On September 8, 2021 Strata Oncology, Inc., a precision oncology company advancing molecular indications for cancer therapies, reported that results from validation studies demonstrating the reliable performance of the StrataNGS test were published in The Journal of Molecular Diagnostics (Press release, Strata Oncology, SEP 8, 2021, View Source [SID1234587442]). StrataNGS is a 429-gene, multiplex PCR-based comprehensive genomic profiling (CGP) test performed on co-isolated DNA and RNA from formalin-fixed paraffin-embedded (FFPE) tissue tumor specimens with >2mm2 tumor surface area.

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Through a unique assay configuration and novel bioinformatics pipeline optimized for multiplex PCR-based sequencing, StrataNGS demonstrated high sensitivity and specificity for detecting single nucleotide variants, insertions/deletions, gene fusions, copy number alterations, microsatellite instability and tumor mutation burden. Designed to provide comprehensive assessment of clinically actionable biomarkers from minute FFPE solid tumor tissue samples, StrataNGS substantially increases patient access to tissue-based CGP. A recent study of >30,000 real-world tumor tissue samples received for CGP found that only 41.6% of samples met minimum tissue requirements (>25mm2 tumor surface area) for several leading hybrid capture-based CGP tests.

"StrataNGS demonstrated the sensitivity and specificity required for routine clinical practice while reporting all CGP variant classes from minute FFPE tissue samples," said Scott Tomlins, M.D., Ph.D., co-founder and Chief Medical Officer, Strata Oncology. "Conventional tissue-based CGP and liquid biopsy tests often fail to meet either the sensitivity or success rate needed to deliver reliable results for each patient. StrataNGS nearly eliminates the insufficient tissue problem, while delivering the sensitivity required to confidently rule-in or out a treatment. This approach directly translates into treatment insights for more patients."

About StrataNGS
StrataNGS is a comprehensive genomic profiling (CGP) test that features the lowest tumor tissue requirements in the industry (≥2mm2 surface area). The 429-gene assay is performed on co-isolated RNA and DNA. Single-/multi-nucleotide variants (SNVs), short insertions and deletions (indels), copy number alterations (CNAs; amplifications and deep deletions), microsatellite instability (MSI) status, gene fusions, and tumor mutation burden (TMB) are assessed simultaneously.

On September 8, 2021 Ryvu Therapeutics (WSE: RVU) reported its 2021 semiannual financial results and provided a corporate update (Press release, Ryvu Therapeutics, SEP 8, 2021, View Source [SID1234587441]).

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"The first half of 2021 was a time to remember for Ryvu. After some initial setbacks we reported major developments in our both clinical programs confirming single agent efficacy of RVU120 and SEL24/MEN1703, and we entered H2 2021 with a solid foundation for the further advancement of our pipeline" – commented Pawel Przewiezlikowski, Chief Executive Officer of Ryvu.

"In April 2021 the FDA placed a partial clinical hold on our Phase Ib clinical trial of RVU120 in AML/HR-MDS patients which was then successfully lifted in July. We also received a full approval to initiate Phase I/II trial with RVU120 in solid tumors, and the first patient has been already dosed in that trial. EHA (Free EHA Whitepaper) 2021 Congress was an exceptional one for us, as we got to present the first positive Phase I data for RVU120, along with the positive follow-up Phase II data presented for SEL24/MEN1703 by our partner, Menarini. We also enjoyed the first ever oral presentation of RVU120 at a major oncology conference, demonstrating its potential in promoting cell differentiation. Additionally, we welcomed Vatnak Vat-Ho, our new Chief Business Officer, on board" – adds Przewiezlikowski.

