On September 21, 2021 858 Therapeutics reported that stealth mode with a $60 million Series A financing led by founding investor Versant Ventures, with participation from NEA, Cormorant Asset Management and Logos Capital (Press release, 858 Therapeutics, SEP 21, 2021, View Source [SID1234590053]). Proceeds are being used to develop a portfolio of small molecules directed against novel therapeutic targets in oncology, including proteins that regulate RNA and the innate immune response.

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858’s founding team includes CEO Jeffrey Stafford, Ph.D., CSO James Veal, Ph.D., and VP of Biology Gretchen Bain, Ph.D. They previously held leadership roles at three other San Diego-based discovery companies co-founded by Versant including Jecure Therapeutics, Quanticel Pharmaceuticals and Amira Pharmaceuticals. Jecure developed therapies against innate immunity targets to treat serious inflammatory diseases and was acquired by Genentech in 2018. Quanticel developed DNA-modulating drug candidates and was acquired by Celgene in 2015. Amira developed anti-fibrotic small molecules and was acquired by Bristol-Myers Squibb in 2011.

The management team has been responsible for the discovery of over 20 drug candidates that have advanced into clinical trials, including the approved drugs VotrientTM (pazopanib) and NesinaTM (alogliptin).

858’s lead programs focus on the emerging fields of RNA modulation and innate immune pathways in cancer and expand upon insights from over 10 years of work by the founders at predecessor companies. Behind these, the company is pursuing emerging cancer targets in other pathways.

"The launch of 858 builds on our long-standing relationship with Versant in San Diego and leverages our prior experiences in both nucleic acid metabolism and innate immunity," said Dr. Stafford. "We are also pleased to have such strong support from NEA, Cormorant, and Logos as we develop an internal pipeline of precision oncology drug candidates."

RNA modulation as a broad and untapped field

There is an increasing appreciation that RNA modifications play a large role in numerous forms of cancer due to their direct influence on gene expression, tumorigenesis and immune-sensing pathways. Until now, however, it has been challenging to address many of these enzyme targets because of a dearth of assays and challenges in creating chemical matter with drug-like properties.

858 has built a platform to assess the impact of RNA-modifying proteins on disease biology and has generated a pipeline of small molecules against priority targets. The platform includes a suite of proprietary in vitro biochemical and biophysical assays, as well as cellular assays.

To accelerate the development of its pipeline, 858 has acquired Gotham Therapeutics for undisclosed terms. Gotham is a pioneer in the field of RNA biology, translating novel biological discoveries from the lab of Samie Jaffrey, M.D., Ph.D., co-founder of Gotham and now an advisor to 858.

"We are confident that 858 is well-positioned to capitalize on recent scientific discoveries in the field of RNA modification and translate them into a pipeline of drug candidates," said Clare Ozawa, Ph.D., Managing Director at Versant and an 858 board member. "Moreover, we are very pleased to again be working with Jeff, Jim, and Gretchen on a new company, given their proven track records in achieving successful outcomes for patients, employees and investors."

Operating plans

The Series A proceeds will allow 858 to enter the clinic with its lead candidates that have potential to treat cancers that are resistant to current therapies. 858 is headquartered in San Diego and will maintain lab operations both in San Diego and New York City. With this financing, the company plans to expand its team to about 40 people within the next 18 months. In addition, the 858 team will engage leading academic scientists who have made important contributions in the field and can bring specialized know-how to the company.

"858’s discovery platform provides a unique opportunity to interrogate this novel area of biology and make an impact for patients with intractable forms of cancer," said Ali Behbahani, M.D., general partner at NEA and an 858 board member. "We look forward to working with management to translate these recent discoveries into a pipeline of important therapeutics."

On September 21, 2021 Novartis reported that it has acquired Arctos Medical, adding a pre-clinical optogenetics-based AAV gene therapy program and Arctos’ proprietary technology to its ophthalmology portfolio (Press release, Novartis, SEP 21, 2021, View Source [SID1234588089]). The acquisition underscores the Novartis commitment to finding treatments for patients with vision loss and the potential of optogenetics as the basis of successful therapeutics.

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"Optogenetics is emerging as a promising therapeutic approach that might restore sight to patients who are legally blind," said Jay Bradner, President of the Novartis Institutes for BioMedical Research. "The Arctos technology builds on our conviction that optogenetic gene therapies may meaningfully help patients battling devastating eye diseases."

