Agios Pharmaceuticals 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 5, 2022 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a late clinical-stage biopharmaceutical company focused on oncology, reported it will release its unaudited financial results for the quarter ended March 31, 2022 on Wednesday, May 11, 2022, before the US markets open (Press release, BioLineRx, MAY 5, 2022, View Source [SID1234613612]).

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The Company will host a conference call on Wednesday, May 11, 2022 at 10:00 a.m. EDT featuring remarks by Philip Serlin, Chief Executive Officer. The conference call will be available via webcast and can be accessed through the Investor Relations page of BioLineRx’s website. Please allow extra time prior to the call to visit the site and download any necessary software to listen to the live broadcast.

To dial into the conference call, please dial +1-866-744-5399 from the U.S. or +972-3-918-0644 internationally. A replay of the conference call will be available approximately two hours after completion of the live conference call on the Investor Relations page of BioLineRx’s website. A dial-in replay of the call will be available until May 13, 2022; please dial +1-888-295-2634 from the U.S. or +972-3-925-5904 internationally.

On May 5, 2022 Cassava Sciences, Inc. (Nasdaq: SAVA), a clinical-stage biotechnology company focused on Alzheimer’s disease, reported financial results for the first quarter ended March 31, 2022 and provided a clinical update on its Phase 3 clinical program of simufilam in Alzheimer’s disease (Press release, Pain Therapeutics, MAY 5, 2022, View Source [SID1234613641]). Simufilam is Cassava Sciences’ lead drug candidate for the proposed treatment of Alzheimer’s disease.

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Net loss was $17.5 million, or $0.44 per share, compared to a net loss of $3.5 million, or $0.09 per share, for the same period in 2021. Net cash used in operations was $23.5 million during the first quarter of 2022, including over $10 million of contractual pre-payments. Pre-payments are amounts paid for in advance of future research and development (R&D) services.

"We’re seeing an uptick in the rate of patient enrollment," said Remi Barbier, President & CEO. "In part, the pandemic challenged some clinical sites, from staffing shortages to operational gaps. We hope these challenges are in the rearview mirror as we continue to move forward with our Phase 3 studies of simufilam in Alzheimer’s disease, while keeping an eye on the balance sheet."

Financial Results for First Quarter 2022

At March 31, 2022, cash and cash equivalents were $209.7 million, with no debt.

Net loss was $17.5 million, or $0.44 per share. This compares to a net loss of $3.5 million, or $0.09 per share, for the same period in 2021. Net loss increased compared to the prior period due primarily to a significant increase in our R&D activities and G&A expenses.

Net cash used in operations was $23.5 million during the first quarter of 2022.

Net cash use in operations for the first half 2022 is now expected to be approximately $35 to $40 million, including significant (over $10 million) contractual pre-payments made to R&D vendors for future services, such as our contract research organization for the Phase 3 clinical program of simufilam in Alzheimer’s disease.

Research and development (R&D) expenses were $14.9 million. This compared to $2.5 million for the same period in 2021. R&D expenses increased compared to the prior period due primarily to increased activities and expenses related to clinical studies, clinical trial supplies and support functions and personnel expenses.

General and administrative (G&A) expenses were $2.9 million. This compared to $1.0 million for the same period in 2021. G&A expenses increased compared to the prior period due primarily to increased activities and expenses related to legal services and depreciation and amortization.
Overview of On-going Phase 3 Clinical Program
Our Phase 3 program consists of two double-blind, randomized, placebo-controlled studies of simufilam in patients with mild-to-moderate Alzheimer’s disease. Both Phase 3 studies have Special Protocol Assessments (SPA) from the U.S. Food and Drug Administration.

A total of over 120 subjects have now been enrolled in our Phase 3 studies. Studies are being conducted in over 115 clinical trial sites across the U.S., Canada and Puerto Rico.

Overview of Each On-going Phase 3 Study – RETHINK-ALZ and REFOCUS-ALZ
Our Phase 3 study called "RETHINK-ALZ" is designed to evaluate the safety and efficacy of oral simufilam 100 mg in enhancing cognition and slowing functional decline over 52 weeks. This randomized, double-blind, placebo-controlled study plans to enroll approximately 750 patients with mild-to-moderate Alzheimer’s disease.

