On February 26, 2025 Geron Corporation (Nasdaq: GERN), a commercial-stage biopharmaceutical company aiming to change lives by changing the course of blood cancer, reported financial results for the fourth quarter and full year 2024 and recent business highlights (Press release, Geron, FEB 26, 2025, View Source [SID1234650627]).

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"2024 was a terrific year for Geron and for RYTELO, our first-in-class telomerase inhibitor, which we believe represents a highly differentiated treatment with blockbuster potential in the high unmet need, lower-risk MDS patient population. We also continued to progress our development efforts in relapsed/refractory myelofibrosis, which could potentially double our commercial opportunity if our IMpactMF Phase 3 trial reads out positively and we are approved in this indication. From a financial perspective, we ended the year with a strong cash position, and Q3 and Q4 revenues exceeded our expectations. Heading into 2025, we are excited by the strategic and leadership changes we put in place early in the launch, which we believe will position us to increase our revenue growth trajectory and more fully capture the significant commercial opportunity over the next several quarters," said John A. Scarlett, M.D., Geron’s Chairman and Chief Executive Officer.

Recent Business Highlights

Continued execution on U.S. commercial launch, with net product revenue for RYTELO (imetelstat) of $47.5 million in the fourth quarter of 2024 and $76.5 million since launch at the end of June 2024, following approval by the U.S. Food and Drug Administration (FDA).
Received positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) in December 2024 recommending approval of RYTELO for the treatment of certain adult patients with transfusion-dependent anemia due to lower-risk MDS. Subject to receiving regulatory approval, which is expected in the first half of 2025, Geron is preparing to commercialize RYTELO in select EU countries in 2026.
Achieved approximately 80% enrollment in the Phase 3 IMpactMF trial evaluating imetelstat in patients with relapsed/refractory myelofibrosis (R/R MF). Based on our current planning assumptions for enrollment and event (death) rates in the trial, we now expect the interim analysis for overall survival may occur in the second half of 2026 (when approximately 35% of planned enrolled patients have died) and the final analysis may occur in the second half of 2028 (when approximately 50% of planned enrolled patients have died).
Presented new data at the 66thAmerican Society for Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2024, including analyses of IMerge Phase 3 data suggesting clinical activity of imetelstat in patients with lower-risk MDS regardless of type or number of prior therapies and Phase 1 findings from IMproveMF suggesting tolerability of imetelstat in combination with ruxolitinib as a potential frontline therapy in patients with MF.
Fourth Quarter 2024 Financial Results

As of December 31, 2024, we had approximately $502.9 million in cash, cash equivalents, restricted cash and marketable securities.

Net Loss

For the three and twelve months ended December 31, 2024, the Company reported a net loss of $25.4 million, or $0.04 per share, and $174.6 million, or $0.27 per share, respectively, compared to $52.0 million, or $0.09 per share, and $184.1 million, or $0.32 per share, respectively, for the three and twelve months ended December 31, 2023.

Revenues

Total product revenue, net for the three and twelve months ended December 31, 2024, was $47.5 million and $76.5 million, respectively. There was no product revenue in the prior year periods, given that RYTELO was approved by the FDA in June 2024.

Total net revenue for the three and twelve months ended December 31, 2024, was $47.5 million and $77.0 million, respectively, compared to $23,000 and $237,000 for the same periods in 2023. Total net revenue includes license fees and royalties in addition to any product revenue, net. The increase in revenue is due to product revenue from U.S. sales of RYTELO, which was approved by the FDA in June 2024.

Operating Expenses

Total operating expenses for the three and twelve months ended December 31, 2024, were $67.6 million and $250.7 million, respectively, compared to $54.3 million and $194.2 million for the same periods in 2023.

Cost of goods sold was approximately $783,000 and $1.3 million for the three and twelve months ended December 31, 2024, respectively, which consisted of costs to manufacture and distribute RYTELO, compared to nil in the prior year periods.

