Vincerx Pharma Provides American Society of Hematology Annual Meeting 2022 Poster Highlights on Enitociclib in Multiple Tumor Types

On December 12, 2022 Vincerx Pharma, Inc. (Nasdaq: VINC), a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, reported two poster presentations of preclinical and clinical data on the Company’s lead asset, enitociclib (also known as VIP152) at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting 2022 and additional data-related updates in the enitociclib program (Press release, Vincerx Pharma, DEC 12, 2022, View Source [SID1234625149]).

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Enitociclib is a potent and selective CDK9 inhibitor currently in clinical development. The small molecule inhibitor targets P-TEFb/CDK9 and has shown robust pathway modulation as well as efficacy and safety in several preclinical tumor models and in early phase clinical studies.

"We are excited with the progress we have made in our enitociclib program," said Ahmed Hamdy, M.D., Chief Executive Officer of Vincerx Pharma. "At ASH (Free ASH Whitepaper), we reported robust pathway modulation in MM cell lines and antitumor efficacy data in vivo. We also presented preliminary clinical data in patients with relapsed/refractory chronic lymphocytic leukemia (CLL) and Richter syndrome (RS), continuing to show that enitociclib 30 mg is more efficacious than 15 mg, and we have definitively showed that 15 mg does not modulate the target in patients."

Dr. Hamdy added, "We believe the potential paths forward for this program are promising, including studying enitociclib in combination given its favorable safety profile. Earlier this year, we established a partnership with the National Institutes of Health (NIH) to study enitociclib in combination with venetoclax and prednisone in MYC DLBCL, non-GCB DLBCL, and peripheral T-cell lymphoma. We will also look at studying enitociclib in combination with a BTK inhibitor in Q1 2023. Currently, patients with CLL receive multiple rounds of combination therapies only to relapse or stay on a BTK inhibitor for the rest of their lives. We believe that enitociclib in combination with a BTK inhibitor could drive patients into a deeper remission allowing them to stop treatment. The benefit of time-limited treatment is reducing unwanted toxicities and the potential for developing resistance mutations."

Dr. Wyndham Wilson, Head, Lymphoma Therapeutics Section and Senior Investigator of the Lymphoid Malignancy Branch, Center for Cancer Research at the National Cancer Institute, added, "The partnership with Vincerx brings together our shared level of expertise, and we are excited about the potential of this combination study of enitociclib with venetoclax and prednisone. The ability of these agents to inhibit multiple important survival pathways suggests this combination could show significant activity in aggressive B-cell lymphomas."

Dr. Aru Narendran, Professor at the departments of Oncology and Pediatrics at the University of Calgary further added, "The preclinical data presented at ASH (Free ASH Whitepaper) demonstrated the efficacy of enitociclib as a monotherapy and in combination with anti-MM agents across a range of in vitro MM cell lines, as well as in vivo models of MM, which provide rationale and biological reasoning for further optimization studies of CDK9 inhibitors for clinical applications and early phase clinical studies to improve outcomes in MM. In addition, going forward, these findings also lay the foundation for further preclinical studies to examine the efficacy of enitociclib and combinations in other refractory hematological malignancies driven by similar oncogenic mechanisms, including high-risk pediatric leukemias."

Dr. Mazyar Shadman, M.D., Fred Hutchinson Cancer Center, Seattle, WA, USA, noted that, "Clinical data presented at ASH (Free ASH Whitepaper) demonstrated evidence of monotherapy clinical activity in DH-DLBCL and a favorable safety profile across a range of B-cell malignancies. The favorable safety profile of enitociclib makes it a viable combination partner."

Key ASH (Free ASH Whitepaper) 2022 Presentation Highlights:

Poster presentation titled, "Preclinical Study of Enitociclib, a Selective CDK9 Inhibitor, in Combination with Bortezomib, Lenalidomide, Pomalidomide, or Venetoclax in the Treatment of Multiple Myeloma", presented by Andy Son Tran, BSc, Department of Oncology, University of Calgary, AB, Canada, include:

Enitociclib was identified as a top hit in small molecule inhibitor screening and exposure to enitociclib for 96 hours against a representative panel of MM cell lines (NCI-H929, MM.1S, OPM-2, and U266B1) demonstrated significant cytotoxic activity, with IC50 values ranging from 36 to 78 nM.
Induction of apoptosis was observed with cleavage of pro-caspase-3 and PARP by western blotting in a time- and dose-dependent manner with enitociclib as a single-agent, in addition to the depletion of phosphorylated RNAPII (Ser 2/5), MYC, MCL1, and PCNA proteins.

