On April 8, 2024 Nested Therapeutics, a biotechnology company pioneering a next-generation precision medicine platform to address hard-to-treat cancers, reported that preclinical data for the company’s lead program, NST-628, were featured in an oral presentation in the "New Drugs on the Horizon" series at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting (Press release, Nested Therapeutics, APR 8, 2024, View Source [SID1234641890]). The presentation, titled "NST-628 is a Novel, Potent, Fully Brain-Penetrant MAPK Pathway Molecular Glue that Inhibits RAS- and RAF-Driven Cancers," was given by Klaus Hoeflich, Ph.D., chief scientific officer and co-founder of Nested. The data were published concurrently online in the journal Cancer Discovery.

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"Dysregulation of RAS-MAPK pathway signaling is one of the most frequently occurring events in tumor development, impacting one in three newly diagnosed patients in the U.S. every year, the vast majority having no approved targeted treatment alternatives. While therapies have been developed for every node of the pathway, tolerability and durability of response continue to be challenging for patients with these difficult-to-treat cancers," said Dr. Hoeflich. "NST-628 was developed as a fully brain-penetrant, non-degrading molecular glue targeting the RAF and MEK nodes of the RAS-MAPK pathway. The preclinical data presented at AACR (Free AACR Whitepaper) show that NST-628 induces broad efficacy in tumor models and demonstrated the potential to overcome limitations of existing MEK and RAF inhibitors and RAS inhibitors in development. With a half-life and metabolic profile optimized to achieve a superior therapeutic index on a daily dosing schedule, as well as full intrinsic blood brain barrier penetrance, these data support NST-628’s potential as a best-in-class treatment for RAS- and RAF-driven cancers."

Preclinical data presented at AACR (Free AACR Whitepaper) and published in Cancer Discovery highlight the differentiated mechanism and drug-like properties of NST-628. Specifically:

In cellular and patient-derived tumor models harboring diverse KRAS, NRAS and BRAF alterations, NST-628 induced potent, deep and durable inhibition of the RAF-MEK signaling complex with broad efficacy without sacrificing tolerability at clinically achievable exposures over other MAPK-targeted compounds administered as either single agents or combinations.
With a predicted clinical half-life of 10-12 hours, the pharmacokinetic and metabolic profile of NST-628 has been optimized to broaden the therapeutic window and be clinically efficacious with once-daily oral dosing.
In mouse models with an intact blood brain barrier as well as central nervous system (CNS) models with RAS-MAPK alterations, NST-628 demonstrated full intrinsic CNS permeability. These data suggest NST-628 has the potential to treat brain metastases and primary CNS malignancies with MAPK pathway alterations. Approximately 40% of patients with metastatic cancer will develop symptomatic brain metastases, in particular in RAS-MAPK pathway dysregulated tumors such as lung, breast and melanoma.
Good laboratory practices (GLP) toxicology studies demonstrate significantly improved exposure margins when compared to other MEK inhibitors in non-clinical species.
Preclinical data support NST-628 as an ideal combination partner for upstream inhibitors including KRAS inhibitors by effectively preventing pathway reactivation.
In totality, the data validate NST-628’s potential to provide transformative clinical benefit as both a monotherapy or vertical combination anchor.
About the Phase 1 Study of NST-628
The ongoing Phase 1 open-label, single-arm, two-part study (NCT06326411) is intended to investigate the safety, pharmacokinetics (PK), pharmacodynamics (PD) and preliminary efficacy of single agent NST-628 in adult patients with RAS-MAPK pathway mutated/dependent advanced solid tumors, especially diverse KRAS, NRAS and BRAF alternations, who have exhausted standard treatment options. The study includes two parts: dose escalation (Part A) followed by dose expansion (Part B). The primary objectives for Part A, which recently initiated, are delineating NST-628’s safety profile and establishing the recommended dose for Part B. For more information, visit clinicaltrials.gov.