Research and development expenses for the three and twelve months ended December 31, 2024, were $23.4 million and $103.7 million, respectively, and $32.9 million and $125.0 million, for the same periods in 2023. The decrease is primarily due to manufacturing and quality costs that were capitalized in the current period due to FDA approval of RYTELO, compared to being expensed in the prior period. The decrease is partially offset by an increase in labor costs due to higher headcount and incentive and stock-based compensation expense recognized due to the vesting of performance-based stock options upon FDA approval of RYTELO.

Selling, general and administrative expenses for the three and twelve months ended December 31, 2024, were $43.4 million, and $145.7 million, respectively, and $21.4 million and $69.1 million for the same periods in 2023. The increase in general and administrative expenses in 2024 as compared to 2023 primarily reflects higher personnel-related expenses related to increased headcount to support commercial launch of RYTELO in the U.S. and stock-based compensation expense recognized upon FDA approval of RYTELO due to the vesting of performance-based stock options.

Interest income was $5.2 million and $19.6 million for the three and twelve months ended December 31, 2024, respectively, compared to $4.6 million and $18.2 million for the same periods in 2023. The increase in interest income in 2024 compared to 2023 primarily reflects a larger marketable securities portfolio due to the receipt of net cash proceeds from the underwritten offering completed in March 2024, as well as higher yields from marketable securities purchases. Interest earned in future periods will depend on the size of our marketable securities portfolio and prevailing interest rates.

Interest expense was $8.7 million and $18.5 million for the three and twelve months ended December 31, 2024, respectively, compared to $2.3 million and $8.3 million or the same periods in 2023. The increase in interest expense primarily reflects $5.3 million in non-cash interest expense related to our agreement with Royalty Pharma and an increased principal debt balance under the Pharmakon loan agreement and the Hercules loan agreement which was repaid in the fourth quarter of 2024. Interest expense reflects interest owed under the loan agreements, interest expense recognized under the Royalty Pharma agreement, as well as amortization of associated debt issuance costs and debt discounts using the effective interest method and accrual for an end of term charge.

Loss on extinguishment of debt

We recorded a loss on the extinguishment of debt of $1.7 million for the twelve months ended December 31, 2024. The loss is related to the settlement of debt outstanding under the terminated Hercules loan agreement.

2025 Financial Guidance

For fiscal year 2025, we expect total operating expenses to be in the range of approximately $270 million to $285 million, which includes non-cash items such as stock-based compensation expense, amortization of debt discounts and issuance costs, and depreciation and amortization.

We expect to reach profitability without additional financing if our current internal sales and operating expense expectations are met. Based on our current operating plans and assumptions, we believe that our existing cash, cash equivalents, and marketable securities, together with anticipated net revenues from U.S. sales of RYTELO, will be sufficient to fund our projected operating requirements for the foreseeable future.

Conference Call

Geron will host a conference call at 8:00 a.m. ET on Wednesday, February 26, 2024, to discuss business updates and fourth quarter and full year 2024 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 RYTELO (imetelstat)

RYTELO (imetelstat) is an FDA-approved oligonucleotide telomerase inhibitor for the treatment of adult patients with low-to-intermediate-1 risk myelodysplastic syndromes (LR-MDS) with transfusion-dependent anemia requiring four or more red blood cell units over eight weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESAs). It is indicated to be administered as an intravenous infusion over two hours every four weeks.

A marketing authorization application for RYTELO is under review by the European Commission as a monotherapy treatment for adult patients with transfusion-dependent anemia due to very low, low or intermediate risk myelodysplastic syndromes (MDS) without an isolated deletion 5q cytogenetic (non-del 5q) abnormality and who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy.

