On January 24, 2022 Ranok Therapeutics, a biopharmaceutical company that is developing a novel approach to targeted protein degradation for the treatment of cancer and other serious diseases, reported that the U.S. Food and Drug Administration (FDA) has cleared the company’s investigational new drug application (IND) for RNK05047, a novel treatment for patients with advanced solid tumors and lymphomas (Press release, Ranok Therapeutics, JAN 24, 2022, View Source [SID1234606737]). Ranok expects to begin enrollment of the Phase 1/2 study entitled CHAMP-1 in the first half of 2022.

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RNK05047 is a first-in-class, small-molecule, BRD4-selective protein degrader that was discovered and developed using Ranok’s proprietary approach to targeted protein degradation, CHAMP (Chaperone-mediated Protein Degradation). This technology leverages the company’s expertise in protein homeostasis and the cellular chaperone network to degrade disease-associated proteins, and is designed to increase drug safety and efficacy through selective targeting of disease tissues.

"We are looking forward to initiating this clinical study of the first small-molecule therapy based on our CHAMP platform technology," said Weiwen Ying, Ph.D., Founder and Chief Executive Officer of Ranok Therapeutics. "The bromodomain transcription factor BRD4 is a master regulator of oncogenes involved in diverse cancer types. RNK05047 is designed to selectively degrade BRD4 protein in a tumor-targeted fashion, which differentiates it from other investigational therapies. We are optimistic that RNK05047 will provide a beneficial new therapeutic option for patients."

On January 24, 2022 Deciphera Pharmaceuticals, Inc. (NASDAQ: DCPH), a commercial-stage biopharmaceutical company developing innovative medicines to improve the lives of people with cancer, reported the presentation of results from the INTRIGUE Phase 3 study of QINLOCK (ripretinib) in patients with gastrointestinal stromal tumor (GIST) previously treated with imatinib at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Plenary Series Session (Press release, Deciphera Pharmaceuticals, JAN 24, 2022, View Source [SID1234606721]). The presentation, which follows the announcement in November 2021 of the top-line results, is titled "INTRIGUE: A phase III, randomized, open-label study to evaluate the efficacy and safety of ripretinib vs sunitinib in patients with advanced gastrointestinal stromal tumor previously treated with imatinib" and is available on the Company’s website at www.deciphera.com/presentations-publications.

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ASCO will be hosting a livestream event on Tuesday, January 25, 2022 at 3:00 PM ET featuring presentation of the abstract by Michael Heinrich, M.D., FACP, Professor of Medicine at Oregon Health & Science University followed by a discussion of the abstract by George D. Demetri, M.D., FASCO, FACP, Dana-Farber Cancer Institute, as well as a panel discussion with Drs. Heinrich and Demetri and Vicki Keedy, M.D., MSCI, Vanderbilt University Medical Center. To participate in the free and open session participants may register and login at View Source

"Patients with GIST in the post-imatinib setting are in need of additional treatment options for their disease, and the results from INTRIGUE demonstrate that ripretinib is an active and well-tolerated agent. Although the INTRIGUE study did not meet its primary endpoint of superiority in progression-free survival versus sunitinib, the efficacy of ripretinib appears comparable to sunitinib in 2nd line patients. In addition, ripretinib had a more favorable safety profile than sunitinib with fewer Grade 3/4 adverse events and patients reported less deterioration in role functioning and several other key patient-reported outcome measures of tolerability," said Dr. Heinrich. "It is important to provide the detailed results of this study to the oncology community to help treating physicians make well-informed decisions on the best treatment options for their patients with advanced GIST."

