On April 28, 2020 CytoSorbents Corporation (NASDAQ: CTSO), a critical care immunotherapy leader commercializing its CytoSorb blood purification technology to treat deadly inflammation in critically-ill and cardiac surgery patients around the world, reported that it will report Q1 2020 financial results after the market close on Tuesday, May 5, 2020 (Press release, Cytosorbents, APR 28, 2020, View Source [SID1234556723]).

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CytoSorbents’ management will host a live conference call and presentation webcast that will recount both operational and financial progress during Q1 2020 followed by a question and answer session.

Conference Call Details:

Date:

Tuesday, May 5, 2020

Time:

4:45 PM Eastern

Toll Free:

877-451-6152

International:

201-389-0879

Conference ID:

13701769

Live Presentation Webcast:

View Source

It is recommended that participants dial in approximately 10 minutes prior to the start of the call. There will also be a simultaneous live webcast of the conference call that can be accessed through the following audio feed link: View Source

An archived recording of the conference call will be available under the Investor Relations section of the Company’s website at View Source

On April 28, 2020 Arvinas, Inc. (Nasdaq: ARVN), a clinical-stage biotechnology company creating a new class of drugs based on targeted protein degradation, reported financial results for the first quarter ended March 31, 2020 and provided a corporate update (Press release, Arvinas, APR 28, 2020, View Source [SID1234556668]).

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"Throughout the first quarter, we continued to execute our plan and have made steady progress in our clinical pipeline. We are excited to present updated clinical data from our Phase 1/2 dose escalation trial of ARV-110, as a potential treatment for men with metastatic castration-resistant prostate cancer (mCRPC), at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting this quarter. We look forward to further discussing the potential of ARV-110 in this vulnerable patient population," said John Houston, Ph.D., President and Chief Executive Officer of Arvinas.

"As we move through the year, we remain committed to implementing a strategy that leverages our innovative PROTAC protein degradation platform and continuing to invest in advancing our entire pipeline," added Dr. Houston.

Anticipated Milestones and Expectations

For the ARV-110 program, Arvinas expects to share updated data from the dose escalation portion of its Phase 1/2 clinical study in men with mCRPC in a presentation at the virtual ASCO (Free ASCO Whitepaper) annual meeting in the second quarter of 2020.
For the ARV-471 program, which is being studied in patients with locally advanced or metastatic ER+/HER2- breast cancer, Arvinas expects to share data from the dose escalation portion of its Phase 1/2 clinical trial in the second half of 2020.
In the second half of 2020, Arvinas expects to provide information about the advancement of additional programs in its robust preclinical pipeline.
Financial Guidance
Based on its current operating plan, Arvinas expects its cash, cash equivalents, and marketable securities will be sufficient to fund its planned operating expenses and capital expenditures into 2022.

Cash, Cash Equivalents, and Marketable Securities Position: As of March 31, 2020, cash, cash equivalents, and marketable securities were $262.8 million as compared with $280.9 million as of December 31, 2019. The decrease of $18.1 million primarily related to cash used to fund operations of $20.9 million and cash used to purchase fixed assets and leasehold improvements of $1.4 million, partially offset by receipts from collaborators of $4.0 million.
Research and Development Expenses: Research and development expenses were $21.7 million for the quarter ended March 31, 2020 as compared with $14.2 million for the quarter ended March 31, 2019. The increase of $7.5 million in research and development expenses for the quarter primarily related to increasing investments in platform research, exploratory and lead optimization programs, our estrogen receptor (ER) clinical program and our androgen receptor (AR) clinical program.
General and Administrative Expenses: General and administrative expenses were $7.9 million for the quarter ended March 31, 2020 as compared with $5.6 million for the quarter ended March 31, 2019. The increase of $2.3 million in general and administrative expenses for the quarter was primarily related to increases in personnel and facility-related costs.
Revenue: Revenue was $6.2 million for the quarter ended March 31, 2020 as compared with $4.0 million for the quarter ended March 31, 2019. The increase of $2.2 million in revenue is primarily due to revenue related to the Bayer collaboration agreement, which was initiated in July 2019, and an increase in license and rights to technology fees and research and development activities related to the Pfizer collaboration agreement.
Net Loss: Net loss was $21.7 million for the quarter ended March 31, 2020 as compared with $14.4 million for the quarter ended March 31, 2019. The increase of $7.3 million in net loss for the quarter primarily related to our increasing investments in platform research, exploratory and lead optimization programs, our ARV-471 clinical program, our ARV-110 clinical program, and increases in general and administrative personnel costs partially offset by an increase in revenue primarily related to the Bayer collaboration agreement that was initiated in July 2019 and an increase in license and rights to technology fees and research and development activities related to the Pfizer collaboration agreement.
About ARV-110
ARV-110 is an orally bioavailable PROTAC protein degrader designed to selectively target and degrade the androgen receptor (AR). ARV-110 is being developed as a potential treatment for men with metastatic castration-resistant prostate cancer (mCRPC).

