On May 6, 2021 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, 2021, and provided an update on recent accomplishments and upcoming events (Press release, Syros Pharmaceuticals, MAY 6, 2021, View Source [SID1234579406]).

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"We continue to make substantial progress toward our vision of building Syros into a fully integrated company, with leading portfolios in targeted hematology and selective CDK inhibition and a robust gene control discovery engine," said Nancy Simonian, M.D., Chief Executive Officer of Syros. "We began enrolling patients in our Phase 3 clinical trial of SY-1425 in RARA-positive higher-risk MDS, marking the start of our first registration-enabling trial and an important step toward our goal of setting new standards of care in the treatment of hematologic malignancies. Looking ahead, we are poised to continue building on this momentum. We look forward to initiating additional clinical trials with SY-1425 and SY-2101 in the second half of the year and reporting new dose-escalation data, including clinical activity, from our Phase 1 trial of SY-5609 in the third quarter, while continuing to invest in our earlier-stage programs in cancer and monogenic diseases."

Syros reported plans to host a three-part KOL webinar series focused on its portfolio of investigational targeted hematology therapies. This series will consist of presentations from key opinion leaders and clinical collaborators who will review the unmet need and evolving landscapes in higher-risk myelodysplastic syndrome (HR-MDS), newly diagnosed unfit acute myeloid leukemia (AML), and acute promyelocytic leukemia (APL), as well as Syros leaders who will review recent progress for SY-1425 and for SY-2101 in these diseases. Each event will be webcast live on the Investors & Media section of Syros’ website, www.syros.com. More details for the series are forthcoming.

Upcoming Milestones
SY-1425: Oral RARa agonist

Initiate randomized Phase 2 trial of SY-1425 in combination with venetoclax and azacitidine in the second half of 2021 in RARA-positive newly diagnosed AML patients who are not suitable candidates for standard intensive chemotherapy.
Report initial data from the randomized Phase 2 trial in 2022.
SY-2101: Oral arsenic trioxide (ATO)

Initiate dose-confirmation study of SY-2101 in the second half of 2021.
Report confirmatory dose and pharmacokinetic data in first half of 2022.
Initiate Phase 3 trial in patients with newly diagnosed APL in 2022.
SY-5609: Oral CDK7 inhibitor

Report additional dose-escalation data, including clinical activity data, in the third quarter of 2021 from the ongoing Phase 1 trial of SY-5609 in patients with breast, colorectal, lung, ovarian and pancreatic cancers, as well as in patients with solid tumors of any histology harboring Rb pathway alterations.
Initiate expansion portion of Phase 1 trial in the second half of 2021.
Gene control discovery engine

Nominate next development candidate in 2022.
Recent Pipeline Highlights

Syros began dosing patients in its Phase 3 trial evaluating SY-1425 in combination with azacitidine in RARA-positive patients with newly diagnosed HR-MDS. The double-blind, placebo-controlled trial is expected to enroll approximately 190 patients. Patients will be randomized 2:1 to receive SY-1425 in combination with azacitidine or placebo, respectively. The primary endpoint of the trial is complete response (CR) rate.
Recent Corporate Highlights

In January 2021, Syros completed an underwritten public offering of 5,400,000 shares of common stock, at a public offering price of $14.00 per share, resulting in gross proceeds of approximately $75.6 million, before underwriting discounts and commissions.
First Quarter 2021 Financial Results

Revenues were $4.8 million for the first quarter of 2021, consisting of $4.0 million in revenue recognized under Syros’ collaboration with Global Blood Therapeutics, Inc. (GBT) and $0.8 million recognized under its collaboration with Incyte Corporation (Incyte). Syros recognized $2.4 million in revenue in the first quarter of 2020, including $2.2 million under its collaboration with GBT and $0.2 million under its collaboration with Incyte.
Research and development expenses were $20.0 million for the first quarter of 2021, as compared to $14.6 million for the first quarter of 2020. This increase was primarily attributable to the continued advancement of Syros’ clinical programs, including the addition of SY-2101, and an increase in employee-related expenses.
General and administrative (G&A) expenses were $5.7 million for the first quarter of 2021, as compared to $5.1 million for the first quarter of 2020. This increase was primarily attributable to an increase in employee-related expenses.
For the first quarter of 2021, Syros reported a net loss of $14.2 million, or $0.23 per share, compared to a net loss of $17.2 million, or $0.39 per share, for the same period in 2020.
Cash and Financial Guidance
Cash, cash equivalents and marketable securities as of March 31, 2021 were $222.1 million, as compared with $174.0M million on December 31, 2020. This increase reflects the gross proceeds of $75.6 million that Syros received from its January 2021 public offering, partially offset by cash used to fund its operations.

