On May 5, 2025 ImmunityBio, Inc. (NASDAQ: IBRX), a leading immunotherapy company, reported that the Company received a Refusal to File (RTF) letter from the U.S. Food and Drug Administration (FDA) for the supplemental biologics license application (sBLA) for use of ANKTIVA plus Bacillus Calmette-Guerin (BCG) in BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) for the indication of papillary disease (Press release, ImmunityBio, MAY 5, 2025, View Source [SID1234652509]). This RTF letter was received despite reaching unanimous guidance and encouragement at the in-person January 2025 meeting from the leadership of the Agency, including from CBER, CDER and OCE to submit this sBLA. At this meeting all key decision makers were specifically asked and unanimously confirmed that ImmunityBio should submit the sBLA as soon as possible based on the data in the single-arm trial. Relying on this unanimous guidance the Company submitted the sBLA in March 2025. The Company has already requested an urgent meeting to resolve the inconsistencies between the directives provided at the January Meeting and receipt of the RTF letter.

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ANKTIVA was approved by the FDA in 2024 with BCG for the treatment of BCG unresponsive NMIBC with Papillary tumors with CIS (Cohort A). In the same clinical trial (QUILT-3.032) long term results of patients with Papillary tumors without CIS (Cohort B), was submitted as a sBLA. The RTF letter referenced herein does not impact this prior approval of CIS +/- Papillary in BCG unresponsive patients.

The Agency leaders present at the January meeting unambiguously invited the Company to expeditiously submit an sBLA for the NMIBC papillary indication based on the strength of the clinical response and long-term duration of follow-up data of the Papillary without CIS cohort of the QUILT 3.032 study. Data was also presented of the long term follow-up Phase 1 results which demonstrated Complete Response and Disease Free Survival in both CIS and Papillary disease, with patients in on-going cystectomy free state at 10 years. In addition disease-specific overall survival rate of 99% at 12 months and 96% at 36 months, in the BCG unresponsive patients with Papillary disease without CIS was discussed. To our knowledge, this is the most durable data to date in the Papillary setting for bladder preservation.

In March 2025, the Company completed its submission to the FDA of the sBLA for the use of ANKTIVA plus BCG in BCG-unresponsive NMIBC in the papillary indication. On May 2, 2025, and notwithstanding the FDA’s invitation to submit the sBLA articulated by its leadership at the January Meeting, the FDA delivered an RTF letter. The Company together with its consultants who attended the January 2025 meeting were shocked by this inconsistent response and have requested an urgent meeting with the Agency to resolve this issue.

"Our commitment to NMIBC patients in the papillary indication and our belief in ANKTIVA’s potential based on the strength of the clinical response and long duration of 5-year follow-up remains unchanged, despite our receipt of a refusal to file letter regarding our supplemental BLA," said Dr. Patrick Soon-Shiong, the Company’s Founder, Executive Chairman and Global Chief Scientific and Medical Officer. "We are fully determined to work with the FDA as quickly as possible, including having already requested a Type A meeting, to explore the best path forward. We would also welcome an FDA Advisory Committee meeting as part of the regulatory process going forward. We presented our data at the recent 2025 American Urological Association (AUA) meeting and ANKTIVA+BCG was considered best in class and best in disease by the thought leaders in attendance, when compared to all the therapies currently approved or in development. Patients with BCG Unresponsive Papillary disease, face a life changing and life-threatening prospect of a total radical cystectomy, as well as the danger of the disease progressing from non-muscle invasive to muscle invasion with consequent progression and mortality. With the data presented of 99% disease specific survival at 12 months and over 82% patients not requiring bladder removal, it is essential that these patients be provided this treatment option, especially with the safety profile of ANKTIVA+BCG and the already approved indication of Papillary disease with CIS. The Company presented these data to the Agency in person in January and the Agency leaders present unanimously encouraged the company to expeditiously submit a supplemental BLA for this indication."

Dr. Soon-Shiong further stated: "The Agency must explain the confounding inconsistency of approving ANKTIVA+BCG for patients with Papillary with CIS disease, while refusing to file the sBLA for patients with Papillary without CIS disease—even though both groups were part of the same trial, in the same high-risk population of BCG-unresponsive non-muscle invasive bladder cancer. In both cases, patients received the same surgical procedure for the Papillary component, the same therapy at the same dose, with the same excellent tolerability and meaningful clinical benefit: over 82% bladder-sparing and over 96% disease-specific survival at 36 months, as shown in Figure 1. On behalf of patients facing a potential loss of a vital organ and high risk of progression of disease, I urge the Agency to reconsider and act now."

