

(a) See the tables included in the "Results of Operations – Non-GAAP" section below for reconciliations of these GAAP and non-GAAP financial measures.
1

Capital expenditures for free cash flow purposes were $36 million for Q1 2024. Cash flow provided by operations was $77 million, compared to cash flow provided by operations of $10 million in the prior year period. Free cash flow (cash flow provided by operations less capital expenditures) was $41 million for the quarter, compared to $(42) million in the prior year period. Depreciation and amortization expenses were $108 million for Q1 2024. At the close of the quarter, the company held $1,115 million in cash, cash equivalents and short-term investments.

First quarter segment results
Illumina has two reportable segments, Core Illumina and GRAIL.

Core Illumina
GAAP Non-GAAP (a)
Dollars in millions Q1 2024 Q1 2023 Q1 2024 Q1 2023
Revenue (b)
$ 1,056 $ 1,076 $ 1,056 $ 1,076
Gross margin (c)
65.7 % 63.8 % 67.1 % 65.2 %
R&D expense $ 241 $ 259 $ 237 $ 257
SG&A expense $ 336 $ 286 $ 254 $ 257
Operating profit
$ 116 $ 142 $ 218 $ 187
Operating margin 11.0 % 13.2 % 20.6 % 17.4 %

(a) See Table 3 included in the "Results of Operations – Non-GAAP" section below for reconciliations of these GAAP and non-GAAP financial measures.
(b) Core Illumina revenue for Q1 2024 was down 2% as compared to Q1 2023 and down 2% on a constant currency basis. Amounts for Q1 2024 and Q1 2023 included intercompany revenue of $7 million and $9 million, respectively, which is eliminated in consolidation.
(c) The year-over-year increase in gross margin was primarily driven by a more favorable mix of sequencing consumables and execution of our operational excellence priorities that delivered cost savings, including freight and improved productivity. This was partially offset by certain strategic partnership revenue that is lower margin and increased warranty and field service costs.

GRAIL
GAAP Non-GAAP (a)
In millions Q1 2024 Q1 2023 Q1 2024 Q1 2023
Revenue $ 27 $ 20 $ 27 $ 20
Gross (loss) profit $ (22) $ (25) $ 12 $ 9
R&D expense $ 101 $ 86 $ 101 $ 86
SG&A expense $ 104 $ 93 $ 96 $ 87
Operating loss $ (227) $ (204) $ (185) $ (164)

(a) See Table 3 included in the "Results of Operations – Non-GAAP" section below for reconciliations of these GAAP and non-GAAP financial measures.

Key announcements by Illumina since Illumina’s last earnings release
•Released XLEAP-SBS chemistry on NextSeq 1000 and NextSeq 2000 Systems, our fastest, highest quality, and most robust sequencing by synthesis (SBS) chemistry to date
•Launched Illumina Complete Long Reads (ICLR) with Enrichment, a flexible, cost-effective solution to enhance coverage of known challenging-to-map regions with targeted long reads where they provide the most value
•A recent literature review, published in the Nature journal Genomic Medicine, showed that short-read genomic sequencing (GS) reduces the time it takes to diagnose and treat pediatric patients who may have a rare genetic condition
•Appointed Ankur Dhingra as Chief Financial Officer and Jakob Wedel as Chief Strategy and Corporate Development Officer
•Appointed Jenny Zheng as Head of Region, Greater China
2

A full list of recent Illumina announcements can be found in the company’s News Center.

Key announcements by GRAIL since Illumina’s last earnings release
•Presented new data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting that:
•Demonstrated the first real-world evaluation of repeat multi-cancer early detection (MCED) / Galleri testing showing the potential value of adding repeat MCED screening
•Exhibited 4-year overall survival follow-up demonstrating the prognostic significance of detecting cancer with a methylation-based cfDNA platform like Galleri
•Illustrated data demonstrating the power of Galleri to preferentially detect high grade, clinically significant prostate cancer over indolent cases.
•Announced a collaboration with AstraZeneca in which participants from Japan will have their samples tested using GRAIL’s novel risk classification test on its Methylation Platform

A full list of recent GRAIL announcements can be found in GRAIL’s Newsroom.

Financial outlook and guidance
For fiscal year 2024, the company continues to expect Core Illumina revenue to be approximately flat compared to fiscal year 2023 and Core Illumina non-GAAP operating margin to be approximately 20%. While Illumina continues to move as quickly as possible to resolve GRAIL, the company is focusing its financial outlook on Core Illumina, as the specific timing and impact of the GRAIL divestment remains uncertain.

