On September 22, 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 it will present new data from its pivotal Phase 3 ROSELLA trial of relacorilant plus nab-paclitaxel in patients with platinum-resistant ovarian cancer at the 2025 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Annual Meeting (Press release, Corcept Therapeutics, SEP 22, 2025, https://ir.corcept.com/news-releases/news-release-details/corcept-present-new-late-breaking-data-pivotal-rosella-trial [SID1234656144]). The data, which will be presented in a late-breaking oral presentation on Sunday, October 19, 2025, focuses on trial participants who were previously treated with a PARP inhibitor, a patient population with particularly poor prognosis.

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The ROSELLA trial is being conducted in collaboration with The GOG Foundation, Inc. (GOG-F), the European Network of Gynaecological Oncological Trial groups (ENGOT), the Asia-Pacific Gynecologic Oncology Trials Group (APGOT), the Latin American Cooperative Oncology Group (LACOG) and the Australia New Zealand Gynaecological Oncology Group (ANZGOG).

Presentation Details

LBA45 – ROSELLA (GOG3073, ENGOTov72, APGOT-OV10): Relacorilant + Nab-paclitaxel in the Subgroup of Patients With Platinum-Resistant Ovarian Cancer (PROC) Previously Exposed to a PARP Inhibitor

Mini Oral session: Gynaecological cancers
October 19, 2025, 10:15 AM – 11:45 AM CEST
About Relacorilant

Relacorilant, an oral therapy, is a selective glucocorticoid receptor (GR) antagonist that modulates cortisol activity by binding to the GR but not to the body’s other hormone receptors. Corcept is developing relacorilant in ovarian cancer and a variety of other serious disorders, including endogenous hypercortisolism and prostate cancer. Relacorilant is proprietary to Corcept and is protected by composition of matter, method of use and other patents. It has been designated an orphan drug by the FDA and the European Commission (EC) for the treatment of hypercortisolism and by the EC for the treatment of ovarian cancer. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) date of December 30, 2025 for relacorilant as a treatment for patients with hypercortisolism, and a PDUFA date of July 11, 2026 for relacorilant as a treatment for patients with platinum-resistant ovarian cancer.

About Cortisol’s Role in Oncology

Cortisol plays a role in tumor growth through several mechanisms. It helps solid tumors resist chemotherapy by inhibiting cellular apoptosis — the tumor-killing effect chemotherapy is meant to stimulate. In some cancers, cortisol promotes tumor growth by activating oncogenes in the cells to which it binds. Cortisol also suppresses the body’s immune response, which weakens its ability to fight all diseases, including cancer.

About Platinum-Resistant Ovarian Cancer

Ovarian cancer is the fifth most common cause of cancer death in women. Patients whose disease returns less than six months after receiving platinum-containing therapy have "platinum-resistant" disease. There are few treatment options for these women. Median overall survival following recurrence is approximately 12 months with single-agent chemotherapy. Approximately 20,000 women with platinum-resistant disease are candidates to start a new therapy each year in the United States, with at least an equal number in Europe.

On September 22, 2025 Congruence Therapeutics, a computationally-driven biotechnology company building a unique pipeline of pharmacological correctors for diseases of protein misfolding, reported that Fierce Biotech has named it as one of 2025’s Fierce 15 biotechnology companies, honoring it as one of the most innovative and promising private biotechnology companies in the industry (Press release, Congruence Therapeutics, SEP 22, 2025, View Source [SID1234656143]).

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"This recognition from Fierce Biotech serves as further validation of the impactful science we are undertaking to develop transformative medicines for significant unmet medical needs and underscores what matters most to us: the patients and families waiting for treatment options," commented Dr. Clarissa Desjardins, co-founder and CEO of Congruence. "We remain committed and laser-focused on advancing small molecule pharmacological drugs that have the potential to transform care for people affected by MC4R-driven genetic obesity, GBA-driven Parkinson’s disease, and α1-Antitrypsin-deficiency. This award strengthens our resolve to deliver these novel therapies to the bedside of those who need them most."

The Fierce 15 honor is intended to celebrate the spirit of being "fierce" – championing innovation and creativity, even in the face of intense competition. This is Fierce Biotech’s 23rd annual Fierce 15 selection.

"Over the past 23 years, our Fierce 15 special report has spotlighted the private biotechs pushing science into uncharted territory, whose approaches and discoveries represent genuine leaps forward," said Ayla Ellison, Editor-in-Chief, Fierce Life Sciences and Healthcare, "This year we’ve selected 15 pre-commercial biotechs that are advancing transformative science in an unrelenting industry landscape. These companies exemplify the innovation and determination driving the next generation of therapies that will hopefully benefit patients worldwide."

An internationally recognized daily report reaching a network of over 300,000 biotech and pharma industry professionals, Fierce Biotech provides subscribers with an authoritative analysis of the day’s top stories. Every year Fierce Biotech evaluates hundreds of early-stage companies from around the world for its annual Fierce 15 list, which is based on a variety of factors such as the strength of its technology, partnerships, venture backers and a competitive market position.

