On October 17, 2022 Fennec Pharmaceuticals Inc. (NASDAQ: FENC; TSX: FRX), a commercial stage specialty pharmaceutical company, reported the U.S. commercial launch and availability of PEDMARK (sodium thiosulfate injection) to reduce the risk of ototoxicity associated with cisplatin in pediatric patients one month of age and older with localized, non-metastatic solid tumors (Press release, Fennec Pharmaceuticals, OCT 17, 2022, View Source [SID1234622058]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are proud to announce that PEDMARK is now available to pediatric patients and their physicians who are in need of options to help reduce the risk of cisplatin-induced hearing loss," said Rosty Raykov, chief executive officer of Fennec Pharmaceuticals. "Children who get platinum-based chemotherapy, such as cisplatin, are at risk for permanent hearing loss, a condition that previously could only be managed with cochlear implants and hearing aids. PEDMARK provides the first and only FDA-approved treatment specifically designed to help protect hearing in children and young adults after receiving cisplatin. We look forward to engaging with healthcare providers and supporting patient access to this breakthrough therapy with our assistance program, Fennec HEARS."

The Company has established Fennec HEARS, a comprehensive single source program designed to connect PEDMARK patients to both patient financial and product access support. The program offers assistance and resources, regardless of insurance type, that can address co-pays or lack of coverage when certain eligibility requirements are met. Fennec HEARS also provides access to care coordinators that can answer insurance questions about coverage for PEDMARK and provide tips and resources for managing treatment. For more information about product availability and patient support, please contact the Fennec HEARS program at 1-833-7PEDMARK (1-833-773-3627).

PEDMARK was approved by the U.S. Food and Drug Administration (FDA) in September 2022 based upon safety and efficacy data from two pivotal open-label, randomized Phase 3 trials (Clinical Oncology Group [COG] Protocol ACCL0431 and SIOPEL 6), which compared PEDMARK plus cisplatin-based regimen to cisplatin-based regimens alone for the reduction of cisplatin-induced hearing loss in pediatric patients.

Advances in chemotherapy-based treatment approaches for pediatric patients with localized, solid tumors have improved, resulting in an 85 percent or higher five-year survival rate for these patients.1 However, use of platinum-based chemotherapy, still the treatment of choice in many cases, can be toxic to the ears and cisplatin treatment frequently causes permanent and irreversible bilateral (affecting both ears) hearing loss. Permanent hearing loss can be seen in approximately 60 percent of children treated with cisplatin and can be as high as 90 percent.1,2 Until now, interventions with management strategies such as cochlear implants and hearing aids only occurred after hearing loss had been detected and these interventions do not return normal hearing.3

The Marketing Authorization Application (MAA) for sodium thiosulfate (tradename PEDMARQSI) is currently under evaluation by the European Medicines Agency (EMA). Prior to its approval in the U.S., the FDA granted PEDMARK Priority Review designation in 2020 and Orphan Drug designation in 2004.

About Cisplatin-Induced Ototoxicity
Cisplatin and other platinum compounds are essential chemotherapeutic agents for the treatment of many pediatric malignancies. Unfortunately, platinum-based therapies can cause ototoxicity, or hearing loss, which is permanent, irreversible, and particularly harmful to the survivors of pediatric cancer.4

The incidence of ototoxicity depends upon the dose and duration of chemotherapy, and many of these children require lifelong hearing aids or cochlear implants, which can be helpful for some, but do not reverse the hearing loss and can be costly over time.5 Infants and young children that are affected by ototoxicity at critical stages of development lack speech and language development and literacy, and older children and adolescents often lack social-emotional development and educational achievement.6

PEDMARK (sodium thiosulfate injection)
PEDMARK is the first and only U.S. Food and Drug Administration (FDA) approved therapy indicated to reduce the risk of ototoxicity associated with cisplatin treatment in pediatric patients with localized, non-metastatic, solid tumors. It is a unique formulation of sodium thiosulfate in single-dose, ready-to-use vials for intravenous use in pediatric patients.7 PEDMARK is also the only therapeutic agent with proven efficacy and safety data with an established dosing paradigm, across two open-label, randomized Phase 3 clinical studies, the Clinical Oncology Group (COG) Protocol ACCL0431 and SIOPEL 6.