Recent Achievements

First patient dosed in Phase I/II study of RVU120 (SEL120) in patients with relapsed/refractory (R/R) metastatic or advanced solid tumors
On August 25, Ryvu announced that the first patient was dosed in the Phase I/II clinical trial investigating RVU120 in relapsed/refractory metastatic or advanced solid tumors. The single-agent, open-label Phase I/II trial is currently enrolling patients in Poland, and site expansion to Spain is in progress.
Initial positive clinical data presented at the EHA (Free EHA Whitepaper) Virtual Congress in June 2021; RVU120 Phase Ib study in AML and high-risk MDS patients resumed enrollment;
The initial safety and efficacy data from the first four cohorts in the trial were presented at the Virtual EHA (Free EHA Whitepaper) Congress on June 11, 2021. RVU120 demonstrated acceptable safety profile and two clinically relevant responses were observed in the first five AML and high-risk MDS patients treated: one complete response (CR) and one erythroid response. During the partial clinical hold, two patients continued on RVU120 treatment, and one MDS patients currently remains on study after more than 12 months of treatment with RVU120.
On July 14, Ryvu announced that the U.S. Food and Drug Administration lifted a partial clinical hold, previously announced on April 8, 2021, on the first-in-human (FIH) Phase Ib, dose escalation clinical trial of RVU120 in patients with R/R acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (high-risk MDS), being conducted in the United States.
Positive Phase I/II data for SEL24 (MEN1703) presented by development partner Menarini at EHA (Free EHA Whitepaper) 2021
A clinical poster on the first-in-human study of SEL24/MEN1703, the DIAMOND-01 trial conducted by Ryvu’s partner Menarini Group, reported four objective responses across the dose escalation (n=25) and cohort expansion (n=23) in patients with AML. Three out of five AML patients harboring IDH mutations treated at 75-125 mg achieved CR/CRi. A follow-up study in IDH-mutated patients was initiated by Menarini in July 2021.
Synthetic lethality platform highlighted in investor event: WRN, PRMT5 and novel targets advancing in the pipeline
On June 09, Ryvu held a live webinar called "Synthetic lethality in cancer treatment" with leading researchers in the field. The webinar targeted the Polish investor community and was well attended. Highlights of the webinar included Ryvu’s WRN/MSI-high and PRMT5/MTAP-deletion programs, as well as an overview of Ryvu’s discovery pipeline including novel synthetic lethality targets.
Publication of research on MCT4 inhibitors in the Journal of Medicinal Chemistry
On August 08, a joint publication of researchers from Ryvu Therapeutics and Merck KGaA on the discovery, development, and optimization of selective MCT4 inhibitors was published in the "Journal of Medicinal Chemistry": "Discovery of 5-{2-[5-Chloro-2-(5-ethoxyquinoline-8-sulfonamido)phenyl]ethynyl}-4-methoxypyridine-2-carboxylic Acid, a Highly Selective in Vivo Useable Chemical Probe to Dissect MCT4 Biology."
Ryvu is scheduled to participate in the following upcoming investor conferences:
Morgan Stanley 19th Annual Global Healthcare Conference on Thursday, September 9, 2021 – Wednesday, September 15, 2021. Ryvu will participate in a fireside chat on Friday, September 10 at 2:45 PM (ET), and host investor meetings during the conference.
H.C. Wainwright 23rd Annual Global Investment Conference, on Monday, September 13 – Wednesday, September 15, 2021. Ryvu’s corporate presentation will be available on-demand starting on September 13 at 7:00 AM (ET), and Ryvu will host investor meetings during the conference.
Ryvu 2021 Half Year, Financial Results

In the first Half Year of 2021, the Company reported PLN 12.2 million (USD 3.2 million) of revenues. Over 90% of the revenues were grants (PLN 11.3 million or USD 3.0 million) and the remainder consisted of income from partnering (PLN 0.4 million or $0.1 million), other revenues (PLN 0.3 million or USD 0.1 million) and other operating revenues in total PLN 0.2 million (USD 0.04 million).