Arctos developed its technology as a potential method for treating inherited retinal dystrophies (IRDs) and other diseases that involve photoreceptor loss, such as age-related macular degeneration (AMD). Existing gene therapy treatments aim to correct a specific gene, so only a small subset of patients can benefit. The Arctos technology is not limited to a specific gene, and thus can potentially address many forms of IRDs regardless of the underlying mutation. Arctos’ proprietary, light-sensitive optogene is delivered to specific retinal cells using gene therapy, thus turning the targeted cells into replacement photoreceptor-like cells. If successful, a therapeutic based on such a technology could be used to treat any disease that causes blindness due to photoreceptor death.

"We’ve watched this technology develop and mature into a therapeutic program that complements our existing portfolio and gives us new optogenetics technology to wield in our efforts to bring desperately needed therapeutic options to patients for these blinding diseases," said Cynthia Grosskreutz, Global Head of Ophthalmology at the Novartis Institutes for BioMedical Research.

IRDs, which impact more than 2 million people globally and often result in complete blindness, can be caused by mutations in over 100 different genes.1 AMD is the leading cause of visual disability, affecting an estimated 170 million people globally.2 There are no curative therapies currently available for AMD.

The Arctos technology was based on discoveries by its scientific co-founders Drs. Sonja Kleinlogel and Michiel van Wyk of University of Bern, Switzerland. Arctos was originally incubated by +ND Capital and was later supported by Novartis Venture Fund through a Series A financing round led by +ND Capital.

On September 21, 2021 Vivoryon Therapeutics N.V. (Euronext Amsterdam: VVY; NL00150002Q7) (Vivoryon), a clinical stage company focused on discovery and development of small molecule medicines to modulate the activity and stability of pathologically altered proteins, reported financial results for the first six months of 2021 and provided an update on clinical and corporate progress (Press release, Vivoryon Therapeutics, SEP 21, 2021, View Source [SID1234588088]).

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"In the first half of 2021, we made significant progress towards bringing our lead candidate varoglutamstat to patients suffering from Alzheimer’s disease. Despite the ongoing pandemic, recruiting into our European Phase 2b study VIVIAD is on track and we’re particularly excited about the initiation of our complementary Phase 2 study VIVA-MIND in the US. Furthermore, by entering into a strategic regional partnership with Simcere, we have made substantial headway towards making varoglutamstat available to AD patients in China in the future," said Dr. Ulrich Dauer, CEO of Vivoryon. "Beyond AD, we are pleased to see our diverse preclinical pipeline of oral small molecule inhibitors maturing in a number of indications with exceptionally high medical need."

Corporate Highlights and R&D Updates
Varoglutamstat

Vivoryon’s US Phase 2a/b VIVA-MIND study for varoglutamstat in patients with early AD is being initiated as planned. VIVA-MIND is a combined Phase 2a/b study expecting to enroll 180 patients into the Phase 2a adaptive dose finding part, with an interim futility analysis planned for H1/2023. If predefined criteria are fulfilled, the trial is stage-gated into the Phase 2b part, enrolling an additional 234 patients treated at the selected dose for ³72 weeks. The primary endpoint for this study is CDR-SB (clinical dementia rating scale – sum of boxes), an established approvable endpoint measuring a combination of cognitive abilities and activities of daily living. The VIVA-MIND study is sponsored by Vivoryon and the study director is Dr. Howard Feldman, Professor of Neurosciences and Director of the Alzheimer’s Disease Cooperative Study (ADCS) at the University of California San Diego School of Medicine. The study is coordinated by the ADCS, and supported by the National Institute on Aging (NIA), part of the National Institutes of Health (NIH) (NIA award number R01AG061146). The study’s first site is now approved to initiate screening of its first participant.
On June 29, 2021, Vivoryon and Simcere Pharmaceutical Group Ltd announced that they have entered into a strategic regional licensing partnership to develop and commercialize medicines targeting the neurotoxic amyloid species N3pE (pGlu-Abeta) to treat AD in Greater China. The agreement grants Simcere a regional license to develop and commercialize varoglutamstat (PQ912), Vivoryon’s Phase 2b-stage N3pE amyloid-targeting oral small molecule glutaminyl cyclase (QPCT) inhibitor with disease-modifying potential for AD, as well as the Company’s preclinical monoclonal N3pE‑antibody PBD-C06 in the Greater China region.
Enrollment into the ongoing European Phase 2b VIVIAD study in patients with mild cognitive impairment (MCI) and mild AD is on track, with an interim safety readout anticipated in mid-2022. A number of additional study centers were opened to balance the effects of COVID-19 related patient and staff protection policies implemented at German study sites. Details on the study led by Prof. Dr. Philip Scheltens, University Medical Center, Amsterdam, were recently published in a peer-reviewed journal as Vijverberg et al., Alzheimer’s Research & Therapy (2021) 13:142 (View Source).
Patent Portfolio