Details of the RETHINK-ALZ Phase 3 study include:

Subjects are randomized (1:1) to simufilam 100 mg or placebo twice daily.
The co-primary efficacy endpoints are ADAS-Cog12 (a cognitive scale) and ADCS-ADL (a functional scale). A secondary efficacy endpoint is iADRS, a clinical tool that combines cognitive and functional scores from ADAS-Cog & ADCS-ADL.
Our Phase 3 study called "REFOCUS-ALZ" is designed to evaluate the safety and efficacy of oral simufilam 100 mg and 50 mg over 76 weeks. This randomized, double-blind, placebo-controlled study plans to enroll approximately 1,000 patients with mild-to-moderate Alzheimer’s disease.

Details of the REFOCUS-ALZ Phase 3 study, include:

Subjects are randomized (1:1:1) to simufilam 100 mg, 50 mg, or placebo twice daily.
The co-primary efficacy endpoints are ADAS-Cog12 (a cognitive scale) and ADCS-ADL (a functional scale). A secondary efficacy endpoint is iADRS, a clinical tool that combines cognitive and functional scores from ADAS-Cog & ADCS-ADL.
Open-label Study – closed enrollment

In March 2020, we initiated a long-term, open-label study to evaluate simufilam, our lead drug candidate, in patients with mild-to-moderate Alzheimer’s disease. The study is intended to monitor the long-term safety and tolerability of simufilam 100 mg twice daily for 12 or more months. The open-label study has reached its final target enrollment of approximately 200 subjects with Alzheimer’s disease. We expect to announce full study results second half 2022.

Cognition Maintenance Study (CMS) – on-going
In May 2021, we initiated a Cognition Maintenance Study (CMS). This is a double-blind, randomized, placebo-controlled study of simufilam in patients with mild-to-moderate Alzheimer’s disease. Study participants are randomized (1:1) to simufilam or placebo for six months. To enroll in the CMS, patients must have previously completed 12 months or more of open-label treatment with simufilam. The CMS is designed to evaluate simufilam’s effects on cognition and health outcomes in Alzheimer’s patients who continue with drug treatment versus patients who discontinue drug treatment. The target enrollment for the CMS is approximately 100 subjects. Over 75 subjects have been enrolled in the CMS and 35 have completed the study.

SavaDx – on-going
SavaDx, is an early-stage program focused on detecting the presence of Alzheimer’s disease from a small sample of blood. For business, technical and personnel reasons, we continue to prioritize the development of simufilam, our lead drug candidate, over SavaDx.

About Simufilam
Simufilam (sim-uh-FILL-am) is a proprietary, small molecule (oral) drug that restores the normal shape and function of altered filamin A (FLNA) protein in the brain. Cassava Sciences owns worldwide development and commercial rights to its research programs in Alzheimer’s disease, and related technologies, without royalty obligations to any third party.

On March 5, 2022 Alector, Inc. (Nasdaq: ALEC), a clinical-stage biotechnology company pioneering immuno-neurology, reported first quarter 2022 financial results and recent portfolio and business updates (Press release, Alector, MAY 5, 2022, View Source [SID1234613656]). As of March 31, 2022, Alector’s cash, cash equivalents, and marketable securities totaled $868.6 million.

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"We made significant progress in the first quarter, highlighted by the presentation of 12-month data from our INFRONT-2 Phase 2 clinical trial of latozinemab in patients with symptomatic FTD-C9orf72, in which treatment with latozinemab demonstrated target engagement and resulted in increases in progranulin levels above physiological levels in all patients," said Arnon Rosenthal, Ph.D., Chief Executive Officer of Alector. "Data from the FTD-C9orf72 cohort builds upon the results observed in our studies to date in patients with symptomatic FTD-GRN. The totality of the data supports our approach of elevating progranulin levels to address a range of neurodegenerative diseases including FTD, ALS, Parkinson’s disease and Alzheimer’s disease. We look forward to evaluating latozinemab and AL101 in multiple indications as part of our progranulin franchise in partnership with GlaxoSmithKline."