RYTELO is a first-in-class treatment that works by inhibiting telomerase enzymatic activity. Telomeres are protective caps at the end of chromosomes that naturally shorten each time a cell divides. In LR-MDS, abnormal bone marrow cells often express the enzyme telomerase, which rebuilds those telomeres, allowing for uncontrolled cell division. Developed and exclusively owned by Geron, RYTELO is the first and only telomerase inhibitor approved by the U.S. Food and Drug Administration.

On February 26, 2025 CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, reported that members of its senior management team will present at the TD Cowen 45th Annual Health Care Conference on Monday, March 3, 2025, at 10:30 a.m. ET (Press release, CRISPR Therapeutics, FEB 26, 2025, View Source [SID1234650626]).

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A live webcast of the fireside chat will be available on the "Events & Presentations" page in the Investors section of the Company’s website at View Source A replay of the webcast will be archived on the Company’s website for 14 days following the presentation.

On February 26, 2025 Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of medications to treat severe endocrinologic, oncologic, metabolic and neurologic disorders by modulating the effects of the hormone cortisol, reported its results for the quarter and year ended December 31, 2024 (Press release, Corcept Therapeutics, FEB 26, 2025, https://ir.corcept.com/news-releases/news-release-details/corcept-therapeutics-announces-fourth-quarter-and-full-year-2024 [SID1234650625]).

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Financial Results

"Once again, we had a record number of new Korlym prescribers and a record number of patients receiving Korlym in the quarter. Physicians are increasingly aware of hypercortisolism’s true prevalence and the poor health outcomes for patients who go untreated. Screening is becoming more common and the number of patients receiving appropriate care continues to increase. We are confident that our Cushing’s syndrome business will grow for many years," said Joseph K. Belanoff, M.D., Corcept’s Chief Executive Officer.

Corcept’s fourth quarter 2024 revenue was $181.9 million, compared to $135.4 million in the fourth quarter of 2023. Revenue for the full year was $675.0 million, compared to $482.4 million in 2023.

Net income was $30.7 million in the fourth quarter, or $0.26 diluted net income per common share, compared to net income of $31.4 million, or $0.28 diluted net income per common share, in the fourth quarter of 2023. Net income was $141.2 million for the full year, or $1.23 diluted net income per common share, compared to net income of $106.1 million, or $0.94 diluted net income per common share, in 2023.

Cash and investments were $603.2 million at December 31, 2024 compared to $425.4 million at December 31, 2023. In 2024, Corcept paid $38.0 million to purchase its common stock pursuant to the company’s stock repurchase program, net exercise of employee stock options and net vesting of restricted stock grants.

Clinical Development

"We made substantial progress in all of our clinical development programs in 2024," added Dr. Belanoff. "We submitted a New Drug Application (NDA) for our proprietary, selective cortisol modulator, relacorilant, as a treatment for hypercortisolism, based on compelling results from our GRACE, GRADIENT, long-term extension and Phase 2 studies. Our CATALYST study demonstrated that hypercortisolism is much more common than previously assumed in patients with difficult-to-control diabetes and that treatment with a cortisol modulator can significantly improve their glucose control. We expect data from ROSELLA, our pivotal study in women with platinum-resistant ovarian cancer, this quarter."

Hypercortisolism (Cushing’s Syndrome)