About NST-628
NST-628 is a fully brain-penetrant, mechanistically novel non-degrading molecular glue that targets multiple nodes in the RAS/MAPK pathway. NST-628 was developed based on Nested’s proprietary structural insights of how signaling complexes form and function in cancer and addresses common pitfalls of other MAPK-targeted compounds, which remain unable to circumvent the risk of intrinsic resistance via signaling pathway reactivation. Preclinical data evaluating oncology biomarkers relevant to RAS/MAPK-driven cell and patient-derived models collectively demonstrate superior anti-tumor activity, including in RAS and central nervous system-implanted tumor models, and tolerability of NST-628 compared to other MAPK-targeted compounds administered as either single agents or in combination. With a half-life and metabolic profile optimized to achieve a superior therapeutic index on a daily dosing schedule, as well as full intrinsic blood brain barrier penetrance, these data support NST-628’s potential as a best-in-class treatment especially for KRAS, NRAS and BRAF-driven cancers.

On April 8, 2024 Phanes Therapeutics, Inc. (Phanes), a clinical stage biotech company focused on innovative drug discovery and development in oncology, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to PT217 for the treatment of patients with extensive-stage small cell lung cancer (ES-SCLC) with disease progression following platinum chemotherapy with or without a checkpoint inhibitor (Press release, Phanes Therapeutics, APR 8, 2024, View Source [SID1234641889]). PT217 was also granted orphan drug designation for the treatment of small cell lung cancer by the FDA in 2022.

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PT217, a first-in-class native IgG-like bispecific antibody (bsAb) targeting DLL3 and CD47, is being developed for the treatment of patients with small cell lung cancer (SCLC), large cell neuroendocrine carcinoma of the lung (LCNEC), and extrapulmonary neuroendocrine carcinomas (EP-NECs).

SCLC is an aggressive form of cancer characterized by high mortality rates and significant morbidities. The average survival for patients who present with ES-SCLC without treatment is 2-4 months after diagnosis; the median survival for patients with treatment is 7-11 months.

"PT217 has the potential to be a transformative treatment option for patients with SCLC, LCNEC and EP-NECs in various settings," said Ming Wang, Founder and CEO of Phanes Therapeutics. "Now we have two Fast Track designations in our pipeline. Last month, PT886 was granted Fast Track designation for the treatment of patients with metastatic claudin 18.2-positive pancreatic adenocarcinoma. Both assets are from our optimized anti-CD47 bispecific antibody franchise and currently in clinical studies. We believe the optimized anti-CD47 bispecific antibody approach can unlock the full potential of the innate immunity in targeting solid tumors."

The multi-center Phase I clinical trial of PT217 (NCT05652686), known as the SKYBRIDGE study, is currently evaluating the safety, tolerability, pharmacokinetics and preliminary efficacy of PT217 in patients with advanced or refractory cancers expressing DLL3.

On April 8, 2024 Accent Therapeutics, a biopharmaceutical company pioneering a novel class of small molecule precision cancer therapeutics reported advances in cancer targeting, including data on DHX9 and KIF18A inhibitor activity in multiple tumor types, at the 2024 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, taking place April 5-10 in San Diego, California (Press release, Accent Therapeutics, APR 8, 2024, View Source [SID1234641888]).

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"Accent has expanded the potential of our two lead programs in preclinical investigations, and we are excited for the promise they hold for addressing cancers with high unmet clinical need," said Robert A. Copeland, Ph.D., President, Founder, and Chief Scientific Officer of Accent Therapeutics. "Our results reveal that DHX9 and KIF18A inhibition are potent therapeutic strategies that stand to benefit large patient populations. These findings validate our systematic approach to tapping the rich therapeutic potential of our target space."

In the presentation entitled "RNA-Modifying Enzyme Inhibitors as Precision Cancer Therapeutics," Dr. Copeland delineates the promise of RNA-modifying enzyme (RME) inhibitors as precision cancer therapeutics that confer cancer-selective growth inhibition in vitro and in vivo. The potential of RME inhibitors is exemplified by inhibitors of the novel cancer target, DHX9, which display great promise as a novel treatment modality in BRCA1 and/or BRCA2 loss-of-function (LOF) cancers across multiple tumor types, including breast cancers.