Sara Kenkare-Mitra, Ph.D., President and Head of Research and Development at Alector, added, "In parallel to advancing and expanding our progranulin franchise, we continue to make progress in our Alzheimer’s and oncology programs. Our INVOKE-2 Phase 2 clinical trial evaluating AL002 in slowing disease progression in individuals with early Alzheimer’s disease is ongoing, and we also expect to initiate a Phase 1 trial for AL044, which targets MS4A, a major risk locus for Alzheimer’s disease, in the second half of 2022. Additionally, we presented exciting preclinical data from our AL009 immuno-oncology program at the 2022 AACR (Free AACR Whitepaper) Annual Meeting, with a Phase 1 trial slated to begin within the next year. This clinical momentum, coupled with the addition of veteran neurodegeneration expert, Gary Romano, as Chief Medical Officer, puts Alector in a strong position to execute our goal of halting the destruction caused by neurodegenerative diseases."

Recent Clinical Updates

Progranulin Franchise Portfolio

Twelve-month data from the INFRONT-2 Phase 2 clinical trial of latozinemab in frontotemporal dementia patients (FTD) with a C9orf72 genetic mutation (FTD-C9orf72) were presented at the AD/PD 2022 International Conference on Alzheimer’s and Parkinson’s Diseases and related neurological disorders. Latozinemab treatment was well tolerated and resulted in a two- to three-fold increase in progranulin above physiological levels in cerebrospinal fluid (CSF) and plasma respectively, and a decrease in the neurodegeneration biomarker GFAP. When compared to a matched control cohort from the ALLFTD consortium, treatment with latozinemab in FTD-C9orf72 patients resulted in a trend towards an annual delay of disease progression of approximately 54 percent as measured by the CDR plus NACC FTLD-SB scale. These data support the company’s efforts to expand the progranulin franchise into additional neurodegenerative disease indications.
Enrollment is ongoing in INFRONT-3, a randomized, placebo-controlled, pivotal Phase 3 trial evaluating the efficacy and safety of latozinemab in at-risk and symptomatic patients with FTD due to a progranulin gene mutation (FTD-GRN). Participants in the trial will be given the option to continue receiving treatment in an open-label extension study.
In partnership with GSK, the company made a strategic, non-safety related decision to close enrollment in the Phase 2a biomarker trial of latozinemab in people with amyotrophic lateral sclerosis (ALS) who carry a C9orf72 mutation. In light of the evolving ALS landscape, the company is currently evaluating plans for a potential Phase 2b study for patients with all forms of ALS, including the C9orf72 mutation.
Alector completed enrollment in the ongoing Phase 1 clinical trial to test multiple doses of AL101 administered intravenously and subcutaneously. AL101, the second drug candidate in the company’s progranulin franchise, is designed to elevate progranulin levels, similar to AL001, with the potential for easier administration and/or less frequent dosing for the treatment of more prevalent neurodegenerative diseases, including Alzheimer’s disease and Parkinson’s disease. The company expects to report data from the Phase 1 trial in the second half of 2022.
Alzheimer’s Disease Portfolio

The INVOKE-2 Phase 2 clinical trial evaluating the efficacy and safety of AL002 in slowing disease progression in individuals with early Alzheimer’s disease is ongoing. AL002 targets Triggering Receptor Expressed on Myeloid cells 2 (TREM2) to increase TREM2 signaling and the functionality of the microglia brain specific immune cells. It is being developed in collaboration with AbbVie.
AL003 is being developed to treat patients with Alzheimer’s disease in collaboration with AbbVie. AL003 focuses on modulating checkpoint receptors on the brain’s immune cells, targeting sialic acid binding Ig-like lectin 3 (SIGLEC 3, also called CD33). In 2021, we presented data from the Phase 1 trial of AL003 in healthy volunteers and Alzheimer’s disease patients. Alector is currently reviewing potential next steps for the AL003 program together with AbbVie.
Alector expects to initiate a Phase 1 clinical trial for AL044 in the second half of 2022. AL044 targets MS4A, a major risk locus for Alzheimer’s disease. MS4A gene family members encode a transmembrane protein that is expressed selectively in myeloid cells in the brain and is associated with control of microglia functionality and potentially with microglia viability.
Immuno-oncology Portfolio

