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Sarclisa recommended for EU approval by the CHMP to treat transplant-ineligible newly diagnosed multiple myeloma

On November 18, 2024 European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) reported to have adopted a positive opinion recommending the approval of Sarclisa in combination with bortezomib, lenalidomide, and dexamethasone (VRd) for the treatment of adult patients with newly diagnosed multiple myeloma (NDMM) who are ineligible for autologous stem cell transplant (ASCT) (Press release, Sanofi, NOV 18, 2024, View Source [SID1234648476]). A final decision is expected in the coming months.

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Dietmar Berger, M.D., Ph.D.

Chief Medical Officer, Global Head of Development at Sanofi

"The positive CHMP opinion is an important step forward for people with transplant-ineligible newly diagnosed multiple myeloma for whom effective front-line therapy may improve long-term outcomes. If approved, this Sarclisa-based combination could establish a new standard-of-care treatment approach for patients in the EU, helping to address a critical care gap in multiple myeloma treatment, and reinforcing Sarclisa’s potential as the anti-CD38 therapy of choice."

In September 2024, the US Food and Drug Administration (FDA) approved Sarclisa in combination with VRd for the treatment of adult patients with NDMM who are not eligible for ASCT, representing the first global approval for Sarclisa in the first line setting. In addition, the FDA granted orphan drug exclusivity for Sarclisa in the approved indication.

Sarclisa is currently approved in two indications for the treatment of certain adult patients with relapsed or refractory MM in more than 50 countries, including the US and EU.

First positive global phase 3 study combining anti-CD38 therapy with VRd to significantly improve PFS versus VRd alone in transplant-ineligible NDMM supports CHMP decision

The positive CHMP opinion is based on data from the IMROZ phase 3 study, which was presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2024 annual meeting, European Hematology Association (EHA) (Free EHA Whitepaper) 2024 meeting, and published in The New England Journal of Medicine. IMROZ is the first global phase 3 study of a CD38 monoclonal antibody in combination with standard-of-care VRd to significantly improve progression-free survival (PFS) versus VRd alone. The safety and tolerability of Sarclisa observed was consistent with the established safety profile of Sarclisa and VRd with no new safety signals.

About Sarclisa

Sarclisa (isatuximab) is a CD38 monoclonal antibody that binds to a specific epitope on the CD38 receptor on MM cells, inducing distinct antitumor activity. It is designed to work through multiple mechanisms of action including programmed tumor cell death (apoptosis) and immunomodulatory activity. CD38 is highly and uniformly expressed on the surface of MM cells, making it a target for antibody-based therapeutics such as Sarclisa. In the US, the non-proprietary name for Sarclisa is isatuximab-irfc, with irfc as the suffix designated in accordance with nonproprietary naming of biological products guidance for industry issued by the US FDA.

Currently Sarclisa is approved in more than 50 countries, including the US and EU, across two indications; Sarclisa is approved under an additional indication in the US. In Europe, based on the ICARIA-MM phase 3 study, Sarclisa is approved in combination with pomalidomide and dexamethasone for the treatment of patients with relapsed refractory MM (RRMM) who have received ≥2 prior therapies, including lenalidomide and a proteasome inhibitor and who progressed on last therapy. Based on the IKEMA phase 3 study, Sarclisa is also approved in 50 countries in combination with carfilzomib and dexamethasone, including in the US for the treatment of patients with RRMM who have received 1–3 prior lines of therapy and in the European Union for patients with MM who have received at least 1 prior therapy. In the US, Sarclisa is also approved in combination with VRd as a first line treatment option for adult patients with NDMM who are not eligible for ASCT, based on the IMROZ phase 3 study.

Sanofi continues to advance Sarclisa as part of a patient-centric clinical development program, which includes several phase 2 and phase 3 studies across the MM treatment continuum spanning six potential indications. In addition, the company is evaluating a subcutaneous administration method for Sarclisa in clinical studies. The safety and efficacy of Sarclisa has not been evaluated by any regulatory authority outside of its approved indications and methods of delivery.