In the U.S. and Europe, it is estimated that, annually, more than 10,000 children may receive platinum-based chemotherapy. The incidence of ototoxicity depends upon the dose and duration of chemotherapy, and many of these children require lifelong hearing aids. There is currently no established preventive agent for this hearing loss and only expensive, technically difficult, and sub-optimal cochlear (inner ear) implants have been shown to provide some benefit. Infants and young children that suffer ototoxicity at critical stages of development lack speech language development and literacy, and older children and adolescents lack social-emotional development and educational achievement.

PEDMARK has been studied by co-operative groups in two Phase 3 clinical studies of survival and reduction of ototoxicity, COG ACCL0431 and SIOPEL 6. Both studies have been completed. The COG ACCL0431 protocol enrolled childhood cancers typically treated with intensive cisplatin therapy for localized and disseminated disease, including newly diagnosed hepatoblastoma, germ cell tumor, osteosarcoma, neuroblastoma, medulloblastoma, and other solid tumors. SIOPEL 6 enrolled only hepatoblastoma patients with localized tumors.

Indications and Usage
PEDMARK (sodium thiosulfate injection) is indicated to reduce the risk of ototoxicity associated with cisplatin in pediatric patients 1 month of age and older with localized, non-metastatic solid tumors.

Limitations of Use
The safety and efficacy of PEDMARK have not been established when administered following cisplatin infusions longer than 6 hours. PEDMARK may not reduce the risk of ototoxicity when administered following longer cisplatin infusions, because irreversible ototoxicity may have already occurred.

Important Safety Information
PEDMARK is contraindicated in patients with history of a severe hypersensitivity to sodium thiosulfate or any of its components.

Hypersensitivity reactions occurred in 8% to 13% of patients in clinical trials. Monitor patients for hypersensitivity reactions. Immediately discontinue PEDMARK and institute appropriate care if a hypersensitivity reaction occurs. Administer antihistamines or glucocorticoids (if appropriate) before each subsequent administration of PEDMARK. PEDMARK may contain sodium sulfite; patients with sulfite sensitivity may have hypersensitivity reactions, including anaphylactic symptoms and life-threatening or severe asthma episodes. Sulfite sensitivity is seen more frequently in people with asthma.

PEDMARK is not indicated for use in pediatric patients less than 1 month of age due to the increased risk of hypernatremia or in pediatric patients with metastatic cancers.

Hypernatremia occurred in 12% to 26% of patients in clinical trials, including a single Grade 3 case. Hypokalemia occurred in 15% to 27% of patients in clinical trials, with Grade 3 or 4 occurring in 9% to 27% of patients. Monitor serum sodium and potassium levels at baseline and as clinically indicated. Withhold PEDMARK in patients with baseline serum sodium greater than 145 mmol/L.

Monitor for signs and symptoms of hypernatremia and hypokalemia more closely if the glomerular filtration rate (GFR) falls below 60 mL/min/1.73m2.

Administer antiemetics prior to each PEDMARK administration. Provide additional antiemetics and supportive care as appropriate.

The most common adverse reactions (≥25% with difference between arms of >5% compared to cisplatin alone) in SIOPEL 6 were vomiting, nausea, decreased hemoglobin, and hypernatremia. The most common adverse reaction (≥25% with difference between arms of >5% compared to cisplatin alone) in COG ACCL0431 was hypokalemia.

On October 17, 2022 Evotec SE (Frankfurt Stock Exchange: EVT, MDAX/TecDAX, ISIN: DE0005664809; NASDAQ: EVO) and Toronto Innovation Acceleration Partners ("TIAP") reported that the two companies have expanded LAB150, their translational BRIDGE partnership, to include Amgen as a strategic partner (Press release, Evotec, OCT 17, 2022, View Source [SID1234622057]). The expansion goes along with a combined investment of US$ 14 m to expedite LAB150 programmes towards the formation of new companies.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

LAB150 was created by Evotec and TIAP in 2017 to accelerate Toronto’s academic research into market-ready products. The expanded agreement builds upon existing partnerships between Evotec, TIAP, and Amgen to support the development of disruptive therapeutics by TIAP’s member base and draws upon Evotec’s industrialised drug discovery platforms. Amgen will provide financial support for chosen LAB150 projects along with significant mentorship from their drug discovery and development teams. In addition, Amgen Ventures will evaluate LAB150-derived companies for venture investment. These combined efforts will amplify the efficiency and translational potential of academic research to develop Canadian intellectual property, novel therapies, and accelerate commercialisation by Canada’s next generation of life science companies.

