On May 16, 2022 Syros Pharmaceuticals (NASDAQ:SYRS), a leader in the development of medicines that control the expression of genes, reported financial results for the quarter ended March 31, 2022 and provided a corporate update (Press release, Syros Pharmaceuticals, MAY 16, 2022, View Source [SID1234614679]).

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"The first quarter of 2022 was very productive for Syros as our team focused on executing across our portfolio in preparation for three data readouts this year, including from the safety lead-ins of the SY-5609 trial in pancreatic cancer and of the SELECT-AML-1 Phase 2 trial of tamibarotene as well as from the dose confirmation study of SY-2101 in APL," said Nancy Simonian, M.D., Chief Executive Officer of Syros. "Given the current market conditions, we will not be starting the Phase 3 trial of SY-2101 until we secure additional capital. In addition, at this time we are deprioritizing the evaluation of SY-5609 in hematologic malignancies. We are focusing our resources to continue our ongoing clinical trials in order to deliver benefit to patients as well as maximize value for our shareholders."

Tamibarotene: Oral RARα agonist

Higher-Risk Myelodysplastic Syndrome (HR-MDS)

Syros is continuing to advance the ongoing SELECT-MDS-1 Phase 3 trial in newly diagnosed RARA-positive patients with HR-MDS and remains on track to report data in the fourth quarter of 2023 or the first quarter of 2024, with a potential new drug application (NDA) filing expected in 2024.

Acute Myeloid Leukemia (AML)

Syros is evaluating tamibarotene in combination with venetoclax/azacitidine compared to venetoclax/azacitidine alone in the ongoing SELECT-AML-1 Phase 2 trial in newly diagnosed unfit RARA-positive patients with AML. Syros expects to report data, including clinical activity, from the safety lead-in portion of the study in the second half of 2022.

SY-2101: Oral arsenic trioxide (ATO)

Syros is advancing the ongoing dose confirmation trial of SY-2101 in patients with newly diagnosed acute promyelocytic leukemia (APL) and expects to announce pharmacokinetic (PK) and safety data from the trial in mid-2022. Given the current market conditions, Syros does not plan to advance SY-2101 into a Phase 3 trial until additional capital is secured.

SY-5609: Oral selective CDK7 inhibitor

Syros is evaluating SY-5609 in combination with chemotherapy in relapsed/refractory metastatic pancreatic cancer patients. The company remains on track to report data, including clinical activity, from the safety lead-in portion of the trial in the second half of 2022. Given the current market conditions, Syros is deprioritizing the development of SY-5609 for the treatment of hematologic malignancies at this time.

In addition, Roche plans for the arm of its ongoing Phase 1/1b INTRINSIC trial arm evaluating SY-5609 in combination with atezolizumab in BRAF-mutant colorectal cancer (CRC) to be open for enrollment in the first half of this year. Under the terms of Syros’ agreement with Roche, Roche is the sponsor of the trial and Syros is supplying SY-5609.

Gene Control Discovery Engine

At the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022, Syros presented promising CDK12 inhibitor data demonstrating strong anti-tumor activity as a single agent as well as in combination with a DNA damaging agent and a PARP inhibitor in models of breast, lung, and ovarian cancer. Additionally, the CDK12 inhibitor showed anti-tumor activity in a PARP inhibitor resistant patient-derived model (PDX) of ovarian cancer. Syros expects to nominate its next development candidate from the CDK12 inhibitor program in the second half of 2022.

First Quarter 2022 Financial Results

Revenues were $5.5 million for the first quarter of 2022, consisting of $5.1 million in revenue recognized under Syros’ collaboration with Global Blood Therapeutics, Inc. (GBT) and $0.4 million recognized under its collaboration with Incyte Corporation (Incyte). Syros recognized $4.8 million in revenue in the first quarter of 2021, consisting of $4.0 million in revenue recognized under its collaboration with GBT and $0.8 million recognized under its collaboration with Incyte.
Research and development expenses were $25.2 million for the first quarter of 2022, as compared to $20.0 million for the first quarter of 2021. This increase was primarily due to the advancement of the Company’s clinical and preclinical programs and increases in employee-related expenses due to headcount growth.
General and administrative (G&A) expenses were $6.9 million for the first quarter of 2022, as compared to $5.7 million for the first quarter of 2021. This increase was primarily due to increases in employee-related expenses and an increase in patent prosecution costs and consulting fees.
For the first quarter of 2022, Syros reported a net loss of $25.1 million, or $0.40 per share, compared to a net loss of $14.2 million, or $0.23 per share, for the same period in 2021.
Cash and Financial Guidance

Cash, cash equivalents and marketable securities as of March 31, 2022 were $112.9 million, as compared with $143.4 million on December 31, 2021.

