1stOncology™: Cancer Intelligence Service – Page 5773 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

Verismo Therapeutics Receives U.S. FDA Orphan Drug Designation for SynKIR-110™, a First-in-Class KIR-CAR T Cell Immunotherapy Candidate, for the Treatment of Mesothelioma

On September 28, 2022 Verismo Therapeutics, a clinical-stage CAR-T company and Penn spinout, and pioneer of the novel KIR-CAR platform technology, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to SynKIR-110 for the treatment of patients with mesothelin-expressing mesotheliomas (Press release, Verismo Therapeutics, SEP 28, 2022, View Source [SID1234621530]).

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!

SynKIR-110 is a next generation approach to cell therapy targeting solid tumors, and the first product to use the novel KIR-CAR platform, a modified NK-like receptor designed to improve persistence and efficacy against aggressive solid tumors. Phase 1 clinical trials of SynKIR-110 in patients with mesothelin-expressing ovarian cancer, mesothelioma and cholangiocarcinoma will commence in Q1 2023.

"Orphan Drug Designation is another major milestone for Verismo following the acceptance of our SynKIR-110 IND," said Dr. Bryan Kim, CEO of Verismo. "Mesothelioma is a rare and deadly disease for which few other treatment options currently exist. We look forward to working with the mesothelioma community to advance SynKIR-110 as a potential treatment while we continue to expand clinical investigation of this novel platform in other cancers in the solid tumor space."

Orphan Drug Designation is a status given to drugs intended to treat a rare disease that affects fewer than 200,000 people in the United States and show promise in the treatment, prevention, or diagnosis of that disease. Benefits to the study sponsor include seven years of marketing exclusivity, federal tax credits, waiver of Prescription Drug User Fee Act (PDUFA) fees and increased regulatory assistance from the FDA.

The Phase 1 trial for SynKIR-110, STAR-101 (SynKIR T cell Advanced Research), will begin enrolling patients in the first quarter of 2023 at the initial clinical site, the Hospital of the University of Pennsylvania.

About the KIR-CAR Platform
The KIR-CAR platform is a dual-chain CAR T cell therapy and has been shown in preclinical models to be capable of maintaining antitumor T cell activity even in challenging solid tumor environments. DAP12 costimulatory chains aid additional T cell stimulating pathways, further improving cell persistence. This continued function and persistence can lead to ongoing regression of solid tumors in preclinical models, including those refractory to traditional CAR T cell therapies. Furthermore, the KIR-CAR platform can be combined with many additional emerging technologies, such as in vivo gene editing, advanced T cell selection and modulation, combination therapies, and even allogeneic cellular therapies to provide an adaptable targeted anti-tumor therapy for patients in need.

About Mesothelioma
Mesothelioma is a rare type of cancer that affects the lining of the chest or abdominal cavities. More than 2,000 cases of this aggressive cancer are diagnosed in the U.S. each year. While life expectancy can differ depending on a variety of factors, such as type and location of the disease, stage at diagnosis, and general patient health, overall, patients usually survive only four to eighteen months after diagnosis.

Ryvu Therapeutics Reports 2022 Half-Year Financial Results and Provides Corporate Update

On September 28, 2022 Ryvu Therapeutics (WSE: RVU), a clinical-stage drug discovery and development company focusing on novel small molecule therapies that address emerging targets in oncology, reported financial results for the first half of 2022 and provided a corporate update (Press release, Ryvu Therapeutics, SEP 28, 2022, View Source [SID1234621529]).

In August, Ryvu secured €22 million in funding from the European Investment Bank to support Ryvu’s pipeline development.
In July, Ryvu signed an exclusive license agreement with Nasdaq-listed Exelixis to develop novel STING agonist-based targeted cancer therapies.
RVU120 clinical and preclinical data were presented at the 2022 European Hematology Association (EHA) (Free EHA Whitepaper) Congress.
In August, Ryvu presented its development plans, including broad development for RVU120 in hematologic and solid tumors and preclinical advancement of the synthetic lethality and immuno-oncology pipeline.
On September 19, Ryvu’s shareholders voted in favor of a resolution granting the Management and Supervisory Board the right to issue authorized capital, providing an important potential source of future financing.
– The first half of 2022 has been productive and progressive for Ryvu. Investors have appreciated the progress in our research and partnering and have supported our proposal to issue authorized capital at our discretion to finance development plans over the coming years. We are happy to have a quality investor base with such a great trust and understanding of our needs and potential – said Pawel Przewiezlikowski, co-founder, largest shareholder and CEO of Ryvu Therapeutics.

