On September 12, 2023 XENOTHERA reported that R&D teams have demonstrated, in vitro and in vivo, the efficacy of LIS1 treatment on several T lymphomas (Press release, Xenothera, SEP 12, 2023, View Source [SID1234635110]). Given the heterogeneity of this disease, XENOTHERA’s researchers have explored the potential of LIS1 on different subtypes and shown that up to 90% of patients’ tumors are likely to be sensitive to LIS1. XENOTHERA’s antibody thus represents a new therapeutic hope for T lymphoma patients, whose prognosis is particularly unfavorable.

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Orphan drug designation is a major step, as formal confirmation by a regulatory agency; by its decision, the FDA affirms the therapeutic interest of a future drug for patients for whom no treatment is available. In addition to this external confirmation of the biotech’s results and strategy, the ODD will enable XENOTHERA’s LIS1 to benefit from free and accelerated regulatory processes, tax advantages and a 7-year marketing exclusivity once the market authorization is obtained.

"Recognition of the quality and therapeutic value of a drug candidate is always an important milestone for a biotech. Here, the analysis of our data by the FDA confirms the soundness of our strategy in the field of cancer, onco-hematology for LIS1, and solid tumors for XON7. We are more than happy to bring new hope to T lymphoma patients, for whom the treatments available today are unfortunately of limited efficacy. LIS1 will be an additional weapon in the arsenal of treatments available to hematologists, who are keenly interested in innovative therapies such as XENOTHERA’s. The first clinical trial in T-lymphoma is scheduled early 2024; this ODD confirms the importance of moving forward, for the good of patients, and we will put all our energy into making the treatment available to patients as quickly as possible," comments Odile Duvaux, President and co-founder of XENOTHERA.

About T lymphomas:

T lymphomas are a heterogeneous group of pathologies, divided into several categories: primary cutaneous, peripheral lymph node, leukemic and extra-lymph node. Depending on their molecular structure, they are classified into several sub-categories, all belonging to the "non-Hodgkin’s lymphoma" class. Treatment approaches and prognosis vary according to subcategory. In the USA, they account for 15% of non-Hodgkin’s lymphomas, or around 12,000 new cases per year. In France, they are estimated to account for around 1% of all cancers. Their course is generally aggressive. About LIS1: LIS1 is a humanized polyclonal antibody (GH-pAb) from XENOTHERA’s platform, in development since 2014, targeting lymphocyte antigens. It is presented as a solution for intravenous administration. LIS1 was introduced in human in 2019, with a first exploratory clinical trial in kidney transplant patients in Europe, completed in 2022. This trial confirmed the product’s safety. LIS1 first indication is the prevention of acute rejection in solid organ transplantation, for which an ODD has already been granted by the FDA and EMA. T lymphomas represent a second indication for the product. The first LIS1 clinical trial in T lymphoma is scheduled early 2024.

On September 12, 2023 Verastem Oncology presented its corporate presentation (Presentation, Verastem, SEP 12, 2023, View Source [SID1234635109]).

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On September 12, 2023 Perspective Therapeutics, Inc. ("Perspective" or "the Company") (NYSE AMERICAN: CATX), reported the presentation of encouraging early clinical results from an open-label, single-arm, investigator-initiated study investigating the safety and efficacy of [212Pb]VMT-α-NET, ​a targeted alpha therapy (TAT), in patients with metastatic neuroendocrine tumors (NETs) and medullary thyroid carcinomas (Press release, Perspective Therapeutics, SEP 12, 2023, View Source [SID1234635108]). The results were presented at the 36th Annual Congress of the European Association of Nuclear Medicine (EANM), held in Vienna, Austria, from September 9-13, 2023.

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Highlights of the presented results include:

Ten patients who failed at least 1 prior line of standard of care therapy have received [212Pb]VMT-α-NET therapy to date, with initial responses observed in 7 of 9 evaluable patients. Responses were observed across both peptide receptor radionuclide therapy (PRRT)-naïve and PRRT-refractory disease. Of the 10 patients enrolled in the study, 3 presented with gastrointestinal NETs, 5 presented with pancreatic NETs, and 2 presented with medullary thyroid carcinoma. Four patients (1 with gastro-intestinal NETs; 3 with pancreatic NETs) were previously treated with [177Lu]DOTATATE PRRT, one of which also received 3 prior administrations of [225Ac]DOTATATE.

Improvements in patients’ symptoms and quality of life trended strongly positive with consecutive [212Pb]VMT-α-NET doses.

