On November 12, 2021 Biosight Ltd., a pharmaceutical development company developing innovative therapeutics for hematological malignancies and disorders, reported the initiation of a Phase 2 trial to evaluate aspacytarabine (BST-236), Biosight’s proprietary antimetabolite, as a second line treatment for patients with relapsed or refractory myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) (Press release, Advaxis, NOV 12, 2021, View Source;soc_trk=ma [SID1234595728]). The multi-center study will be conducted across 18 leading U.S. and Israeli sites including Memorial Sloan Kettering Cancer Center and The University of Texas MD Anderson Cancer Center.

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"Biosight has built a strong foundation of compelling data that suggests that aspacytarabine may serve as a more tolerable and effective standard of care treatment for patients with AML," said Dr. Ruth Ben Yakar, Chief Executive Officer of Biosight. "The initiation of this multi-center, U.S. based, Phase 2 study is an important step forward in the development of aspacytarabine, seeking to address unmet needs in the treatment of patients with relapsed or refractory AML and MDS. We are proud to be continuing our momentum in the clinic and look forward to collaborating with leading academic medical centers as we progress the study."

Eytan Stein, M.D., Hematologic Oncologist at Memorial Sloan Kettering Cancer Center and lead investigator of the study said "I’m encouraged by the results from Biosight’s Phase 2b trial to be presented at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. The efficacy achieved in a challenging population, across key measures including complete remission and MRD (-) rates, duration of response and overall survival, are noteworthy. Furthermore, these results were particularly impressive as they were achieved with a favorable safety and tolerability profile in patients who are unfit for intensive chemotherapy. Patients with relapsed or refractory AML and MDS have limited treatment options and poor prognoses, with many patients unable to tolerate intensive chemotherapy. I look forward to leading this new Phase 2 study to evaluate the potential of aspacytarabine which may ultimately become the standard of care for relapsed or refractory MDS and AML patients for whom currently there are no effective treatments."

The Phase 2 open label multi-center study will assess the safety and efficacy of BST-236 as a single agent in adult patients unfit for standard therapy with AML or higher-risk (HR)MDS who fail to respond to, or have relapsed following, first line therapy. A similar study in patients with relapsed/refractory AML and MDS is ongoing in collaboration with the European cooperative group, Groupe Francophone des Myélodysplasies (GFM). Approximately 40 adult patients with relapsed and/or refractory AML and approximately 40 adult patients with relapsed and/or refractory HR MDS will be enrolled into the two studies. Primary endpoints include complete remission (CR) rate in AML patients and Overall Response Rate (ORR) in MDS patients, with ORR defined as the proportion of patients who achieve a CR or partial response (PR) per proposal for modification of the International Working Group (IWG) criteria for MDS, 2006.

About Aspacytarabine (BST-236)

Aspacytarabine is a novel proprietary anti-metabolite. It is composed of cytarabine covalently bound to asparagine, acting as a pro-drug of cytarabine. Cytarabine has served as the backbone of AML therapy for over 45 years due to its superior efficacy. However, it is associated with severe bone marrow, gastrointestinal, and neurological toxicities, which significantly limit its use, especially in older and medically compromised patients. Due to its unique pharmacokinetics and metabolism, aspacytarabine enables high-dose therapy with lower systemic exposure to free cytarabine and relative sparing of normal tissues. As such, aspacytarabine may serve as a new therapy for AML and other hematological malignancies and disorders, including for older adults who are unfit for intensive therapy.

Aspacytarabine was granted FDA Fast Track Designation for treatment of AML patients unfit for standard chemotherapy, and FDA and EMA Orphan Drug Designations, which entitle Biosight to seven and ten years of market exclusivity in the U.S. and Europe, respectively, upon aspacytarabine marketing approval for the treatment of AML in each territory.

Interim results from an ongoing Phase 2b study evaluating aspacytarabine as a single-agent first-line AML therapy demonstrate safety and single-agent activity, and additional studies are ongoing to evaluate aspacytarabine as a second line treatment for patients with relapsed or refractory MDS or AML. For more information regarding the Phase 2b clinical study of BST-236, please visit www.clinicaltrials.gov.