Based on its current plans, 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 2023.

Conference Call and Webcast
Syros will host a conference call today at 8:30 a.m. ET to discuss these first quarter 2021 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 5770919. A webcast of the call will also be available on the Investors & Media section of the Syros website at www.syros.com. An archived replay of the webcast will be available for approximately 30 days following the presentation.

On May 6, 2021 X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel therapies targeting diseases resulting from dysfunction of the CXCR4 pathway, reported financial results for the first quarter ended March 31, 2021 (Press release, X4 Pharmaceuticals, MAY 6, 2021, View Source [SID1234579444]). The company also provided an update on its lead product candidate, mavorixafor, a novel small molecule currently being evaluated in a Phase 3 clinical trial (4WHIM) for patients with WHIM (warts, hypogammaglobulinemia, infections, and myelokathexis) syndrome and in two Phase 1b trials for patients with Waldenström’s macroglobulinemia and Severe Congenital Neutropenia (SCN), respectively.

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"We are excited by the strong progress we have achieved to date in 2021 in the development of mavorixafor across multiple rare disease indications," said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. "In addition, we recently completed an at-the-market PIPE financing that provided $55 million in gross proceeds to the company. We believe that the significant level of new and existing investor interest demonstrates confidence in the clinical and commercial potential of mavorixafor while also extending our expected cash runway into late 2022, as well as supporting our additional pipeline programs. We look forward to continuing to report both clinical enrollment and data milestones throughout the rest of 2021 and beyond."

Recent Highlights
•Mavorixafor Phase 1b Data in Waldenström’s to be Presented at EHA (Free EHA Whitepaper) 2021. X4 has received notification of acceptance of an abstract for a poster presentation at this year’s annual congress of the European Hematology Association (EHA) (Free EHA Whitepaper), taking place virtually from June 9-17, 2021. The abstract and poster will include initial clinical safety and efficacy data from the ongoing Phase 1b clinical trial of mavorixafor in combination with ibrutinib in the treatment of a subset of Waldenström’s macroglobulinemia patients with mutations to both the MYD88 and CXCR4 genes.
•Enrollment Update in 4WHIM Clinical Trial Expected to be Announced in Mid-2021. The 4WHIM Phase 3 trial is a randomized, double-blind, placebo-controlled, multicenter study designed to evaluate the safety and efficacy of mavorixafor in approximately 18-28 genetically confirmed WHIM patients over the course of a 52-week study. The primary endpoint for the trial will compare the level of circulating neutrophils relative to a clinically meaningful threshold in response to treatment with mavorixafor versus placebo. Secondary endpoints will assess infection rates, wart burden, and markers of immune system function and quality of life among others. Based on the current rate of enrollment, X4 expects to provide an important enrollment update on the trial in the second or third quarter of 2021.
•Raised $55.0 Million in an At-the-Market PIPE Financing. In March 2021, X4 priced a $55 million at-the-market private placement financing at $8.70 per common share. The financing transaction included participation from leading biotechnology investors that are both new and existing X4 shareholders. The financing extended the company’s guidance on its expected cash runway into the fourth quarter of 2022.