The company provides forward-looking guidance on a non-GAAP basis. The company is unable to provide a reconciliation of forward-looking non-GAAP financial measures to the most directly comparable GAAP reported financial measures because it is unable to predict with reasonable certainty the financial impact of items such as acquisition-related expenses, gains and losses from our strategic investments, fair value adjustments related to contingent consideration and contingent value rights, potential future asset impairments, restructuring activities, and the ultimate outcome of pending litigation without unreasonable effort. These items are uncertain, inherently difficult to predict, depend on various factors, and could have a material impact on GAAP reported results for the guidance period. For the same reasons, the company is unable to address the significance of the unavailable information, which could be material to future results.

Conference call information
The conference call will begin at 2 p.m. Pacific Time (5 p.m. Eastern Time) on Thursday, May 2, 2024. Interested parties may access the live teleconference through the Investor Info section of Illumina’s website at investor.illumina.com. Alternatively, individuals can access the call by dialing 866.400.0049 or +1.323.701.0225 outside North America, both using conference ID 9170634. To ensure timely connection, please dial in at least ten minutes before the scheduled start of the call.

A replay of the conference call will be posted on Illumina’s website after the event and will be available for at least 30 days following.

On May 2, 2024 Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company developing investigational first-in-class telomerase inhibitor, imetelstat, to treat hematologic malignancies, reported financial results and business highlights for the first quarter 2024 (Press release, Geron, MAY 2, 2024, View Source [SID1234642586]).

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"Since the FDA ODAC’s 12 to 2 vote in favor of the clinical benefit/risk profile of imetelstat for the treatment of transfusion-dependent anemia in patients with lower-risk MDS in March, we have continued working with the FDA as they complete their review of our New Drug Application, which has a June 16, 2024 PDUFA target action date," said John A. Scarlett, M.D., Chairman and Chief Executive Officer. "We are actively preparing for a successful launch of imetelstat in the U.S., if approved, including most recently onboarding our sales force last month, refining our market research and completing buildout of our enterprise capabilities and systems to support our transition from a clinical to commercial-stage company."

U.S. Commercial Preparation

Geron has now completed onboarding its commercial team, with the buildout of the full sales organization in April. Other commercial preparations for the U.S. are ongoing and on target, including enhancing and/or establishing company processes and systems to support an expected commercial launch, refining market research in TD LR-MDS, and engaging in marketing, commercial access, payer, and reimbursement preparatory efforts.

Clinical Development Update

The Phase 3 IMpactMF clinical trial, which has a primary endpoint of overall survival, is ongoing in myelofibrosis patients who are relapsed/refractory to JAK inhibitors. Last month, the data monitoring committee evaluated unblinded data and recommended the clinical trial continue. In addition, the Company reviewed enrollment rates and blinded death rates, which are lower than anticipated based on initial planning assumptions. Accordingly, guidance is being updated to extend the timelines by half a year, with the interim analysis now expected in early 2026 and the final analysis expected in early 2027.

First Quarter 2024 Financial Results

As of March 31, 2024, the Company had approximately $465 million in cash and marketable securities, including proceeds from an underwritten public offering of common stock and a pre-funded warrant in March 2024 for net proceeds of approximately $141 million.

For the first quarter of 2024, the Company reported a net loss of $55.4 million, or $0.09 per share, compared to $38.1 million, or $0.07 per share, for the first quarter of 2023.

Revenues for the first quarter of 2024 were $304,000, compared to $21,000 for the same period in 2023. Royalty revenues in 2024 and 2023 primarily reflect estimated royalties from sales of cell-based research products from the Company’s divested stem cell assets.

Total operating expenses for the first quarter of 2024 were $56.4 million, compared to $40.1 million for the same period in 2023. Research and development expenses for the first quarter of 2024 were $29.4 million, compared to $27.2 million for the same period in 2023. The increase in research and development expenses for the three months ended March 31, 2024, compared to the same period in 2023, primarily reflects the net result of higher manufacturing costs due to the timing of imetelstat manufacturing batches and increased personnel-related expenses for additional headcount. We expect research and development expenses to remain consistent in the future as we support IMpactMF, IMproveMF and IMpress, as well as the long-term treatment and follow-up of remaining patients in IMerge. General and administrative expenses for the first quarter of 2024 were $27.1 million, compared to $12.9 million for the same period in 2023. The increase in general and administrative expenses for the three months ended March 31, 2024, compared to the same period in 2023, primarily reflects new costs for commercial preparatory activities and higher personnel-related expenses for additional headcount.

Interest income for the first quarter of 2024 was $4.2 million, compared to $3.9 million for the same period in 2023. The increase in interest income for the three months ended March 31, 2024, compared to the same period in 2023, primarily reflects a larger marketable securities portfolio with the receipt of net cash proceeds from the underwritten offering completed in March 2024, as well as higher yields from recent marketable securities purchases.