ARV-110 has demonstrated activity in preclinical models of AR mutation or overexpression, both common mechanisms of resistance to currently available AR-targeted therapies.

About ARV-471
ARV-471 is an orally bioavailable PROTAC protein degrader designed to specifically target and degrade the estrogen receptor (ER) for the treatment of patients with locally advanced or metastatic ER+/HER2- breast cancer.

In preclinical studies, ARV-471 demonstrated near-complete ER degradation in tumor cells, induced robust tumor shrinkage when dosed as a single agent in multiple ER-driven xenograft models, and showed superior anti-tumor activity when compared to a standard of care agent, fulvestrant, both as a single agent and in combination with a CDK4/6 inhibitor.

On April 28, 2020 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported the presentation of a new off-the-shelf, iPSC-derived, chimeric antigen receptor (CAR)-targeted cell-based cancer immunotherapy program at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 23rd Annual Meeting to be virtually hosted on May 12 – 15, 2020 (Press release, Fate Therapeutics, APR 28, 2020, View Source [SID1234556688]). The new preclinical program targets MHC class I related proteins A (MICA) and B (MICB), and is supported by an exclusive license from the Dana-Farber Cancer Institute to intellectual property covering novel antibody fragments binding MICA/B for iPSC-derived cellular therapeutics. MICA and MICB are stress proteins that are selectively expressed at high levels on many solid tumors.

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"MICA and MICB are emerging as exciting pan-cancer immunotherapy targets, and we are pleased that our engineered iPSC-derived CAR-MICA/B NK cells have shown potent anti-tumor activity in preclinical studies," said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. "Importantly, since the shedding of MICA/B is a common escape mechanism deployed by many tumors to evade immune cell recognition, we are encouraged that our novel CAR design, which uniquely targets the α3 domain of MICA/B, prevents protein shedding and promotes NK and T cell-mediated tumor immunity."

While it is well known that tumor resistance to cytotoxic T cells is mediated by loss of MHC Class I expression, proteolytic shedding of the α1 and α2 domains of MICA/B expressed on tumor cells is a common mechanism of NK cell evasion. A recent publication in Science (DOI:10.1126/science.aao0505) by Kai W. Wucherpfennig, M.D., Ph.D., Chair of the Department of Cancer Immunology and Virology at the Dana-Farber Cancer Institute and co-leader of the Cancer Immunology Program at Dana-Farber / Harvard Cancer Center, demonstrated that antibody targeting of the MICA/B α3 domains specifically prevents MICA/B shedding and restores NK cell-mediated immunity. Additionally, in a more recent publication in Cancer Immunology Research (DOI: 10.1158/2326-6066.CIR-19-0483), Dr. Wucherpfennig also demonstrated that cancers with B2M and JAK1 inactivating mutations resulting in loss of MHC Class I expression can be effectively targeted with MICA/B α3 domain-specific antibodies to restore NK cell-mediated immunity against solid tumors resistant to cytotoxic T cells.

Using a clonal master induced pluripotent stem cell (iPSC) line previously engineered with an expression cassette introduced into the CD38 locus that encodes for a high-affinity, non-cleavable CD16 Fc receptor and an IL-15 receptor fusion, scientists from Fate Therapeutics introduced a second expression cassette encoding for the novel CAR MICA/B construct. In vitro differentiation of the clonal iPSC lines produced homogeneous NK cell populations with uniform expression of CAR MICA/B, hnCD16, and IL15RF, and without any expression of CD38. The iPSC-derived CAR NK cells displayed enhanced cytokine production and cytotoxicity against MICA/B positive tumor cells compared to non-engineered, iPSC-derived NK cells.

