On September 24, 2024 pharmaand GmbH (pharma&) and Tolmar, Inc. (Tolmar) reported an exclusive agreement for Tolmar to promote Rubraca (rucaparib) in the U.S. for the treatment of metastatic castration-resistant prostate cancer (mCRPC). pharma& will continue to promote Rubraca in the U.S. and Europe in approved indications for advanced ovarian cancer.

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Under the terms of the Agreement, Tolmar will be responsible for U.S. marketing and sales activities to promote Rubraca in mCRPC. pharma& will be responsible for U.S. supply of product and regulatory responsibilities, including product labeling for Rubraca in mCRPC and retains the global rights to Rubraca.

"We are pleased to have finalized our Agreement with Tolmar to provide commercial support for Rubraca in the U.S. for the treatment of metastatic castration-resistant prostate cancer," said Frank Rotmann, Founder and Managing Director of pharma&. "We are confident that partnering with Tolmar will further expand the use of Rubraca to eligible patients with mCRPC."

"We are excited to add Rubraca to our Prostate Cancer Portfolio in the U.S.," said Anil D’Souza, CEO of Tolmar, Inc. "Along with our flagship advanced prostate cancer treatment Eligard, we are now building on that foundation of care with Rubraca. Tolmar is proud of its dedication to patients, healthcare providers, and the Urology-Oncology community. We look forward to providing Rubraca as an option for eligible men with mCRPC."

Rubraca (rucaparib) U.S. Prostate Cancer FDA Approved Indication

Rubraca is indicated for the treatment of adult patients with a deleterious BRCA mutation (germline and/or somatic)-associated metastatic castration-resistant prostate cancer (mCRPC) who have been treated with androgen receptor-directed therapy

and a taxane-based chemotherapy. This indication is approved under accelerated approval based on objective response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Select Important Safety Information

Myelodysplastic Syndrome (MDS)/Acute Myeloid Leukemia (AML) have occurred in patients treated with Rubraca, and are potentially fatal adverse reactions. In TRITON2, MDS/AML was not observed in patients with mCRPC (n=209) regardless of homologous recombination deficiency (HRD) mutation.

Do not start Rubraca until patients have recovered from hematological toxicity caused by previous chemotherapy (≤ Grade 1). Monitor complete blood counts for cytopenia at baseline and monthly thereafter for clinically significant changes during treatment. For prolonged hematological toxicities (> 4 weeks), interrupt Rubraca or reduce dose and monitor blood counts weekly until recovery. If the levels have not recovered to Grade 1 or less after 4 weeks or if MDS/AML is suspected, refer the patient to a hematologist for further investigations, including bone marrow analysis and blood sample for cytogenetics. If MDS/AML is confirmed, discontinue Rubraca.

Based on its mechanism of action and findings from animal studies, Rubraca can cause fetal harm when administered to a pregnant woman. Apprise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment and for 6 months following the last dose of Rubraca.

For males on Rubraca treatment who have female partners of reproductive potential or who are pregnant, effective contraception should be used during treatment and for 3 months following the last dose of Rubraca. Advise male patients on Rubraca treatment, who have female partners of reproductive potential or who are pregnant to use effective contraception during treatment and for 3 months following the last dose of Rubraca.

Most common adverse reactions in patients with BRCA-mutated mCRPC in TRITON2 (≥ 20%; Grade 1-4) were fatigue/asthenia (62%), nausea (52%), anemia (43%), AST/ALT elevation (33%), decreased appetite (28%), rash (27%), constipation (27%), thrombocytopenia (25%), vomiting (22%), and diarrhea (20%).

Co-administration of rucaparib can increase the systemic exposure of CYP1A2, CYP3A, CYP2C9, or CYP2C19 substrates, which may increase the risk of toxicities of these drugs. Adjust dosage of CYP1A2, CYP3A, CYP2C9, or CYP2C19 substrates, if clinically indicated. If co-administration with warfarin (a CYP2C9 substrate) cannot be avoided, consider increasing frequency of international normalized ratio (INR) monitoring.

Because of the potential for serious adverse reactions in breast-fed children from Rubraca, advise lactating women not to breastfeed during treatment with Rubraca and for 2 weeks after the last dose.

