On April 21, 2021 Sermonix Pharmaceuticals Inc., a privately held biopharmaceutical company developing innovative therapeutics to treat ESR1-mutated metastatic breast and gynecological cancers, reported a preclinical collaboration with researcher Jay Gertz, Ph.D., to examine the potential effects of lasofoxifene, Sermonix’s lead investigational drug, on endometrial cancer (Press release, Sermonix Pharmaceuticals, APR 21, 2021, View Source [SID1234578341]).

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The project will investigate the effects of lasofoxifene on unique models of endometrial cancer that carry ESR1 mutations. Lasofoxifene has shown novel activity in ESR1 mutations, and Sermonix is currently enrolling patients in two Phase 2 Evaluation of Lasofoxifene in ESR1 Mutations (ELAINE) studies within the breast cancer arena.

"Sermonix is committed to exploring ESR1 mutations across gynecological tumor types and examining lasofoxifene’s potential to serve the unmet medical needs of more women fighting cancer," said Barry Komm, Ph.D., chief scientific officer of Sermonix. "The unique ESR1 model system is state-of-the-art for this exploration and Dr. Gertz is an esteemed partner with whom it is an honor for Sermonix to collaborate."

Dr. Gertz is a cancer researcher at Huntsman Cancer Institute and associate professor of oncological sciences at the University of Utah. His lab studies how transcription regulation is altered in cancer with a particular focus on the roles of steroid hormone signaling in endometrial and breast cancers.

"The Gertz Lab has a major emphasis in advancing the study of new drugs for endometrial cancer, an aggressive disease with limited treatment options," said Dr. Gertz. "We look forward to learning more about the efficacy of lasofoxifene in the context of ESR1-mutated endometrial cancer."

About Lasofoxifene
Lasofoxifene is an investigational, nonsteroidal selective estrogen receptor modulator (SERM), which Sermonix licensed globally from Ligand Pharmaceuticals Inc. (NASDAQ: LGND) and has been studied in previous comprehensive Phase 1-3 non-oncology clinical trials in more than 15,000 postmenopausal women worldwide. Lasofoxifene’s bioavailability and activity in mutations of the estrogen receptor could potentially hold promise for patients who have acquired endocrine resistance due to ESR1 mutations, a common finding in the metastatic setting and an area of high unmet medical need. Lasofoxifene’s novel activity in ESR1 mutations was discovered at Duke University and Sermonix has exclusive rights to develop and commercialize the product in this area. Lasofoxifene, a potent, oral SERM could, if approved, play a critical role in the targeted precision medicine treatment of advanced ER+ breast cancer.

On April 21, 2021 Recursion Pharmaceuticals, Inc. ("Recursion" or "we") reported the closing on April 20, 2021 of its initial public offering of 27,878,787 shares of its Class A common stock, which includes the exercise in full of the underwriters’ option to purchase 3,636,363 additional shares of its Class A common stock, at a price to the public of $18.00 per share (Press release, Recursion Pharmaceuticals, APR 21, 2021, View Source [SID1234578289]). Including the option exercise, the gross proceeds from the offering were $501.8 million, before deducting underwriting discounts and commissions and other offering expenses payable by Recursion. All of the shares were offered by Recursion. The shares began trading on the Nasdaq Global Select Market on April 16, 2021 under the symbol "RXRX."

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Goldman Sachs & Co. LLC and J.P. Morgan acted as lead book-running managers for the offering. BofA Securities, SVB Leerink and Allen & Company LLC also acted as active book-running managers. KeyBanc Capital Markets acted as book running manager for the offering.

Registration statements relating to this offering have been filed with the Securities and Exchange Commission and became effective on April 15, 2021. This offering was made only by means of a prospectus. Copies of the final prospectus may be obtained from: Goldman Sachs & Co. LLC, Attention: Prospectus Department, 200 West Street, New York, NY 10282, by telephone at (866) 471-2526 or by email at [email protected]; or J.P. Morgan Securities LLC, Attention: Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717 or by telephone at (866) 803-9204 or by email at [email protected]. Copies of the final prospectus related to the offering are also available at www.sec.gov.

This press release does not constitute an offer to sell or the solicitation of an offer to buy these securities, nor will there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On April 21, 2021 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, 2021 and guidance regarding the release of new clinical data with simufilam (Press release, Pain Therapeutics, APR 21, 2021, View Source [SID1234578308]). Simufilam is the Company’s lead drug candidate to treat Alzheimer’s disease.

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"Alzheimer’s is a progressive disease, so a patient’s cognition is expected to worsen over time," said Remi Barbier, President & CEO. "Patients’ cognition scores actually improved following 6 months of open-label treatment with simufilam. Showing similar drug effects following 9 months of open-label treatment would be remarkable, yet consistent with simufilam’s mechanism of action. Eventually, we’d like this drug candidate to benefit cognition for a year or longer."