FIRST HALF 2022 AND RECENT HIGHLIGHTS

– Despite challenging market conditions, our ability to secure additional debt financing and establish an important new corporate partnership reflects the quality of our scientific expertise and clinical development capabilities – said Pawel Przewiezlikowski.

European Investment Bank (EIB) €22 million financing to accelerate the development of blood cancers and solid tumor therapies: The funding is provided under the EIB’s venture debt instrument and will finance Ryvu’s pipeline development of new cancer treatments. The funding is guaranteed by the European Fund for Strategic Investments, the financial pillar of the Investment Plan for Europe.

Exclusive License Agreement with Exelixis to develop new targeted therapies utilizing Ryvu’s STING (STimulator of INterferon Genes) technology: Under the terms of the agreement, Exelixis paid Ryvu an upfront fee of $3 million in exchange for certain rights to Ryvu’s STING agonist small molecules. Exelixis will be responsible for all research, development, and commercialization activities for which Ryvu will be eligible to receive milestone payments, as well as tiered royalties on the annual net sales of any products that are successfully commercialized under the collaboration.

Promotion of Ryvu’s Chief Medical Officer, Hendrik Nogai, and Chief Business Officer, Vatnak Vat-Ho to the Management Board: Dr. Nogai has served as Chief Medical Officer at Ryvu since February 2022 and Mr. Vat-Ho has been Ryvu’s Chief Business Officer since April 2021. The promotions reflect each individual’s significant contributions toward advancing Ryvu’s lead candidate, RVU120, and expanding Company’s business development activities.

Presentation of Clinical and Preclinical Data on RVU120 at the 2022 European Hematology Association (EHA) (Free EHA Whitepaper) Congress: Data from the ongoing Phase 1b dose-escalation study of RVU120 in patients with acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (HR-MDS) demonstrated single-agent activity with one complete remission (CR) and stable diseases (SD) with blast reductions in several ongoing patients who failed multiple prior lines of therapy.

Additionally, data on SEL24/MEN1703, an orally available dual PIM/FLT3 inhibitor was presented. SEL24/MEN1703 was well tolerated, with no drug discontinuations or deaths due to treatment-related adverse events (TRAEs) in patients with relapsed or refractory (R/R) IDHm AML. Promising efficacy was observed, with overall response rates (ORR) and complete remission (CR) / CR with incomplete hematologic recovery (CRi) / CR with partial hematologic recovery (CRh) of 13% for the IDHm cohort.

Key Opinion Leader Webinar on RVU120 for the Treatment of Hematological Malignancies and Solid Tumors: The webinar featured a presentation by KOL Dr. Michael Savona, MD, from Vanderbilt University School of Medicine, who discussed the current treatment landscape and unmet medical need in treating patients with AML and HR-MDS. Additionally, Ryvu’s leadership team discussed the underlying mechanism of action of RVU120 and provided an update on the Phase I data for RVU120.

Project updates from RVU120 and SEL24 programs presented at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting: Updates from the Phase 1/2 study of SEL24/MEN1703, a first-in-class dual PIM/FLT3 kinase inhibitor, in patients with IDH1/2-mutated acute myeloid leukemia and Phase I/II trial of RVU120, a CDK8/CDK19 inhibitor in patients with relapsed/refractory metastatic or advanced solid tumors were presented.

Poster Presentations at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting: Data were presented from the RVU120 Trial-In-Progress in patients with relapsed/refractory metastatic or advanced solid tumors and in vitro and in vivo data demonstrating RVU120 efficacy against hormone-independent breast cancer. Additionally, a novel MTA-cooperative PRMT5 inhibitor as a targeted therapeutic for MTAP deleted cancer was presented.

Shareholders meeting convened and approved authorized capital on September 19: At an extraordinary shareholders meeting, shareholders voted in favor of a resolution to approve authorized capital, which provides the Management and Supervisory Board the flexibility to issue equity at its discretion when optimal.

– We are really appreciative of the overwhelming support that we received at our shareholders’ meeting, which further enables Ryvu to execute our mission of discovering and developing drugs that will improve the lives of cancer patients and their families while maximizing the Company’s value – underlined Pawel Przewiezlikowski.