Other Company abstracts selected for presentation at ASGCT (Free ASGCT Whitepaper) include the derivation of clonal master engineered iPSC lines for the Company’s FT576 product candidate, an off-the-shelf, multi-antigen targeted CAR-BCMA NK cell engineered with four anti-tumor modalities for the treatment of multiple myeloma; and a study of various expression systems for the generation, engineering and cryopreservation of clonal master engineered iPSC lines.

About MICA and MICB Proteins
The major histocompatibility complex (MHC) class I related proteins A (MICA) and B (MICB) are induced by cellular stress, damage or transformation, and the expression of MICA and MICB proteins has been reported for many tumor types. Cytotoxic lymphocytes, such as NK cells and CD8+ T cells, can detect and bind the membrane-distal α1 and α2 domains of MICA/B, activating a potent cytotoxic response. However, advanced cancer cells frequently evade immune cell recognition by proteolytic shedding of the α1 and α2 domains of MICA/B. The clinical importance of proteolytic shedding is reflected in the association of high serum concentrations of shed MICA/B with disease progression in many solid tumors. Several recent publications have shown that therapeutic antibodies targeting the membrane-proximal α3 domain strongly inhibited MICA/B shedding, resulting in a substantial increase in the cell surface density of MICA/B and restoration of NK cell-mediated tumor immunity. Therapeutic approaches aimed at targeting the α3 domain of MICA/B therefore represent a potentially promising novel strategy to overcome this prominent evasion mechanism as a means of restoring anti-tumor immunity.

About Fate Therapeutics’ iPSC Product Platform
The Company’s proprietary induced pluripotent stem cell (iPSC) product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that can be administered with multiple doses to deliver more effective pharmacologic activity, including in combination with cycles of other cancer treatments. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Company’s first-of-kind approach involves engineering human iPSCs in a one-time genetic modification event and selecting a single engineered iPSC for maintenance as a clonal master iPSC line. Analogous to master cell lines used to manufacture biopharmaceutical drug products such as monoclonal antibodies, clonal master iPSC lines are a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, can be mass produced at significant scale in a cost-effective manner, and can be delivered off-the-shelf for patient treatment. As a result, the Company’s platform is uniquely capable of overcoming numerous limitations associated with the production of cell therapies using patient- or donor-sourced cells, which is logistically complex and expensive and is subject to batch-to-batch and cell-to-cell variability that can affect clinical safety and efficacy. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 300 issued patents and 150 pending patent applications.

On April 28, 2020 F-star Therapeutics Ltd., a clinical-stage biopharmaceutical company focused on transforming the lives of patients with cancer through the development of innovative tetravalent bispecific (mAb2) antibodies, reported the publication of preclinical data on the focused, potent and safe immune response shown with FS222 in leading peer-reviewed journal Clinical Cancer Research (Press release, F-star, APR 28, 2020, View Source [SID1234556706]). FS222 is a PD-L1 and CD137 targeting, potentially best-in-class, conditional agonist tetravalent antibody.

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The preclinical data show the synergistic benefit of F-star’s tetravalent mAb², with evidence of robust CD4+ and CD8+ T cell activation, which outperformed combinations of monoclonal antibodies in multiple in vitro assays. FS222 showed no signs of liver toxicity with doses up to 30 mg/kg in a non-human primate dose-range finding study. In a mouse tumor model resistant to PD-L1 and CD137, in both mono and combination therapy, FS222 caused complete tumor eradication, concomitant with CD8+ T cell activation.

FS222 targets PD-L1 (programmed death-ligand 1), the immune checkpoint protein which regulates the balance of activated T cells in the immune system and is expressed on many solid tumors, and CD137, a co-stimulatory molecule from the tumor necrosis factor receptor superfamily (TNFRSF), which is widely known to be upregulated on CD8+ T cells following activation. Currently, only a fraction of patients respond to monotherapies that block the PD-1/PD-L1 pathway, and CD137-targeting molecules have yet to demonstrate significant responses in patients without toxicity. FS222 is designed to simultaneously target the two modalities, combining PD-L1 blockade and provoking strong CD137 agonism in a safe and efficacious manner that does not rely on a combination of antibodies approach. A regulatory application to commence clinical development of FS222 is expected to be submitted later this year.