In July 2021, Cassava Sciences plans to announce results of a pre-specified interim analysis that summarizes safety and cognition data on approximately the first 50 subjects to complete at least 9 months of open-label drug treatment. The Company will present these data July 26 – 29th at the 2021 Alzheimer’s Association International Conference (AAIC). AAIC’s scientific committee has invited the Company’s scientists to present the dataset as an oral presentation.

About the Open-label Study with Simufilam
In March 2020, Cassava Sciences initiated a long-term, open-label study to evaluate simufilam in patients with Alzheimer’s disease. This study is funded by a research grant award from the National Institutes of Health (NIH). The open-label study is intended to monitor the long-term safety and tolerability of simufilam 100 mg twice-daily for 12 months or longer in patients with Alzheimer’s disease. Another study objective is to measure changes in cognition on ADAS-Cog, a standard test of cognition in Alzheimer’s disease. The study’s clinical protocol has pre-specified cognition measurements at 6, 9 and 12 months.

The study’s target enrollment is approximately 150 subjects with mild-to-moderate Alzheimer’s disease (recently increased by 50 subjects). One-hundred subjects have enrolled in this study across multiple clinical sites in the U.S. and Canada.

On February 2, 2021, Cassava Sciences announced positive results of a first interim analysis that summarizes clinical data on the first 50 subjects to complete 6 months of open-label treatment. Patients’ cognition scores improved from baseline following 6 months of simufilam treatment, with no safety issues. Six months of simufilam treatment improved cognition scores by 1.6 points on ADAS-Cog11, a 10% mean improvement from baseline to month 6.

In September 2021, Cassava Sciences plans to announce results of an interim analysis that summarizes safety and cognition data on approximately the first 50 subjects to complete at least 12 months of open-label drug treatment.

About the Cognition Maintenance Study (CMS)
In June 2021, Cassava Sciences plans to initiate a double-blind, randomized, placebo-controlled study in patients with Alzheimer’s disease. Patients who have completed at least one year of open-label treatment with simufilam qualify to enroll in the Cognition Maintenance Study (CMS). Study subjects in the CMS will be randomized (1:1) to simufilam or placebo for six months. The CMS is designed to compare simufilam’s effects on cognition in Alzheimer’s patients who continue with drug treatment versus patients who discontinue drug treatment.

About the Phase 3 Clinical Program
Cassava Sciences plans to initiate a Phase 3 program of simufilam in Alzheimer’s disease in the second half of 2021. The Phase 3 program consists of two large, double-blind, randomized, placebo-controlled studies in patients with mild-to-moderate Alzheimer’s disease dementia.

Cassava Sciences’ first Phase 3 study is designed to evaluate disease-modifying effects of simufilam in Alzheimer’s disease. The goal is to demonstrate a slower rate of decline in cognition and health function in subjects treated with simufilam compared to placebo. Approximately 1,000 subjects to be enrolled, randomized (1:1:1) to simufilam 100 mg, 50 mg or placebo BID, and treated for 18 months. The co-primary efficacy endpoints are ADAS-Cog, a cognitive scale, and ADCS-ADL, a functional scale, both widely used clinical tools in trials of Alzheimer’s disease.

Cassava Sciences’ second Phase 3 study is designed to evaluate symptomatic improvement in Alzheimer’s disease. The goal is to demonstrate improved cognition and health function in subjects treated with simufilam compared to placebo. Approximately 600 subjects to be enrolled, randomized (1:1) to simufilam 100 mg or placebo BID, and treated for 12 months. The co-primary efficacy endpoints are ADAS-Cog, a cognitive scale, and ADCS-ADL, a functional scale.

Slide Deck
Cassava Sciences’ latest corporate presentation is available on its website under the Investors/Presentations page: View Source

Financial Results for First Quarter 2021
Net loss was $3.5 million, or $0.09 per share, compared to a net loss of $1.2 million, or $0.05 per share, for the same period in 2020. Net cash used in operations was $2.3 million during the first quarter of 2021.

Net cash use for operations for full-year 2021 is expected to be approximately $20 to $25 million. Cash and cash equivalents were $282.2 million as of March 31, 2021, with no debt.

Financial Highlights for First Quarter 2021

At March 31, 2021, cash and cash equivalents were $282.2 million, compared to $93.5 million at December 31, 2020, with no debt. Cash balance included net proceeds of approximately $189.8 million from the sale of 4.1 million shares of common stock completed February 2021. Cash balance also included $0.7 million from exercise of common stock warrants in the quarter. There were no remaining common stock warrants outstanding as of March 31, 2021.

Net cash used in operations during the quarter ended March 31, 2021 was $2.3 million, net of reimbursements received from NIH grant awards.