No significant renal or hepatic function adverse events have been observed to date​. Most adverse events were mild and included Grade 1 anemias, alopecia, and fatigue, which usually resolved within 1 week of [212Pb]VMT-α-NET administration. Two patients experienced serious adverse events (SAEs) that were deemed unrelated to [212Pb]VMT-α-NET treatment. One patient who developed myelodysplastic syndromes (MDS) discontinued treatment and the other patient, who was heavily pre-treated, died (patient was deemed not evaluable).

"We have safely administered a total of 25 doses of [212Pb]VMT-α-NET across the 10 patients enrolled in the trial, with two of these patients having received all 4 doses to date," commented Dr. Dharmender Malik, Consultant Nuclear Medicine and PET-CT, MBBS, DNB, FANMB & RSO-II, at the Fortis Memorial research institute (FMRI), Gurugram India. "[212Pb]VMT-α-NET shows encouraging initial efficacy and safety across different neuroendocrine and medullary thyroid tumor types in patients who failed prior lines of therapy, including standard of care [177Lu]DOTATATE, and [225Ac]DOTATATE."

"While still early, these initial high objective response rates are promising in light of the 13% response rate for LUTATHERA (www.novartis.com/us-en/sites/novartis_us/files/lutathera.pdf, Table 8). We are especially encouraged by the responses at low [212Pb]VMT-α-NET activity doses as these are in the starting range of our IND-enabled dose escalation study which is currently screening patients in the US," said Thijs Spoor, Chief Executive Officer at Perspective. "Additionally, these early responses among multiple tumor types suggest the potential to expand into other indications including small cell lung cancers, meningiomas, pheochromocytomas, paragangliomas, and neuroblastomas – especially given the compelling preclinical neuroblastoma [212Pb]VMT-α-NET therapy data presented at EANM on Sunday. We will continue to monitor patients’ progress, and we look forward to providing a more extensive data readout in the upcoming quarters."

About the study

Ten adult subjects with histologically confirmed NETs and metastatic medullary thyroid carcinomas who failed at least one prior line of treatment were treated as part of a compassionate use program. All patients were planned to receive [212Pb]VMT-α-NET peptide at intervals of 8 weeks up to 4 doses or until evidence of radiographic progression, unacceptable toxicity or the patient’s decision to discontinue. ​All patients were to be co-infused with an amino acid solution for renal protection. The primary objective of the study is to evaluate the safety of low doses of [212Pb]VMT-α-NET in this patient population. Secondary assessments will include objective response rate measured by RECIST 1.1 criteria, and the number of patients with treatment-related adverse events as assessed by CTCAE v.4.0​. Both will be measured at 24 months after the last administered dose of [212Pb]VMT-α-NET. Isotope was provided using Perspective’s proprietary VMT-α-GEN generator.

About VMT-α-NET

VMT-α-NET is being developed for the treatment and diagnosis of somatostatin receptor subtype 2 (SSTR2) expressing neuroendocrine tumors, which are a rare and difficult-to-treat type of cancer. The dosed patients were diagnosed with confirmed-advanced somatostatin expressing NETs. In early December 2022, VMT-α-NET, the Company’s lead drug for the treatment of neuroendocrine tumors was administered to patients in India on a compassionate use basis, independent of a planned U.S. trial.

On September 12, 2023 Nuvectis Pharma, Inc. (NASDAQ: NVCT) ("Nuvectis" or the "Company"), a clinical-stage biopharmaceutical company focused on the development of innovative precision medicines for the treatment of serious conditions of unmet medical need in oncology, reported the initiation of a Phase 1a dose escalation clinical trial of NXP900, its novel inhibitor of the SRC/YES1 kinase family ("SFK") (Press release, Nuvectis Pharma, SEP 12, 2023, View Source [SID1234635107]). The study is designed to evaluate the safety, tolerability and pharmacokinetic properties of NXP900 in patients with advanced solid tumors.

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SFKs are aberrantly activated in various cancer types and they are central mediators of various oncogenic processes such as proliferation, survival, cell adhesion, invasion, and angiogenesis. NXP900 is a potent and highly selective SFK inhibitor, including low nanomolar IC50 against YES1 and SRC (0.5nM and 2.4nM, respectively) that demonstrated robust single agent anti-cancer activity against several solid tumor types in preclinical models.

"Advancing NXP900 into its first in human clinical trial is an important milestone for Nuvectis," said Ron Bentsur, Co-Founder, Chairman and Chief Executive Officer of Nuvectis, who added, "We believe that NXP900’s unique mechanism of action, which completely shuts down signaling via SFK, in conjunction with its high target specificity, differentiates it from other multi-kinase inhibitors that also inhibit SFK and provide it with the potential to become the first SFK inhibitor for the treatment of solid tumors."

On September 13, 2023 Nanostring technologies presented its corporate presentation (Presentation, NanoString Technologies, SEP 12, 2023, View Source [SID1234635106]).

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