"I actively participated in the January 2025 meeting at which the leadership of the Agency present at that meeting unanimously supported and encouraged ImmunityBio to submit results from the single arm trial, QUILT 3.032 as a supplemental BLA because of the high-risk of progression and metastasis these patients with BCG unresponsiveness face. The consensus was reached by all present, including me, because of the lack of satisfactory alternatives in this desperately ill population at high-risk of losing their bladder – a life threatening and life changing procedure. Thus, I was startled to learn the ImmunityBio had received a ‘Refuse to File’ letter which is in my opinion rife with regulatory inaccuracies. I recommend that the company seek a rapid meeting with the Agency to resolve this issue and minimize the delay and threat to well-being of patients who could benefit significantly from avoiding a cystectomy," said Dr. Rachel Sherman, former FDA Principal Deputy Commissioner, who pioneered single arm trials at the height of the HIV epidemic, and was responsible for developing the expedited pathways at the Agency to address life threatening and serious diseases on behalf of the American public during her 30 year career at the agency. "Furthermore, it is incomprehensible to me that the FDA refuses to file a supplemental BLA, stating the study is not sufficient to support a regulatory review, when it has already approved a product based on that very same study in essentially the same indication and population. As stated above, these patients are suffering from a disease with a high risk of morbidity and mortality in the very short term – no delay should be tolerated," added Dr. Sherman.

About the QUILT-3.032 Study

QUILT-3.032 (NCT03022825) is a Phase II/III, open-label, single-arm, multicenter study of intravesical BCG plus ANKTIVA or ANKTIVA only in patients with BCG unresponsive high grade non-muscle invasive bladder cancer (NMIBC). All participants receive BCG plus ANKTIVA (Cohorts A and B) or ANKTIVA only (Cohort C) via a urinary catheter in the bladder for 6 consecutive weeks (initial induction treatment period). After the first disease assessment, eligible patients receive either a 3-week maintenance course or a 6-week re-induction course (second treatment period) at Month 3. Eligible patients will continue to receive maintenance treatment in the third treatment period at Months 6, 9, 12, and 18. Eligible patients have the option to receive maintenance treatment in the fourth treatment period at Months 24, 30, and 36. The study duration is 60 months.

Cohort A (N=100) includes patients with histologically confirmed BCG-unresponsive NMIBC CIS with or without papillary disease. The primary endpoint is biopsy-confirmed complete response (CR) rate at any time. Secondary endpoints include duration of CR, progression-free survival (PFS), time to cystectomy, safety and overall survival. FDA Approved this indication in 2024 in which eligibility included patients with Papillary disease.

Cohort B (N=80) enrolled participants with histologically confirmed BCG-unresponsive high-grade Papillary disease without CIS. The primary endpoint is a response of a disease-free rate at 12 months. Secondary endpoints include disease-free survival DFS and disease-specific survival, PFS, time to cystectomy, safety and overall survival.

About ANKTIVA

The cytokine interleukin-15 (IL-15) plays a crucial role in the immune system by affecting the development, maintenance, and function of key immune cells—NK and CD8+ killer T cells—that are involved in killing cancer cells. By activating NK cells, ANKTIVA overcomes the tumor escape phase of clones resistant to T cells and restores memory T cell activity with resultant prolonged duration of complete response.

ANKTIVA is a first-in-class IL-15 receptor agonist IgG1 fusion complex, consisting of an IL-15 mutant (IL-15N72D) fused with an IL-15 receptor alpha, which binds with high affinity to IL-15 receptors on NK, CD4+, and CD8+ T cells. This fusion complex of ANKTIVA mimics the natural biological properties of the membrane-bound IL-15 receptor alpha, delivering IL-15 by dendritic cells and drives the activation and proliferation of NK cells with the generation of memory killer T cells that have retained immune memory against these tumor clones. The proliferation of the trifecta of these immune killing cells and the activation of trained immune memory results in immunogenic cell death, inducing a state of equilibrium with durable complete responses. ANKTIVA has improved pharmacokinetic properties, longer persistence in lymphoid tissues, and enhanced anti-tumor activity compared to native, non-complexed IL-15 in-vivo.