Studies in breast cancer models demonstrate the potential impact of DHX9 inhibitor treatment for patients with defective DNA repair. These data are highlighted in a presentation entitled "DHX9 inhibition as a novel therapeutic for cancer with loss-of-function mutations in DNA damage repair genes BRCA1 and BRCA2." Additional key takeaways from this talk are:

DHX9 inhibition shows selective growth inhibition in breast cancer cell lines that exhibit BRCA LOF
DHX9 inhibition results in DNA damage and catastrophic increase in replication stress in BRCA LOF cell lines, causing cell cycle arrest prior to onset of apoptosis
In vivo, DHX9 inhibitors were well tolerated and demonstrate robust tumor growth inhibition in multiple BRCA LOF cell line xenograft models
Accent scientists further reinforce KIF18A – a kinesin that plays a critical role in facilitating mitosis – as a compelling therapeutic target in chromosomally instable tumors. In a poster entitled "Inhibition of KIF18A leads to mitotic arrest and robust anti-tumor activity in chromosomally instable tumors," they show data that Accent’s novel, potent and selective small molecule inhibitors of KIF18A:

Upregulate biomarker proteins, indicating specific induction of mitotic arrest, DNA damage, and apoptosis
Exhibit robust anti-proliferative activity, and result in fragmented nuclei and malformed mitotic spindles in chromosomally instable ovarian cancer cell lines
Induce cell cycle arrest in G2/M for sensitive cell lines, consistent with the role of KIF18A in facilitating mitosis
Lead to dose-dependent tumor growth inhibition in an OVCAR-3 cancer cell line-derived xenograft model, while sparing tumors that are insensitive to KIF18A loss
Data describing a DHX9-specific pharmacodynamic biomarker of inhibition are presented in a poster entitled "circBRIP1 RNA as a non-invasive target engagement pharmacodynamic biomarker for DHX9 inhibition." Key takeaways include:

Positive correlation of circBRIP1 induction with DHX9 biochemical activity, dose, and exposure for DHX9 inhibitors
Similar potency of dose-dependent induction of circBRIP1 across cell lines that are sensitive or insensitive to DHX9 inhibition, indicating that circBRIP1 is a unique target engagement PD biomarker, but not a predictor of DHX9 sensitivity
circBRIP1 induction correlates with DHX9 inhibitor concentration within individual tumors in mouse xenografts, providing strong evidence of a predictable PK/PD relationship
Select posters and presentations will be shared on Accent’s website following the meeting.

About DHX9
Accent’s lead program is a first-in-class DHX9 inhibitor with the potential to address high unmet need indications not adequately served by existing therapies, including tumors with BRCA loss of function (breast, ovarian, and others), mismatch repair deficient (dMMR) or microsatellite instability-high (MSI-H) cancers (colorectal, endometrial, gastric) and additional undisclosed cancer types representing large patient populations. DHX9 is a DNA/RNA helicase that has been reported to play important roles in replication, transcription, translation, RNA splicing, RNA processing, and maintenance of genomic stability. Hence, this enzyme represents a compelling novel oncology target as inhibition of DHX9 exploits key tumor vulnerabilities, resulting in cancer-specific death. Accent is currently conducting IND-enabling studies for this program.

About KIF18A
Accent’s second lead program is a potential best-in-class inhibitor for KIF18A which may address a large patient population across several cancer indications, including ovarian and triple negative breast cancer (TNBC). KIF18A is a mitotic kinesin motor protein critical for cell division in select tumors with chromosomal instability. A subset of tumor cells with an abnormal number of chromosomes (aneuploid) are reliant on KIF18A and show rapid cell killing in vitro and in vivo upon KIF18A inhibitor treatment, while cells with normal numbers of chromosomes (euploid) are unaffected. Accent is planning to initiate IND-enabling studies for KIF18A in the first half of 2024.