In patients with GIST who progressed on or were intolerant to imatinib, the efficacy of QINLOCK and sunitinib were comparable, although progression-free survival (PFS) of QINLOCK as determined by independent radiologic review using modified Response Evaluation Criteria in Solid Tumors (RECIST) did not meet the study’s primary endpoint of superiority compared to sunitinib. The statistical analysis plan included a hierarchical testing sequence that included evaluation of patients with a KIT exon 11 primary mutation (Exon 11) and then of the all patient (AP) population. Key highlights from the study presented today include the following:

An international, multicenter study conducted in 122 active sites across 22 countries, 453 patients were randomized to ripretinib (n = 226; Exon 11, n = 163) or sunitinib (n = 227; Exon 11, n = 164).
In patients with a KIT exon 11 primary mutation, ripretinib demonstrated a median PFS (mPFS) of 8.3 months compared to 7.0 months for the sunitinib arm (Hazard Ratio [HR] 0.88, p=0.36). In the AP population (n=453), ripretinib demonstrated an mPFS of 8.0 months compared to 8.3 months for the sunitinib arm (HR 1.05, nominal p value=0.72).
In patients with a KIT exon 11 primary mutation, ripretinib demonstrated an objective response rate (ORR) of 23.9% (n=39 of 163) compared to 14.6% (n=24 of 164) for sunitinib (nominal p value=0.03). In the AP population ripretinib demonstrated an ORR 21.7% (n=49 of 226) compared to 17.6% (n=40 of 227) for sunitinib (nominal p value=0.27).
Ripretinib was generally well tolerated. Fewer patients in the ripretinib arm experienced Grade 3-4 treatment-emergent adverse events compared to sunitinib (41.3% vs 65.6%).
Patients receiving sunitinib were three times more likely to develop Grade 3 hypertension compared to patients receiving ripretinib (26.7% vs. 8.5%) and patients receiving sunitinib were seven times more likely to develop Grade 3 palmar-plantar erythrodysesthesia compared to patients receiving ripretinib (10.0% vs. 1.3%).
Patient reported outcome measures also showed a more favorable tolerability profile for patients receiving ripretinib compared to patients receiving sunitinib. Patients receiving ripretinib experienced less deterioration in their ability to engage in either work or leisure activities during treatment and fewer patients receiving ripretinib experienced moderate to extremely large impact on their lives due to skin toxicity across treatment cycles compared to patients receiving sunitinib.
QINLOCK is approved by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with advanced GIST who have received prior treatment with three or more kinase inhibitors, including imatinib. The new drug application (NDA) for QINLOCK was based on positive results from the Phase 3 INVICTUS trial in patients with fourth-line and fourth-line plus GIST1. QINLOCK is also approved for the treatment of fourth-line GIST in Australia, Canada, China, the European Union, Hong Kong, Switzerland, Taiwan, and the United Kingdom.

About QINLOCK (ripretinib)

QINLOCK is a switch-control tyrosine kinase inhibitor that was engineered to broadly inhibit KIT and PDGFRA mutated kinases by using a dual mechanism of action that regulates the kinase switch pocket and activation loop. QINLOCK inhibits primary and secondary KIT mutations in exons 9, 11, 13, 14, 17, and 18 involved in GIST, as well as the primary exon 17 D816V mutation2,3. QINLOCK also inhibits primary PDGFRA mutations in exons 12, 14, and 18, including the exon 18 D842V mutation, involved in a subset of GIST2,3.

On January 24, 2022 SeqOne Genomics, provider of next genomic analysis solutions for personalized medicine reported a Series A funding round of €20M (Press release, SeqOne, JAN 24, 2022, View Source [SID1234606738]).

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The round, led by Omnes, Merieux Equity Partners, together with the Software Club and existing investors, Elaia and IRDI Capital Investissement, will enable SeqOne Genomics to accelerate its international sales and the development of new collaborative genomic analysis tools to facilitate communications between different disciplines in the medical team, in order to improve patient outcomes in cancer and hereditary disease.

The company will intensify investments in the development of its genomic-aware data lake to improve the use of big data and machine learning approaches in genomic analysis with the aim of better addressing the fast-evolving needs of molecular biology labs that provide genomic analysis in clinical routine environments, as well as to biopharma companies developing new therapies.

