On May 12, 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 that the data from the Phase 1b dose-escalation study of RVU120 (SEL120) in patients with AML or high-risk myelodysplastic syndromes (HR-MDS) and the Phase 1/2 study of SEL24 (MEN1703) in Patients with IDH1/2-Mutated AML will be presented at the Annual European Hematology Association (EHA) (Free EHA Whitepaper) 2022 Hybrid Congress, June 9-17 in Vienna, Austria and online (Press release, Ryvu Therapeutics, MAY 12, 2022, View Source [SID1234614433]).

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"The data presented at this year’s EHA (Free EHA Whitepaper) Congress demonstrate Ryvu’s commitment to providing first-in-class and effective treatment options for people with hematologic malignancies," said Hendrik Nogai, M.D., Chief Medical Officer at Ryvu Therapeutics. "We are excited to share promising results from our ongoing Phase 1b dose-escalation study in AML and HR-MDS, in which we have seen single-agent activity of RVU120. Notably, our translational data confirm the proposed MoA in a molecularly-defined subset of patients with DNMT3A and NPM1 mutations. Based on the encouraging data, we plan to further advance the clinical development of RVU120 in both biomarker-driven AML patients as well as in the unselected broader AML population.

We are also proud that the SEL24 (MEN1703) program, developed in collaboration with our partner Menarini, is making progress in the clinic. We look forward to future updates from our clinical programs throughout 2022, as we continue to advance new and innovative therapeutics for patients suffering from cancer."

Details on the poster presentations are as follows:

RVU120: orally available CDK8/19 inhibitor

Abstract Title: "Preclinical and Clinical Signs of RVU120 Efficacy, a Specific CDK8/19 Inhibitor in DNMT3A Mutation Positive AML and HR-MDS"

Abstract Number: #P450

Session date and time: Friday, June 10 at 16:30 – 17:45 CEST

Preclinical data demonstrate differential sensitivity to RVU120 treatment in DNMT3 and NPM1 mutated AML Patient-Derived Cells (PDCs) in vitro and in vivo. Anti-cancer efficacy of RVU120 was associated with transcriptomic reprogramming involving inhibition of homeobox genes and induction of lineage commitment markers. Preliminary evidence of clinical response to RVU120 has also been shown in relapsed/refractory (R/R) AML and HR-MDS patients positive for DNMT3A and NPM1 mutations. Both mutations cooperate and define molecular subset of AML, characterized by elevated expression of homeobox genes. Further molecular studies confirming molecular mechanism of action and potential stratification markers are ongoing in more patients treated with RVU120.

Abstract Title: "CLI120-001 Phase1b Dose Escalation Study of RVU120 in Patients with AML or High-Risk MDS Safety and Efficacy Data Update"

Abstract Number: #P501

Session date and time: Friday, June 10 at 16:30 – 17:45 CEST

The presentation includes preliminary results from the first six patient cohorts, which demonstrated a favorable safety and a predictable pharmacokinetic (PK) profile of RVU120. Meaningful pharmacodynamic (PD) activity and clinical efficacy have been observed at the 50 and 75 mg doses. Enrollment in the trial continues with highest dose cohort now receiving 85 mg of RVU120 (NCT04021368).

SEL24/MEN1703: orally available dual PIM/FLT3 inhibitor

Abstract Title: "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: The DIAMOND-01 Trial"

Abstract Number: #P520

Session date and time: Friday, June 10 at 16:30 – 17:45 CEST

Ryvu’s partner Menarini Group reports the updated safety and efficacy results from an additional expansion cohort of the DIAMOND-01 trial which enrolled patients with relapsed or refractory (R/R) IDHm AML treated with the dual PIM/FLT3 inhibitor SEL24 (MEN1703). SEL24/MEN1703 demonstrated a manageable safety profile and single-agent activity in patients with R/R IDHm AML.

All abstracts are now available online and can be obtained from the conference site: View Source

On May 12, 2022 Harpoon Therapeutics, Inc. (Nasdaq: HARP), a clinical-stage immunotherapy company developing novel T cell engagers, reported financial results for the first quarter ended March 31, 2022 and provided a corporate update (Press release, Harpoon Therapeutics, MAY 12, 2022, View Source [SID1234614479]).