ANKTIVA was approved by the FDA in 2024 with BCG for the treatment of adult patients with BCG-unresponsive non-muscle invasive bladder cancer with CIS with or without papillary tumors. For more information, visit ImmunityBio.com (Founder’s Vision) and Anktiva.com.

On May 5, 2025 Immuneering Corporation (Nasdaq: IMRX), a clinical-stage oncology company seeking to develop and commercialize more effective and better tolerated therapies for cancer patients, reported financial results for the first quarter ended March 31, 2025, and provided several business updates (Press release, Immuneering, MAY 5, 2025, View Source [SID1234652508]).

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"In Q1, we showed that IMM-1-104 can drive exceptional efficacy for patients with pancreatic cancer, including a third-line monotherapy patient with over 13 months progression-free survival, a second-line monotherapy patient with a confirmed partial response, and a first-line combination therapy patient with a confirmed complete response. These outcomes are particularly striking given the excellent tolerability observed with IMM-1-104. In January, we reported encouraging overall response rates and disease control rates for IMM-1-104 in combination with chemotherapy in first-line pancreatic cancer. We are excited to share survival data in a larger group of patients in the coming weeks," said Ben Zeskind, Ph.D., Co-founder and Chief Executive Officer of Immuneering.

"Our corporate progress in Q1 has matched the pace of our clinical progress. We announced a clinical supply agreement with Regeneron, expanded our cash runway into 2026, and hired an outstanding Chief Medical Officer as we plan for the initiation of a Phase 3 trial in first-line pancreatic cancer patients," Zeskind concluded.

Corporate Highlights

Pancreatic Cancer Patient Passes 13-month Mark on IMM-1-104 Monotherapy: In April, Immuneering provided an update on a Phase 1 pancreatic cancer patient in the third-line setting who has been receiving IMM-1-104 monotherapy for more than 13 months and is still on treatment. The patient – who previously experienced disease progression on first-line FOLFIRINOX and second-line Gem/Cis/nab-Pac – has been on IMM-1-104 monotherapy at 240 mg once daily and maintained stable disease including a RECIST SLD change of -28% and a 91% reduction in peak CA 19-9 levels. Treatment continued to be well tolerated by the patient, with approximately 16% weight gain.
Dr. Igor Matushansky Named as Chief Medical Officer: In March, Immuneering announced that Igor Matushansky, MD, PhD, an industry veteran with extensive global oncology drug development expertise and experience in clinical treatment of cancer patients, has been appointed the Company’s Chief Medical Officer. In this role, Dr. Matushansky is directing Immuneering’s clinical activities and is providing medical and operational leadership for the company’s development programs, including the ongoing Phase 2a trial of IMM-1-104 in pancreatic cancer, lung cancer, and melanoma, and plans to initiate a pivotal Phase 3 clinical trial in pancreatic cancer.
Clinical Trial Supply Agreement Announced with Regeneron for Libtayo (cemiplimab): In February, Immuneering announced a clinical trial supply agreement with Regeneron Pharmaceuticals for its anti-PD-1 therapy, Libtayo. The agreement supports the evaluation of IMM-1-104, in combination with Libtayo in patients with unresectable or metastatic RAS-mutant non-small cell lung cancer (NSCLC).
Provided a Positive Data Update from Three Pancreatic Cancer Arms of Ongoing Phase 2a Trial of IMM-1-104: In January, Immuneering announced positive data updates from three pancreatic cancer arms of its ongoing Phase 2a trial of lead program IMM-1-104 and confirmed plans to expand the Phase 2a trial to include additional combination arms.
Near-Term Milestone Expectations

IMM-1-104

Initial progression-free survival data from the IMM-1-104 Phase 2a trial expected in the second quarter of 2025.
Additional IMM-1-104 combination arms in planning.
Initiation of Phase 3 trial of IMM-1-104+mGnP in first-line pancreatic cancer planned for 2026.
First Quarter 2025 Financial Highlights