Enitociclib synergizes with several anti-MM agents (bortezomib, lenalidomide, pomalidomide, and venetoclax [synergy scores >10]) at pharmacologically relevant concentrations across several MM cell lines.
Enitociclib enhances the efficacy of lenalidomide and venetoclax as demonstrated by robust apoptosis induction through caspase-3 activation and PARP cleavage at 2 hours.

In a JJN-3 MM xenograft mouse model, intravenous administration of enitociclib transiently inhibits the transcription of MYC and MCL1 and promotes apoptosis by induction of pro-caspase-3 and PARP cleavage with the onset of drug-induced effects seen as early as 1 hour after enitociclib treatment. Tumor volumes were reduced by 96% to 99% as compared with control mice on day 20 after the commencement of treatment. Increased efficacy of enitociclib in combination with lenalidomide was observed.

Overall, these studies present evidence that enitociclib has significant antitumor activity against MM cell lines and provides specific pharmacologic targetability of several key oncogenic pathways involving proteins such as MYC, MCL1, and PCNA, leading to growth inhibition and apoptosis.

Poster presentation titled, "Enitociclib (VIP152/formerly BAY1251152) Is a Selective and Active CDK9 Inhibitor: Preliminary Safety and Early Signs of Efficacy in Patients with Non-Hodgkin Lymphoma (NHL) and Chronic Lymphocytic Leukemia (CLL)", presented by Mazyar Shadman, M.D., Fred Hutchinson Cancer Center, Seattle, WA, USA, include:

Updated clinical data on 20 patients, 16 NHL from study VNC-152-101 (NCT02635672) and 4 CLL patients from study VNC-152-102 (NCT04978779) were presented. Safety and preliminary efficacy data for 5 newly enrolled patients with MYC+ tumors (one each: DH-DLBCL, Richter syndrome [RS], transformed follicular lymphoma [tFL], Burkitt lymphoma, and mantle cell lymphoma [MCL]), in addition to the previously reported 11 patients, making a total of 16 NHL patients in VNC-152-101.

Patients with NHL were heavily pretreated with >3 prior therapies with a median number of 4 enitociclib doses administered. As part of prior therapy, patients with CLL had received an approved BTKi, 3 patients had received venetoclax, and 2 patients had received CAR-T.

Monotherapy treatment with enitociclib (doses of 10 to 30 mg) has a favorable safety profile in a pooled safety analysis for NHL/CLL (n=20) that is consistent with previously reported safety data:
Mainly Grade 1 and 2 gastrointestinal adverse events (AEs).

Neutropenia was observed in 8 patients with supportive care (G-CSF) administered to 3 patients.
There were no discontinuations due to an AE; 3 deaths due to disease progression.
Enitociclib monotherapy treatment showed 1 stable disease (SD), 3 radiologic disease progression, and 1 clinical progression.

The patient with tFL with SD had a 16% reduction in tumor burden at the end of cycle 2 and at cycle 7 a 22% reduction in tumor burden (this patient is currently still on study); this response kinetics is consistent with the 2 previously reported DH-DLBCL patients who achieved SD at cycle 2 and metabolic CRs at cycle 10.
Enitociclib does not inhibit T-cell dependent antibody response in a rat model.
AUC0-24 was 390 ng*hr/mL and 1820 ng*mg/mL following enitociclib doses of 0.33 mg/kg/week and 1.25 mg/kg/week, respectively.

The exposure observed for the 1.25 mg/kg group was comparable to the exposure observed in patients who received 30 mg enitociclib.

Results from this preclinical study suggests enitociclib is not expected to hamper vaccine response.
In patients with NHL and CLL, enitociclib PK properties are comparable at the same dose (Cmax, AUC0-t) and across doses (CL, VSS and t1/2). Enitociclib PK are consistent with previously reported data in patients with NHL.
Pharmacodynamic data show enitociclib 10/15 mg does not deliver adequate control of oncogenic signals of MYC, MCL1, and PCNA mRNA as compared with other NHL patients (MCL and DH-DLBCL) dosed at 30 mg.
The 30-mg dose of enitociclib is well-tolerated by patients with CLL/RS, consistent with observations of 30 mg in NHL/solid tumor indications.