On April 8, 2024 Transcenta Holding Limited ("Transcenta") (HKEX: 06628), a clinical stage biopharmaceutical company with fully-integrated capabilities in discovery, research, development and manufacturing of antibody-based therapeutics, and Agilent Technologies, reported a collaboration to develop a Claudin18.2 (CLDN18.2) companion diagnostic to support TranStar301 global Phase III pivotal trial of Osemitamab (TST001) in combination with Nivolumab and chemotherapy as first-line treatment in patients with CLDN18.2 expressing locally advanced or metastatic gastric or gastroesophageal (G/GEJ) adenocarcinoma (Press release, Transcenta, APR 8, 2024, View Source [SID1234641887]).

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Transcenta has developed a mouse anti-CLDN18.2 monoclonal antibody, clone 14G11 which specifically binds to CLDN18.2 but not CLDN18.1. This antibody, generated against a linear epitope located on the extracellular domain of loop 1, has a binding site that overlaps with the binding site of therapeutic antibody Osemitamab (TST001).

Transcenta has been collaborating with Agilent, a world leader in CDx development, to further develop this antibody for use in a companion diagnostic assay. Agilent is developing Claudin18.2 IHC 14G11 pharmDx, an immunohistochemistry (IHC) assay for the detection of CLDN18.2 protein in gastric and gastroesophageal junction (GEJ) adenocarcinoma with the potential for other indications. Agilent and Transcenta presented the early results of the Claudin18.2 IHC 14G11 pharmDx assay at AACR (Free AACR Whitepaper) Annual Meeting. Claudin18.2 IHC 14G11 pharmDx for Investigational Use Only/for Performance Evaluation Only will be used for patient selection in the phase III trial of gastric/GEJ adenocarcinoma where applicable ethics committee and regulatory approvals have been granted.

"Agilent’s expertise in the development of companion diagnostics is impressive, as is their strong track record of developing companion diagnostics across the precision oncology sector," said Dr. Caroline Germa, Transcenta’s Executive Vice President, Global Medicine Development and Chief Medical Officer. "We are excited about the collaboration and look forward to working together to pave the way for enhanced patient health outcomes."

"We are excited to be working with Transcenta on the development of the Claudin18.2 IHC 14G11 pharmDx companion diagnostic assay," said Dr. Paul Beresford, VP/GM of CDx, Agilent. "This partnership with Transcenta will further pave the way for enhanced precision medicine products for those with gastric and gastroesophageal junction adenocarcinoma, and continue transforming diagnostics, treatments, and patient health outcomes."

About Osemitamab (TST001)

Osemitamab (TST001) is a high affinity humanized anti-CLDN18.2 monoclonal antibody with enhanced antibody-dependent cellular cytotoxicity ("ADCC"). It has shown potent anti-tumor activities in tumor xenograft models. Osemitamab (TST001) is the second most advanced CLDN18.2 targeting antibody being developed globally. Osemitamab (TST001) was generated using Transcenta’s Immune Tolerance Breaking Technology (IMTB) platform. Osemitamab (TST001) kills CLDN18.2 expressing tumor cells by mechanisms of ADCC. Leveraging advanced bioprocessing technology, the fucose content of Osemitamab (TST001) was significantly reduced during the production, which further enhanced NK cells mediated ADCC activity of Osemitamab (TST001). Clinical trials for Osemitamab (TST001) are ongoing in the U.S. and China (NCT05190575, NCT04396821, NCT04495296, NCT05608785/CTR20201281). Osemitamab (TST001) has been granted Orphan Drug Designation in the U.S. by FDA for the treatment of patients with gastric or gastroesophageal junction (G/GEJ) and pancreatic cancer.

On April 8, 2024 Vincerx Pharma, Inc. (Nasdaq: VINC), a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, reported positive preliminary Phase 1 data for VIP236 and updates on pipeline progress at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2024 (Press release, Vincerx Pharma, APR 8, 2024, View Source [SID1234641886]).