On September 22, 2025 CatalYm , a world-leader in neutralizing GDF-15 in cancer and cachexia, reported two upcoming oral presentations highlighting the company’s lead-candidate visugromab at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress, being held October 17th-21st, 2025, in Berlin, Germany (Press release, Catalym, SEP 22, 2025, View Source [SID1234656142]). Visugromab is a humanized, monoclonal antibody designed to neutralize the tumor-derived cytokine Growth Differentiation Factor-15 (GDF-15) that plays a central role in immune suppression and anti-PD(L)-1 treatment resistance.

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A late-breaking abstract featuring first results from the Phase 2 GDFATHER-NEO trial (NCT06059547) was accepted for oral presentation. The trial investigates visugromab as neoadjuvant treatment in combination with nivolumab in patients with muscle-invasive bladder cancer (MIBC) compared to nivolumab administered with placebo.

In addition, a second oral presentation will cover long-term follow-up data from the GDFATHER-01 Phase 1/2 trial (NCT04725474) demonstrating deep and durable remissions in heavily pretreated, anti-PD1/-L1 relapsed/refractory non-squamous non-small cell lung cancer (NSCLC), urothelial cancer (UC) and hepatocellular cancer (HCC) patients.

Presentation Details

Late-breaking Oral Presentation

Abstract Title: A blinded, exploratory phase 2 trial of nivolumab and the GDF-15 neutralizing antibody visugromab or placebo as neoadjuvant treatment of patients with muscle-invasive bladder cancer (MIBC): Primary results of the GDFATHER-NEO trial

Session Category & Title: Proffered paper session 1: GU tumours, renal & urothelial

Presentation Date & Time: October 17, 2025; 3:00 – 3:10 pm CEST

Presenter: Prof. Andrea Necchi, MD, Vita-Salute San Raffaele University, IRCCS San Raffaele Hospital

Location: Dortmund Auditorium – Hall 7.1a

Proffered Paper Presentation

Abstract Title: Long-term follow-up GDFATHER-01 trial: GDF-15 neutralization combined with nivolumab can enable deep, long-term remission in heavily pretreated, anti-PD1/-L1 relapsed/refractory non-squamous non-small cell lung cancer (NSCLC), urothelial cancer (UC) and hepatocellular cancer (HCC)

Session Category & Title: Proffered paper session: Investigational immunotherapy

Presentation Date & Time: October 20, 2025; 10:55 – 11:05 am CEST

Presenter: Prof. Ignacio Melero, MD, PhD, Co-Director of Immunology and Immunotherapy (CIMA) at the Universidad de Navarra, Pamplona/Spain

Location: Hanover Auditorium – Hall 7.2c

About Visugromab

Visugromab is a monoclonal antibody that neutralizes Growth Differentiation Factor-15 (GDF-15), a locally acting immunosuppressant produced by tumors which fosters immunotherapy resistance and drives cachexia in people with cancer. Neutralizing GDF-15 with visugromab reverses key cancer resistance mechanisms to reinstate an efficient anti-tumor response by re-enabling immune cell activation, proliferation and induction of interferon-γ. In addition, visugromab also mitigates cancer cachexia, a severe condition affecting a significant number of advanced cancer patients by inhibiting the activation of the GFRAL pathway in the brainstem, a key driver of weight loss and appetite suppression in cancer patients.

On September 22, 2025 Avenzo Therapeutics, Inc. ("Avenzo"), a clinical-stage biotechnology company developing next-generation oncology therapies, reported the closing of a $60 million Series B financing (Press release, Avenzo Therapeutics, SEP 22, 2025, View Source [SID1234656141]). This funding, which follows Avenzo’s $386 million Series A/A-1 financing announced in November 2024, brings the total capital raised to $446 million. The proceeds will be used to support the advancement of Avenzo’s pipeline of potential best-in-class oncology drug candidates.

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The Series B financing was led by OrbiMed and SR One, with participation from new investor Longwood Fund. All existing investors, including Foresite Capital, Lilly Asia Ventures, Surveyor Capital (a Citadel company), New Enterprise Associates (NEA), Deep Track Capital, Sofinnova Investments, Sands Capital, INCE Capital, TF Capital, Delos Capital, and Quan Capital, participated in this financing.

"We are excited to add Longwood to our syndicate and are grateful to all of our investors for their continued partnership as we strive to develop oncology therapies that help fight cancers with high unmet medical needs," said Athena Countouriotis, M.D., Co-founder, President and CEO of Avenzo. "Avenzo has made tremendous progress this year as we have advanced our pipeline from one to four clinical stage assets. This Series B financing enables us to build on this momentum as we continue the development of our potential best-in-class oncology programs."

On September 22, 2025 Astrazeneca reported that Koselugo (selumetinib), an oral, selective MEK inhibitor, has been recommended for approval in the European Union (EU) for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in adult patients with neurofibromatosis type 1 (NF1) (Press release, AstraZeneca, SEP 22, 2025, View Source [SID1234656140]).