A link to the full study can be found here.

Neil Brewis, CSO of F-star, said: "Considering the broad expression of PD-L1 on many solid tumors, we believe FS222 has the potential to provide best-in-class benefit for patients with cancer who remain challenging to treat. By targeting CD137 agonism to areas of PD-L1 expression, predominantly found in the tumor microenvironment, FS222 has the potential to leverage a focused, potent and safe immune response, enhancing the PD-L1 blockade. These data support our view that FS222 could outperform CD137 and PD-L1 monospecific antibodies in a very safe way, providing greater benefit to patients than a combination approach against both targets in solid tumors. We look forward to progressing this tetravalent bispecific antibody into the clinic, targeting tumors that are tough to treat."

On April 28, 2020 Decipher Biosciences, a commercial-stage precision oncology company committed to improving patient care, initially focused on urologic cancers, reported that patients with the molecular subtype Basal Claudin Low, identified by the Decipher Bladder test and studied in the PURE-01 clinical trial, received the most benefit in progression-free survival from neoadjuvant KEYTRUDA (pembrolizumab) immunotherapy in patients with muscle-invasive bladder cancer (MIBC) (Press release, Decipher Biosciences, APR 28, 2020, View Source [SID1234556724]).

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Identification of patients who benefit from immune checkpoint inhibitors, such as KEYTRUDA (a PD-1 inhibitor), is an unmet need in numerous disease settings for clinicians and pharmaceutical development teams. Currently, the use of KEYTRUDA is approved for patients with locally advanced or metastatic bladder cancer who are either platinum-ineligible or who have disease progression on platinum-based chemotherapy, and also for patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high risk, non-muscle-invasive bladder cancer. The PURE-01 clinical trial examined the use of KEYTRUDA prior to surgical removal of the bladder in platinum-eligible patients with non-metastatic MIBC.

"Identification of a predictive biomarker to a PD-1 inhibitor is essential for us to move the use of immunotherapy earlier in bladder cancer," said Andrea Necchi, MD, medical oncologist at the Fondazione IRCCS Istituto Nazionale dei Tumori in Milan. "The results of this study support the further examination of subtyping classifiers and their inclusion in ongoing and future immunotherapy clinical trials."

Standard of care for patients with MIBC is cisplatin-based neoadjuvant chemotherapy (NAC) followed by surgical removal of the bladder, however, many patients suffer from comorbidities prohibiting the use of NAC, resulting in low utilization. Immunotherapy is a promising alternative, as demonstrated in the PURE-01 clinical trial, with rates of response and progression-free survival with neoadjuvant KEYTRUDA similar to rates reported for NAC.

In the PURE-01 clinical trial, the Basal Claudin Low molecular subtype represented 13% of patients with a unique tumor profile and RNA expression enriched for immune activity. These patients demonstrated exceptional improvement in two-year progression-free survival. Other Basal Claudin Low patients treated with the standard of care NAC had significantly worse outcomes with ~ 50% rate of disease progression.

The study, led by Dr. Necchi and titled "Impact of Molecular Subtyping and Immune Filtration on Pathological Response and Outcome Following Neoadjuvant Pembrolizumab in Muscle-Invasive Bladder Cancer," was published in the peer-reviewed journal European Urology on March 9, 2020. Additionally, the website UroToday published a "Beyond the Abstract" commentary by Dr. Necchi highlighting the findings from the study.

About Decipher Bladder

Decipher Bladder is a genomic test that measures the molecular profile of bladder cancer using gene expression analysis from transurethral resected bladder cancer specimens. It was developed in bladder cancer patients with muscle-invasive disease who face the question of immediate cystectomy or systemic treatment in the neoadjuvant setting prior to cystectomy. The assay results are reported as one of five molecular subtypes (Luminal, Luminal-Infiltrated, Basal, Basal Claudin Low or Neuroendocrine-like), each of which has distinct biological composition, clinical behavior and predicted benefit from NAC.