Patients will CLL (n=2) treated with 10/15 mg did not respond to enitociclib, in line with the data showing that the 10/15-mg dose level does not appear to be able to deliver downregulation of MYC, MCL1 and PCNA mRNA.
In blood, the maximum extent of inhibition for MYC, MCL1, and PCNA mRNAs was 98%, 76%, and 92%, respectively. The maximal inhibition of all mRNAs in the blood occurred within 4 hours of enitociclib treatment in patients with DH-DLBCL and other B-cell malignancies (CLL, tFL, MCL, and Burkitt lymphoma) with the once weekly 30-mg dose of enitociclib.
Copies of the presentation materials can be accessed on the Investors section of the Company’s website at www.vincerx.com.

Tessa Therapeutics Announces Updated Safety, Efficacy and Biomarker Data from Phase 2 Trial of Autologous CD30.CAR-T Therapy (TT11) in Relapsed or Refractory Classical Hodgkin Lymphoma

On December 12, 2022 Tessa Therapeutics Ltd. (Tessa), a clinical-stage cell therapy company developing next-generation cancer treatments for hematological malignancies and solid tumors, reported updated clinical data from the pilot stage of the ongoing Phase 2 CHARIOT trial (NCT04268706) of TT11, the Company’s autologous CD30 chimeric antigen receptor T-cell (CAR-T) therapy, were presented in a poster and oral podium presentation at the 64rd Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) (Press release, Tessa Therapeutics, DEC 12, 2022, View Source [SID1234625148]). The results demonstrated TT11 to be well-tolerated with promising efficacy in relapsed or refractory (r/r) CD30-positive classical Hodgkin lymphoma (cHL). Moreover, research identified circulating tumor DNA (ctDNA) analysis as a potential measure of response in cHL after CD30 targeted CAR T-cell therapy.

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The ASH (Free ASH Whitepaper) poster, titled, "Updated Results and Correlative Analysis: Autologous CD30.CAR-T Cell Therapy in Patients with Relapsed or Refractory Classical Hodgkin Lymphoma (CHARIOT Trial)," reported expanded data from the pilot segment of a Phase 2 trial of autologous CD30.CAR-T in patients with r/r cHL. The CD30.CAR-T cell therapy was demonstrated to be well tolerated with no unexpected safety signals. An ORR of 73.3% and a CR of 60% was observed in 15 heavily pre-treated r/r cHL patients, suggesting strong anti-tumor responses. Additionally, CD30.CAR-T expansion and persistence was observed after CD30.CAR-T infusion.

In an oral podium presentation on December 12, titled, "Ultrasensitive ctDNA Dynamics after Autologous CD30.CAR-T Cell Therapy for Relapsed or Refractory (r/r) Classical Hodgkin Lymphoma (CHARIOT Trial)," research examining the potential of ctDNA as a biomarker in r/r cHL after CD30 CAR-T therapy were reported. Foresight Diagnostics’ PhasED-Seq MRD assay was used to measure ctDNA at multiple timepoints throughout therapy. Data showed that ctDNA responses mirrored radiographic responses, suggesting that ctDNA levels could be predictive of patient response to CAR-T therapy. Researchers also determined that PhasED-Seq ctDNA analysis is a viable biomarker to monitor responses and predict outcomes in patients with r/r cHL treated with CD30.CAR-T Cell Therapy.

"The data presented at ASH (Free ASH Whitepaper) 2022 continue to demonstrate the CD30.CAR-T cell therapy to be well tolerated with excellent anti-tumor responses in patients with relapsed or refractory classical Hodgkin lymphoma. This includes an overall response rate of 73.3% in heavily pretreated patients, as well as good expansion and persistence after infusion," stated Sairah Ahmed, M.D., principal investigator, lead presentation author, and Associate Professor, The University of Texas MD Anderson Cancer Center.

"We are pleased to present research demonstrating the potential of minimally invasive ctDNA analysis as a viable method to monitor responses, rapidly risk stratify, and predict outcomes of patients with r/r cHL treated with CD30.CAR-T therapy," stated David M. Kurtz, M.D., Ph.D., lead presenter and Assistant Professor, Department of Medicine (Oncology), Stanford University.

CD30 is a well validated lymphoma target with homogeneous expression in 98% of cHL and a significant proportion of subsets of non-Hodgkin lymphoma (NHL). TT11 is an autologous CD30 chimeric antigen receptor T-cell (CAR-T) therapy that harvests the patient’s own T-cells and modifies them to target cancer cells expressing the CD30 protein.