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"We continue to advance our robust pipeline of T cell engagers and explore their therapeutic potential in areas of unmet medical need," said Julie Eastland, President and Chief Executive Officer of Harpoon Therapeutics. "We anticipate upcoming milestones in the second half of the year for our lead programs from our TriTAC platform and our next generation ProTriTAC T cell engager HPN601 in solid tumors. We look forward to sharing our progress as we work to bring these important therapeutic options to patients."

First Quarter 2022 Recent Highlights and Upcoming Milestones

Enrollment continues across Harpoon’s portfolio of novel T cell engagers for the treatment of cancer:

Tri-specific T cell Activating Construct (TriTAC) Platform

HPN328 (DLL3) Phase 1/2 trial in small cell lung cancer (SCLC) and neuroendocrine cancers

Harpoon plans to present interim clinical results from the ongoing Phase 1 part of the study in a poster presentation at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting 2022 on June 6 at 8:00 a.m. CT.
In May 2022, Harpoon entered into a Master Clinical Supply Agreement with F. Hoffmann-La Roche Ltd for the supply of atezolizumab (Tecentriq) for use in the Company’s planned clinical trials to evaluate HPN328 in combination with atezolizumab for the treatment of patients with SCLC.
In March 2022, the U.S. Food and Drug Administration (FDA) granted Orphan Drug designation to HPN328 for the treatment of patients with SCLC.
Harpoon is continuing dose escalation with the goal to identify expansion dose(s) by year-end 2022.
HPN217 (BCMA) Phase 1/2 trial for relapsed, refractory multiple myeloma

In March 2022, the FDA granted Fast Track designation to HPN217 for the treatment of patients with relapsed, refractory multiple myeloma.
Compelling initial clinical activity observed in dose escalation phase of ongoing trial. Maximum tolerated dose (MTD) has not been reached and enrollment in escalation cohorts continues in first half of 2022.
Harpoon plans to initiate a Phase 2 dose expansion trial in the second half of 2022.
HPN536 (MSLN) Phase 1/2a trial for tumors expressing mesothelin

The dose escalation phase of the ongoing Phase 1/2a clinical trial for cancers expressing mesothelin is ongoing and is expected to be complete by year-end 2022.
ProTriTAC

ProTriTAC is a conditionally active T cell engager platform that is designed to be preferentially active in the tumor. This enables our T cell engagers to address more broadly expressed solid tumor targets across multiple tumor types.

HPN601 (EpCAM)

HPN601 is the first conditionally active T cell engager based on the ProTriTAC platform. EpCAM is expressed in a broad range of solid tumors, including gastrointestinal cancers, potentially enabling HPN601 to address multiple indications with high unmet medical need.
Harpoon expects to advance HPN601 with an IND submission in the second half of 2022.

TriTAC-XR

The proprietary TriTAC-XR extended-release T cell engager platform is designed to minimize on-target cytokine release syndrome (CRS), a characteristic of many T cell engagers that can lead to dose limiting toxicities and can reduce the efficacy of these potent anti-tumor drugs.

In April 2022, preclinical data supporting Harpoon’s TriTAC-XR platform were highlighted in a poster presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, demonstrating improved safety by minimizing CRS.
First Quarter 2022 Financial Results

Harpoon ended the first quarter of 2022 with $112.5 million in cash, cash equivalents and marketable securities compared to $136.6 million as of December 31, 2021. Current cash is expected to fund operations through the first half of 2023.

Revenue for the quarter ended March 31, 2022 was $5.9 million, compared to $9.0 million for the quarter ended March 31, 2021. The decrease in revenue was primarily due to lower revenue recognized from the Amended and Restated Discovery Collaboration Agreement with AbbVie.

Research and development (R&D) expense for the quarter ended March 31, 2022 was $20.8 million, compared to $16.2 million for the quarter ended March 31, 2021. The increase primarily arose from higher clinical development and personnel-related expense, which included conducting preclinical studies and clinical trials for HPN328, HPN217 and HPN536.