Please Click here for full Prescribing Information for Rubraca.

For medical information inquiries within the U.S., contact pharma& at [email protected].

You may report adverse events to the FDA at 1‑800‑FDA‑1088 or www.fda.gov/medwatch.

Alternatively, to report an adverse event or reaction, contact pharma& at [email protected].

To report a product complaint, contact pharma& at [email protected].

Eligard (leuprolide acetate) for injectable suspension Indication

Eligard is used for the palliative treatment of advanced prostate cancer. Eligard is a prescription medication that must be administered by a health care professional.

Important Safety Information

Eligard should not be used by anyone who is allergic to any of the ingredients in Eligard or to any similar drugs.

Eligard causes an increase in testosterone during the first few weeks of therapy and some men may experience new or worsening symptoms of prostate cancer e.g., bone pain, urinary symptoms, or nerve problems such as numbness, during this period. If your cancer has spread to the urinary tract or spine, urinary blockage or pressure on the spine that can lead to paralysis may occur. Your doctor will discuss with you the benefits and risks of taking Eligard.

Increased risk of heart attack, sudden death due to heart problems and stroke have also been reported in men taking Eligard. Eligard may also affect electrical activity in the heart that can cause an irregular heartbeat. Your doctor will monitor you for heart conditions.

Elevated blood sugar and an increased risk of developing diabetes have been reported in men receiving Eligard. Your doctor will monitor blood sugar levels.

Convulsions have been observed in patients taking leuprolide acetate, including patients who have a history of seizures, epilepsy, or brain disorders (related to blood vessels, nerves, or tumors), and in those taking medications associated with convulsions. Convulsions have also been reported in patients without any of these conditions.

Eligard may cause fetal harm when administered to a pregnant woman. Expected hormonal changes that occur with Eligard treatment increase the risk for pregnancy loss.

Eligard may impair fertility in males of reproductive potential. The most common injection site reactions are transient burning and stinging, pain, bruising, and redness. The most common side effects include hot flashes/sweats, fatigue, weakness, muscle pain, dizziness, clamminess, testicular shrinkage, decreased erections and enlargement of breasts.

Other side effects, including thinning of bones that may lead to fracture, and rare but serious problems with the pituitary gland in the brain, have been reported with Eligard.

Call your doctor for medical advice about side effects. You may report side effects to the FDA.

Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Please Click here for full Prescribing Information for Eligard

For medical information inquiries within the U.S., contact Tolmar at [email protected]

You may report adverse events to the FDA at 1‑800‑FDA‑1088 or www.fda.gov/medwatch

Alternatively, to report an adverse event or reaction or to report a product complaint, contact Tolmar at 1-844-4TOLMAR

(Press release, pharmaand, SEP 24, 2024, View Source [SID1234661571])

On September 24, 2024 Shanghai Junshi Biosciences Co., Ltd (Junshi Biosciences, HKEX: 1877; SSE: 688180), a leading innovation-driven biopharmaceutical company dedicated to the discovery, development, and commercialization of novel therapies, and its wholly-owned subsidiary, TopAlliance Biosciences Inc. (TopAlliance Biosciences), reported that the European Commission (EC) has approved toripalimab (European trade name: LOQTORZI) for the treatment of two indications (Press release, Shanghai Junshi Bioscience, SEP 24, 2024, View Source [SID1234656134]):

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Toripalimab in combination with cisplatin and gemcitabine for the first-line treatment of adult patients with recurrent, not amenable to surgery or radiotherapy, or metastatic nasopharyngeal carcinoma (NPC);
Toripalimab in combination with cisplatin and paclitaxel for the first-line treatment of adult patients with unresectable advanced, recurrent, or metastatic oesophageal squamous cell carcinoma (ESCC).
In July, a positive opinion was issued by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) for the marketing authorization application (MAA) of these two indications. This approval applies to all 27 member states of the European Union, Iceland, Norway and Liechtenstein, making toripalimab the first and only drug in Europe for the treatment of NPC and the only first-line treatment for advanced or metastatic ESCC, regardless of PD-L1 status.