On November 12, 2021, Shattuck Labs, Inc. presented at the 2021 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting (Presentation, Shattuck Labs, NOV 12, 2021, View Source [SID1234595579]).

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On November 12, 2021 Curaleaf Holdings, Inc. (CSE: CURA / OTCQX: CURLF) ("Curaleaf" or the "Company"), a leading international provider of consumer products in cannabis, reported that entered into a definitive agreement to acquire Tryke Companies ("Tryke") (dba as Reef Dispensaries), a privately held vertically integrated, multi-state cannabis operator, in a cash and stock transaction valued at approximately US$286 million (Press release, Curaleaf Holdings, NOV 12, 2021, View Source [SID1234595546]).1 The transaction is expected to close in the second half of 2022, subject to customary approvals and conditions.

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Under the terms of the agreement, Curaleaf will pay US$40 million in cash at closing, with a remaining US$75 million in cash to be paid in equal installments on the first, second and third anniversaries of the closing. The stock portion of the transaction, which consists of 17 million subordinate voting shares of Curaleaf ("Curaleaf Shares"), will also be paid in three equal installments on the first, second and third anniversaries of the closing. An incremental earnout of up to 1 million Curaleaf Shares may be paid in 2023 based on the business exceeding certain EBITDA targets for the year 2022.

Founded in Arizona in 2014, Tryke has focused on growing and producing the finest and most consistent cannabis products on the market. The company helped pioneer Nevada’s legal cannabis market from its inception in 2015, and continues to lead the industry in Utah where it has worked since 2019 to help establish the state’s medical cannabis program. Tryke has refined processes to craft an ever-evolving selection of products and brands at multiple price points. The company’s dispensaries have served more than 7.6 million customers, offering a wide variety of in-house and third-party flower, concentrates, vape cartridges, edibles, topicals and CBD products. Upon closing, Curaleaf will assume ownership of Tryke’s extensive portfolio of processing licenses and expects to significantly expand its cultivation capacity from 30,000 square feet to over 80,000 square feet over the next three years.

Boris Jordan, Founder and Executive Chairman of Curaleaf, said, "On Behalf of the Board of Directors and management team, I look forward to welcoming Tryke to the Curaleaf family as we expand our offerings and operations and bolster our competitive position in three key growth markets. We believe that Tryke represents a unique opportunity to join forces with another industry leading pioneer that shares Curaleaf’s commitment to legalization and expansion. This strategically and financially compelling transaction will expand our US presence by bringing additional premium products to our consumers and retailers in Nevada, Arizona and Utah, all while yielding meaningful benefits for all of our stakeholders. We expect the acquisition to be immediately accretive to our EBITDA margins and free cash flow generation upon closing."

1 Based on the closing price of Curaleaf’s subordinate voting shares on the OTC market as of November 5, 2021.

"This is a tremendous opportunity for Tryke and, as a combined entity, we will continue to deliver significant value for our consumers and retailers in Arizona, Nevada and Utah," said Adam Ryan, Chief Executive Officer of Tryke Companies. "As a part of Curaleaf’s growing network of dispensaries, Tryke is excited to bring its full suite of multi-price point products to an expanded base of consumers across the country. We are excited to join forces with the industry leader at such a pivotal moment in the United States’ legalization efforts. We share Curaleaf’s optimism for the future and are excited to become investors alongside the Company’s talented leadership team."

Compelling Strategic and Financial Benefits

·Enhances Curaleaf’s operations in Arizona, Nevada and Utah: Tryke currently owns and operates six heavily trafficked dispensaries under the Reef brand, with two retail stores in Arizona and four in Nevada, including the Phoenix metropolitan area, Las Vegas strip and North Las Vegas. The company’s products are sold in over 50 additional locations across its footprint.

·Enriches Curaleaf’s product offerings: Tryke currently offers a wide variety of in-house and third-party flower, concentrates, vape cartridges, edibles, topicals and CBD products at a range of price points. Tryke’s product portfolio is highly complementary to Curaleaf’s, allowing the Company to offer consumers and retailers in Arizona, Nevada and Utah an even broader selection of premium cannabis products.