General and administrative (G&A) expense for the quarter ended March 31, 2022 was $5.4 million, compared to $4.6 million for the quarter ended March 31, 2021. The increase was primarily attributable to an increase in personnel expenses related to an increase in headcount and other professional services to support Harpoon’s operations as a public company.

Net loss for the quarter ended March 31, 2022 was $20.3 million, compared to $61.7 million for the quarter ended March 31, 2021. The prior year period included a $50 million legal settlement expense.

On May 12, 2022 NeoTX reported the acquisition of InterX. InterX, is a drug discovery company utilizing advanced proprietary tools of biomolecular computation for the discovery and design of novel therapeutic molecules based on Nobel Prize Laureate Prof Michael Levitt’s discoveries (Press release, NeoTX, MAY 12, 2022, View Source [SID1234640355]). These tools allow a detailed quantum mechanical process assessment of biochemical interactions resulting in faster, more accurate and efficient drug discovery. In addition to the discovery engine, this acquisition also comes with a pipeline of early drugs.

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"We are excited to be expanding NeoTX capabilities to include a suite of proprietary drug discovery technologies. InterX is a world class team that includes three Nobel Laureates who have developed technologies that have the potential to increase the speed of drug discovery. These technologies augment traditional Computer Assisted Drug Discovery (CADD) and are designed to replace much of the typical synthesis and testing cycles that are needed after obtaining results from the traditional CADD process, while potentially saving three-plus years of the typical drug discovery timeline" said CEO, Asher Nathan.

"InterX’s technology is the most advanced in the industry, and when combined with the development expertise of NeoTX, we will be able to advance best-in-class drug candidates, increase the value of our pipeline, and strive to contribute to improvement of quality of life within society," said Nobel Prize Laureate Prof. Roger Kornberg, Chief Scientist of NeoTX and cofounder of InterX. InterX will benefit from the experience and infrastructure of NeoTX while maintaining a culture of innovation and creativity. Post-merger, Prof. Kornberg has also assumed the role of Chairman of the Board of NeoTX.

On May 12, 2022 Pulmatrix (NASDAQ: PULM), a clinical-stage biopharmaceutical company developing innovative inhaled therapies to address serious pulmonary and non-pulmonary disease using its patented iSPERSE technology, reported first quarter financial results for 2022 and provided a corporate update (Press release, Pulmatrix, MAY 12, 2022, View Source [SID1234614311]).

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Ted Raad, Chief Executive Officer of Pulmatrix commented, "We have prioritized capital towards extending our cash runway through the Pulmazole Phase 2b top-line data anticipated in Q2 2024. We anticipate dosing for the Phase 2b study to begin in Q1 2023. Also in 2022, we will further analyze the PUR1800 Phase 1b data to finalize a potential Phase 2 study design while we execute to deliver PUR3100 Phase 1 top-line data in Q4 2022."

First Quarter 2022 and Recent Program Highlights

Pulmazole (PUR1900)

The Pulmazole Phase 2b efficacy study will include a 16-week dosing regimen with potential registration efficacy endpoints, is on track to begin dosing patients in Q1 2023. With a focus on capital conservation, we have extended our projected cash runway through the anticipated top-line data readout in Q2 2024.
PUR3100

On January 25, 2022, the Company conducted a Type C meeting with the FDA to add additional clarification around some of the written pre-IND responses in relation to the overall non-clinical and clinical program. Management concluded that conducting the Phase 1 study in Australia should allow the Company to generate the most comprehensive dataset for inclusion in an IND for Phase 2 in the United States, while also providing the most time efficient path to Phase 1 data in 2022.
PUR1800

On March 21, 2022, the Company announced top-line data from a Phase 1b clinical study of PUR1800 assessing the safety, tolerability and pharmacokinetics of PUR1800 in patients with stable COPD after dosing the first patient in February 2021. We are analyzing the Phase 1b clinical study data for future publication and to finalize design of a potential Phase 2 efficacy study in treatment of AECOPD.
First Quarter Corporate Highlights

On February 28, the Company completed a reverse stock-split at a ratio of 1-for-20 which reduced the number of outstanding shares of the Company’s common stock from approximately 65.9 million shares to 3.3 million shares.