Throughout the first half of 2021, Vivoryon has significantly expanded its patent portfolio with eight additional patents granted for the Company’s small molecule inhibitors and antibody-based medicines in development to treat AD and other diseases with exceptionally high medical need. Year to date (as of September 21, 2021) a total of 14 additional patents have been granted.
Corporate Developments

On June 28, 2021, Vivoryon held its 2021 Annual General Meeting as a virtual event. All items presented for resolution by the Board of Directors were approved with a large majority and can be found on the Company’s website.
On April 15, 2021, Vivoryon hosted a virtual event covering next steps in AD treatment options with leaders and experts in the field. The interactive session covered discussions surrounding current hurdles and exciting, novel approaches to the challenging AD space, including varoglutamstat, the Company’s small molecule inhibitor of QPCT designed to target all three hallmarks of AD: amyloid-beta, tau, and neuroinflammation.
On April 1, 2021, Florian Schmid joined Vivoryon as Chief Financial Officer. He joined the Company from InflaRx, where he served as Director Finance & Controlling. Prior to Vivoryon Mr. Schmid led the Global Deal & Business Support department at T‐Systems International GmbH. He began his career as certified Tax Advisor and Public Accountant at Arthur Andersen and Ernst & Young. Mr. Schmid holds a business degree from the Ludwig‐Maximilian‐University, Munich.
Financial Results for the First Six Months of 2021
In the first two quarters of 2021, research and development expenses amounted to EUR 9,456 k (H1 2020: EUR 6,380 k). This increase was mainly driven by higher expenses for production (H1 2021: EUR 4,194 k, H1 2020: EUR 1,876 k), expenses for share-based payments (H1 2021: EUR 464 k, H1 2020: EUR 3 k) and higher costs associated with basic research projects in connection with Meprin (H1 2021: EUR 220 k, H1 2020: nil) and cancer (H1 2021: EUR 162 k, H1 2020: nil).

General and administrative expenses increased to EUR 2,337 k (H1 2020: EUR 1,138 k). This increase is largely attributable to costs for consulting (H1 2021: EUR 1,030 k, H1 2020: EUR 481 k) and expenses for share based payments (2021: EUR 464 k, 2020: nil). The increase in consulting costs resulted from the transformation of the Company’s legal form, subsequent adaption of administrative structures and preparations for potential future capital measures.

The Company did not generate any licensing revenues in the reporting period. Revenues deriving from the strategic regional licensing partnership with Simcere will be recognized starting in the third quarter of 2021.

Net loss of the period was EUR 11,671 k compared to EUR 7,572 k in the first half of 2020.

The Company held EUR 19,832 k in cash and cash equivalents as of June 30, 2021, compared to EUR 26,306 k as of December 31, 2020.

Conference Call and Webcast
Vivoryon will host a conference call and webcast today, September 21, 2021, at 3:00 pm CEST / 9:00 am EDT. A Q&A session will follow the presentation of the half year results.

Please dial in ten minutes prior to commencement.

A live webcast and slides will be made available at: www.vivoryon.com/investors-news/news-and-events/presentations-webcasts/.

Approximately one day after the call, a slide-synchronized audio replay of the conference will be available on: www.vivoryon.com/investors-news/news-and-events/presentations-webcasts/.

The statement for the first six months of 2021 is available on the Company’s website www.vivoryon.com/investors-news/financial-information.

On September 21, 2021 Selecta Biosciences, Inc. (NASDAQ: SELB), a biotechnology company leveraging its clinically validated ImmTOR platform to develop tolerogenic therapies that selectively mitigate unwanted immune responses, reported that Company’s Management will participate in a fireside chat and one-on-one investor meetings at the Cantor Virtual Global Healthcare Conference to be held September 27-30, 2021 (Press release, Selecta Biosciences, SEP 21, 2021, View Source [SID1234588087]).