Syros believes that its existing cash, cash equivalents and marketable securities will be sufficient to fund its planned operating expenses and capital expenditure requirements into the second quarter of 2023.

Conference Call and Webcast

Syros will host a conference call today at 8:30 a.m. ET to discuss these first quarter 2022 financial results and provide a corporate update.

To access the live conference call, please dial (866) 595-4538 (domestic) or (636) 812-6496 (international) and refer to conference ID 4664097. A webcast of the call will also be available on the Investors & Media section of the Syros website at. An archived replay of the webcast will be available for approximately 30 days following the presentation.

On May 16, 2022 Catamaran Bio, Inc., a biotechnology company developing off-the-shelf chimeric antigen receptor (CAR)-NK cell therapies to treat cancer, reported that it is presenting new preclinical data at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting, being held in Washington, DC (Press release, Catamaran Bio, MAY 16, 2022, View Source [SID1234614678]). The data demonstrate successful engineering of CAR-NK cells to preserve and enhance NK cell activity in the immunosuppressive tumor microenvironment.

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"The ability to enable robust functioning of CAR-NK cells in the immunosuppressive solid tumor microenvironment will be critical to the success of cell therapies," said Vipin Suri, PhD, MBA, Chief Scientific Officer of Catamaran Bio. "We are presenting data on a TGFβ receptor that lacks an intracellular signaling domain and acts as a TGFβ trap, as well as proprietary next generation switch receptors that reconfigure inhibitory TGFβ signaling to be stimulatory. Both approaches lead to protection from TGFβ immunosuppression of CAR-NK cells as well as neighboring immune cells. The approaches we are presenting at ASGCT (Free ASGCT Whitepaper) are part of our innovative switch receptor platform which includes multiple strategies to convert other immunosuppressive signals into beneficial signals that stimulate and enhance the therapeutic activity of NK cells."

Highlights of the data presented on TGFβ switch receptors for CAR-NK cell therapies are as follows:

Abstract #313: Dominant Negative Receptor for TGFβ

A TGFβ dominant negative receptor (DNR) was shown to protect the function of both the engineered CAR‑NK cells and neighboring NK cells from the immunosuppressive effects of TGFβ.

CAR-NK cells engineered with this TGFβ DNR demonstrated reduction of downstream TGFβ activity as shown by >90% reduction of SMAD2 phosphorylation (a downstream signal of TGFβ activity) even at supraphysiological doses of TGFβ.
By acting as a sink, the TGFβ DNR also protected neighboring NK cells from immunosuppressive TGFβ signaling, resulting in approximately a 3-fold reduction in potency of TGFβ in inducing SMAD2 phosphorylation.
Co-expression of the TGFβ DNR with a HER2-CAR construct restored CAR-dependent cytotoxicity in the presence of TGFβ.
Abstract #331: Next Generation TGFβ Switch Receptors

Building on its success using TGFβ-DNRs to prevent immunosuppression, Catamaran also engineered synthetic receptors that effectively converted TGFβ signals into cell stimulatory signals.

A diverse set of switch receptors were engineered with a TGFβ receptor extracellular domain, coupled with various intracellular domains.
These proprietary next generation TGFβ switch receptors successfully reversed TGFβ-mediated inhibition, promoted NK cell expansion in vitro, and enhanced NK cell functional responses.
Chronic TGFβ stimulation strengthened NK cell tumor killing and target-induced cytokine secretion, and incorporation of the switch receptors also protected the cells from suppressive TGFβ signaling.