"As our strategic partner since 2019, Amgen has worked closely with TIAP, and we are thankful for their continued support as we drive the commercialisation of breakthrough Canadian innovations. Together with Evotec, we look forward to collaborating with Amgen to bring enhanced expertise and capital to LAB150 to enable life science company creation," said Parimal Nathwani, President and CEO of TIAP. "To have two globally recognised industry leaders, Amgen and Evotec, come together under the LAB150 umbrella bodes very well for the life science community in Canada and improving global health."

According to Philip Tagari, Vice President of Research at Amgen, "Amgen’s ongoing four-year partnership with TIAP to support very early innovation in the Greater Toronto biotechnology ecosystem has revealed both the unique expertise within TIAP to curate novel projects with significant future medical and commercial potential, as well as the outstanding quality of the biomedical research performed in the network of world-class institutions that LAB150 accesses. This multi-year investment, in partnership with the long-term collaborators at Evotec, will provide a streamlined path for cutting-edge academic science in Ontario to transition into early-stage company formation and identification of novel candidates for clinical development, with the intent of providing meaningful medicines for patients with grievous illness."

Dr Thomas Hanke, EVP Head of Academic Partnerships at Evotec, added: "The LAB150 BRIDGE partnership has demonstrated its ability to successfully identify and validate high-potential therapeutic projects and drive them towards the formation of new companies. We very much look forward to accelerating our efforts further by leveraging Amgen’s impressive track record in the drug development space across therapeutic areas as well as in the formation of successful life science companies."

With over $ 7 m invested, more than 150 projects evaluated, and ten projects currently being supported, LAB150 has established a strong track record. Having Amgen as a new partner in this programme will ensure a robust 360-degree selection process and scale up LAB150’s capital-efficient investments to fast-track therapeutics. Moreover, the symbiotic partnership between the three partners around LAB150 will bring crucial expertise to the development and financing of emerging technologies.

On October 17, 2022 Gilead Sciences, Inc. (NASDAQ: GILD) and MacroGenics (NASDAQ: MGNX) reported an exclusive option and collaboration agreement to develop MGD024, an investigational, bispecific antibody that binds CD123 and CD3 using MacroGenics’ DART platform, and two additional bispecific research programs (Press release, MacroGenics, OCT 17, 2022, View Source [SID1234622055]). The collaboration agreement grants Gilead the option to license MGD024, a potential treatment for certain blood cancers, including acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

A leader in the bispecific antibody space, MacroGenics has extensive experience applying its proprietary DART platform to develop novel therapeutics. MGD024 is a next-generation, bispecific that incorporates a CD3 component that is designed to minimize cytokine-release syndrome (CRS), a potentially life-threatening toxicity, while increasing the magnitude of antitumor activity with a longer half-life to permit intermittent dosing.

"MacroGenics’ bispecific expertise naturally complements Gilead’s portfolio strengths in immuno-oncology and our growing hematology franchise," said Bill Grossman, MD, PhD, Senior Vice President, Oncology Clinical Development, Gilead Sciences. "We believe MGD024, with its potential to reduce CRS and permit intermittent dosing through a longer half-life, could translate to more patient-friendly dosing and enhanced clinical outcomes for people living with AML and MDS. This partnership is the latest in our efforts to develop and advance transformative new cancer therapies as we deepen our portfolio across oncology indications."

Scott Koenig, MD, PhD, President, and CEO, MacroGenics said, "Rapid advances over the last decade have made CD123 a very promising target in oncology research. Advancing our bispecific DART molecule, MGD024, through a strategic collaboration with the team at Gilead will accelerate our ability to drive further development of MGD024 to the potential benefit of people living with blood cancers."

MacroGenics will be responsible for the ongoing Phase 1 study for MGD024 during which Gilead may elect to exercise its option to license the program at predefined decision points. The Phase 1 study will include a dose escalation segment and an expansion segment that is intended to evaluate MGD024 as monotherapy and in combination with other therapies across multiple indications.