At the 2022 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, Alector presented preclinical data from its first-in-class AL009 innate immuno-oncology program. AL009 is a dual function biologic that inhibits multiple Siglec receptors on myeloid cells and simultaneously activates a stimulating receptor on the same cells. The findings demonstrated that AL009 led to repolarization of myeloid cells and activation of innate and adaptive immunity against tumors. Pharmacologically relevant doses of AL009 appeared well-tolerated in initial non-human primate studies. IND enabling studies are ongoing, and a Phase 1 clinical study of AL009 in patients with advanced solid tumors is expected to begin within the next year.
Recent Corporate News

Gary Romano, M.D., Ph.D., will join Alector’s executive leadership team as Chief Medical Officer (CMO) on May 23, 2022, and Marc Grasso, M.D., joined as Chief Financial Officer (CFO).
Dr. Romano is a board-certified neurologist, neurodegeneration expert and recognized clinical leader in the industry with a demonstrated track record in progressing the development of therapeutics for multiple neuroscience indications. As CMO, Dr. Romano will lead the company’s global clinical development strategy, including oversight of the clinical development, clinical operations, biometrics and digital science and medical affairs functions.
Dr. Grasso brings extensive biotechnology industry leadership experience, including a successful track record in finance and corporate development. As CFO, Dr. Grasso leads all aspects of the company’s financial operations and plays a critical role in supporting corporate strategy.
First Quarter 2022 Financial Results

Revenue. Collaboration revenue for the quarter ended March 31, 2022, was $24.5 million, compared to $4.1 million for the same period in 2021. This increase was primarily due to revenue recognized from the GSK Agreement.

R&D Expenses. Total research and development expenses for the quarter ended March 31, 2022, were $53.0 million, compared to $45.7 million for the quarter ended March 31, 2021. The increase in R&D expenses was mainly driven by increased personnel-related expenses, as well as increased spending to support advancement of several clinical and preclinical programs.

G&A Expenses. Total general and administrative expenses for the quarter ended March 31, 2022, were $15.6 million, compared to $11.0 million for the same period in 2021 was primarily due to an increase in personnel-related expenses and increase in legal expenses from the arbitration award in 2021 that reduced expenses.

Net Loss. For the quarter ended March 31, 2022, Alector reported a net loss of $44.6 million, or $0.54 per share, compared to a net loss of $52.2 million, or $0.66 per share, for the same period in 2021.

Cash Position. Cash, cash equivalents, and marketable securities were $868.6 million as of March 31, 2022. Management expects that this will be sufficient to fund current operations into mid-2024.

On May 5, 2022 Jounce Therapeutics, Inc. (NASDAQ: JNCE), a clinical-stage company focused on the discovery and development of novel cancer immunotherapies and predictive biomarkers, reported financial results for the first quarter ended March 31, 2022 and provided a corporate update (Press release, Jounce Therapeutics, MAY 5, 2022, View Source [SID1234613672]).

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"Jounce made significant progress this quarter as we focused on the continued advancement of our two proof of concept studies, INNATE and SELECT, in addition to advancing candidates from our discovery engine, such as JTX-1484, our LILRB4 (or ILT3) inhibitor. With our cash runway extending beyond our key inflection points, we are poised for an important second half of this year," said Richard Murray, Ph.D., chief executive officer and president of Jounce Therapeutics. "I’m very pleased to share that we recently met the prespecified response criteria in two combination cohorts to continue the Phase 2 expansion in the INNATE trial, and, in our SELECT trial, we have achieved target enrollment. These achievements represent significant progress in building our pipeline of diverse immunotherapy candidates. We continue to focus on our mission of delivering meaningful and long-lasting benefit to cancer patients through the discovery and pursuit of therapies that target new mechanisms of immune suppression across different types of immune cells, and bringing the right immunotherapies to the right patients."

Pipeline Update & Highlights:
JTX-8064 (LILRB2/ILT4)

Expanded two of seven combination cohorts in INNATE Phase 2 trial. Jounce is evaluating JTX-8064 in the ongoing Phase 2 portion of the INNATE clinical trial, which is comprised of indication specific expansion cohorts, including one monotherapy cohort and seven combination therapy cohorts. Each cohort is a Simon 2-stage design, in which we enroll 10 patients, wait for initial response data, and then further expand to a total of 29 patients if prespecified response criteria are met. Today, Jounce is announcing that the first two combination cohorts have met their response criteria for expansion within INNATE and are now continuing enrollment to 29 patients each. Jounce has seen an acceptable safety profile for both the monotherapy and combination therapy to date.

