On December 1, 2020 LianBio, a biotechnology company focused on bringing paradigm-shifting medicines to patients in China and other major Asian markets, reported that the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) has cleared the Company’s Clinical Trial Application (CTA) to conduct the Phase 2a trial of infigratinib in patients with locally advanced or metastatic gastric cancer or gastroesophageal junction adenocarcinoma with FGFR2 gene amplification (Press release, LianBio, DEC 1, 2020, View Source [SID1234572042]).

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LianBio has in-licensed the oncology rights for infigratinib from QED Therapeutics, an affiliate of BridgeBio Pharma, Inc., and is responsible for the clinical development, registration application and future commercial operations of the product candidate in Mainland China, Hong Kong and Macau.

Infigratinib is an investigational oral, selective inhibitor of fibroblast growth factor receptor (FGFR) 1-3 that has shown activity in clinical measures, such as overall response rate, in patients with chemotherapy-refractory cholangiocarcinoma with FGFR2 fusions and advanced urothelial carcinoma with FGFR3 genomic alterations [1][2]. The Phase 2a trial is a multicenter, single-arm study designed to explore and evaluate the pharmacokinetic profile, efficacy and safety of infigratinib in patients with locally advanced or metastatic gastric cancer or gastroesophageal junction adenocarcinoma with FGFR2 gene amplification. Infigratinib is also currently under Phase 3 global development by LianBio and BridgeBio for patients with FGFR2 fusion positive cholangiocarcinoma.

"With strong support from our partner, BridgeBio, the exploratory study highlights LianBio’s continued commitment to expand its global footprint and develop potential breakthrough therapeutics in China," said Dr. Bing Li, Chief Executive Officer of LianBio. "Gastric cancer is the third most common cancer in China, causing approximately 300,000 deaths every year. This clearance by the China NMPA to conduct the Phase 2a trial of infigratinib in gastric cancer will enable LianBio to work towards addressing the significant unmet medical need for this growing patient population."

References

Javle M. et al. A phase II study of infigratinib (BGJ398), an FGFR-selective tyrosine kinase inhibitor (TKI), in patients with previously-treated advanced cholangiocarcinoma containing FGFR2 fusions; ESMO (Free ESMO Whitepaper) 2018 Annual Meeting. Poster #LBA28.
Pal K. et al. Efficacy of BGJ398, a Fibroblast Growth Factor Receptor 1-3 inhibitor, in patients with previously treated advanced urothelial carcinoma with FGFR3 alterations; Cancer Discovery 2018.

On December 1, 2020 Turnstone Biologics Corp., a clinical-stage biotechnology company pioneering the development of engineered viral immunotherapies, reported enrollment of the first patients in a Phase 1/2a clinical trial (RAPTOR) of its RIVAL-01/TAK-605 candidate in patients with solid tumors, conducted in collaboration with Takeda Pharmaceutical Company Limited ("Takeda") (Press release, Turnstone Biologics, DEC 1, 2020, View Source [SID1234572041]). RIVAL-01 consists of Turnstone’s proprietary oncolytic vaccinia virus backbone encoding transgenes for Flt3 ligand, anti-CTLA-4 antibody and IL-12 cytokine, purposefully designed to work together to drive immune activity and re-program the microenvironment to be best suited for tumor eradication.

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"The enrollment of the first patients into our Phase 1/2a trial is a key milestone for Turnstone and meaningfully advances our aim of transforming the treatment paradigm for people with cancer," said Mike Burgess, Ph.D., President of R&D, Turnstone. "The meticulous design of our differentiated vaccinia virus platform enabled us to engineer RIVAL-01 to deliver three powerful immune modulating agents locally to primary and metastatic tumor sites, by either intratumoral or systemic administration. We look forward to sharing future updates as the study progresses."

Turnstone’s RIVAL therapeutic pipeline is based on its proprietary vaccinia virus platform, which has been engineered for enhanced immune-stimulation and tumor cell selectivity, potent oncolysis and large transgene carrying capacity. The transgenes are designed to be expressed when the vaccinia virus, delivered either intratumorally or intravenously, enters and replicates in cancer cells. The resulting local production of these therapeutics at the site of tumors adds to the inherent oncolytic and microenvironment-modifying properties of the virus to form a powerful multi-modal attack on the disease. RIVAL-01 is being co-developed and co-commercialized under a global collaboration with Takeda.

"Takeda’s partnership with Turnstone continues to gain momentum. Our teams successfully collaborated to establish the RIVAL-01/TAK-605 clinical development plan while simultaneously working together to advance additional novel next-generation multi-payload oncolytic virus concepts to the clinic", said Chris Arendt, Ph.D., Head, Oncology Therapeutic Area Unit, Takeda. "The trial enrollment represents an important step forward in our collaboration and ongoing efforts to leverage innate and adaptive immunity to overcome the limitations of current immuno-oncology therapies."