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"The positive preliminary data we’ve reported for VIP236 and VIP943, coupled with preclinical findings leveraging our VersAptx platform to improve the efficacy of TRODELVY and ENHERTU, two marketed ADCs, underscore the power of our platform approach for hematologic malignancies and solid tumors," said Ahmed Hamdy, M.D., Chief Executive Officer of Vincerx. "The early VIP236 data demonstrated positive clinical activity, including tumor reductions. This represents significant promise for patients who have exhausted standard anticancer therapy options with many different tumor types, including tumors not usually responsive to camptothecin-derived therapies. Dose escalation continues in the VIP236 and VIP943 first-in-human studies. As we advance into higher dose levels, we look forward to sharing more clinical data for VIP236 later this summer and for VIP943 on or around the 2024 European Hematology Association (EHA) (Free EHA Whitepaper) annual meeting."

Raquel Izumi, Ph.D., President and Chief Operating Officer of Vincerx added, "The main objectives of a Phase 1 dose-escalation study are to assess safety and tolerability while establishing an optimal dose and schedule, so seeing dose-dependent clinical activity at this point in the development of VIP236 is exciting. We are still in dose escalation and are starting to see tumor reduction after only two doses. We expect to see responses deepen with more time on treatment and as we continue to escalate."

Vivek Subbiah, M.D., Chief of Early-Phase Drug Development at Sarah Cannon Research Institute commented, "In the oncology landscape, ADCs have emerged as a new and encouraging treatment option for people facing cancer. With a decade of invaluable insights in ADC drug development, we are transitioning from first to second to third generations, and the imperative now is for novel mechanisms of action. Innovations like VIP236’s optimized camptothecin, which can potentially circumvent some of the known camptothecin liabilities and issues with drug resistance, deliver a potent payload from a clinically validated drug class. The goal is to deliver best-in-class therapies to meet the urgent needs of patients battling advanced cancers."

VIP236 Updates

Study VNC-236-101 is an open-label, multicenter, Phase 1 dose-escalation study with monotherapy VIP236 for the treatment of patients with metastatic tumors who have exhausted all standard therapy options. The study’s main objective is to determine a safe dose and schedule for VIP236 for further clinical development.
Fifteen patients have been dosed to date on the once every three weeks (Q3W) schedule. Sequential dose-escalation cohorts with the Q3W schedule were 0.2 mg/kg (n=2), 0.4 mg/kg (n=5), 0.6 mg/kg (n=5), and 0.8 mg/kg (n=3). Results of the Q3W schedule (n=15) show:
The patient population is typical of a Phase 1 study with heavily pretreated patients and a wide range of tumor types.
The Q3W schedule is well tolerated with no dose-limiting toxicity (DLT) in any patients, and no patients have discontinued VIP236 due to an adverse event. Importantly, no severe or life-threatening diarrhea has been observed, validating the purposeful design of VIP236’s optimized camptothecin payload.
First efficacy assessment was at the end of cycle 2 (i.e. after only two doses on the Q3W schedule). Seven patients have achieved objective stable disease, including tumor reduction. Four patients remain on study with the longest treated patient on study for 168 days.
Dose escalation continues on the Q3W schedule. Vincerx anticipates presenting additional Phase 1 data for VIP236 later this summer.
VIP943 Updates

Study VNC-943-101 is an open-label, multicenter, Phase 1 dose-escalation study with monotherapy VIP943 for the treatment of patients with CD123+ acute myeloid leukemia (AML), B-cell acute lymphocytic leukemia (B-ALL) or myelodysplastic syndromes (MDS) who have exhausted standard therapeutic options. The study’s main objective is to determine a safe dose and schedule for VIP943 for further clinical development.
VIP943 is administered once per week. Three patients were dosed in Cohort 1 (0.2 mg/kg) and four patients were dosed in Cohort 2 (0.4 mg/kg).
Despite the initial low doses in the study, all seven sequentially enrolled patients completed the 28-day DLT evaluation period. Five out of seven received a cycle 2 dose and two of these patients started cycle 3. One patient with MDS is still on study on cycle 3.
No DLTs occurred in Cohort 1 and 2. Four patients have been enrolled in Cohort 3 (0.7 mg/kg) and are undergoing DLT assessment.
VIP943 PK data shows very little free payload in circulation, consistent with the favorable safety profile observed preclinically and clinically.
As the study progresses through the dose escalation, Vincerx will present additional Phase 1 data for VIP943 on or around the 2024 European Hematology Association (EHA) (Free EHA Whitepaper) Annual Meeting in June 2024.
New In Vitro Solid Tumor Data