On track to report preliminary clinical data in the second half of 2022. The INNATE clinical trial is studying three distinct patient populations across the 7 indications: (1) PD-1 inhibitor naïve patients with tumors for which there are approved PD-1 or PD-L1 inhibitors, (2) PD-(L)1 inhibitor naïve patients who have tumors for which there are no PD-1 or PD-L1 inhibitors approved and (3) patients who were previously treated with a PD-1 inhibitor and are PD-1 inhibitor resistant. Jounce remains on track to report preliminary clinical data, including all 31 dose escalation patients and at least 60 Phase 2 patients from INNATE, in the second half of 2022.
Vopratelimab (ICOS) and Pimivalimab (PD-1)

Patient screening finished in the Phase 2 SELECT trial of vopratelimab. Patient screening is complete with the target enrollment of at least 60 evaluable patients having been met in SELECT, a randomized Phase 2 trial evaluating vopratelimab in combination with pimivalimab versus pimivalimab alone in immunotherapy naïve, TISvopra biomarker-selected, second line non-small cell lung cancer (NSCLC) patients. Jounce is on track to report data from the SELECT trial in the second half of 2022, including additional single agent data for pimivalimab.
JTX-1484 (LILRB4/ILT3)

JTX-1484 is the most recent product candidate to emerge from our Translational Science Platform. JTX-1484 is a monoclonal antibody designed to block human LILRB4 expressed on myeloid cells in the tumor microenvironment with the potential to reduce immune suppression and enhance T cell functionality. JTX-1484 is currently in IND-enabling activities, with the goal of filing an investigational new drug application ("IND") in 2023.
Discovery Pipeline

Productive discovery engine with the goal of an IND every 12 to 18 months: Jounce continues to invest in and advance its growing immuno-oncology pipeline. Its discovery engine is built upon the capability to thoroughly interrogate different cell types in the tumor microenvironment, including T cells and myeloid cells. This approach has resulted in four clinical stage programs, with a fifth in IND enabling studies, over the last 6 years.
First Quarter 2022 Financial Results:

Cash position: As of March 31, 2022, cash, cash equivalents and investments decreased to $186.4 million, compared to $220.2 million as of December 31, 2021. The decrease was due to operating expenses incurred during the period.
License and collaboration revenue: Jounce did not recognize any revenue during the first quarter of 2022. License and collaboration revenue of $1.5 million was recognized during the first quarter of 2021 and was comprised solely of non-cash revenue related to the performance of research and transition services under the Gilead License Agreement.
Research and development expenses: Research and development expenses were $30.1 million for the first quarter of 2022, compared to $20.5 million for the same period in 2021. The increase in research and development expenses was primarily due to increased manufacturing activities performed for Jounce’s development programs, increased clinical and regulatory costs for INNATE, and increased payroll and stock-based compensation expense.
General and administrative expenses: General and administrative expenses were $7.3 million for the first quarter of 2022, compared to $7.6 million for the same period in 2021. The decrease in general and administrative expenses was primarily due to decreased external consulting and stock-based compensation expense.
Net loss: Net loss was $37.4 million for the first quarter of 2022, resulting in basic and diluted net loss per share of $0.72. Net loss was $26.5 million for the same period in 2021, resulting in a basic and diluted net loss per share of $0.58. The increase in net loss was primarily attributable to increased operating expenses incurred during the first quarter of 2022.
Financial Guidance:
Based on its current operating and development plans, Jounce reiterates its financial guidance for 2022. Gross cash burn on operating expenses and capital expenditures for the full year 2022 is expected to be approximately $115.0 million to $130.0 million. Given the strength of its balance sheet, Jounce expects its existing cash, cash equivalents and investments to be sufficient to enable the funding of its operating expenses and capital expenditure requirements through the third quarter of 2023.

Conference Call and Webcast Information:
Jounce Therapeutics will host a live conference call and webcast today at 8:00 a.m. ET. To access the conference call, please dial (866) 916-3380 (domestic) or (210) 874-7772 (international) and refer to conference ID 9072989. The live webcast can be accessed under "Events & Presentations" in the Investors and Media section of Jounce’s website at www.jouncetx.com. The webcast will be archived and made available for replay on Jounce’s website approximately two hours after the call and will be available for 30 days.