Improves Curaleaf’s margins and free cash flow generation: Tryke has a strong financial profile, with a history of delivering significant revenue growth and compelling EBITDA margins in excess of 35%. Tryke is expected to record nearly US$110 million in full year 2021 revenue. Curaleaf expects the acquisition will be immediately accretive to the Company’s EBITDA margins and free cash flow generation.

The closing of the transaction is expected to occur in the second half of 2022 subject to customary closing conditions, including the receipt of approval from the applicable state regulators, including the Nevada Cannabis Compliance Board.

Transaction Advisors

In a separate press release to be issued today after the market closes, Curaleaf will announce its financial results for the third quarter of 2021, and it will be available at View Source

Conference Call & Webcast

Curaleaf will hold a conference call today, November 8, at 5:00 p.m. Eastern Time to discuss this announcement, as well as its third quarter 2021 results. Investors who wish to participate in the call should dial 1-888-317-6003 (U.S.) or 1-866-284-3684 (Canada) or 1-412-317-6061 (International) approximately 15 minutes before the call begins and provide conference ID number 2599473.

On November 12, 2021 Prelude Therapeutics Inc. (Nasdaq: PRLD), a clinical-stage precision oncology company, reported its financial results for the third quarter ended September 30, 2021 and provided an update on recent clinical and development pipeline progress (Press release, Prelude Therapeutics, NOV 12, 2021, View Source [SID1234595536]).

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"We continue to make significant progress advancing our novel pipeline of therapeutic candidates, most notably with the recent presentation of dose escalation data from the Phase 1 trials of our lead PRMT5 inhibitors, PRT543 and PRT811," said Kris Vaddi, PhD, Chief Executive Officer. "We were pleased by these initial data in unselected patients, which demonstrated key points of differentiation for our molecules, including good tolerability and potency, and a desirable therapeutic window. In addition, evidence of preliminary clinical activity was observed in multiple tumor types displaying preclinically validated genomic features. We look forward to leveraging learnings from these data as we execute on the dose escalation portion of the trials and evaluate PRT543 and PRT811 in biomarker-selected patient populations, with data readouts from these cohorts anticipated in 2022. Beyond our PRMT5 inhibitors, the balance of our pipeline continues to advance. During the quarter we received IND clearance from the FDA for PRT2527, our CDK9 inhibitor, positioning us to commence a Phase 1 study of this molecule before year-end."

Recent Highlights and Upcoming Milestones

PRT543

Phase 1 Dose Escalation Study Data Presented at the AACR (Free AACR Whitepaper)-NCI-EORTC Annual Meeting; Data from Expansion Cohorts to be Presented in 2022: In October 2021, the Company presented data from the dose escalation portion of its Phase 1 trial of PRT543, which is designed to be a potent and selective inhibitor of PRMT5, in unselected patient populations. PRT543 demonstrated target engagement and inhibition of PRMT5 functional activity, as well as preliminary clinical activity. PRT543 was generally well tolerated. Patient enrollment is ongoing in specific biomarker-selected solid tumor and hematologic malignancy expansion cohorts. The Company expects to present data from the expansion cohorts in 2022.

Phase 1 Dose Escalation Data for PRT543 in Patients with Myeloid Malignancies to be Presented at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting: Data from the dose escalation portion of the ongoing Phase 1 clinical trial of PRT543 in patients with myelodysplastic syndrome (MDS) and myelofibrosis (MF), including safety, pharmacokinetics, pharmacodynamics, and preliminary clinical activity, will be featured during a poster session at the 63rd ASH (Free ASH Whitepaper) Annual Meeting and Exposition being held December 11-14, 2021.

PRT811

Phase 1 Dose Escalation Study Data Presented at the AACR (Free AACR Whitepaper)-NCI-EORTC Annual Meeting; Dose Expansion Portion of Phase 1 Trial to Commence 4Q21: In October 2021, the Company presented data from the dose escalation portion of its Phase 1 trial of PRT811, which is designed to be a potent, selective, and brain penetrant PRMT5 inhibitor, in patients with unselected advanced solid tumors. PRT811 demonstrated dose dependent inhibition of PRMT5 activity and demonstrated signs of preliminary clinical activity. PRT811 was generally well-tolerated. Prelude will shortly commence the dose expansion portion of the Phase 1 trial in selected patients with central nervous system cancers (CNS) and non-CNS cancers. The Company expects to present data from the expansion cohorts in 2022.