"We continue to be intrigued by the data being generated from our ongoing Phase 2 CHARIOT trial of TT11, which show the therapy to be safe and well tolerated, along with clear signals of efficacy, in patients with relapsed or refractory CD30-positive classical Hodgkin lymphoma," said Ivan Horak, M.D., Chief Medical Officer and Chief Scientific Officer of Tessa Therapeutics.

TT11 was granted Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. Food and Drug Administration (FDA) and PRIority MEdicines (PRIME) designation by the European Medicines Agency (EMA).

An Expression makes a world of difference

On December 12, 2022 Syros Pharmaceuticals presented its corporate presentation (Presentation, Syros Pharmaceuticals, DEC 12, 2022, View Source [SID1234625147]).

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Step Pharma Announces Promising Data on the Therapeutic Potential of Selective Small Molecule Inhibition of CTPS1 Presented at the 64th American Society of Hematology Annual Meeting

On December 12, 2022 Step Pharma, the world leader in CTPS1 inhibition for the targeted treatment of cancer, reported that encouraging pre-clinical data on its first-in-class, highly selective, orally bioavailable CTPS1 inhibitor, STP938, were presented at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting held from the 10-13 December, 2022 in New Orleans, Louisiana and virtually (Press release, Step Pharma, DEC 12, 2022, View Source [SID1234625146]).

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The data presented by Step Pharma collaborators demonstrates the potential of STP938, a highly selective CTPS1 inhibitor, as a therapeutic for the treatment of mantle cell lymphoma and multiple myeloma both as a single agent and in combination with other targeted inhibitors.

In pre-clinical models of mantle cell lymphoma, STP938 showed strong anti-tumour effects, which were further enhanced by combined administration with venetoclax, a selective BCL-2 inhibitor currently in clinical development for treatment of this lymphoma type.

Single agent STP938 also showed activity in preclinical models of multiple myeloma and demonstrated strong synergy when combined with an inhibitor of the DNA damage response pathway (ATR, CHEK1 or WEE1).

"We are encouraged by the promising pre-clinical data produced by our collaborators showing the anti-tumour effects of STP938 in mantle cell lymphoma and multiple myeloma models",

said Andrew Parker, Chief Executive Officer of Step Pharma.

"These data reinforce our conviction that by inhibiting CTPS1, a key component of the pyrimidine synthesis pathway to which all cancers are addicted, STP938 has broad therapeutic potential. We have commenced clinical trials to test the safety and efficacy of STP938 in T cell and B cell lymphomas to bring this potentially lifesaving therapy to patients in our efforts to drive a step change in the way we treat cancer."

Full details of the poster presentations, which were both presented on Sunday, December 11, 2022 at 6:00 PM-8:00 PM EST in Hall D (Ernest N. Morial Convention Center), are:

Title: STP938, a Selective CTPS1 Inhibitor, Shows Single Agent Activity and Synergy with BCL2 Inhibition in Preclinical Models of Mantle Cell Lymphoma
Authors: David Chiron, PhD, Emmanuelle Menoret, PhD, Charlotte Kervoelen, PhD, Sophie Maïga, Celine Bellanger, Benoit Tessoulin, MD, Cyrille Touzeau, Hélène Asnagli, PhD, Andy Parker, PhD, Philip Beer, MD, PhD and Catherine Pellat-Deceunynck, PhD

Title: CTPS1 is a Novel Therapeutic Target in Multiple Myeloma that Synergizes with Inhibition of ATR, CHEK1 or WEE1
Authors: Christina Pfeiffer, MSc, Alexander Michael Grandits, PhD, Hélène Asnagli, PhD, Anja Schneller, MSc, Julia Huber, MSc, Niklas Zojer, MD, Martin Schreder, MD, Andrew Parker, PhD, Arnold Bolomsky, PhD, Philip Beer, MD, PhD, Heinz Ludwig, MD

Step Pharma also recently entered clinical development with STP938 in a Phase I/II trial for T cell and B cell lymphomas. An abstract outlining the study design was published by ASH (Free ASH Whitepaper) in the journal Blood. Full details are:

Title: A Phase 1/2 Study of STP938, a First in Class Inhibitor of CTP Synthase 1, in Patients with Relapsed/Refractory B or T Cell Lymphoma
Authors: Philip Beer, Maureen Higgins, Hélène Asnagli, Eef Hoeben, Michael Hubank, Andrew Parker, Brian Schwartz

Harnessing the power of Engineered Macrophages

On December 12, 2022 Carisma therapeutics presented its Corporate presentation (Press release, Sesen Bio, DEC 12, 2022, View Source [SID1234625145]).

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