SeqOne’s cloud-based solution manages the entire genomic analysis process from raw data to final report presented to clinicians. The solution’s end-to-end approach affords better analytic performance, high levels of traceability and improved operational efficiency and has already been adopted by a score of hospitals and central labs and biopharmas as well as spawning partnerships with leading manufacturers of genomics analysis hardware and reagents.

Nicolas Philippe, Ph.D., Co-founder and CEO of SeqOne Genomics stated "We are extremely happy to have closed this round with leading investors who bring extensive experience in deep-tech, healthcare and biotherapy and who share our vision of building SeqOne into a global leader in personalized medicine. The funding will give us the resources we need to enhance and commercialize our solution to make genomic analysis more accessible and affordable so that each patient can benefit from personalized medicine recommandations."

"The genomics analysis market is experiencing exponential growth driven by the needs of personalized medicine. With the rapid expansion in the available genomic-linked treatments, the complexity of treatment interactions, and the staggering volume of biological and medical data to be factored into each medical decision, biologists and doctors must have access to reliable and actionable analyses in real-time," stated Fabien Collangettes, Director at Omnes. "We were particularly impressed by SeqOne’s innovative technological approach that enables improved accuracy of genomic test while reducing turnaround time and cost, thus delivering a key competitive advantage in this fast-growing market."

With the closing of this round, SeqOne’s board of directors will be: Sacha Loiseau, Ph.D. independent board member and Chairman of the Board, Fabien Collangettes, Director at Omnes, Yoann Bonnamour, Investment Manager at Merieux Equity Partners, Marc Rougier, Partner at Elaia, Nicolas Philippe, co-founder and CEO of SeqOne Genomics and Jean-Marc Holder, Co-founder and Chief Strategy and Innovation officer of SeqOne Genomics.

This new funding round brings the total amount raised by SeqOne Genomics since its founding in 2017 to €23M.

The company currently employs over 40 staff, primarily experts in genomic medicine, data science, bioinformatics, software development and regulatory compliance / quality assurance. It plans to double its staff within the year to execute its ambitious plans.

On January 24, 2022 MaxCyte, Inc., (NASDAQ: MXCT; LSE: MXCT, MXCN), a leading commercial cell-engineering company focused on providing enabling platform technologies to advance innovative cell-based research as well as next-generation cell therapeutic discovery, development and commercialization, reported a preliminary update on revenue results for the fourth quarter and full year 2021 (Press release, MaxCyte, JAN 24, 2022, View Source [SID1234606722]).

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Preliminary Unaudited Fourth Quarter 2021 and Full Year Revenue

Management expects total revenue for the fourth quarter of 2021 to be more than $10.0 million, up from $8.5 million of total revenue in the fourth quarter of 2020, reflecting growth of at least 17% in total revenue and at least 37% in core business revenue.

MaxCyte’s revenue for the fourth quarter of 2021 was derived from its core business, which is defined as sales or leases of instruments, sales of single-use disposables, and sales of consumables (buffer) to the cell therapy and drug discovery markets.

MaxCyte also generates revenue under Strategic Platform License agreements (SPLs) with cell therapy developers, such as precommercial milestone payments. These revenues are categorized as program-related revenue and are excluded from core business revenue.

Preliminary revenue for the full year ended December 31, 2021 is expected to be more than $33.7 million, up from $26.2 million in full year 2020, reflecting growth of at least 28% in total revenue and at least 36% in core business revenue. Revenue for the full year ended December 31, 2021 includes $2.5 million of program-related revenue, compared to $3.3 million of program-related revenue in 2020.

MaxCyte ended the year with 15 SPLs, including 4 SPLs added during 2021: Nkarta, Inc., Myeloid Therapeutics, Celularity, Inc. and Sana Biotechnology, Inc.

Doug Doerfler, President and CEO of MaxCyte said: "We are proud of our performance in the fourth quarter as well as the full year, which has been a year of key achievements for the company. This includes raising $257.2 million in gross equity proceeds, the completion of an IPO in the United States and commencement of trading in our common stock on the Nasdaq, continuing significant organic growth in our core business, and our ongoing success in signing SPLs with innovative cell therapy developers. We are also excited to confirm that our ExPERT VLx instrument became available for sale at the end of December."