Cash Position: Cash and cash equivalents as of March 31, 2025, were $35.9 million, compared with $36.1 million as of December 31, 2024.
Research and Development (R&D) Expenses: R&D expenses for the first quarter of 2025 were $11.5 million compared with $11.2 million for the first quarter of 2024. The increase in R&D expenses was primarily attributable to higher clinical costs related to the Company’s lead program.
General and Administrative (G&A) Expenses: G&A expenses for the first quarter of 2025 were $4.0 million compared with $4.1 million for the first quarter of 2024. The decrease in G&A was primarily attributable to a decrease in the Company’s employee-related costs in connection with the general and administrative functions supporting the business.
Net Loss: Net loss attributable to common stockholders was $15.0 million, or $0.42 per share, for the first quarter ended March 31, 2025, compared to $14.3 million, or $0.49 per share, for the first quarter ended March 31, 2024.
2025 Financial Guidance

Based on cash and cash equivalents, as of March 31, 2025, and current operating plans, the Company expects its cash runway to be sufficient to fund operations into 2026.

On May 5, 2025 IGI, a global, fully integrated clinical-stage biotechnology company focused on developing multispecificsTM in oncology, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for ISB 2001 (Press release, Ichnos Sciences, MAY 5, 2025, View Source;utm_medium=rss&utm_campaign=ichnos-glenmark-innovation-igi-receives-usfda-fasttrack-designation-for-isb-2001-for-relapsed-refractory-multiple-myeloma [SID1234652507]). This important designation was granted for the treatment of adult patients with relapsed or refractory multiple myeloma (RRMM) who have received at least three prior lines of therapy including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. ISB 2001 is an investigational trispecific antibody therapeutic that targets BCMA and CD38 on myeloma cells and CD3 on T cells. ISB 2001 is currently being evaluated in a Phase 1 dose-expansion study.

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"A growing number of patients with multiple myeloma have been heavily pretreated, have exhausted currently approved therapies, and continue to face disease progression," said Cyril Konto, M.D., President and CEO of IGI. "At IGI, we have long recognized the urgent need for novel treatment options – particularly for patients who have already received first-generation bispecifics or CAR T-cell therapies. Our trispecific candidate is designed to enhance tumor targeting while reducing on-target, off-tumor toxicity. We are honored to receive this Fast Track designation and look forward to working closely with the FDA to advance our MultispecificTM T-cell engager, with the goal of delivering a first-in-class therapy for patients with relapsed or refractory multiple myeloma."

IGI recently completed the dose-escalation portion of its Phase 1 clinical study in patients with heavily pretreated multiple myeloma. Initial study results, presented in an oral session at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2024, demonstrated a high overall response rate (ORR) with durable responses and a favorable safety profile. Complete results from the dose-escalation portion will be presented in a rapid oral session at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting on Monday, June 2, 2025.

The FDA’s Fast Track designation is designed to enable the development and expedite the review of drugs that treat serious conditions and address unmet medical needs, with the ultimate goal of getting important new drugs to patients earlier. A drug that receives Fast Track designation may be eligible for more frequent meetings and communications with the FDA and rolling review of any application for marketing approval. A drug receiving Fast Track designation also may be eligible for Priority Review if relevant criteria are met. ISB 2001 was previously granted Orphan Drug Designation by the FDA in July 2023.

ASCO Rapid Oral Presentation Details:
Session title: Phase 1, first-in-human study of ISB 2001: A BCMAxCD38xCD3-targeting trispecific antibody for patients with relapsed/refractory multiple myeloma (RRMM)—Dose escalation results. (Abstract # 7514)

Session Name: Hematologic Malignancies—Plasma Cell Dyscrasia

Date & Time: June 2, 2025, 8 AM – 9:30 AM CDT

About ISB 2001 and Relapsed/Refractory Multiple Myeloma

ISB 2001 is a first-in-class trispecific T-cell engager that targets BCMA and CD38 on myeloma cells and CD3 on T cells. Developed using IGI’s proprietary BEAT protein platform, ISB 2001 was engineered with two distinct binders against myeloma-associated antigens to enhance avidity, even at low target expression levels, while aiming to improve safety over first-generation bispecific antibodies. The dose-expansion portion of the ongoing Phase 1 trial in patients with RRMM (NCT05862012) is currently enrolling patients across 9 sites in the United States and Australia.

Nearly all patients with relapsed or refractory multiple myeloma (RRMM) ultimately experience disease progression. With no cure currently available and limited treatment options once approved therapies are exhausted, there remains a significant unmet need. IGI is developing ISB 2001 to address this gap, specifically for patients who have previously received T-cell–directed therapies, including CAR T-cell treatments and bispecific antibodies.