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) based its positive opinion on results from KOMET, the largest and only placebo-controlled global Phase III trial in this patient population, which were presented at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and published in The Lancet.1

In the primary analysis of the trial, Koselugo showed a statistically significant objective response rate (ORR) of 20% (n=14/71, 95% CI: 11.2, 30.9) compared to 5% with placebo (n=4/74, 95% CI: 1.5, 13.3; p=0.01) by cycle 16.1

NF1 is a rare, progressive, genetic condition usually diagnosed in early childhood, but often progressing into adulthood, that can impact every organ system.2,3 Up to 50% of people living with NF1 may develop a type of non-malignant tumour called PN that may affect the brain, spinal cord and nerves.3,4 PN may appear later in a person’s life and can grow and become large, leading to pain, disfigurement and muscle weakness, among other debilitating symptoms.3,4

Prof. Pierre Wolkenstein, MD, PhD, Head of the Department of Dermatology at Henri Mondor Hospital, APHP, Paris East University (UPEC), and National Coordinating Investigator of the KOMET trial in Europe, said: "For adults with NF1, tumour growth doesn’t stop at childhood and can rapidly progress or develop into adulthood, impacting physical, emotional and social well-being. The positive recommendation by the CHMP for Koselugo in adults underscores the urgent need for additional targeted treatments for this patient population. When approved, Koselugo could offer a treatment option for adult patients and continuity of care across age groups, supported by established clinical experience and practice among doctors managing this lifelong condition."

Marc Dunoyer, Chief Executive Officer, Alexion, said: "The CHMP positive opinion of Koselugo in adults with NF1 PN builds on more than a decade of global clinical and real-world experience, reflecting the unmatched body of evidence supporting the safety profile and efficacy of Koselugo. As demonstrated by the results from KOMET, the largest and only placebo-controlled, double-blind global Phase III trial in an adult population, we continue to advance the pioneering legacy of Koselugo, which set the treatment standard in NF1 PN, to reach even more people worldwide."

The safety profile of Koselugo in the KOMET Phase III trial was consistent with its known profile and established use in paediatric patients.1

Koselugo has been recently approved in Japan and other countries for the treatment of adult patients with NF1 who have symptomatic, inoperable PN based on data from the KOMET Phase III trial, and additional regulatory reviews are ongoing.

Notes

NF1
NF1 is a rare, progressive, genetic condition that is caused by a spontaneous or inherited mutation in the NF1 gene.2,3 NF1 is associated with a variety of symptoms, including soft lumps on and under the skin (cutaneous neurofibromas) and, in up to 50% of patients, tumours called plexiform neurofibromas (PN) may develop on the nerve sheaths.3,4 These PN can cause clinical issues such as disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment and bladder or bowel dysfunction.3,4

KOMET
KOMET is a global Phase III randomised, double-blind, placebo-controlled, multicentre trial designed to evaluate the efficacy and safety of Koselugo in adults with NF1 who have symptomatic, inoperable PN. The trial enrolled 145 adults from 13 countries across North America, South America, Europe, Asia and Australia, with participants’ baseline characteristics, including gender and distribution of PN, reflective of the global adult NF1 patient population. Patients were enrolled and randomised to receive Koselugo or placebo (1:1) for 12 28-day cycles. Participants were required to have diagnosis of NF1, at least one symptomatic, inoperable PN measurable by volumetric MRI analysis, chronic PN pain score documented during screening, adequate organ and marrow function and stable chronic PN pain medication use at enrolment.1,5

The primary endpoint is confirmed objective response rate (ORR) by cycle 16 as assessed by ICR. ORR is defined as the percentage of patients with confirmed complete response (disappearance of PNs) or partial response (at least 20% reduction in tumour volume). Secondary endpoints include improved PN-related pain and health-related quality of life (HRQoL) at cycle 12.1,5

After 12 cycles, patients on placebo were switched to Koselugo and patients on Koselugo remained on treatment for an additional 12 cycles. Patients who had the opportunity to complete 24 cycles of treatment have the option to participate in a long-term extension period and continue to receive Koselugo.1,5

Koselugo
Koselugo (selumetinib) is a kinase inhibitor that blocks specific enzymes (MEK1 and MEK2), which are involved in stimulating cells to grow. In NF1, these enzymes are overactive, causing tumour cells to grow in an unregulated way creating so-called plexiform neurofibromas (PN). By blocking these enzymes, Koselugo slows down the growth of tumour cells and, therefore, the PN growth. 
 
Koselugo is approved in the US, EU, Japan, China and other countries for the treatment of certain paediatric patients with NF1 who have symptomatic, inoperable PN.

Koselugo is approved in Japan and other countries for the treatment of adult patients with NF1 who have symptomatic, inoperable PN, and additional regulatory reviews are ongoing

Koselugo has been granted Orphan Drug Designation in the US, EU, Japan and other countries for the treatment of NF1.

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