Financial Considerations

As part of the agreement, Gilead will pay MacroGenics an upfront payment of $60 million and MacroGenics will be eligible to receive up to $1.7 billion in target nomination, option fees, and development, regulatory and commercial milestones. MacroGenics will also be eligible to receive tiered, double-digit royalties on worldwide net sales of MGD024 and a flat royalty on worldwide net sales of products under the two research programs.

Beginning in the first quarter of 2022, consistent with recent industry communications from the U.S. Securities and Exchange Commission (SEC), Gilead no longer excludes acquired IPR&D expenses from its non-GAAP financial measures and expects the transaction with MacroGenics to reduce Gilead’s GAAP and non-GAAP 2022 EPS by approximately $0.04.

On October 17, 2022 Kineta, Inc. ("Kineta" or the "Company"), a clinical-stage biotechnology company focused on the development of novel immunotherapies in oncology, reported that it has entered into a clinical trial collaboration and supply agreement with Merck (known as MSD outside the U.S. and Canada) (Press release, Kineta, OCT 17, 2022, View Source;utm_medium=rss&utm_campaign=kineta-announces-clinical-collaboration-with-merck-to-evaluate-kva12123-in-combination-with-keytruda-pembrolizumab-in-cancer-patients-with-advanced-solid-tumors [SID1234622054]). Under this collaboration, Kineta will evaluate the safety, tolerability, pharmacokinetics and anti-tumor activity of KVA12123 (formerly KVA12.1), its novel anti-VISTA monoclonal antibody, alone and in combination with KEYTRUDA (pembrolizumab), Merck’s anti-PD-1 therapy, in patients with advanced solid tumors.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

VISTA is a key driver of immunosuppression in the tumor microenvironment. VISTA is up-regulated after checkpoint inhibitor therapy that has been associated with treatment failure and poor outcomes in cancer patients. KVA12123 is a fully human engineered IgG1 monoclonal antibody that was designed to bind to VISTA through a unique epitope and at physiologic and acidic pH levels. In preclinical studies, KVA12123 demonstrated promising anti-tumor activity both as a single agent and in combination with PD-1 checkpoint inhibitor therapy.

"Treatment with PD-1 inhibitors as a monotherapy or in combination with other treatments has become a recommended first-line treatment and has dramatically improved outcomes for many cancer patients. However, for patients who failed to achieve a response or who relapse, treatment options are limited. We believe that KVA12123 is a potential best-in-class VISTA blocking immunotherapy that may drive strong anti-tumor efficacy and deliver a highly differentiated product profile across a range of solid tumors," said Shawn Iadonato, Ph.D., Chief Executive Officer of Kineta. "We are excited to enter into this collaboration with Merck to evaluate KVA12123 in combination with KEYTRUDA in cancer patients later this year."

Kineta is planning to conduct a Phase 1/2 clinical study evaluating KVA12123 as a single agent and in combination with KEYTRUDA in patients with advanced solid tumors. The objectives of the study are to evaluate the safety, tolerability, pharmacokinetics and anti-tumor responses of KVA12123 alone and in combination with KEYTRUDA with interim results anticipated in late 2023. Kineta is responsible for conducting this study, which it expects to initiate in the fourth quarter of 2022.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.

On October 12, 2022 ANDARIX Pharmaceuticals, a leader in the discovery and development of targeted peptide therapy for cancer reported that it will present its technology for treating lung cancer patients at a clinical provider conference (Press release, Andarix Pharmaceuticals, OCT 12, 2022, View Source [SID1234622053]). The conference will focus on the broad array of clinical trial technologies and provider capabilities. The technology will be presented at the OCT New England Conference in Boston, Massachusetts on October 12-13, 2022.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

About Tozaride
Tozaride is a novel, best-in-class cancer therapy based on a radio-labeled somatostatin peptide analogue. Early clinical studies of Tozaride demonstrated that it is well tolerated and may produce prolonged stable disease and improved overall survival in advanced lung cancer patients whose disease has continued to progress after failing other therapies. Tozaride targeted radiotherapy represents a new treatment paradigm which is expected to yield significant clinical benefit for both lung cancer (SCLC, NSCLC), and pancreatic cancer patients. Along with its companion diagnostic that helps identify patients most likely to respond – those with sufficient expression of the peptide’s target – Tozaride could provide another treatment option for patients who are not eligible for, or who have not responded to current therapies.