On May 5, 2025 Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of medications to treat serious endocrinologic, oncologic, metabolic and neurologic disorders by modulating the effects of the hormone cortisol, reported its results for the quarter ended March 31, 2025 (Press release, Corcept Therapeutics, MAY 5, 2025, https://ir.corcept.com/news-releases/news-release-details/corcept-therapeutics-announces-first-quarter-financial-results-2 [SID1234652506]).

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Financial Results

"In the first quarter, we had another record number of prescriptions from new and existing prescribers, broadly distributed throughout the country. Growing physician awareness of hypercortisolism has resulted in increased screening and treatment of patients with this devastating disease. Our specialty pharmacy vendor began the quarter unable to fulfill this surge in demand, which negatively affected our first quarter financial results. Pharmacy operations improved substantially in March and April, with each month setting a record for tablets dispensed. We are reiterating our 2025 revenue guidance of $900 – $950 million," said Joseph K. Belanoff, M.D., Corcept’s Chief Executive Officer.

Corcept’s first quarter 2025 revenue was $157.2 million, compared to $146.8 million in the first quarter of 2024. First quarter 2025 operating expenses were $153.8 million, compared to $117.3 million in the same period last year. Net income was $20.5 million in the first quarter of 2025, compared to $27.8 million in the first quarter of 2024.

Cash and investments were $570.8 million at March 31, 2025, compared to $603.2 million at December 31, 2024. The balance at March 31, 2025 reflects the acquisition of $43.3 million of common stock in the first quarter pursuant to the company’s stock repurchase program, net exercise of employee stock options and net vesting of restricted stock grants.

Clinical Development

"Our New Drug Application (NDA) for relacorilant in hypercortisolism is progressing towards approval by the end of this year. We will submit our NDA next quarter for relacorilant in platinum-resistant ovarian cancer. We expect that relacorilant will have a role in helping treat earlier stages of ovarian cancer and other tumors that express the glucocorticoid receptor and have already begun our next clinical trial, BELLA. Meanwhile, we are making progress in understanding the role of cortisol modulation to treat a broad range of other serious disorders, including ALS and Metabolic Dysfunction-Associated Steatohepatitis (MASH)," added Dr. Belanoff.

Hypercortisolism (Cushing’s Syndrome)

Relacorilant for patients with hypercortisolism – U.S. Food and Drug Administration (FDA) accepted NDA for filing; Prescription Drug User Fee Act (PDUFA) target action date of December 30, 2025
GRACE – Pivotal Phase 3 trial of relacorilant in 152 patients with all etiologies of hypercortisolism – Results presented at Endocrine Society (ENDO) annual meeting and Heart in Diabetes (HiD) conference in June 2024
GRADIENT – Randomized, double-blind, placebo-controlled, Phase 3 trial of relacorilant in 137 patients with hypercortisolism caused by adrenal gland pathology – Results will be presented at American Association of Clinical Endocrinology (AACE) annual meeting in May 2025
Phase 3 long-term extension study of 116 patients who completed the GRACE, GRADIENT or Phase 2 relacorilant studies – Results presented at World Congress on Insulin Resistance, Diabetes and Cardiovascular Disease (WCIRDC) in December 2024
CATALYST Part 1 – Prevalence of hypercortisolism in patients with difficult-to-control type 2 diabetes – Results published in Diabetes Care in April 2025
CATALYST Part 2 – Randomized, double-blind, placebo-controlled study of Korlym in 136 patients with hypercortisolism – Results to be presented at the American Diabetes Association’s 85th Scientific Sessions (ADA) in June 2025
MOMENTUM – Enrollment continues in 1,000-patient trial examining the prevalence of hypercortisolism in patients with resistant hypertension – Results expected by year-end
"The positive results from our pivotal GRACE, GRADIENT, long-term extension and Phase 2 studies provide powerful support for the NDA for relacorilant in hypercortisolism. Patients in these studies experienced clinically significant improvements in a wide array of the signs and symptoms of hypercortisolism, without the off-target effects and toxicities that accompany currently available treatments. Relacorilant has the potential 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 diabetes resists treatment with the best available medications have hypercortisolism; hyperglycemia in these patients responds powerfully to treatment with a cortisol modulator. We reached an important milestone with the publication of CATALYST’s prevalence phase results in Diabetes Care and look forward to presenting the full results of the study’s treatment phase at ADA next month," added Dr. Guyer. "Building on the insights from CATALYST, our MOMENTUM study will deepen physicians’ understanding of hypercortisolism as a cause of resistant hypertension."