On March 1, the Company announced the hiring of Dr. Margaret Wasilewski as the Company’s Chief Medical Officer. Dr. Wasilewski leverages over 25 years of experience in pharmaceutical drug development.

On March 17, the Company announced that it regained compliance for its listing on Nasdaq, allowing continued access to capital markets and liquidity for its investors.
First Quarter 2022 Financial Results

Revenue was $1.2 million for the first quarter ended March 31, 2022, compared to $1.4 million for the same period in 2021, a decrease of $0.2 million. Revenue for 2022 from the collaboration and license agreement with Cipla on the Company’s Pulmazole program increased, offset by no revenues from a previous JJEI License Agreement for the Company’s PUR1800 kinase inhibitor.

For the three months ended March 31, 2022, research and development expenses were $4.1 million compared to $3.9 million for the same period in 2021, an increase of $0.3 million. The increase was primarily due to increased spend of $0.7 million in employment costs and $0.1 million in rent costs, partially offset by decreased spend of $0.3 million on preclinical costs related to our PUR1800 program and $0.2 million on clinical and manufacturing costs related to the Pulmazole program.

General and administrative expenses were $2.0 million for the three months ended March 31, 2022, compared to $1.6 million for the three months ended March 31, 2021, an increase of $0.4 million. The increase was primarily due to increased spend of $0.1 million in employment costs, $0.3 million on consulting and legal, and $0.1 million on audit, tax and public company expense, partially offset by decreased patent expense of $0.1 million.

Our total cash and cash equivalents balance as of March 31, 2022 was $47.5 million. We expect that our existing cash and cash equivalents as of March 31, 2022 will enable us to fund our projected operating expenses and capital expenditures into Q2 2024.

On May 12, 2022 Paige, a global leader in clinical AI applications in pathology, reported it received CE-IVD and UKCA marks for the Paige Prostate Biomarker Suite, an AI software that is designed to detect the presence of four prostate cancer biomarkers on digitized tissue images stained with hematoxylin and eosin (H&E)* (Press release, Paige AI, MAY 12, 2022, View Source [SID1234614328]). This is the first European regulatory certification of image-based biomarker detection on H&E-stained tissue samples for Paige, potentially expanding the utility of AI to analyze tissues prepared with the most frequently used stain in histology.

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In contrast to traditional molecular biomarker testing, image-based biomarkers can enable faster turnaround time for results, provide new information at the point of diagnosis and reduces the need for unnecessary and expensive testing on definite negatives, while protecting the integrity of the tissue sample. Specifically, the Paige Prostate Biomarker Suite can detect biomarkers from H&E-stained samples and can inform the need for confirmatory tests such as immunohistochemistry (IHC) or fluorescence in situ hybridization (FISH).

The Paige Prostate Biomarker Suite is designed to assist in the detection of Androgen Receptor (AR), TP53, RB1 and PTEN biomarkers that are associated with the development and progression of prostate cancer. The biomarkers can help physicians stratify patients into treatment paradigms and direct targeted enrollment into clinical trials. The Paige Prostate Biomarker Suite was developed using the same underlying technology from Paige Prostate Detect, which was developed with histology image data from tens of thousands of patients and is already CE-IVD and UKCA marked, in addition to being approved by the FDA in the U.S.

"By employing Paige Prostate Biomarker Suite, clinicians can rapidly reduce laboratory turnaround time while providing a broader range of data at the point of diagnosis," said Jill Stefanelli, Ph.D., President and Chief Business Officer at Paige. "We’re excited by this regulatory milestone of our biomarker capabilities built on our robust AI technology platform, which can rapidly screen and develop proof-of-concept biomarkers. As we expand our biomarker portfolio, we also look forward to developing novel biomarkers across indications to identify patients that should receive genomic testing or could potentially respond to targeted therapies. This opens the door to a whole new range of biomarker applications and, in turn, new opportunities for industry collaboration."

For more information about the Paige Prostate Biomarker Suite, contact [email protected].

*In the United States, Paige Prostate Biomarker Suite is available for Research Use Only and not for use in diagnostic procedures.