UPCOMING CLINICAL AND CORPORATE MILESTONES:

RVU120

Data update from the ongoing Phase I clinical study in acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (HR-MDS) and the ongoing Phase I clinical study in solid tumors will be presented in Q4 2022
Synthetic lethality

Data on Ryvu’s novel MTA-cooperative PRMT5 inhibitors as targeted therapeutics for MTAP deleted cancers to be presented in Q4 2022.

In August, Ryvu also highlighted its development plans for 2022-2024, which are focused on rapidly advancing its pipeline to treat cancer patients:

Broad development of RVU120, including a potential fast-to-market strategy in AML/HR-MDS,
Advancing into Phase I clinical trials one additional program,
Strengthening of the Synthetic Lethality Platform and discovery of novel targets,
Acceleration in the early pipeline,
Executing milestones from existing collaborations (i.e. Menarini, Galapagos, Exelixis), and adding at least one new partnering deal per year.
FIRST HALF 2022 FINANCIAL UPDATE:

Cash Position – On September 21, 2022, Ryvu Therapeutics held $9.7M in cash, cash equivalents, and short-term investments, compared to $20.5M at the end of 2021.

Operating costs, excluding the non-cash cost of valuation of the Incentive Program ($3.8M) and valuation of NodThera shares ($1.8M), for the 6 months period ended June 30, 2022, amounted to $13M and related primarily to research and development expenditures, while the operating costs without Incentive Program and valuation of Nodthera shares for the same period last year amounted to $11.5M.

Net Loss Attributable to Common Shareholders – Net loss attributable to common shareholders excluding the non-cash cost of valuation of the Incentive Program was $11M for the 6 months period ended June 30, 2022, as compared to a net loss without the Incentive Program of $8.2M for the same period last year.

Precision Cancer Consortium Adds New Members in Efforts to Improve Access to Comprehensive Genomic Testing for Patients with Cancer

On September 28, 2022 The Precision Cancer Consortium (PCC) reported the addition of two new members, Lilly and the Janssen Pharmaceutical Companies of Johnson & Johnson (Press release, The Precision Cancer Consortium, SEP 28, 2022, View Source [SID1234621528]). The PCC now consists of six member organizations including founding members Bayer, GSK, Novartis, and Roche who are collaborating to support efforts to improve patient access to comprehensive genomic testing in routine care and in clinical trials globally. The PCC will drive a variety of initiatives aimed at increasing patient access to precision diagnostics using comprehensive genomic testing, including next generation sequencing (NGS).

Aligned with one of PCC’s key aims to remove barriers to access and increase awareness of the benefit of genomic testing for healthcare stakeholders, the first educational session "Bringing Precision Oncology Closer to Community Care" will be held virtually on September 29th, 2022 at 16:00 CET. Join us for our interactive peer to peer workshop, the event is open to all, register here.

MorphoSys Presents New Long-Term Data from L-MIND Suggesting Durable Response to Treatment with Monjuvi® (tafasitamab-cxix) for Patients with R/R DLBCL

On September 28, 2022 MorphoSys U.S. Inc., a fully owned subsidiary of MorphoSys AG (FSE: MOR; NASDAQ: MOR), reported data from the ongoing L-MIND study showing that Monjuvi (tafasitamab-cxix) plus lenalidomide followed by Monjuvi monotherapy provided long-term efficacy in patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) treated for at least 2 years, including six patients on treatment for 5 years or more (Press release, MorphoSys, SEP 28, 2022, View Source [SID1234621527]). Additionally, the frequency of adverse events declined after patients transitioned from combination therapy to monotherapy. The data will be presented during a poster session at the Tenth Annual Meeting of the Society of Hematologic Oncology (SOHO 2022) in Houston, Texas.

"The body of data presented at SOHO shows the long-term duration of response to tafasitamab in some patients with relapsed or refractory diffuse large B-cell lymphoma not eligible for autologous stem-cell transplant," said Malte Peters, M.D., MorphoSys Chief Research and Development Officer. "These long-term follow-up results for L-MIND reaffirm our belief that tafasitamab plus lenalidomide remains the in-practice, outpatient targeted immunotherapy of choice for this group of patients."