First Quarter 2021 Financial Results
•Cash, Cash Equivalents & Restricted Cash: X4 had $116.7 million in cash, cash equivalents and restricted cash as of March 31, 2021. X4 expects that its cash and cash equivalents will fund company operations into the fourth quarter of 2022.
•Research and Development Expenses were $12.1 million for the first quarter ended March 31, 2021, as compared to $8.9 million for the comparable period in 2020. R&D expenses include $0.6 million and $0.2 million of certain non-cash expenses for the quarters ended March 31, 2021 and 2020, respectively.
•General and Administrative Expenses were $5.8 million for the first quarter ended March 31, 2021, as compared to $4.7 million for the comparable period in 2020. G&A expenses include $0.7 million and $0.4 million of certain non-cash expenses for the quarters ended March 31, 2021 and 2020, respectively.
•Net Loss: X4 reported a net loss of $18.7 million for the quarter ended March 31, 2021, as compared to a net loss of $11.1 million for the comparable period in 2020. Net losses include $1.3 million and $0.6 million of certain non-cash expenses for the quarters ended March 31, 2021 and 2020, respectively.

Conference Call and Webcast
The Company will host a conference call and webcast today at 8:30 a.m. ET to discuss these financial results and business highlights. The conference call can be accessed by dialing (866) 721-7655 from the United States or (409) 216-0009 internationally, followed by the conference ID: 5658628. The live webcast can be accessed on the investor relations section of X4 Pharmaceuticals’ website at www.x4pharma.com. Following the completion of the call, a webcast replay of the conference call will be available on the website.

On May 6, 2021 Cannabics Pharmaceuticals Inc. (OTCQB: CNBX), a global leader in the development of cancer related cannabinoid-based medicine, reported that it had received "Notice of Allowance" from the Mexican Patent and Trademark Office (IMPI) in relation to the company’s patent titled: ‘System and method for high throughput screening of cancer cells’ (Press release, Cannabics Pharmaceuticals, MAY 6, 2021, View Source [SID1234579654]). The decision of the Mexican patent authority follows the European Patent Office (EPO) "Intention to Grant a European Patent" notice to the company from October 2020, concerning the same patent application.

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Eyal Barad, Cannabics Pharmaceuticals’ co-founder and CEO commented: "This patent application is a great example of Cannabics’s innovative and novel approach to the drug discovery process in our field. The patent was already granted in Israel and in South Africa, and we have been notified of intention to grant in Europe and now also in Mexico. This opens many interesting opportunities for the company in these markets, which we intend to develop and grow."

Exelixis has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission .

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On May 6, 2021 Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases with significant unmet needs, reported financial results for the first quarter ended March 31, 2021 and provided a business update (Press release, Protara Therapeutics, MAY 6, 2021, View Source [SID1234579268]).

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"We remain on track to file an Investigational New Drug (IND) application and initiate our Phase 1 clinical study for TARA-002 in patients with non-muscle invasive bladder cancer (NMIBC) by the end of the year. Patients suffering from NMIBC have limited approved treatment options, and with the current standard-of-care facing a long-term supply shortage, there is an urgent need for effective therapies for these patients," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "We look forward to completing GMP scale up and comparability and then aligning with the Food and Drug Administration (FDA) on the design of a clinical study for TARA-002 in patients with Lymphatic Malformations (LMs). This new study, together with the extensive data for OK-432, the originator compound for TARA-002, should provide a robust data package for TARA-002 in this rare pediatric indication for which there is no U.S. FDA-approved therapy."

Recent Highlights and Upcoming Milestones

TARA-002 in NMIBC

Protara remains on track to submit an IND application in the second half of 2021. Subject to the successful completion of select non-clinical studies, which are expected to be completed in the first half of 2021, and FDA acceptance of the IND application, the Company plans to commence a Phase 1 study by the end of 2021 to assess the safety and tolerability of TARA-002 in patients with NMIBC, including patients with carcinoma in situ (CIS).
TARA-002 in LMs

Based on feedback from the FDA, the Company intends to complete confirmatory, large-scale, GMP manufacturing comparability in the second half of 2021 and, upon alignment with FDA on study design, subsequently initiate a clinical study in pediatric LM patients.
IV Choline Chloride in Intestinal Failure Associated Liver Disease (IFALD)

The Company is currently undertaking a prevalence study in partnership with a large home health organization in the U.S. to enhance understanding of the appropriate patient population and will use this information to define the next steps for the development program.
Corporate Update

In April 2021, the Company announced the appointment of Martín Sebastian Olivo, M.D. as Chief Medical Officer. Dr. Olivo brings to Protara more than 15 years of experience in oncology translational and clinical research and global drug development, most recently serving as Vice President, Breast Cancer Clinical Development Lead at Gilead Sciences, Inc. (formerly Immunomedics, Inc.).
First Quarter 2021 Financial Results

As of March 31, 2021, cash, cash equivalents and investments totaled $155 million.