In striving to become the number one immunoscience company globally, Sanofi remains committed to advancing oncology innovation. Through focused strategic decisions the company has reshaped and prioritized its pipeline, leveraging its expertise in immunoscience to drive progress. Efforts are centered on difficult-to-treat cancers such as select hematologic malignancies and solid tumors with critical unmet needs, including multiple myeloma, acute myeloid leukemia, certain types of lymphomas, as well as gastrointestinal and lung cancers.

Ratio Enters License and Collaboration Agreement with Novartis for SSTR2-targeting Radiotherapeutic Candidate

On November 18, 2024 Ratio Therapeutics Inc. (Ratio), a pharmaceutical company employing innovative technologies to develop best-in-class radiopharmaceuticals for cancer treatment and monitoring, reported to have entered into an exclusive worldwide license and collaboration agreement with Novartis Pharma AG, a subsidiary of Novartis AG (NYSE: NVS) (Press release, Ratio Therapeutics, NOV 18, 2024, View Source [SID1234648475]). The collaboration leverages Ratio’s radioligand therapy discovery and development expertise as well as its technology platforms for the development of a Somatostatin Receptor 2 (SSTR2) radiotherapeutic candidate for cancer.

"The team at Ratio is honored and excited to partner with Novartis on the development of a next-generation SSTR2-targeting therapeutic," said Jack Hoppin, Ph.D., Chief Executive Officer of Ratio. "Together, we aim to develop a best-in-class therapy in the fight against SSTR2-expressing tumors."

"Radioligand therapies hold transformative potential for certain forms of cancer, and Novartis is committed to maximizing their impact by continually improving the benefit for patients," said Fiona Marshall, President of Biomedical Research at Novartis. "We are delighted to collaborate with Ratio to advance this RLT candidate and work together to bring forward additional therapeutic options for patients with difficult-to-treat cancer."

Under the terms of the agreement, Ratio will receive combined upfront and potential milestone payments up to $745m, and is eligible to receive tiered royalty payments. Ratio will collaborate with Novartis to drive preclinical activities to research and select an SSTR2-targeting development candidate. Novartis will assume responsibility for all remaining development, manufacturing, and commercialization activities.

The collaboration combines the expertise and strengths of Ratio and Novartis to further elevate the safety and efficacy of radiopharmaceuticals for patient benefit.

Chestnut Partners served as exclusive financial advisor to Ratio for this transaction.

Orphelia Pharma provides update on the regulatory submission of KIZFIZO® with the European Medicines Agency

On November 18, 2024 Orphelia Pharma, a pharmaceutical company dedicated to the development and marketing of pediatric and orphan medicines, reported that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a negative opinion on the Marketing Authorization Application for KIZFIZO (temozolomide oral suspension) for the treatment of patients with relapsed or refractory high-risk neuroblastoma, because the CHMP considered that a positive benefit-risk balance has not been established at this stage (Press release, ORPHELIA Pharma, NOV 18, 2024, View Source [SID1234648474]). Orphelia will seek a re-examination of the opinion by the CHMP.

"We are extremely disappointed by the CHMP’s negative opinion and understand that this may also be disappointing for the pediatric oncology community as well as for parents of children suffering from cancer." said Jérémy Bastid, Chief Medical Officer at Orphelia Pharma. "There is a significant demand for developing children-adapted formulations. Orphelia Pharma has been developing KIZFIZO in collaboration with Gustave Roussy, the leading European cancer center, for the last seven years specifically to address this unmet medical need for a drinkable temozolomide medication in the treatment of relapsed or refractory neuroblastoma, which affects very young children".

"As part of the re-examination process, we will seek to address the CHMP’s grounds for refusal and we will work diligently to substantiate the clinical benefit of the treatment so that KIZFIZO can be available to pediatric patients in the EU without further delay" commented Laurent Martin, Chief Pharmaceutical Affairs Officer at Orphelia Pharma.

In the meantime, Orphelia Pharma will, subject to agreement by the relevant authorities, continue to make KIZFIZO available to pediatric patients suffering from relapsed or refractory neuroblastoma under compassionate use or early access programs as well as to patients enrolled into ongoing clinical trials.