PRT1419

Phase 1 Dose Escalation Portion of Oral and Intravenous (IV) PRT1419 Trial Ongoing: The dose escalation portion of the Company’s first-in-human Phase 1 study investigating both an oral and IV formulation of MCL-1 inhibitor, PRT1419, the Company’s third clinical candidate, in patients with relapsed/refractory hematologic malignancies, including acute myeloid leukemia and high-risk myelodysplastic syndromes, and solid tumors, remains ongoing. The Company expects to add dose expansion and combination cohorts to the Phase 1 clinical trial in the first half of 2022.

PRT2527

Dose Escalation Phase 1 Trial of PRT2527 on Track to Begin by Year-End: During the third quarter the Company received clearance for an Investigational New Drug (IND) application for PRT2527, which is designed to be a potent and selective CDK9 inhibitor. The Company anticipates beginning a Phase 1 trial of PRT2527 by year-end evaluating IV infusion monotherapy in patients with selected solid tumors.

Preclinical Data Presented at the AACR (Free AACR Whitepaper)-NCI-EORTC Annual Meeting: In October 2021, the Company presented new preclinical data demonstrating that intermittent intravenous administration of PRT2527 demonstrated strong efficacy in hematological malignancies and solid tumor models with MYC dysregulation.

Discovery Programs

The Company continues to expect to initiate IND-enabling studies for PRT-SCA2, which is designed to be a SMARCA2 protein degrader, by year-end. The Company also continues to make progress in the PRT-K4 discovery program and expects to initiate IND-enabling studies by year-end.

Corporate Update

On November 5, 2021, Brian Piper, our Chief Financial Officer, notified the Company that he will be resigning from the Company, effective November 19, 2021, to pursue other opportunities.

The Company reported the appointment of Laurent Chardonnet as its new Chief Financial Officer starting November 29, 2021. Mr. Chardonnet joins from Axcella Health where, since 2019, he served as Senior Vice President, CFO. Prior to Axcella, he spent 15 years at Incyte Corporation where he held roles of increasing responsibility including Vice President Finance, Treasurer and Principal Accounting Officer, Head of Finance and Administration for the company’s European division, and Vice President of Alliances and Global Strategy. Mr. Chardonnet received his Master of Business Administration from Vanderbilt University and his initial business degree from the Institut Supérieur de Gestion in Paris

The Company and Dr. David Mauro, the Company’s Chief Medical Officer, mutually agreed that Dr. Mauro would depart from the Company to pursue other opportunities. Dr. Mauro’s last day with the Company was on November 9, 2021. The Company has an ongoing search for a successor. Dr. Victor Sandor, former Chief Medical Officer of Array Biopharma and current board member and chair of the Science and Technology Committee, will provide strategic and operational oversight of clinical development during this time.

Third Quarter 2021 Financial Results

Cash, Cash Equivalents, and Marketable Securities: Cash, cash equivalents, and marketable securities as of September 30, 2021 were $320.9 million.

Research and Development (R&D) Expenses: For the third quarter of 2021, R&D expense increased by $7.4 million to $22.7 million for the three months ended September 30, 2021 from $15.3 million for the three months ended September 30, 2020. The increase was mainly due to increased clinical research costs to support the advancement of our clinical programs as well as an increase in discovery-stage program expenses. Our chemistry, manufacturing and other costs for the clinical trials also increased.

General and Administrative (G&A) Expenses: For the third quarter of 2021, G&A expense increased by $5.2 million to $8.1 million for the three months ended September 30, 2021 from $2.9 million for the three months ended September 30, 2020. The increase was primarily due to an increase in personnel related expense due to an increase in employee headcount and an increase in our professional fees as we expanded our operations to support our research and development efforts and incurred additional costs to operate as a public company.

Net Loss: For the third quarter of 2021, net loss was $30.7 million, or $0.66 per share, compared with a net loss of $16.8 million, or $5.25 per share, for the same period in 2020.

Financial Guidance: The Company believes that its current cash, cash equivalents and marketable securities will be sufficient to fund operating expenses and capital expenditure requirements into the second half of 2023.