On May 16, 2022 Avacta Group plc (AIM: AVCT), a clinical stage oncology drug company developing innovative cancer therapies and powerful diagnostics based on its proprietary pre|CISION and Affimer platforms, reported that AffyXell, its joint venture with South Korean drug maker Daewoong Pharmaceutical, has expanded its strategic partnership with GenScript ProBio, a leading biopharmaceutical manufacturer (Press release, AffyXell Therapeutics, MAY 16, 2022, View Source [SID1234614677]).

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AffyXell is a joint venture established in January 2020 by Daewoong Pharmaceutical and Avacta, developing the next generation of cell and gene therapies based on mesenchymal stem cells which incorporate Affimer immunotherapies. This new class of stem cell therapy is designed to produce Affimer proteins, in situ in the body, that reduce inflammatory or autoimmune responses to the stem cell therapy to potentially enhance their therapeutic effects.

GenScript ProBio, part of Genscript Biotech Corporation (HKG: 1548), is a leading global contract development and manufacturing organisation providing a best-in-class, one-stop platform for research and development of biological drugs, DNA plasmids and lentiviruses, and for clinical production of cell and gene therapy products.

AffyXell and GenScript ProBio are extending their strategic manufacturing partnership, which they entered into in December 2021, that covers AffyXell’s first drug development programme to include additional future programmes. The partnership covers process development and production of viral vectors required for the production of AffyXell’s future cell therapy products. In addition, as part of this strategic alliance, GenScript has now committed to take an equity position in AffyXell at a future funding round, and the two companies will collaborate in the area of business development, including potential out licensing.

Dr Alastair Smith, Chief Executive Officer of Avacta, commented: "This is an important strategic partnership for AffyXell with a world leading contract development and manufacturing partner. The fact that GenScript ProBio are prepared to take a strategic equity stake in AffyXell is a very strong validation of the potential for AffyXell’s next generation cell and gene therapies, and the future valuation of the business."

"We are delighted with the excellent progress being made by AffyXell with the Affimer immunomodulators provided by Avacta under our joint venture agreement."

Jongsang Ryu, Chief Executive Officer of AffyXell, commented: "We were able to attract strategic investment from the CDMO partner as the excellence and potential of our next-generation cell therapy platform have been recognized."

"This agreement will be an opportunity to accelerate the development of therapies targeting intractable diseases, providing meaningful options for the suffering patients."

Dr Brian Min, Chief Executive Officer of GenScript ProBio commented: "We are pleased to partner with AffyXell and are honored to support this next-generation cell therapy project with GenScript ProBio’s stable and high-yield viral vector platform. We expect many patients to benefit from this innovative genetically modified mesenchymal stem cells therapy soon."

On May 16, 2022 CEL-SCI Corporation (NYSE American: CVM) reported financial results for the quarter ended March 31, 2022, as well as key clinical and corporate developments (Press release, Cel-Sci, MAY 16, 2022, View Source [SID1234614676]).

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Clinical and Corporate Developments include:

The American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) has accepted two abstracts related to CEL-SCI’s pivotal Phase 3 Multikine (Leukocyte Interleukin, Injection)* head and neck cancer clinical trial for presentation at the 2022 ASCO (Free ASCO Whitepaper) Annual Meeting to be held June 3-7, 2022 in Chicago, Illinois. The abstract titles are:
"Novel algorithm for assigning risk/disease-directed treatment (DDT) choice in locally advanced primary squamous cell carcinoma of the head and neck (SCCHN): Using pretreatment data only."
"Leukocyte interleukin injection (LI) immunotherapy extends overall survival (OS) in treatment-naive low-risk (LR) locally advanced primary squamous cell carcinoma of the head and neck: The IT-MATTERS study."
Head and neck cancer patients who are scheduled to receive surgery and radiation as their first treatments have not seen a marked improvement in their treatment outcome in decades. CEL-SCI’s Phase 3 study showed great improvement in survival with no toxicity issues for these patients with a statistically significant, robust, and durable survival benefit of 14.1% at 5 years. This is clearly an unmet medical need for an estimated 211,000 people globally.
Additional results from the Phase 3 study of Multikine in advanced primary head and neck cancer have been submitted to the U.S. government clinical trial website www.clinicaltrials.gov. That data is expected to be released to the public in the near future.
CEL-SCI’s dedicated current Good Manufacturing Practice (cGMP) facility in which it manufactures Multikine is now undergoing validation following the completion of its commercial scale build out during the first quarter of 2022. The construction is designed to ensure the facility will be compliant with all U.S. Food and Drug Administration’s (FDA) and European cGMP regulations.
As of March 31, 2022, CEL-SCI had $34.3 million in cash and cash equivalents.
"ASCO’s annual meeting in June, with over 40,000 oncology professionals from over 100 countries expected, is one of the largest and most prominent oncology conferences. Our scientific team looks forward to presenting two abstracts there, and we expect a high level of interest based on Multikine’s results and the fact that outcomes have not improved for the head and neck cancer population in decades," stated CEL-SCI CEO, Geert Kersten. "The conference is well timed to inform oncologists from around the world about Multikine ahead of our planned regulatory filing for approval."