The RAPTOR trial will evaluate the safety and efficacy of RIVAL-01 (formerly known as TBio-6517) administered intratumorally as a single agent and in combination with Keytruda (pembrolizumab) in patients with solid tumors, including triple negative breast cancer, microsatellite stable colorectal cancer, melanoma and cholangiocarcinoma. Patients are currently enrolling in Phase 1 dose escalation, which will be followed by an expansion phase in specified tumor types in the Phase 2a portion of the trial.

Additional information about the trial, including eligibility criteria and a list of clinical trial sites, can be found at View Source (ClinicalTrials.gov Identifier: NCT04301011).

On December 1, 2020 NanoString Technologies, Inc. (NASDAQ:NSTG), a leading provider of life science tools for discovery and translational research, reported the development of a spatial molecular imaging platform, a next generation solution for multiplexed analysis of RNA and protein expression for individual cells within a tissue sample (Press release, NanoString Technologies, DEC 1, 2020, View Source [SID1234572040]).

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The spatial molecular imager combines the power of high-plex profiling with high-resolution imaging to enable the analysis of hundreds to thousands of RNAs or proteins directly from single cells within morphologically intact tissue samples. The platform utilizes the company’s advanced Hyb & Seq chemistry, which over the last 18 months has been optimized for in situ spatial analysis. The platform is capable of profiling RNA and protein within a single cell down to sub-cellular resolution in multiple tissue types. To date, prototype systems have imaged RNA from as many as 1,000+ genes simultaneously across thousands of individual cells in Formalin-Fixed Paraffin Embedded (FFPE) tissue.

Tissue biology takes place on several spatial scales including multi-cellular, single cell and the sub-cellular levels. The GeoMx Digital Spatial Profiler (DSP) enables multi-cellular analysis at the whole transcriptome level to elucidate the behavior of populations of cells, such as those within a tumor or the tumor microenvironment. NanoString is developing the spatial molecular imager to address the unmet need for high-plex spatial analysis of single cells and sub-cellular resolution, ideally suited for targeted applications, such as creating cell atlases or studying cell-cell interactions. The GeoMx DSP and the spatial molecular imager are highly synergistic, creating a spatial biology portfolio that spans the continuum from targeted to whole transcriptome analysis, and from multicellular resolutions down to single cell and sub cellular applications.

"Spatial imagers measuring RNA and protein in situ at single-cell and sub-cell resolution are expected to emerge as an important product category over the next several years," said Brad Gray, president and CEO. "Our spatial molecular imaging platform can profile RNA and protein in FFPE with category-leading plex as well as high sensitivity and high resolution. Together, our GeoMx DSP and spatial imager form the leading spatial biology portfolio, spanning from discovery and translational research to clinical diagnostics."

"Researchers and clinicians have gained tremendous insights from the revolution in single cell biology in recent years, but the cellular context was always lost," said Christopher Mason, Ph.D., a geneticist who recently published research using GeoMx. "Now, biological discoveries can be elucidated from high-plex, multi-omic spatial imaging, revealing the key spatial interactions within and between normal or diseased tissues (e.g. COVID-19, cancer) and how these cells communicate and mediate phenotypes, change over time, and respond to therapies."

"Spatial molecular imaging opens up new possibilities to understand the diversity of T-cells infiltrating a tumor at the single cell level without dissociating tissue. Our work under the Technology Access Program has produced results on the location of different T-cell subsets and their interaction with tumors and could reveal which T-cell subsets are most important for controlling tumors," said Evan Newell, Ph.D., Associate Professor, Vaccine and Infectious Disease Division, the Fred Hutchinson Cancer Research Center.

The company plans to provide researchers access to the prototype versions of spatial molecular imaging platform through a Technology Access Program (TAP) beginning in 2021. Under the program, a TAP customer can submit tissue samples to NanoString to be analyzed using the spatial molecular imager and resulting data analysis reported back to the customer by NanoString. Full commercial launch of the spatial imaging solution consisting of instruments, consumables, and software is expected in the second half of 2022.

The Hyb & Seq chemistry was originally developed as part of a hybridization-based sequencing program. The company formed a strategic collaboration with Lam Research, under which Lam Research provided $50 million in funding and access to their industry-leading semi-conductor technology. This collaboration resulted in a robust and scalable chemistry that can be applied to a wide range of applications.

The company is hosting an Investor and Analyst Day webcast at 11:30am ET on Tuesday, December 1st, and will highlight the development program and new capabilities of the platform. Interested parties can access the webcast through the company’s website at View Source The webcast will be available on the company site for 90 days.

Researchers interested in participating in NanoString’s Technology Access Program should contact us at [email protected].

On December 1, 2020 Biodesix, Inc. (Nasdaq: BDSX) a leading data-driven diagnostic solutions company with a focus in lung disease, reported publication of an analysis of the company’s Nodify XL2 lung nodule test (Press release, Biodesix, DEC 1, 2020, View Source [SID1234572039]). The test supports clinical decision-making for suspicious nodules by more accurately identifying patients with a very low risk of malignancy and shifting those patients into surveillance, thereby minimizing invasive procedures on those with benign nodules.