Oncology

Relacorilant for patients with platinum-resistant ovarian cancer – NDA submission expected in the third quarter with submission of Marketing Authorization Application (MAA) in Europe shortly thereafter
ROSELLA – Primary endpoint of improved progression-free survival (PFS) met in pivotal Phase 3 trial of relacorilant plus nab-paclitaxel in 381 patients with platinum-resistant ovarian cancer (hazard ratio: 0.70; p-value: 0.008; median PFS of 6.5 versus 5.5 months); interim evaluation of overall survival (OS) (hazard ratio: 0.69; p-value: 0.01; median OS of 16.0 versus 11.5 months), with no increase in side effect burden – Results to be presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting in June 2025
BELLA – Enrollment underway in Phase 2 trial of relacorilant plus nab-paclitaxel and bevacizumab in 90 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
"The ROSELLA results are an important advance for patients with platinum-resistant ovarian cancer, a disease with few treatment options. The PFS and OS improvements demonstrated in ROSELLA, with no increase in safety burden, bring us closer to delivering a new standard of care for these patients. We look forward to presenting the full results from ROSELLA in a late-breaker session at ASCO (Free ASCO Whitepaper) and submitting our NDA next quarter," said Dr. Guyer. "We are building on the findings from ROSELLA with our BELLA study, which will examine whether combining relacorilant with two medications – nab-paclitaxel and bevacizumab – will offer patients with platinum-resistant ovarian cancer another potent treatment option."

Amyotrophic Lateral Sclerosis (ALS)

DAZALS – In a randomized, double-blind, placebo-controlled Phase 2 study in 249 patients with ALS, dazucorilant did not meet the primary endpoint of improvement in the ALS Functional Rating Scale-Revised (ALSFRS-R)
DAZALS – An exploratory analysis at the one-year mark shows that patients who received 300 mg of dazucorilant at baseline demonstrate significantly improved overall survival, compared to patients who received placebo and did not switch to dazucorilant in the long-term extension study (hazard ratio of 0.16, p-value: 0.0009)
DAZALS – Results to be presented at European Network to Cure ALS (ENCALS) annual meeting in June 2025
"ALS is a devastating disease. Patients who received dazucorilant did not show improvement in the ALS Functional Rating Scale-Revised (ALSFRS-R), which was DAZALS’ primary endpoint. An improvement in overall survival, first seen at the six-month mark, was also observed at year one of the study. An exploratory analysis determined that patients who received 300 mg of dazucorilant at the start of the study lived significantly longer than patients who received placebo and did not switch to dazucorilant in the long-term extension study, with a hazard ratio of 0.16 (p-value: 0.0009). This long-term extension study is on-going. We will immediately seek input from U.S. and European regulatory authorities on the next steps with dazucorilant," said Dr. Guyer.

Metabolic Dysfunction-Associated Steatohepatitis (MASH)

MONARCH – Enrollment continues in randomized, double-blind, placebo-controlled, Phase 2b trial of miricorilant in patients with biopsy-confirmed or 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. First results are expected by the end of next year," said Dr. Guyer.

Conference Call

We will hold a conference call on May 5, 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 May 5, 2025 Certara, Inc. (Nasdaq: CERT), a global leader in model-informed drug development, reported its financial results for the first quarter of fiscal year 2025 (Press release, Certara, MAY 5, 2025, View Source [SID1234652504]).

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First Quarter Highlights:
Revenue was $106.0 million, compared to $96.7 million in the first quarter of 2024, representing growth of 10%.
Software revenue was $46.4 million, compared to $39.3 million in the first quarter of 2024, representing growth of 18%.
Services revenue was $59.6 million, flat compared to $57.3 million in the first quarter of 2024, representing growth of 4%.
Net income was $4.7 million, a $9.4 million increase compared to a net loss of $4.7 million in the first quarter of 2024.
Adjusted EBITDA was $34.8 million, compared to $29.1 million in the first quarter of 2024, representing growth of 20%.
"We are pleased with the first quarter’s strong performance in both core biosimulation and the regulatory services businesses," said William F. Feehery, Chief Executive Officer. "The FDA’s recently announced roadmap for reducing animal testing in preclinical studies further demonstrates the vast opportunities ahead for Biosimulation. Certara is well positioned to support the continued adoption of Biosimulation by industry to gain important insights into new therapies. We continue to work closely with our customers as they explore new approaches to make the drug development process more efficient."