CEL-SCI reported an operating loss of $18.4 million for the six months ended March 31, 2022 versus an operating loss of $17.3 million for the six months ended March 31, 2021. CEL-SCI reported an operating loss of $9.6 million for the three months ended March 31, 2022 versus an operating loss of $8.5 million for the three months ended March 31, 2021. Net cash used in operating activities was $7.5 million for the six months ended March 31, 2022 which represents a decrease of $1.2 million compared to the six months ended March 31, 2021.

On MAY 16, 2022 CG Oncology, Inc., a clinical-stage biotechnology company focused on developing oncolytic immunotherapies for patients with advanced cancer, reported that interim results from the global Phase 2 study (CORE1) of CG0070 in combination with Merck’s anti-PD-1 therapy KEYTRUDA (pembrolizumab), for the treatment of patients with Non-Muscle-Invasive Bladder Cancer (NMIBC) unresponsive to Bacillus Calmette-Guerin (BCG) (Press release, CG Oncology, MAY 16, 2022, View Source [SID1234614675]).

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The results (Session MP54-03) were presented at the 2022 American Urological Association (AUA) Annual Meeting. The preliminary data adds to that presented at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) earlier this year and continues to show both promising early anti-tumor activity and tolerability of CG0070 in combination with pembrolizumab for patients with BCG unresponsive NMIBC.

Summary of Interim Clinical Results

As of the interim analysis, based on a data cutoff on April 28, 2022, 22 patients were evaluable for efficacy with a minimum of 3 months follow up.
91% of patients evaluable for efficacy (n=20/22) have achieved complete response (CR) at the initial 3-month timepoint. Of those patients evaluable for CR at additional timepoints, 87% (n=15) have also maintained a CR through 6 months, 80% (n=10) through 9 months and 75% (n=8) at the 12-month assessment.
Treatment related adverse events were generally limited to transient grade 1-2 local genitourinary symptoms including pollakiuria, bladder spasm, dysuria, fatigue, nocturia, hematuria, chills, and immune-related adverse events including hyperglycemia and hypothyroidism.
About the CORE1 Study

Under a previously announced clinical collaboration with Merck (known as MSD outside the US and Canada) relating to the investigation of CG0070 used in combination with pembrolizumab, the goal of CORE1, which will enroll up to 35 patients, is to evaluate the safety and efficacy of CG0070 plus pembrolizumab for the treatment of NMIBC unresponsive to BCG.

More information about the study can be found at www.clinicaltrials.gov (NCT04387461).

KEYTRUDA is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.

About CG0700

CG0070, a selective oncolytic immunotherapy based on a modified adenovirus type 5 backbone that contains a cancer-selective promoter and a GM-CSF transgene, destroys bladder tumor cells through their defective retinoblastoma (Rb) pathway. CG0070 was designed to replicate inside tumor cells with dysfunctional Rb pathways, causing tumor cell lysis and immunogenic cell death. The rupture of cancer cells releases tumor-derived antigens and GM-CSF, which stimulates a systemic anti-tumor immune response. In advanced clinical trials, CG0070 is a safe and efficacious agent in NMIBC following BCG failure. CG0070 is currently in late-stage clinical trials across a variety of solid cancers, as a monotherapy or in combination with immune checkpoint inhibitors.