Operating costs, excluding the non-cash cost of valuation of the Incentive Program (PLN 6.7 million or USD 1.8), in the audited period amounted to PLN 43.5 million (USD 11.5 million), and related in majority to research and development expenditures, while the operational loss without Incentive Program was PLN 31.3 million (USD 8.3 million) compared to PLN 11.8 million (USD 3.0 million) reported in the corresponding period in 2020. Net loss without Incentive Program for the first Half Year of 2021 amounted to PLN 30.9 million (USD 8.2 million) and the reported loss for the corresponding period of 2020 amounted to PLN 8.6 million (USD 2.1 million).

The results are consistent with the strategy assumptions of Ryvu Therapeutics. The company remains focused on increasing the value of its projects which are intended to be commercialised at later stages of the development cycle and generate substantial financial surpluses.

On August 31, 2021, Ryvu Therapeutics held PLN 96.4 million (USD 25.4 million) in cash, cash equivalents, and short-term investments.

On September 8, 2021 Cardiff Oncology, Inc. (Nasdaq: CRDF), a clinical-stage oncology company, developing new precision medicine treatment options for cancer patients in indications with the greatest unmet medical need including KRAS-mutated colorectal cancer, pancreatic cancer, and castrate-resistant prostate cancer, reported new data from its lead clinical program evaluating onvansertib in combination with standard-of-care (SOC) FOLFIRI/bevacizumab for second-line treatment of patients with KRAS-mutated metastatic colorectal cancer (mCRC) (Press release, Cardiff Oncology, SEP 8, 2021, View Source [SID1234587440]).

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"Our Phase 1b/2 trial continues to generate data suggesting that the addition of onvansertib to SOC results in an objective response rate and median progression-free survival that substantially exceed those previously achieved with SOC alone," said Katherine L. Ruffner, M.D., chief medical officer of Cardiff Oncology. "Radiographic responses have been observed across multiple KRAS mutation variants, which speaks to a key advantage of onvansertib over competing agents targeting individual mutations. These impressive results, which have remained consistent across both academic and community trial sites, highlight the potential for onvansertib to address the unmet need for new second-line therapeutic options to treat patients with KRAS-mutated mCRC. I look forward to the trial’s continued advancement and future data readouts."

Highlights from today’s data announcement include:

Efficacy data in patients evaluable for disease response as of data cutoff date (July 2, 2021):

Patients treated per protocol at the recommended Phase 2 dose (RP2D; 15 mg/m2) across both Phase 1b and Phase 2:
8 of 19 (42%) achieved an initial partial response (PR) as of the data cutoff date
7 of 19 (37%) have achieved a confirmed PR (based on further follow-up of patients with an initial PR as of data cutoff date)
Objective response rates observed in historical control trials in similar patient populations treated with standard of care are 5-13%1-4

Patients evaluable for response treated at all dose levels (12 mg/m2, 15 mg/m2, 18 mg/m2) across both phases of the study
12 of 32 (38%) achieved an initial PR as of the data cutoff date
10 of 32 (31%) have achieved a confirmed PR (based on further follow-up of patients with an initial PR as of data cutoff date)
Median progression free survival (mPFS)

mPFS has not yet been reached in patients treated per protocol at the RP2D
mPFS across all response-evaluable patients (n = 32) is 9.4 months (95% confidence interval: 7.8 – not yet reached)
mPFS of ~4.5-5.7 months has been reported in trials used as historical controls1-4
Biomarker data as of data cutoff date across all patients:

Partial responses (PRs) were observed across different KRAS mutation variants, including the 3 most common observed in colorectal cancer (G12D, G12V, G13D)
Patients achieving a best response of PR showed the greatest decreases in plasma KRAS mutant allelic frequency (MAF) after 1 cycle (28 days) of therapy
Safety data as of data cutoff date across all patients:

The combination of onvansertib and FOLFIRI/bevacizumab was shown to be well-tolerated with only 10% (49/490) of reported treatment-emergent adverse events (TEAEs) being G3/G4
Most reported treatment-related adverse events (TRAEs) were manageable and reversible with supportive care
Mark Erlander, Ph.D., chief executive officer of Cardiff Oncology, commented, "The strong signal of efficacy and favorable tolerability profile observed in this trial bodes well not only for our lead mCRC program, but for each of our KRAS-focused clinical programs. The meaningful improvements we are seeing in treatment response relative to historical controls demonstrate the value of combination therapy and support the synergistic effect observed preclinically when onvansertib is added to standard-of-care irinotecan and 5-FU (FOLFIRI). We are also seeing compelling biomarker results that highlight the potential utility of plasma KRAS MAF as a predictive tool that could aid in the design of subsequent trials. Looking forward, we anticipate the ongoing Phase 2 portion of the trial to provide additional data catalysts that will advance the clinical development of onvansertib, generate value for shareholders and, most importantly, provide new treatment options for patients."

Key Opinion Leader Webinar
The newly announced data are being discussed today at 4:00 PM ET as part of a key opinion leader (KOL) webinar being hosted by Cardiff Oncology. The webinar is featuring the clinical trial principal investigator, Heinz-Josef Lenz, M.D., FACP, USC Norris Comprehensive Cancer Center, key clinical advisor Afsaneh Barzi, M.D., Ph.D., City of Hope Comprehensive Cancer Center, and members of the Cardiff Oncology management team.

To attend the webinar, click here. A replay of the webinar will be available by visiting the "Events" section of the Cardiff Oncology website shortly after its conclusion.

About the Phase 1b/2 Trial of Onvansertib in KRAS-mutated mCRC
This is a multi-center, single-arm, Phase 1b/2 trial of onvansertib in combination with standard-of-care FOLFIRI and Avastin (bevacizumab) to evaluate the safety and preliminary efficacy of the combination regimen in the second-line treatment of patients with KRAS-mutated mCRC. The trial, A Phase 1b/2 Study of Onvansertib (PCM-075) in Combination with FOLFIRI and Bevacizumab for Second–Line Treatment of Metastatic Colorectal Cancer in Patients with a KRAS Mutation, is enrolling patients with histologically confirmed metastatic and unresectable colorectal carcinoma harboring a KRAS mutation. Patients must also have failed treatment with, or be intolerant to, FOLFOX (fluoropyrimidine and oxaliplatin) with or without bevacizumab to be eligible. The trial is being conducted at the following cancer centers across the U.S.: USC Norris Comprehensive Cancer Center, The Mayo Clinic (Arizona, Rochester, and Jacksonville), Kansas University Medical Center (KUMC), CARTI Cancer Center and Inova Schar Cancer Institute. For more information on the trial, please visit View Source

References

Giessen et al., Acta Oncologica 2015, 54: 187-193
Cremolini et al., Lancet Oncol 2020, 21: 497–507
Antoniotti et al., Correspondence Lancet Oncol June 2020
Bennouna et al., Lancet Oncol 2013; 14: 29–37

On September 8, 2021 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported that it will webcast management participation as follows (Press release, Regeneron, SEP 8, 2021, View Source [SID1234587439]):

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Morgan Stanley 19th Annual Global Healthcare Conference at 8:45 a.m. ET on Monday, September 13, 2021

Cantor Fitzgerald Virtual Global Healthcare Conference at 9:20 a.m. ET on Monday, September 27, 2021
The sessions may be accessed from the "Investors & Media" page of Regeneron’s website at View Source Replays of the webcasts will be archived on the Company’s website for at least 30 days.

On September 8, 2021 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that the company will participate in the following upcoming virtual investor conferences (Press release, Jazz Pharmaceuticals, SEP 8, 2021, View Source [SID1234587438]):

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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

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BofA Virtual Global Healthcare Conference 2021 on Wednesday, September 15, 2021

The presentation will be available to registered conference attendees.
Cantor Virtual Global Healthcare Conference on Monday, September 27, 2021

The presentation is scheduled for 4:40 p.m. ET / 9:40 p.m. IST.
An audio webcast of the presentation will be available via the Investors section of the Jazz Pharmaceuticals website at www.jazzpharmaceuticals.com. A replay of the webcast will be archived on the website for at least one week following the conference.