About KIZFIZO 40 mg/ml

KIZFIZO (temozolomide oral suspension, 40 mg/ml) is a ready-to-use oral liquid pediatric formulation of temozolomide developed/designed specifically for use in the treatment of children with relapsed or refractory high-risk neuroblastoma, which carries a very poor prognosis. This age-adapted and taste-masked formulation delivers an accurate dose in a small volume, while avoiding drug handling and caregiver exposure to temozolomide. It is the result of a collaboration between the pharmacists and clinicians at Gustave Roussy hospital and the development team at Orphelia Pharma.

In March 2022, KIZFIZO was granted Early Access Authorization (Autorisation d’Accès Précoce) by the French authorities, for the treatment of refractory and relapsed neuroblastoma as monotherapy or in combination with irinotecan or topotecan.

KIZFIZO has received Orphan Drug Designation (ODD) from the EMA and the FDA, the formulation is covered by granted patents and pending applications in Europe and the US.

The pharmacokinetics of KIZFIZO in children have been evaluated in TEMOkids, a European multicenter population pharmacokinetic acceptability and safety study in pediatric patients in need of temozolomide (NCT04610736).

Efficacy and safety data for temozolomide in relapsed or refractory neuroblastoma submitted in the application includes in particular:

BEACON-Chemo, a sub-analysis of the chemotherapy arms of the BEACON study, a prospective randomized phase II study in refractory or relapsed neuroblastoma. This study was sponsored by Birmingham University (UK).
Retro-TMZ, a multicenter descriptive, retrospective study, assessing the efficacy and tolerability of temozolomide in children with refractory or relapsed neuroblastoma. This study was conducted by Gustave Roussy (France).
About Neuroblastoma

Neuroblastoma is the most common extracranial cancer in early childhood, with approximately 900 new cases diagnosed per year in the European Union. It almost exclusively affects children under five, with a median age at diagnosis of 18 months. Neuroblastoma has a wide diversity of clinical outcomes, which is reflected in the risk stratification. Approximately 40% of patients have the high-risk disease and often face a poor response to first line induction therapy or later relapse. There remains a high unmet need for relapsed or refractory neuroblastoma patients and the best therapeutic strategy is still an intensive area of research. Temozolomide is the standard chemotherapy and is therefore an essential part of the treatment armamentarium for these patients.

OncoSpherix Showcased at 2024 Georgia Bio Life Sciences Summit, Highlights Latest Advances in Cancer treatment

On November 18, 2024 OncoSpherix, Inc., a preclinical-stage cancer drug development company advancing HIF inhibitors for a variety of cancer indications, reported participation in the 2024 Georgia Bio Life Sciences Summit last month from October 21 to October 22, 2024, where the company was featured in the prestigious Company Showcase (Press release, OncoSpherix, NOV 18, 2024, View Source [SID1234648473]). This event provided a valuable platform for OncoSpherix to present its pioneering developments in cancer therapies and underscore its commitment to advancing cancer treatment.

The Georgia Bio Life Sciences Summit, known for bringing together key players in the biotechnology and life sciences sectors, offered OncoSpherix an opportunity to engage with industry leaders, investors, and researchers. The Company Showcase spotlighted OncoSpherix’s unique approach to precision oncology, focusing on therapies designed to block activation of HIF and combat hard-to-treat cancers more effectively.

"We are honored to have been part of the Company Showcase at the 2024 Georgia Bio Life Sciences Summit," said OncoSpherix CEO, Margaret Offermann, MD, PhD. "This platform allowed us to share our breakthroughs in developing new drugs for oncology and our mission to bring innovative, effective therapies to patients in need. The positive response from the community reinforces our commitment to developing solutions that will make a significant impact on cancer treatment."