Interest expense for the first quarter of 2024 was $3.4 million, compared to $1.9 million for the same period in 2023. The increase in interest expense for the three months ended March 31, 2024, compared to the same period in 2023, primarily reflects higher interest rates. Currently, we have $80.0 million in principal debt outstanding. Interest expense reflects interest owed under our loan facility.

2024 Financial Guidance

For fiscal year 2024, the Company expects total operating expenses to be in the range of approximately $270 million to $280 million, which includes non-cash items such as stock-based compensation expense, amortization of debt discounts and issuance costs, and depreciation and amortization.

The fiscal year 2024 financial guidance reflects costs to support regulatory processes with the FDA and EMA in 2024; continued support of ongoing clinical trials; manufacturing of commercial inventory of imetelstat; build out of our commercial organization to support the potential U.S. commercial launch of imetelstat in the U.S.; increases in headcount in preparation for transition to a commercial-stage company; and interest payments on outstanding debt.

Based on our current operating plans and our assumptions regarding the timing of the potential approval and commercial launch of imetelstat in lower risk MDS in the U.S., we believe that our existing cash, cash equivalents, and current and noncurrent marketable securities, together with projected revenues from U.S. sales of imetelstat, if approved, potential proceeds from the exercise of our outstanding warrants, and potential future drawdowns under our loan facility, will be sufficient to fund our projected operating requirements into the second quarter of 2026.

As of March 31, 2024, we had 162 full-time employees, prior to the onboarding of our salesforce in April 2024. Subject to approval of imetelstat in the U.S., the Company plans to grow to a total of approximately 250-300 employees by year-end 2024.

Conference Call

Geron will host a conference call at 8:00 a.m. ET on Thursday, May 2, 2024, to discuss business updates and first quarter financial results.

A live webcast of the conference call and related presentation will be available on the Company’s website at www.geron.com/investors/events. An archive of the webcast will be available on the Company’s website for 30 days.

Participants may access the webcast by registering online using the following link, View Source

About Imetelstat

Imetelstat is a novel, first-in-class investigational telomerase inhibitor exclusively owned by Geron and being developed by Geron in hematologic malignancies. Data from non-clinical studies and clinical trials of imetelstat provide strong evidence that imetelstat targets telomerase to inhibit the uncontrolled proliferation of malignant stem and progenitor cells in myeloid hematologic malignancies, resulting in malignant cell apoptosis. Imetelstat has been granted Fast Track designation by the U.S. Food and Drug Administration for both the treatment of adult patients with transfusion-dependent anemia due to Low or Intermediate-1 risk MDS that is not associated with del(5q) who are refractory or resistant to an erythropoiesis stimulating agent, and for adult patients with Intermediate-2 or High-risk myelofibrosis (MF) whose disease has relapsed after or is refractory to Janus kinase (JAK) inhibitor treatment. Imetelstat is currently not approved by any regulatory authority.

About IMpactMF

IMpactMF is an open label, randomized, controlled Phase 3 clinical trial with registrational intent. The trial is designed to enroll approximately 320 patients with Intermediate-2 or High-risk myelofibrosis (MF) who are refractory to prior treatment with a JAK inhibitor, also referred to as refractory MF. Patients will be randomized to receive either imetelstat or best available therapy. The primary endpoint is overall survival (OS). Key secondary endpoints include symptom response, spleen response, progression free survival, complete remission, partial remission, clinical improvement, duration of response, safety, pharmacokinetics, and patient reported outcomes.

IMpactMF is currently enrolling patients. For further information about IMpactMF, including enrollment criteria, locations, and current status, visit ClinicalTrials.gov/NCT04576156.

On May 2, 2024 Genmab reported Interim Report for the First Quarter Ended March 31, 2024 (Press release, Genmab, MAY 2, 2024, View Source [SID1234642585]).