Relacorilant for patients with hypercortisolism – NDA submitted in December 2024
GRACE – Pivotal Phase 3 trial of relacorilant in 152 patients with all etiologies of hypercortisolism – primary endpoint achieved in randomized withdrawal phase; open-label phase demonstrated clinically meaningful improvements in a broad range of hypercortisolism signs and symptoms; relacorilant was well-tolerated, consistent with its known safety profile, with no cases of endometrial hypertrophy or drug-induced vaginal bleeding, relacorilant-induced hypokalemia, adrenal insufficiency or QT prolongation
GRADIENT – Supportive data for NDA – Patients treated with relacorilant exhibited clinically meaningful improvements in a broad range of hypercortisolism signs and symptoms in randomized, double-blind, placebo-controlled, Phase 3 trial in 137 patients with hypercortisolism caused by adrenal gland pathology; relacorilant was well-tolerated, consistent with its known safety profile, including no cases of endometrial hypertrophy or drug-induced vaginal bleeding, relacorilant-induced hypokalemia, adrenal insufficiency or QT prolongation
Phase 3 long-term extension study – Supportive data for NDA – clinically meaningful and durable cardiometabolic improvements exhibited in 116 patients who completed the GRACE, GRADIENT or Phase 2 relacorilant studies, with no new or notable safety signals observed; treatment duration of up to six years
CATALYST Part 1 – Of 1,057 patients with difficult-to-control type 2 diabetes, 23.8 percent were found to have hypercortisolism
CATALYST Part 2 – Primary endpoint met in randomized, double-blind, placebo-controlled study of 136 patients identified with hypercortisolism in CATALYST Part 1; patients who received Korlym exhibited a clinically meaningful and statistically significant improvement in hemoglobin A1c, with a decrease from baseline of 1.47 percent compared to a decrease of 0.15 percent in patients who received placebo (p-value: < 0.0001); safety profile of Korlym in this study was consistent with the medication’s label; no new adverse events were observed
MOMENTUM – 1,000-patient trial examining the prevalence of hypercortisolism in patients with resistant hypertension to begin this quarter
"The positive results from our pivotal GRACE study, and confirmatory evidence from our GRADIENT, long-term extension and Phase 2 studies, provide powerful support for relacorilant’s NDA in hypercortisolism. Patients in these studies experienced clinically significant improvements in a wide array of hypercortisolism’s signs and symptoms, without the off-target effects and toxicities that accompany currently available treatments. Relacorilant’s strong efficacy and safety profile positions it to become the new standard of care for patients with hypercortisolism," said Bill Guyer, PharmD, Corcept’s Chief Development Officer.

"CATALYST is a landmark study that will change the way physicians treat some of their sickest patients. Its findings are striking: One-in-four patients whose type 2 diabetes resists treatment with the best available medications have hypercortisolism and hyperglycemia in these patients responds powerfully to treatment with a cortisol modulator," added Dr. Guyer. "We plan to build on these findings. Our MOMENTUM study will establish the prevalence of hypercortisolism in patients with resistant hypertension."

Oncology

ROSELLA – Results expected this quarter from pivotal Phase 3 trial of relacorilant plus nab-paclitaxel in 381 patients with platinum-resistant ovarian cancer
Early-stage prostate cancer – Enrollment continues in randomized, placebo-controlled, Phase 2 trial of relacorilant plus enzalutamide in patients with early-stage prostate cancer, conducted in collaboration with the University of Chicago
"If ROSELLA replicates the positive results of our large, controlled, Phase 2 study, it will constitute a major medical advance and serve as the basis for relacorilant’s next NDA. We expect progression-free survival results this quarter," said Dr. Guyer. "Relacorilant has the potential to become the standard of care for patients with platinum-resistant ovarian cancer."

Amyotrophic Lateral Sclerosis (ALS)

DAZALS – In a randomized, double-blind, placebo-controlled Phase 2 study in 249 patients with ALS, dazucorilant did not meet its primary endpoint of improvement in the ALS Functional Rating Scale-Revised (ALSFRS-R); a statistically significant improvement in overall survival at week 24 was observed; an open-label, long-term extension study is ongoing and one-year overall survival results are expected in the second quarter
"ALS is a devastating disease, with few good treatment options. In DAZALS, patients who received dazucorilant did not show improvement in the ALS Functional Rating Scale-Revised (ALSFRS-R), which was the study’s primary endpoint. An improvement in overall survival was observed at week 24 of the study – no deaths (0 of 83 patients) were observed in the 300 mg dazucorilant arm, compared to 5 deaths (5 of 82 patients) in the placebo group (p-value: 0.02). The open-label, long-term extension study is ongoing and we expect one-year overall survival results in the second quarter," said Dr. Guyer.