NPC is a malignant tumor that occurs in the nasopharyngeal mucosal epithelium and is one of the most common types of head and neck cancers globally. According to GLOBOCAN 2022 statistics, the number of newly diagnosed NPC cases in 2022 exceeded 120,000 worldwide. Due to the location of the primary tumor, surgery is rarely an option. The latest European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Guidelines recommend immunotherapy combined with chemotherapy as the first-line treatment for recurrent or metastatic NPC.

The approval of the NPC indication is primarily based on the results from the JUPITER-02 study (a randomized, double-blind, placebo-controlled, multinational multi-center Phase III clinical study, NCT03581786). The JUPITER-02 study is the first international multi-center, double-blind, randomized Phase III clinical study in the field of immunotherapy for NPC with the largest sample size, and the world’s first Phase III clinical study with preset statistical verification (Type I error control) for Overall Survival ("OS") for first-line immunotherapy combined with chemotherapy for NPC compared to chemotherapy alone and demonstrated a survival benefit. The study results were presented in an oral report during the Plenary Session of the 2021 annual meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) (#LBA2) and were subsequently featured on the cover of Nature Medicine. The results were also published in full in the Journal of the American Medical Association (JAMA). The results showed that, compared to chemotherapy alone, toripalimab in combination with chemotherapy reduced the risk of disease progression by 48% and the risk of death by 37%. The median progression-free survival ("PFS") in the toripalimab plus chemotherapy group was prolonged by 13.2 months compared to chemotherapy alone, from 8.2 months to 21.4 months. In addition, patients treated with this combined therapy achieved a higher objective response rate (ORR), longer duration of response (DoR), and higher disease control rate (DCR), and no new safety signal was identified. Long-term survival follow-up data, presented at ASCO (Free ASCO Whitepaper) 2024, reported a 5-year survival rate of 52.0%.

EC is one of the most common malignant tumors in the alimentary tract. According to GLOBOCAN 2022 statistics, esophageal cancer is the 11th most commonly diagnosed cancer and the seventh leading cause of cancer death worldwide, with over 511,000 new cases and over 445,000 deaths in 2022. ESCC and esophageal adenocarcinoma are the two main histological subtypes of esophageal cancer. The ESMO (Free ESMO Whitepaper) Guidelines recommend PD-1 blocking antibodies combined with chemotherapy for the treatment of patients with advanced or metastatic ESCC with PD-L1 positive status.

The approval of the ESCC indication is primarily based on the results from the JUPITER-06 study (a randomized, double-blind, placebo-controlled, multi-center Phase III clinical study, NCT03829969). The study aimed to evaluate the efficacy and safety of toripalimab in combination with paclitaxel/cisplatin (TP) for the first-line treatment of advanced ESCC compared with placebo in combination with chemotherapy. The results were first presented in an oral session during the ESMO (Free ESMO Whitepaper) Congress 2021 and later published in Cancer Cell and Journal of Clinical Oncology, two leading international oncology journals. The results of the study showed that toripalimab in combination with chemotherapy resulted in superior PFS and OS in patients with advanced or metastatic ESCC, the median OS was prolonged by 6 months to 17 months and the risk of disease progression or death in patients was significantly reduced by 42%. Futhermore, there was significant improvement in survival benefits regardless of PD-L1 status.

Professor Ruihua XU, Principal Investigator and President of Sun Yat-sen University Cancer Center, said, "Both NPC and EC are highly prevalent in Asia, while the development of innovative therapies for these cancer types has been slow in Europe and the Americas. The outstanding results from the JUPITER-02 and JUPITER-06 studies reflect the pioneering leadership of Chinese researchers in the diagnosis, treatment, and clinical research of NPC and EC. We hope that this ‘Chinese Solution’ will truly transform the outlook for patients around the world who have long lacked effective treatment options for these cancers, and bring them renewed hope for survival!"

Dr. Jianjun ZOU, General Manager and CEO of Junshi Biosciences, said, "‘In China, For Global’ has been a core strategic goal of Junshi Biosciences since its inception. The approval of toripalimab by the EC signifies that, following our success in China and the US, our global commercial strategy has officially expanded into Europe. It also reflects the international recognition of our research and production quality for innovative drugs. Moving forward, we will continue to collaborate with our partners on the commercialization of toripalimab in Europe, and provide high-quality, innovative therapies from China to more patients worldwide."