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Highlights

The U.S. Food and Drug Administration (U.S. FDA) granted Priority Review for the supplemental Biologics License Application (sBLA) for EPKINLY (epcoritamab-bysp) for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy, with a Prescription Drug User Fee Act (PDUFA) target action date of June 28, 2024
An additional Phase 3 clinical trial was initiated, evaluating epcoritamab in combination with rituximab and lenalidomide compared to chemoimmunotherapy in previously untreated follicular lymphoma
The U.S. FDA accepted for Priority Review the sBLA seeking to convert the accelerated approval of Tivdak (tisotumab vedotin-tftv) to full approval, for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after first-line therapy
Genmab announced the decision of its arbitration appeal under its daratumumab license agreement with Janssen Biotech, Inc. (Janssen)
Genmab revenue increased 46% compared to the first quarter of 2023, to DKK 4,143 million

"The acceptance for Priority Review by the U.S. FDA of the sBLAs for EPKINLY and Tivdak that we received in the first quarter are important events that support our commitment to continue to deliver innovative treatment options that have the potential to profoundly impact the lives of patients. These regulatory acceptances for priority review also reflect our dedication to working with our partners, AbbVie Inc. (AbbVie) and Pfizer Inc. (Pfizer) to expand the labels for EPKINLY and Tivdak, respectively, in order to maximize the potential of both medicines and bring them to as many patients in need of alternative therapeutic options as possible," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

Financial Performance First Quarter of 2024

Revenue was DKK 4,143 million for the first three months of 2024 compared to DKK 2,834 million for the first three months of 2023. The increase of DKK 1,309 million, or 46%, was primarily driven by higher DARZALEX and Kesimpta royalties achieved under our collaborations with Janssen and Novartis Pharma AG (Novartis), respectively, EPKINLY net product sales, and a milestone achieved under our collaboration with AbbVie.
Royalty revenue was DKK 3,104 million in the first three months of 2024 compared to DKK 2,408 million in the first three months of 2023, an increase of DKK 696 million, or 29%. The increase in royalties was driven by higher net sales of DARZALEX and Kesimpta.
Net sales of DARZALEX (daratumumab), including sales of the subcutaneous (SC) product (daratumumab and hyaluronidase-fihj, sold under the tradename DARZALEX FASPRO in the U.S.) by Janssen were USD 2,692 million in the first three months of 2024 compared to USD 2,264 million in the first three months of 2023, an increase of USD 428 million or 19%.
Total costs and operating expenses were DKK 3,342 million in the first three months of 2024 compared to DKK 2,417 million in the first three months of 2023. The increase of DKK 925 million, or 38%, was driven by the expansion of our product pipeline, EPKINLY post launch activities in the U.S. and Japan, the continued development of Genmab’s broader organizational capabilities and related increase in team members to support these activities, as well as profit-sharing amounts payable to AbbVie related to EPKINLY sales.
Operating profit was DKK 801 million in the first three months of 2024 compared to DKK 417 million in the first three months of 2023.
Net financial items resulted in income of DKK 915 million for the first three months of 2024 compared to an expense of DKK 151 million in the first three months of 2023. The increase of DKK 1,066 million was primarily driven by movements in USD to DKK foreign exchange rates impacting Genmab’s USD denominated cash and cash equivalents and marketable securities, with strengthening of the USD/DKK rate in the first three months of 2024 compared to the weakening of the USD/DKK rate in the first three months of 2023.
Subsequent Event

April: Genmab and ProfoundBio, Inc. (ProfoundBio) announced that the companies have entered into a definitive agreement for Genmab to acquire ProfoundBio in an all-cash transaction. The acquisition will give Genmab worldwide rights to three candidates in clinical development, including rinatabart sesutecan (Rina-S), plus ProfoundBio’s novel antibody-drug conjugate (ADC) technology platforms. Genmab will acquire ProfoundBio for USD 1.8 billion in cash, payable at closing (subject to adjustment for ProfoundBio’s closing net debt and transaction expenses). The proposed transaction is expected to close in the first half of 2024. The closing of the proposed transaction is subject to the satisfaction of customary closing conditions.

Outlook
Genmab is maintaining its 2024 financial guidance published on February 14, 2024.

Following the announcement of the proposed acquisition of ProfoundBio, Genmab’s operating expenses before expenses incurred by it in connection with the proposed transaction are now anticipated to be at or moderately above the upper end of the previously disclosed guidance range of DKK 12.4 -13.4 billion. The anticipated increase reflects the incremental R&D investment to support the advancement of ProfoundBio’s clinical programs, primarily Rina-S. Genmab’s revenue guidance is unchanged and expected to be in the previously disclosed guidance range of DKK 18.7 – 20.5 billion.

We expect to update our guidance no later than in connection with our second quarter 2024 earnings.

Conference Call
Genmab will hold a conference call to discuss the results for the first quarter of 2024 today, Thursday, May 2, at 6:00 pm CEST, 5:00 pm BST or 12:00 pm EDT. To join the call please use the below registration link. Registered participants will receive an email with a link to access dial-in information as well as a unique personal PIN: https://register.vevent.com/register/BIcaf0da755ddb4d7f81f4dd2c0229135e. A live and archived webcast of the call and relevant slides will be available at www.genmab.com/investors.