New Findings Show Moleculin’s Annamycin Overcomes Resistance to Venetoclax in AML

On November 18, 2024 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a late-stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat tumors and viruses, reported new findings supporting the ability of Annamycin to overcome resistance to Venetoclax in acute myeloid leukemia ("AML") (Press release, Moleculin, NOV 18, 2024, View Source [SID1234648472]). This includes data from preclinical in vitro studies recently accepted for online publication at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) ("ASH") Annual Meeting, and correlates with efficacy demonstrated by recent preliminary clinical data in subjects who were relapsed from or refractory to first line Venetoclax regimens and were then treated with Annamycin in combination with Ara-C ("AnnAraC").

Jorge Cortes, MD, Director of the Georgia Cancer Center at Augusta University and a member of the Company’s Scientific Advisory Board, commented, "Although Venetoclax has been an important improvement in first line therapy for AML patients who are unfit for intensive chemotherapy, the rate of relapse is high and overall survival post relapse is just a few months. This turns out to be a large percentage of AML patients in total and we clearly need a better treatment option."

Michael Andreeff, MD, PhD, Professor of Medicine, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center and a member of the Company’s Scientific Advisory Board, added, "Many patients get Ven-Aza, not because they are ‘unfit’ but because of the high initial response rates. When they relapse, however, our options are very limited. Annamycin combined with Ara-C could significantly advance the standard of care and provide better outcomes for these high-risk patients. I am excited to be a part of the next step in the development of this important asset."

A prior publication, Outcomes of relapsed or refractory acute myeloid leukemia after frontline hypomethylating agent and venetoclax regimens1, reported that the CR/CRi2 rate for salvage therapy using available standard of care in AML subjects who relapsed from or were refractory to Venetoclax regimens was 12.5% (n=24, 4% CR). The new preliminary clinical findings announced today in the MB-106 clinical trial indicate that relapsed or refractory ("R/R") AML subjects previously treated with Venetoclax regimens achieved a 60% CR/CRi rate (n=5, 40% CR), more than 4 times the rate that would be expected based on the above referenced paper. As previously disclosed in MB-106, there was an overall CR/CRi rate of 60% (50% CR) in 2nd line subjects (n=10) and 41% (36% CR) in all subjects, 1st-7th line (n=22).

An abstract titled, "Annamycin, a non-cardiotoxic anthracycline, demonstrates unique organotropism and activity against Ara-C and Venetoclax resistant AML," supporting the clinical activity of Annamycin was accepted for online publication at the ASH (Free ASH Whitepaper) Annual Meeting being held December 7-10, 2024, in San Diego, CA. The abstract will be published in a supplemental issue of Blood, expected in late November, and will become part of the permanent ASH (Free ASH Whitepaper) and Blood abstracts archive following the conclusion of the Annual Meeting.

Additionally, new preliminary data from the Company’s Phase 1B/2 clinical trial evaluating AnnAraC for the treatment of subjects with AML as both first line therapy and for subjects who were refractory to or relapsed after induction therapy (MB-106) demonstrated median overall survival ("OS") of 9.1 months for subjects with a wide range of (0-6) prior lines of therapy (n=22) and 11.6 months (n=10) for subjects with 1 prior line of therapy (second line therapy).

"Moleculin is focusing on development of Annamycin to address the significant unmet need in R/R AML. The growing body of preliminary data continue to bolster our confidence in the safety and efficacy of Annamycin, and its potential to provide patients and physicians with a promising new treatment option in AML," commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin. "We believe the latest preliminary OS data we are seeing in our MB-106 trial can now be considered exceptional and we look forward to the initiation of our pivotal registration study, especially now that our recent protocol amendment allows for disclosing unblinded data for the first 45 subjects, which we expect within the next 12 months."

The Company is advancing the development of Annamycin in a Phase 3 pivotal trial evaluating AnnAraC for the treatment of AML patients who are refractory to or relapsed after induction therapy (R/R AML) (MB-108). This Phase 3 "MIRACLE" trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) will be a global trial, including sites in the US. The Company remains on track to initiate patient treatment in the first quarter of 2025.

Annamycin currently has Fast Track Status and Orphan Drug Designation from the FDA for the treatment of relapsed or refractory acute myeloid leukemia, in addition to Orphan Drug Designation for the treatment of soft tissue sarcoma. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia from the European Medicines Agency (EMA).