Research grant funding reimbursements of $0.6 million were received from NIH and recorded as a reduction in research and development (R&D) expenses. This compared to $1.3 million of NIH grant receipts received for the same period in 2020.

Net cash use for operations for full year 2021 is expected to be approximately $20 to $25 million, consistent with previous financial guidance. Net cash use in 2021 is expected to be driven by higher headcount and personnel expenses, manufacturing costs around large-scale drug supply, professional services expenses related to clinical programs, and operating costs such as insurance, office space and IT related expenses.

R&D expenses were $2.5 million. This compared to $0.5 million for the same period in 2020, representing a 365% increase. This increase was due primarily to costs related to manufacture of clinical trial supplies in anticipation of launching a Phase 3 clinical program in simufilam, increased personnel expenses, as well as a decrease in grant funding received from NIH compared to the prior year.

General and administrative (G&A) expenses were $1.0 million. This compared to $0.8 million for the same period in 2020, representing a 29% increase. This increase was due primarily to higher insurance costs and professional fees compared to the prior year.
About Simufilam
Simufilam is a proprietary, small molecule (oral) drug that restores the normal shape and function of altered filamin A (FLNA), a scaffolding protein, in the brain. Altered FLNA in the brain disrupts the normal function of neurons, leading to Alzheimer’s pathology, neurodegeneration and neuroinflammation. The underlying science for simufilam is published in peer-reviewed journals, including Journal of Neuroscience, Neurobiology of Aging, Journal of Biological Chemistry, Neuroimmunology and Neuroinflammation and Journal of Prevention of Alzheimer’s Disease. Cassava Sciences is also developing an investigational diagnostic, called SavaDx, to detect Alzheimer’s disease with a simple blood test.

Simufilam and SavaDx were both developed in-house. Both product candidates are substantially funded by peer-review research grant awards from the National Institutes of Health (NIH). 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.

About Alzheimer’s Disease
Alzheimer’s disease is a progressive brain disorder that destroys memory and thinking skills. Currently, there are no drug therapies to halt Alzheimer’s disease, much less reverse its course. As of 2020, there were approximately 50 million people worldwide living with dementia, a figure expected to increase to 150 million by 2050.1 The annual global cost of dementia is now above $1 trillion, according to Alzheimer’s Disease International, a charitable organization.

On April 21, 2021 Versant Ventures reported $950 million in additional capital allocated across a platform of three separate vehicles (Press release, Versant Ventures, APR 21, 2021, View Source [SID1234578394]). These included Versant Venture Capital VIII, a $560 million primary global biotech fund; Versant Voyageurs II, a $140 million booster fund; and Versant Vantage II, a $250 million later-stage opportunity fund.

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All three funds exceeded their initial targets and were heavily oversubscribed. The capital from existing investors and a select number of new top-tier limited partners will support Versant’s highly successful biotech investment strategy and portfolio construction model.

Versant Venture Capital VIII – primary global biotech fund

Fund VIII closed at $560 million and will be allocated to 20 or more biotechnology start-ups in the U.S., Canada and Europe. These newcos will be focused on translating breakthrough innovation into the next generation of therapeutics targeting the most important unmet medical needs. The emphasis will be placed on building sustainable companies that follow business models with dual paths to liquidity.

The fund will be deployed by the same team and will follow the same investment strategy that is consistently producing outlier returns. More specifically, Versant’s three predecessor funds are currently demonstrating top decile performance across the venture industry.

"The pace of scientific discovery and technology development in the healthcare sector continues to accelerate, including advances in engineering new cell and gene therapies, developing the next generation of antibodies and therapeutic proteins, and even conceiving novel small molecule constructs for previously intractable targets in the human proteome," said Brad Bolzon, Ph.D., chairman and managing director of Versant. "These innovations are at the heart of our investment model."

Versant expects the majority of Fund VIII portfolio companies to be created de novo by working directly with talented entrepreneurs or through the firm’s existing Discovery Engines that support about 60 scientists working in wet labs across North America and Europe.

Versant Voyageurs II – strategic booster fund

Versant Voyageurs II closed at $140 million and will co-invest with Fund VIII in a select number of Series A opportunities that show early potential to be breakout companies.

"During the course of constructing any given portfolio, we inevitably generate a select number of deals showing exceptional potential from the outset," said Dr. Bolzon. "In these circumstances, we now have the ability to access additional Versant capital beyond our typical Series A investment allocations to increase momentum."

Rather than extend Versant’s main fund and jeopardize reserves planned for other valuable portfolio companies, the Voyageurs II capital will be available to participate alongside those specific Series A rounds. Thus, Voyageurs II will serve as a booster fund for a handful of select deals.

Versant Vantage II – strategic opportunity fund

Versant Vantage II closed at $250 million and will primarily invest in Series B or later rounds of existing Versant portfolio companies that are nearing a liquidity event.