"We remain optimistic about the potential for our SPLs to generate meaningful revenue over the next 12 to 18 months and beyond. Our partners continue to achieve clinical success – particularly in moving their next-generation product candidates into pivotal trials. We also see the potential for several IND filings by our SPL customers for novel ex vivo engineered cell therapies this year. Finally, we continue to benefit from the ongoing investment in the ex vivo engineered cell therapy space. As a result, we believe our SPL pipeline remains as robust and diverse as ever. We look forward to a strong 2022."

MaxCyte’s fourth quarter and full year 2021 financial results presented in this release are preliminary and unaudited and are subject to revision based on the completion of MaxCyte’s normal quarter and year-end process and year-end audit. As a result, these preliminary results may be different from the actual results that will be reflected in MaxCyte’s consolidated financial statements for the quarter and year ended December 31, 2021, which are expected to be released by the end of March 2022.

On January 24, 2022 Castle Biosciences, Inc. (Nasdaq: CSTL), a leader in transforming disease management and improving patient outcomes through innovative diagnostics, reported that it will present data highlighting its portfolio of skin cancer tests at Maui Derm for Dermatologists 2022, being held Jan. 24-28, 2022, in Maui, Hawaii (Press release, Castle Biosciences, JAN 24, 2022, View Source [SID1234606739]).

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Presentation details are as follows:

Title: Integrating the 31-gene expression profile and clinicopathologic data to determine the risk of sentinel lymph node positivity and recurrence-free survival in cutaneous melanoma
Title: Appropriate utilization of the prognostic 40-gene expression profile (40-GEP) test for cutaneous squamous cell carcinoma (cSCC) demonstrated by clinical reports and physician evaluation of real-world cases
Title: Evidence review of the prognostic 40-gene expression profile test for cutaneous squamous cell carcinoma
Title: A comprehensive diagnostic offering workflow increases the rate of actionable results of the 23- and 35-gene expression profile tests for use as ancillary diagnostic tools for difficult-to-diagnose melanocytic lesions
All posters will be available for viewing digitally at the poster viewing station in the Haleakala Foyer, on monitors throughout the conference and through the conference app and virtual conference platform.

About DecisionDx-Melanoma

DecisionDx-Melanoma is a gene expression profile test that uses an individual patient’s tumor biology to predict individual risk of cutaneous melanoma metastasis or recurrence, as well as risk of sentinel lymph node positivity, independent of traditional staging factors, and has been studied in more than 6,000 patient samples. Using tissue from the primary melanoma, the test measures the expression of 31 genes. The test has been validated in four archival risk of recurrence studies of 901 patients and six prospective risk of recurrence studies including more than 1,600 patients. Impact on patient management plans for one of every two patients tested has been demonstrated in four multicenter and single-center studies including more than 560 patients. The consistent performance and accuracy demonstrated in these studies provides confidence in disease management plans that incorporate DecisionDx-Melanoma test results. To predict risk of recurrence and likelihood of sentinel lymph node positivity, the Company utilizes its proprietary algorithms, i31-ROR and i31-SLNB, to produce an Integrated Test Result. Through Sept. 30, 2021, DecisionDx-Melanoma has been ordered 84,195 times for use in patients with cutaneous melanoma.

About DecisionDx-SCC

DecisionDx-SCC is a 40-gene expression profile test that uses an individual patient’s tumor biology to predict individual risk of cutaneous squamous cell carcinoma metastasis for patients with one or more risk factors. The test result, in which patients are stratified into a Class 1 (low), 2A (moderate) or 2B (high) risk category, predicts individual metastatic risk to inform risk-appropriate management.

Peer-reviewed publications have demonstrated that DecisionDx-SCC is an independent predictor of metastatic risk and that integrating DecisionDx-SCC with current prognostic methods can add positive predictive value to clinician decisions regarding staging and management.