On November 12, 2021 Celularity Inc. (Nasdaq: CELU) ("Celularity"), a clinical-stage biotechnology company developing placental-derived allogeneic cell therapies, reported financial results for the third quarter ended September 30, 2021, and provided a corporate update (Press release, Celularity, NOV 12, 2021, View Source [SID1234595535]).

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"We have continued to make significant progress over the past months, including the establishment of multiple strategic collaborations to extend further the potential uses of our technology platform, with the goal of bringing potentially transformative therapeutics to patients who require them most," said Robert J. Hariri, M.D., Ph.D., Founder, Chairperson and Chief Executive Officer of Celularity. "Moreover, we have advanced multiple preclinical and clinical programs targeting significant unmet medical needs, including acute myeloid leukemia and glioblastoma. We believe we are well-positioned to continue executing our business strategy and developing cellular therapies for cancer, infectious and degenerative diseases that leverage our proprietary placental-based platform technology."

Third Quarter Corporate Highlights

Appointed Andrew L. Pecora, M.D., F.A.C.P., C.P.E., as President.
Entered into a research collaboration with Oncternal Therapeutics (Nasdaq: ONCT) to evaluate placental derived-cellular therapies targeting receptor-tyrosine kinase-like Orphan Receptor 1 (ROR1) in preclinical studies. The collaboration will initially explore the use of Oncternal’s ROR1-targeted chimeric antigen receptor (CAR) gene modification as part of Celularity’s CYNK natural killer cell and CyCART platforms. The partnership will also explore the use of monoclonal antibody, cirmtuzumab, in combination with Celularity’s CYNK natural killer cells.
Third Quarter 2021 Financial Results

Revenues for the three months ended September 30, 2021, increased $7.3 million compared to the prior year period. This was primarily due to an increase in license, royalty and other revenues driven by (i) recognition of previously deferred revenue as a result of the termination of the Sanuwave license agreement of $6.8 million, and (ii) revenues from supply and distribution agreements recognized during the third quarter of 2021 of $1.4 million, partially offset by a decrease in product sales and rentals revenues of $0.6 million resulting from the sale of the MIST/UltraMIST assets in August 2020.
Research and development expenses for the three months ended September 30, 2021, increased $12.5 million compared to the prior year period. The increase in research and development expenses was primarily due to higher stock-based compensation expense resulting from awards granted in connection with the consummation of the business combination as well as higher clinical trial costs related to Celularity’s CYNK-001 clinical trial and higher personnel costs.
Selling, general and administrative expenses for the three months ended September 30, 2021, increased $13.3 million compared to the prior year period, primarily due to higher stock-based compensation expense resulting from awards granted in connection with the consummation of the business combination, and a $5.3 million charge related to an estimated legal settlement and higher personnel costs.
Net income for the third quarter of 2021 was $49.9 million, or $0.47 per share (basic) and $0.40 per share (diluted).
Year to Date Financial Results

Revenue for the nine months ended September 30, 2021, increased $5.4 million compared to the prior year period. The increase was due to an increase of $9.1 million in license, royalty and other revenues primarily driven by (i) recognition of previously deferred revenue as a result of the termination of the Sanuwave license agreement of $6.8 million, and (ii) revenues from supply and distribution agreements recognized during the third quarter of 2021 of $1.4 million, partially offset by a decrease in product sales and rentals revenues of $3.6 million resulting from the sale of the MIST/UltraMIST assets in August 2020.
Research and development expenses for the nine months ended September 30, 2021, increased $24.9 million compared to the prior year period. The increase in research and development expenses was primarily due to higher stock-based compensation resulting from awards granted in connection with the consummation of the business combination, higher cell therapy process development and research expenses related to the CyCART-19 program, and higher personnel costs.
Selling, general and administrative expenses for the nine months ended September 30, 2021, increased $32.8 million compared to the prior year period, primarily due to higher stock-based compensation resulting from awards granted in connection with the consummation of the business combination , a charge related to an estimated legal settlement and higher personnel costs.
Net loss for the nine months ended September 30, 2021, was $96.1 million, or $2.00 per share (basic and diluted).