Dr. Patricia Keegan, Chief Medical Officer of TopAlliance Biosciences, said, "Junshi Biosciences and TopAlliance Biosciences are dedicated to producing innovative therapies that offer survival benefits to patients around the world while consistently addressing the clinical needs of local populations. This approval represents another significant milestone in our entry into the global market. In addition to toripalimab, we have several promising indications and drugs under development internationally. We believe that our commitment to providing patients with more effective treatment options will continually motivate us toward becoming a leading international innovative enterprise."

On September 24, 2024 Araris Biotech AG ("Araris"), a Swiss oncology biotech company developing next-generation antibody drug conjugates (ADCs), reported it has entered into an Agreement with Innate Pharma ("Innate") (Press release, Araris Biotech, SEP 24, 2024, View Source [SID1234651284]). Under the agreement, Innate will assign its portfolio of patents related to its ADC transglutaminase conjugation technology to Araris.

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"We are excited to acquire Innate’s portfolio of transglutaminase related patents, as this transaction further positions Araris as a leader in the development of ADCs using transglutaminase conjugation technology," said Dragan Grabulovski, Ph.D., CEO and co-founder of Araris. "We look forward to continuing to develop next-generation sitespecific ADCs and believe this acquisition not only expands our intellectual property portfolio, but also strengthens our competitive edge."

The newly acquired patents encompass a broad range of intellectual property that cover use of bacterial transglutaminase in conjugating various linker-payloads to antibodies.

On September 24, 2024 Generate:Biomedicines ("Generate") reported a multi-target collaboration with Novartis (NYSE: NVS) to discover and develop protein therapeutics across multiple disease areas (Press release, Generate Biomedicines, SEP 24, 2024, https://generatebiomedicines.com/media-center/generatebiomedicines-announces-multi-target-collaboration-with-novartis [SID1234650142]). The collaboration leverages Generate’s proprietary generative AI platform, ​"The Generate Platform," to create potentially first- and best-in-class molecules through AI-based optimization and de novo generation.

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The collaboration will combine The Generate Platform, which integrates machine learning with high-throughput experimental validation, with Novartis expertise and capabilities in target biology, biologics development, and clinical development to create novel therapeutics and to accelerate the pace of drug discovery and development.

"We are delighted to collaborate with Generate to explore the promise of generative AI in enhancing and accelerating the discovery of next-generation biologics," said Fiona Marshall, President of Biomedical Research at Novartis. ​"This collaboration offers an opportunity to leverage the unique strengths of our respective companies, from target biology and biologics discovery to machine learning/​AI and clinical development, in order to bring forward new medicines with transformative potential for patients."

"Partnering with a world-leading drug discovery and development organization like Novartis allows us to broaden the use of our cutting-edge generative biology platform to tackle even more areas of unmet medical need," said Mike Nally, Chief Executive Officer of Generate:Biomedicines. ​"We look forward to working closely with the team at Novartis to continue to demonstrate the transformative potential of programming biology to create better medicines for patients, faster."

Under the terms of the collaboration agreement, Generate will receive a total upfront payment of $65 million in cash from Novartis, which includes $15 million for the purchase of equity in Generate. Generate is also eligible to receive more than $1 billion in performance-based milestone payments, in addition to tiered royalties up to low double-digits. The number of targets and therapeutic areas are not being disclosed.

On September 24, 2024 Zetagen Therapeutics, a private, clinical-stage, biopharmaceutical company developing breakthrough therapies via local administration for metastatic and primary cancers to bone and other organs, reported publication of clinical data in the peer-reviewed Journal of the American Academy of Orthopaedic Surgeons (JAAOS) on ZetaFuse (Zeta-ZF-002)for the treatment of multi-level DDD in a Stage 4 lung cancer patient (Press release, Zetagen Therapeutics, SEP 24, 2024, View Source [SID1234647538]).