At data cutoff (February 15, 2022), 27 of 80 patients (34%) had undergone treatment for at least 2 years, with a median duration of treatment of 4.3 years. Of those 27, 23 patients were alive at data cutoff, and 13 remained on treatment, including six who were on treatment for at least 5 years.

A complete response was observed in 23 of the 27 patients, including four who were refractory to their primary therapy. A partial response was seen in four patients, two of whom were still on treatment at data cutoff.

The majority of adverse events were grade 1 or 2 during both combination and monotherapy treatment. Patients experienced a lower frequency of all-grade and grade 3 or higher adverse events during monotherapy. The most common adverse events with combination therapy were neutropenia (incidence per person per year, all-grade/Grade ≥3: 3.87/1.91) and diarrhea (1.04/0.04), which declined after patients switched to monotherapy (all-grade/Grade ≥3: 0.87/0.45 and 0.32/0.00, respectively, in the first year of monotherapy). Neutropenia and diarrhea remained the most common adverse events in the first two years of monotherapy.

"The new long-term follow-up results from L-MIND at SOHO 2022 highlight the potential of tafasitamab in providing long-term efficacy in patients with relapsed or refractory diffuse large B-cell lymphoma," said Johannes Duell, M.D., University Hospital Wuerzburg Medical Clinic and Polyclinic. "The fact that six of the 27 patients who responded received treatment for 5 or more years – with another seven patients nearing the 5-year mark – points to the durable response induced by tafasitamab. This response durability represents the type of data that offers oncologists confidence when recommending therapies to their patients."

Atara Biotherapeutics to Receive Additional Near-Term Milestone Payment Under Updated Tabelecleucel (Tab-cel®) Commercialization Agreement with Pierre Fabre

On September 28, 2022 Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leader in T-cell immunotherapy, reported an update to its existing collaboration agreement with Pierre Fabre for the commercialization of tabelecleucel (tab-cel) for Epstein-Barr virus (EBV)-positive cancers (Press release, Atara Biotherapeutics, SEP 28, 2022, View Source [SID1234621526]).

Under the amended terms, Atara will receive an additional USD 30 million milestone payment upon tab-cel EC approval and subsequent filing of the Marketing Authorization Application (MAA) transfer to Pierre Fabre in exchange for reduced tab-cel royalties and supply price mark-up for Pierre Fabre. Tiered royalties on sales remain at a significant double-digit rate following this amendment.

"With an anticipated European approval in Q4 2022, tab-cel is positioned to become the first ever allogeneic off-the-shelf T-cell therapy available for patients with significant need," said Pascal Touchon, President and CEO of Atara. "This update to our agreement with Pierre Fabre reaffirms our strong partnership and shared belief in the transformative potential of tab-cel, while enabling Atara to further extend our cash runway."

In October 2021, Atara entered into an exclusive commercialization agreement with Pierre Fabre for tab-cel in Europe, Middle East, Africa and other select emerging markets for EBV-positive cancers, receiving an upfront payment of USD 45 million, and up to approximately USD 320 million in additional regulatory and sales milestone payments. In addition to responsibility for the pivotal ALLELE study in EBV+ post-transplant lymphoproliferative disease (EBV+ PTLD) and securing European Commission approval, Atara also remains responsible for the Phase 2 multi-cohort study, which is evaluating tab-cel in six additional patient populations with the goal of label expansion in other EBV-driven cancers. Pierre Fabre will lead all commercialization and distribution activities in the territories, as well as medical and regulatory activities after the anticipated MAA approval in Europe. Atara retains full commercialization rights to tab-cel in the United States and other major markets.

"Tab-cel has the potential to be a transformational product for EBV-positive cancers and is eagerly awaited by physicians and patients in Europe with limited treatment options," said Eric Ducournau, CEO of Pierre Fabre. "This update to our agreement with Atara confirms our confidence and commitment to tab-cel in Europe, where the Pierre Fabre team is excited to bring the first allogeneic T-cell therapy to European patients in a rare oncology condition."

Atara is leveraging its first-in-kind allogeneic off-the-shelf EBV T-cell platform to develop transformative therapies for patients including tab-cel, which is the Company’s lead candidate in development for EBV-positive cancers, including EBV+ PTLD, where it is currently being investigated in adults and children in the Phase 3 ALLELE study. Tab-cel has been granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA) and Priority Medicines (PRIME) designation by the European Medicines Agency (EMA).