Research and development expenses for the first quarter of 2021 increased to $7.0 million from $3.1 million during the first quarter of 2020. The increased R&D expenses were primarily due to increases in manufacturing and regulatory expenses associated with TARA-002.

General and administrative expenses for the first quarter of 2021 decreased to $6.5 million from $7.1 million for the prior year period. The decrease was primarily due to one-time expenses related to the reverse merger, which occurred in the first quarter of 2020, off-set by an increase in personnel and related costs supporting the Company’s growth.

For the first quarter of 2021, Protara reported a net loss of $13.5 million, or $1.20 per share, compared with a net loss of $10.1 million, or $1.81 per share, for the same period in 2020. Net loss for the first quarter of 2021 included approximately $2.7 million of stock-based compensation expenses.
About TARA-002

TARA-002 is an investigational cell therapy in development for the treatment of non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations (LMs) for which it has been granted Rare Pediatric Disease Designation by the U.S. Food and Drug Administration. TARA-002 was developed from the same master cell bank of genetically distinct group A Streptococcus pyogenes as OK-432, a broad immunopotentiator marketed as Picibanil in Japan and Taiwan by Chugai Pharmaceutical Co., Ltd. Protara successfully demonstrated initial manufacturing comparability between TARA-002 and OK-432.

When TARA-002 is administered, it is hypothesized that innate and adaptive immune cells within the cyst or tumor are activated and produce a strong immune cascade. Neutrophils, monocytes and lymphocytes infiltrate the abnormal cells and various cytokines, including interleukins IL-6, IL-8, IL-12, interferon (IFN)-gamma, tumor necrosis factor (TNF)-alpha, and vascular endothelial growth factor (VEGF) are secreted by immune cells to induce a strong local inflammatory reaction and destroy the abnormal cells.

About Non-Muscle Invasive Bladder Cancer

Bladder cancer is the 6th most common cancer in the United States, with non-muscle invasive bladder cancer (NMIBC) representing approximately 80% of bladder cancer diagnoses. Approximately 65,000 patients are diagnosed with NMIBC in the United States each year. NMIBC is cancer found in the tissue that lines the inner surface of the bladder that has not spread into the bladder muscle. The current standard of care for high-grade NMIBC includes intravesical Bacillus Calmette-Guerin (BCG).

About Lymphatic Malformations

Lymphatic malformations (LMs) are rare, congenital malformations of lymphatic vessels resulting in the failure of these structures to connect or drain into the venous system. Most LMs are present in the head and neck region and are diagnosed in early childhood during the period of active lymphatic growth, with more than 50% detected at birth and 90% diagnosed before the age of 3 years. The most common morbidities and serious manifestations of the disease include compression of the upper aerodigestive tract, including airway obstruction requiring intubation and possible tracheostomy dependence; intralesional bleeding; impingement on critical structures, including nerves, vessels, lymphatics; recurrent infection, and cosmetic and other functional disabilities.

About IV Choline Chloride and Intestinal Failure-associated Liver Disease (IFALD)

IV Choline Chloride is an investigational, intravenous (IV) phospholipid substrate replacement therapy initially in development for patients receiving parenteral nutrition (PN) who have IFALD. Choline is a known important substrate for phospholipids that are critical for healthy liver function. Because PN patients cannot sufficiently absorb adequate levels of choline and no available PN formulations contain sufficient amounts of choline to correct this deficiency, PN patients often experience a prolonged progression to hepatic failure and death, with the only known intervention being a dual small bowel/liver transplant. If approved, IV Choline Chloride would be the first approved therapy for IFALD. It has been granted Orphan Drug Designations (ODDs) by the FDA for the treatment of IFALD and the prevention of choline deficiency in PN patients.