Today Vincerx also reported preclinical experiments applying the next-generation effector chemistry of its VersAptx platform to the antibodies of approved ADCs, TRODELVY and ENHERTU, demonstrating the potential to improve tumor toxicity of ADCs by orders of magnitude.
In in vitro tumor models, Vincerx’s sacituzumab-legumain-KSPi ADC had a 20-fold improvement in tumor toxicity compared with TRODELVY (sacituzumab-govitecan). The company’s trastuzumab-legumain-KSPi ADC demonstrated an 8-fold increase in tumor toxicity compared with ENHERTU (fam-trastuzumab-deruxtecan).
These findings further support the versatility of VersAptx to address multiple cancer types, including solid tumors, and increase the efficacy and safety of ADCs. Further studies will be conducted in animal models.
Virtual Investor Event

Vincerx will host a virtual investor event featuring company management and key opinion leaders to review preliminary clinical data from its Phase 1 dose-escalation study of VIP236 and provide an update on pipeline progress today at 2:00 PM PDT/ 5:00 PM EDT. To register and view the live webcast, please visit: View Source An archived replay of the webcast will be available on the Vincerx Investor Page website following the conclusion of the live event.

About VIP236

VIP236, the first-in-class small molecule drug conjugate (SMDC) from the VersAptx Platform, consists of an αvβ3 integrin binder, a neutrophil elastase linker cleaved in the tumor microenvironment, and a camptothecin payload optimized for high permeability and low active efflux. VIP236 was designed to deliver its payload to advanced/metastatic tumors that express αvβ3. Preclinical data show enhanced efficacy, independent of HER2 status, in patient-derived and cell line-derived gastric cancer models compared with ENHERTU, an approved ADC. VIP236 is being evaluated in a Phase 1 dose-escalation trial treating patients with advanced or metastatic solid tumors (NTC05371054).

About VIP943

VIP943, the first ADC from the VersAptx platform, consists of an anti-CD123 antibody, a unique linker cleaved intracellularly by legumain, and a novel kinesin spindle protein inhibitor (KSPi) payload enhanced with our CellTrapper technology. The next-generation effector chemistry (linker + payload with CellTrapper) was designed to reduce non-specific release of the payload and ensure payload accumulation in cancer cells versus healthy cells. The increased therapeutic index has the potential to address challenges associated with many ADCs by improving efficacy and reducing severe toxicities. VIP943 is in a Phase 1 dose-escalation trial evaluating patients with relapsed/refractory AML, B-ALL, and MDS who have exhausted standard therapeutic options (NCT06034275).

About VersAptx Platform

VersAptx is a versatile and adaptable next-generation bioconjugation platform. The modular nature of this innovative platform allows for the combination of different targeting, linker, and payload technologies to develop bespoke bioconjugates to address different cancer biologies. With this platform, (i) antibodies and small molecules can be used to target different tumor antigens, (ii) linkers can be designed to reduce non-specific release of the payload, cleave intracellularly or extracellularly, and conjugate to single or multiple payloads, and (iii) payloads can be designed with reduced permeability using the CellTrapper technology to ensure accumulation in cancer cells or to be permeable for release in the tumor microenvironment. The VersAptx platform allows for the optimization of these technologies to a specific target and the development of bioconjugates designed to address the safety and efficacy challenges of many ADCs and the needs of cancer patients.