Metabolic Dysfunction-Associated Steatohepatitis (MASH)

MONARCH – Enrollment continues in randomized, double-blind, placebo-controlled, Phase 2b trial of miricorilant in 120 patients with biopsy-confirmed MASH and in 75 patients with presumed MASH
"In our Phase 1b study, miricorilant reduced liver fat very rapidly, improved liver health and key metabolic and lipid measures and was well-tolerated. We look forward to building on these promising results in our MONARCH study," said Dr. Guyer.

Conference Call

We will hold a conference call on February 26, 2025, at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time). Participants must register in advance of the conference call by clicking here. Upon registering, each participant will receive a dial-in number and a unique access PIN. Each access PIN will accommodate one caller. A listen-only webcast will be available by clicking here. A replay of the call will be available on the Investors / Events tab of Corcept.com.

On February 26, 2025 Boston Scientific Corporation (the "Company") (NYSE: BSX) reported that American Medical Systems Europe B.V. ("AMS Europe"), its wholly owned finance subsidiary, has completed a public offering of €850,000,000 aggregate principal amount of 3.000% notes due 2031 and €650,000,000 aggregate principal amount of 3.250% notes due 2034 (collectively, the "Notes") (Press release, Boston Scientific, FEB 26, 2025, View Source [SID1234650623]). The Notes are fully and unconditionally guaranteed by the Company. Application has been made for the Notes to be admitted to the Official List of the Irish Stock Exchange plc trading as Euronext Dublin and to trading on the Global Exchange Market thereof.

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The Company intends to use the net proceeds from the offering, together with cash on hand, to fund the repayment at maturity of AMS Europe’s 0.750% senior notes due March 8, 2025 and to pay accrued and unpaid interest with respect to such notes, and for general corporate purposes, which may include, among other things, short term investments, reduction of short term debt, funding of working capital and potential future acquisitions.

On February 26, 2025 Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, reported has it has dosed its final patient in the third cohort of its ongoing Phase 1 clinical trial evaluating its novel chimeric antigen receptor-T cell (CAR-T) therapy for recurrent ovarian cancer (Press release, Anixa Biosciences, FEB 26, 2025, View Source [SID1234650622]). The study is being conducted through a research partnership with Moffitt Cancer Center ("Moffitt"). The fourth cohort is expected to commence after a 30-day verification that there continue to be no adverse effects experienced by the third cohort.

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The third cohort in the trial received a dose of one million CAR-positive cells per kilogram of patient weight, which represents a tenfold increase compared to the first cohort. This escalation is an important step in evaluating the safety and efficacy of CAR-T therapy for ovarian cancer patients. The fourth cohort will increase the dosage by another factor of three.

Anixa’s FSHR-mediated CAR-T technology targets the follicle-stimulating hormone receptor (FSHR), which research indicates is exclusively expressed on ovarian cells, tumor vasculature, and certain cancer cells. The first-in-human trial (NCT05316129) is enrolling adult women with recurrent ovarian cancer who have progressed after at least two prior therapies. The study is designed to evaluate safety, identify the maximum tolerated dose, and monitor efficacy.

Dr. Amit Kumar, Chairman and CEO of Anixa, stated, "With the completion of the third cohort, we are gaining important insights into the potential of CAR-T therapy for ovarian cancer. Increasing the dose is a key step in evaluating the therapy’s effectiveness while ensuring patient safety. Additionally, the recent approval of our protocol amendment allows eligible patients to receive a second dose if they may benefit from it. This amendment also expands enrollment criteria to include rare ovarian cancer subtypes, making the therapy accessible to more patients. Now, all eligible participants can receive a second dose without requiring separate IND approvals, helping us better assess the full potential of this treatment. As we move forward with the fourth cohort, we remain committed to advancing our CAR-T program with the goal of improving treatment options for ovarian cancer patients."