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Details on the conference be found below.

Cantor Virtual Global Healthcare Conference
Format: Fireside chat and one-on-one investor meetings
Date: Monday September 27, 2021
Presentation Time: 3:20 p.m. EST
Webcast: Click Here

An archived webcast will also be accessible in the Investors & Media section of the company’s website at www.selectabio.com.

On September 20, 2021 OSE Immunotherapeutics reported that the positive final results of its Phase 3 trial of neoepitope-based cancer vaccine Tedopi, called Atalante 1, in HLA-A2 positive patients with advanced non-small cell lung cancer (NSCLC) after immune checkpoint inhibitor (PD-1/PD-L1) failure, were presented in a late-breaking oral presentation(1) at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Virtual Congress being held on September 16 – 21, 2021 (Press release, OSE Immunotherapeutics, SEP 20, 2021, View Source [SID1234646971]).

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Pr. Benjamin Besse, Director of Clinical Research at Gustave Roussy (Villejuif, France), and Principal Investigator of the Atalante 1 study, commented: "There was a lot learned during this trial about Tedopi and its potential clinical benefit that will help inform future studies in the immunotherapy field. Applying the 2020 SITC (Free SITC Whitepaper)(2) guidelines defining resistance categories for PD-1/PD-L1 checkpoint inhibitors to our trial, developed while this trial was ongoing, suggest there is great potential for Tedopi in patients with secondary resistance(3) . Therefore, we are very pleased to share such very promising results demonstrating the substantial benefits of Tedopi for NSCLC patients with secondary resistance to anti-PD-1 treatments, a hard to treat patient population with high medical need."

The Atalante 1 clinical trial evaluated the benefit of Tedopi in an HLA-A2 positive patient population with NSCLC at invasive stage IIIB or metastatic stage IV, in 2nd or 3rd line treatment following checkpoint inhibitor failure. The Tedopi treatment was compared to docetaxel or pemetrexed chemotherapy (CT) treatments in this patient population, with overall survival as the primary endpoint of the trial.

The Atalante 1 clinical trial evaluated the benefit of Tedopi in an HLA-A2 positive patient population with NSCLC at invasive stage IIIB or metastatic stage IV, in 2nd or 3rd line treatment following checkpoint inhibitor failure. The Tedopi treatment was compared to docetaxel or pemetrexed chemotherapy (CT) treatments in this patient population, with overall survival as the primary endpoint of the trial.

A total of 219 patients were enrolled in Atalante 1. 183 (84%) of these patients received sequential CTimmunotherapy (IO), of which 118 patients (54%) met the definition of PoI, with otherwise similar other baseline characteristics to the overall Atalante 1 population.

Tedopi demonstrated a favorable benefit/risk ratio versus standard of care (SoC) docetaxel or pemetrexed in advanced HLA-A2+ NSCLC patients with secondary resistance to immune checkpoint inhibitors.

The main results were:
Improved efficacy
1. Overall survival (primary endpoint) was statistically significantly improved for Tedopi: HR=0.59 (95% CI: 0.38, 0.91) in favor of the Tedopi arm. A clinically meaningful gain in median overall survival of 3.6 months in favor of the Tedopi arm with Tedopi OS at 11.1 months versus 7.5 months for SoC (p=0.017).

2. The objective response rate and progression free survival (PFS) were lower in the Tedopi arm, as
expected for a therapeutic vaccine versus a cytotoxic drug while at 6 months, the disease control
rate (DCR) was similar (25 % Tedopi versus 24 % SoC).

3. Post progression survival was also significantly longer in the Tedopi arm (7.7 months versus 4.6 months; p=0.004).

Improved safety profile
1. A good ECOG performance status(5), with time to ECOG deterioration significantly longer in the Tedopi arm (8.6 months versus 3.3 months; p=0.0005).

2. A maintained quality of life was observed with Tedopi (p= 0.04).

3. A good tolerance profile of Tedopi with fewer Severe Adverse Events (Tedopi 38% vs SoC 68%, p<0.001). No Treatment Emergent Adverse Effects of concern in the Tedopi arm.