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"We are seeing promising consistency between our pre-clinical and clinical data with our ‘Zeta’ oncology platform," said Joe C. Loy, Chief Executive Officer of Zetagen. "This new data published in JAAOS further validates that our drugs in development with our proprietary formulations are producing the desired outcomes."

JAAOS Report Overview

The report, entitled Treating Multi-level Cervical Degenerative Disc Disease (DDD) in a Stage IV Lung Cancer Patient with Significant Comorbidities Using a Breakthrough Therapy focused on a 64-year-old, Stage 4, non-small cell lung cancer (NSCLC) patient with cervical DDD at three levels of the spine. The patient has several comorbidities which includes obesity and long-term smoking.

Due to a recent work-related accident which resulted in degenerative changes in the neck, the patient was referred for surgical consultation. Under an FDA Expanded Access protocol, (Compassionate Use) ZetaFuse (Zeta-ZF-002) was administered to promote bone formation in the patient’s three-level ACDF procedure. Despite the compromised health, ongoing chemotherapy treatments and poor bone physiology of the patient, case study results exceeded expectations, confirming radiographic fusion at all three cervical levels by eight months.

"Knowing the patient had been treated previously for his lung cancer with cytotoxic therapy and immunotherapy, which are known to negatively impact bone healing and, in spite of extensive exposure to these agents over the last three (3) years, our patient showed an excellent bone healing response to the novel, drug eluting biomaterial of the ZetaFuse," said Pedro Sanz-Altamira, MD, PhD, hematologist and oncology specialist at Dana Farber Cancer Institute who has been treating the case study patient for several years.

"Spine surgeons have no approved resources to treat cancer patients which have such orthopedic fusion needs, and we are pleased that this patient has seen outcomes which surpassed expectations in such a short period of time," said Nikhil Thakur, MD, Chief Medical Officer of Zetagen and Orthopaedic spine surgeon in Boston, MA.

View the JAAOS publication via open access here.

A New Approach to Treating Metastatic Cancer

Zetagen is dedicated to developing breakthrough therapies via local administration for metastatic and primary cancers to bone and other organs which may provide increased survival rates. The Company’s proprietary "Zeta" oncology platform is based on a novel, opioid growth factor receptor (OGFR) antagonist pathway which targets the management of the p21 transcript.

ZetaFuse (Zeta-ZF-002), the focus of the JAAOS publication, shares the same mechanism of action as Zetagen’s lead oncology drug candidate, ZetaMet (Zeta-BC-003), and were awarded separate Breakthrough Designations by the U.S. Food and Drug Administration (FDA) based upon their clinical indication. ZetaMet (Zeta-BC-003) is a synthetic, small-molecule, developed via a proprietary control release carrier developed to resolve metastatic breast cancer bone lesions, inhibiting pain while regenerating bone, with the potential to increase survival rates.

Peer-reviewed 2-year follow up data published in 2023 on ZetaMet (Zeta-BC-003) demonstrated resolution of lytic lesions, significant reduction in pain, prevention of vertebral fracture, and increased survival rate in a patient living with Stage 4 breast cancer.[i]

This patient had two groups of spinal lesions, a total of seven spinal lesions in all, one-group had two advanced lesions (T7 & L1) treated initially with fractionated radiation and when the cancer and pain returned, treated via an outpatient procedure with ZetaMet (Zeta-BC-003). Sixty (60) days later a second group of five new lesions appeared on the patient’s sacrum. She decided to forego fractionated radiation, the FDA approved a second procedure with ZetaMet (Zeta-BC-003), patient experienced significant reduction in pain (4x), and nine (9) months later independent radiologist reports showed no active tumor, no skeletal related events (SREs- fractures) in either lesion group, and the patient experienced complete resolution with the second lesion group with an increased survival rate of 36 months. View this publication via open access here.

"I am delighted to see these results being shared with our peers in scientific community," said Bryan Margulies, PhD, Chief Scientific Officer of Zetagen. "These observations build off of the extensive preclinical data we have generated over several years and we look forward to further advancing our ongoing human clinical trials as we continue to develop the ‘Zeta’ platform for patients living with metastatic and primary cancers."

[i] Pain Management. Volume 13, Issue 10, October 2023, Pages 569-577 View Source