"Our first quarter performance reflected strong commercial execution from our team across software and services. Despite the challenges facing our customers in the biopharma industry, we are reiterating guidance for 2025," said John Gallagher, Chief Financial Officer.

First Quarter 2025 Results
Total revenue for the first quarter of 2025 was $106.0 million, representing year-over-year growth of 10% on a reported basis and on a constant currency basis. Total revenue included $5.9 million of Chemaxon revenue. The overall increase in revenue was primarily due to growth in our biosimulation software portfolio and contribution from M&A. Please see note (1) in the section "A Note on Non-GAAP Financial Measures" below for more information on constant currency revenue.

Software revenue for the first quarter of 2025 was $46.4 million, representing year-over-year growth of 18% on a reported basis and 19% on a constant currency basis. Software growth was driven by biosimulation software and contribution from M&A.

Services revenue for the first quarter of 2025 was $59.6 million, representing year-over-year growth of 4% on a reported basis and on a constant currency basis. Services growth was driven by biosimulation services.

Total Bookings for the first quarter of 2025 were $118.2 million representing a year-over-year growth of 12% on a reported basis. Total Bookings included $4.9 million of Chemaxon bookings.

Software Bookings for the first quarter of 2025 were $40.8 million, representing a year-over-year growth of 23%. The increase in software bookings was primarily due to strength in Certara’s core biosimulation software and contribution from Chemaxon.

Services Bookings for the first quarter of 2025 were $77.4 million, representing a year-over-year growth of 7%. The increase in services bookings was driven by demand for biosimulation and regulatory services.

Total cost of revenue for the first quarter of 2025 was $41.5 million, an increase of $2.3 million from $39.3 million in the first quarter of 2024, primarily due to an increase in software amortization expense.

Total operating expenses for the first quarter of 2025 were $56.9 million, which decreased by $1.8 million from $58.7 million in the first quarter of 2024. Lower operating expenses were primarily due to a $3.1 million decrease in the change in fair value of a contingent consideration, which was offset by higher sales and marketing expense and intangible asset amortization.

Adjusted EBITDA for the first quarter of 2025 was $34.8 million compared to $29.1 million for the first quarter of 2024, an increase of $5.7 million. See note (2) in the section A Note on Non-GAAP Financial Measures below for more information on adjusted EBITDA.

Diluted earnings per share for the first quarter of 2025 was $0.03, as compared to a diluted loss per share of $0.03 in the first quarter of 2024.

Net income for the first quarter of 2025 was $4.7 million, compared to a net loss of $4.7 million in the first quarter of 2024. The $9.4 million increase was primarily due to an increase in gross profit and lower operating expenses.

Adjusted net income for the first quarter of 2025 was $22.2 million compared to $16.5 million for the first quarter of 2024, an increase of $5.7 million. Adjusted diluted earnings per share for the first quarter 2025 was $0.14 compared to $0.10 for the first quarter of 2024. See note (3) in the section A Note on Non-GAAP Financial Measures below for more information on adjusted net income and adjusted diluted earnings per share.

2025 Financial Outlook
Certara is reiterating its guidance for the full year 2025:

Full year 2025 revenue to be in the range of $415 million to $425 million.
Full year adjusted EBITDA margin to be in the range of 30-32%.
Full year adjusted diluted earnings per share is expected to be in the range of $0.42 – $0.46.
Fully diluted shares are expected to be in the range of 162 million to 164 million.
Please note that the Company has not reconciled adjusted EBITDA (including its related margin) or adjusted diluted earnings per share forward-looking guidance included in this press release to the most directly comparable GAAP measures because this cannot be done without unreasonable effort due to the variability and low visibility with respect to costs related to acquisitions, financings, and employee stock compensation programs, which are potential adjustments to future earnings. We expect the variability of these items to have a potentially unpredictable, and a potentially significant, impact on our future GAAP financial results.

Webcast and Conference Call Details
Certara will host a conference call today, May 5, 2025, at 5:00 p.m. ET to discuss its first quarter 2025 financial results. Investors interested in listening to the conference call are required to register online in advance of the call. A live and archived webcast of the event will be available on the "Investors" section of the Certara website at View Source