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In previously published findings from the Pulmonary Nodule Plasma Proteomic Classifier (PANOPTIC) Trial, the Nodify XL2 test was shown to accurately identify patients with lung nodules who have a pre-test risk of malignancy less than 50% as "likely benign." After one year of follow-up, the test demonstrated a sensitivity of 97%, specificity of 44%, and negative predictive value of 98%, which is more accurate than other commonly used lung nodule risk assessment calculators.

The new paper, published in the American College of Chest Physicians (CHEST) Journal, presents findings that all nodules in the study group that were established as benign after one year remained benign after two years of follow-up. This data confirms the performance of the Nodify XL2 test over the guideline-recommended two-year surveillance period to radiologically confirm a benign diagnosis. Additionally, a new analysis suggests that the classifier performs similarly regardless of the whether the nodule of concern was solitary or there were other nodules present.

"This assessment demonstrates our commitment to providing long-term follow-up for patients and to continuously study the performance of our tests," said Scott Hutton, CEO of Biodesix. "Central to our mission is the drive to improve patient outcomes while reducing ineffective and unnecessary treatments and procedures. Nodify XL2 exemplifies this. With this test, part of our Nodify LungTM testing strategy, physicians are equipped with vital and time-sensitive information to help efficiently determine the appropriate course of treatment for each patient."

About Nodify XL2 Lung Nodule Test

The Nodify XL2 blood-based proteomic test helps identify patients who have a suspicious lung nodule that is likely benign or at a reduced risk of being cancerous. Results help physicians to identify patients who may be better candidates for routine CT surveillance to monitor for growth or shrinkage of the nodule over time instead of an invasive diagnostic procedure. The Nodify XL2 test is used for patients who are 40 years or older, have nodules between 8mm and 30mm, and have a pre-test risk of lung cancer of less than or equal to 50%.

The test is performed in Biodesix’s COLA-accredited laboratory in De Soto, Kansas.

On December 1, 2020 MaaT Pharma reported that the company secured an additional €7.35 million ($8.7 million) in an extension of its Series B financing round, bringing the total raised in this round to €25.35 million (Press release, MaaT Pharma, DEC 1, 2020, View Source [SID1234572038]). The new capital from the PSIM Fund managed by Bpifrance on behalf of the French State, with participation from SkyViews Life Science and Celeste Management, underscores the potential of MaaT Pharma’s microbiome restoration biotherapeutics platform and the progress the company has made towards developing drugs that harness the rich diversity of the gut microbiome to improve overall survival in blood cancers and Graft-versus-Host Disease (GvHD). In February, the company announced the initial closing of an €18 Million Series B Financing Round, led by US investor SymBiosis, LLC, with support from Seventure Partners, Crédit Mutuel Innovation, and Biocodex.

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The final closing of the Series B financing round will primarily support the advancement of an innovative fermentation production platform for the development of next-generation, large-scale, fully controlled microbiome ecosystem therapeutics. By leveraging MaaT Pharma’s GutPrint computational data science platform, the new industrial fermentation platform will enable to develop and manufacture both "standard" high-richness, high-diversity fermented product MaaT034, and a pipeline of indication-specific candidates. In particular, the fermentation technology supports pipeline extension towards solid tumors indications, for combination with immunotherapy. The resulting new product candidates will complement the company’s donor-derived lead products MaaT013 (enema) and MaaT033 (oral formulation) for the treatment of GvHD and liquid tumors.

The funding will also support the production of MaaT013 to meet the demand from clinicians wanting to access the product for the treatment of their GvHD patients with no other therapeutic options; this compassionate use is permitted by the French regulator under an "ATUn" program.

"Raising additional capital through an extension of our Series B is a testament to the relevance of our innovative full-ecosystem microbiome therapy approach. We are now supported by a syndicate of seven highly respected investors, enabling us to further advance and expand our portfolio with confidence. Serving patients has always been the cornerstone of our purpose and we look forward to meeting their needs through our microbiome therapeutics development and in our early-access-program," commented Hervé Affagard, CEO and Co-founder of MaaT Pharma. "I would like to thank all of our investors for their trust and for sharing our company’s commitment to improving patients’ lives and treatment outcomes in a variety of cancer indications by restoring gut microbiome function and immune system homeostasis."

"MaaT Pharma has developed an innovative portfolio of microbiome-based biotherapeutics that we believe offers tremendous short-term and long-term potential, addressing the needs of advanced-stage cancer patients with limited options," commented Muriel Prudent, Senior Investment Manager at Bpifrance. "We look forward to working with MaaT Pharma’s team to expand its leadership position and contribute to advancing its innovative pipeline."

Dr Stefan Catsicas, Managing Partner at SkyViews Life Science said: "SkyViews Life Science is delighted to join a strong group of investors collectively committed to supporting MaaT Pharma, a leader in the field of microbiome functionality, as they address severe unmet needs of cancer patients."

"MaaT Pharma has brought together a strong team grounded in scientific and manufacturing excellence with a compelling vision for microbiome-based therapies, which convinced us to support this endeavor," added Dimitri Boulanger, Chief Executive Officer, Celeste Management.