The remit of Vantage II is similar to its predecessor fund, Versant Vantage I, which was designed to provide later-stage investment opportunities in portfolio companies believed to be within 12 months of an exit.

"Vantage I launched in March 2019 and has exceeded expectations. Of the fund’s 15 later-stage commitments, there already are 11 liquid companies with an average time to IPO or M&A event of about six months," noted Dr. Bolzon. "We expect similar outcomes from Vantage II, which already has a deep lineup of potential Versant companies in which to invest."

Managing directors leading investments for the new funds include Dr. Bolzon, Jerel Davis, Ph.D., Alex Mayweg, Ph.D., Clare Ozawa, Ph.D., and Tom Woiwode, Ph.D.

On April 20, 2021 Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing novel cell transplants for serious medical conditions, reported a worldwide license and development collaboration agreement with Immunomic Therapeutics, Inc., ("ITI"), a privately-held clinical-stage biotechnology company pioneering the study of nucleic acid immunotherapy platforms (Press release, Lineage Cell Therapeutics, APR 20, 2021, View Source [SID1234578238]). The collaboration will generate a novel product candidate derived from Lineage’s VAC allogeneic cancer immunotherapy platform and targeting a proprietary Tumor Associated Antigen (TAA) construct provided by ITI, for the treatment of glioblastoma multiforme (GBM). Lineage and ITI will collaborate in the manufacturing and clinical development of a novel VAC product candidate. Following the full development and delivery of Current Good Manufacturing Practice (cGMP) VAC product material, ITI will assume full and independent clinical and commercial responsibility and further advancement of the program. Under the terms of the agreement, Lineage will be entitled to upfront payments totaling $2 million anticipated in the first year and up to $67 million in development and commercial milestones across multiple indications and territories. Lineage also will be eligible to receive royalties up to 10% on net sales of future products.

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"The VAC platform provides us with the opportunity to generate a broad pipeline of product candidates, each targeting a different type of cancer," stated Brian Culley, Lineage CEO. "This collaboration represents the first of many partnerships we hope to enter into with our platform and we believe it helps further validate VAC as a promising new therapeutic vaccine platform. Our objective is to leverage our technology to generate additional VAC-derived cell therapies for our pipeline, as well as in collaboration with partners, capitalizing on the strength of Lineage’s recent manufacturing and cell transplant success. These alliances also will diversify our oncology pipeline across more programs, providing new opportunities for success without the financial burden of independent development. We appreciate ITI selecting our antigen delivery platform for this collaboration and look forward to a productive partnership on this new VAC-derived product candidate. We also are eager to collaborate with additional partners on future versions of VAC."

"We’re very pleased to collaborate with Lineage, a well-recognized cell therapy company, to expand our pipeline with the development of a novel product candidate to treat GBM," commented Dr. William Hearl, CEO of ITI. "Over the last several years, ITI has invested significant capital and development resources to identifying multiple novel paths forward in GBM. By teaming up with Lineage, we are hoping to expand our efforts in this difficult to treat indication and look forward to the benefit that the VAC immunotherapy platform can bring to our antigen constructs."

About Glioblastoma multiforme (GBM)

Glioblastoma multiforme (GBM) (also called glioblastoma) is a fast-growing glioma that develops from star-shaped glial cells (astrocytes and oligodendrocytes) that support the health of the nerve cells within the brain. GBM is often referred to as a grade IV astrocytoma. These are the most invasive type of glial tumors, rapidly growing and commonly spreading into nearby brain tissue. GBMs can arise in the brain "de novo" or evolve from lower-grade astrocytomas or oligodendrogliomas. In adults, GBM occurs most often in the cerebral hemispheres, especially in the frontal and temporal lobes of the brain. GBM is a devastating brain cancer that typically results in death in the first 15 months after diagnosis, with only 25% of glioblastoma patients surviving more than one year, and only 5% of patients surviving more than five years.

About VAC2

VAC2 is an allogeneic, or non-patient specific "off-the-shelf," cancer vaccine product candidate designed to stimulate patient immune responses to an antigen commonly expressed in cancerous cells but not in normal adult cells. VAC2, which is produced from a pluripotent cell technology using a directed differentiation method, is comprised of a population of nonproliferating mature dendritic cells. As the most potent type of antigen presenting cell in the body, dendritic cells instruct the body’s immune system to attack and eliminate harmful pathogens and unwanted cells. Because the tumor antigen is loaded exogenously into the dendritic cells prior to administration, VAC2 is a platform technology that can be modified to carry selected antigens, including patient-specific tumor neo-antigens or viral antigens. VAC2 is currently being tested in a Phase 1 study in adult patients with non-small cell lung cancer (NSCLC) in the advanced and adjuvant settings (NCT03371485), conducted by Cancer Research UK.