On August 3, 2022 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported that financial results for the second quarter of 2022 and provided a business update (Press release, Regeneron, AUG 3, 2022, View Source [SID1234617391]).

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"The second quarter of 2022 was distinguished by record net product sales of EYLEA, Dupixent, and Libtayo, as well as multiple regulatory achievements for Dupixent, including U.S. approvals for atopic dermatitis among very young patients and for eosinophilic esophagitis in adults and adolescents, as well as European approval for pediatric asthma," said Leonard S. Schleifer, M.D., Ph.D., President and Chief Executive Officer of Regeneron. "In addition, we have continued to strengthen our oncology franchise, including through the purchase of worldwide rights to Libtayo as well as encouraging but preliminary anti-tumor activity observed at higher doses of our novel PSMAxCD28 costimulatory bispecific in combination with Libtayo for advanced metastatic castration-resistant prostate cancer."

Financial Highlights

"We are pleased with our second quarter 2022 financial performance, including 20% revenue growth when excluding contributions from REGEN-COV. This demonstrates the continued strength of our core business," said Robert E. Landry, Executive Vice President, Finance and Chief Financial Officer of Regeneron. "Additionally, we updated our full-year 2022 financial guidance primarily to reflect the recently completed acquisition of Libtayo global rights from Sanofi, a transaction that we believe will deliver significant shareholder value over time. In the second half of 2022, we look forward to advancing our pipeline with important clinical data readouts in oncology and ophthalmology as well as continued commercial execution and prudent capital allocation to drive value creation for shareholders."

Business Highlights

Key Pipeline Progress
Regeneron has approximately 35 product candidates in clinical development, including a number of marketed products for which it is investigating additional indications. Updates from the clinical pipeline include:

Dupixent (dupilumab)

In June 2022, the U.S. Food and Drug Administration (FDA) approved Dupixent as the first biologic medicine for children aged 6 months to 5 years with moderate-to-severe atopic dermatitis.
In May 2022, the FDA approved Dupixent for adults and adolescents aged 12 years and older with eosinophilic esophagitis (EoE).
In April 2022, the European Commission (EC) approved Dupixent for the treatment of severe asthma in children aged 6 to 11 years.
The Company and Sanofi announced positive results from a Phase 3 trial in children aged 1 to 11 years with EoE. The trial met its primary endpoint of histological disease remission at 16 weeks with both higher and lower dose weight-tiered regimens.
The FDA accepted for priority review the supplemental Biologics License Application (sBLA) for Dupixent for adults with prurigo nodularis, with a target action date of September 30, 2022. Regulatory applications have also been submitted in the European Union (EU) and Japan.
EYLEA (aflibercept) Injection

The FDA accepted for review the sBLA for EYLEA for an every-16-weeks dosing regimen in patients with diabetic retinopathy (DR), with a target action date of February 28, 2023.
REGN5678, a PSMAxCD28 costimulatory bispecific antibody

Reported preliminary, first-in-human data in combination with Libtayo in patients with advanced metastatic castration-resistant prostate cancer.
Antibodies to SARS-CoV-2 virus

The Company is continuing to progress investigational "next generation" antibodies that are active against multiple variants including those of Omicron-lineage.
REGN5381, an agonist antibody to NPR1

A Phase 2 study in heart failure was initiated.
Corporate and Business Development Updates

In May 2022, the Company completed its acquisition of Checkmate Pharmaceuticals, Inc. for a total equity value of approximately $250 million. In connection with the acquisition, the Company obtained the rights to vidutolimod (immune activator targeting TLR9), which is in clinical development for oncology.
Effective July 1, 2022, the Company obtained the exclusive right to develop, commercialize, and manufacture Libtayo worldwide under an Amended and Restated Immuno-oncology License and Collaboration Agreement with Sanofi. Under the terms of the agreement, the Company made a $900 million up-front payment, and Sanofi is eligible to receive a $100 million regulatory milestone and up to an aggregate of $100 million in sales-based milestones upon achieving certain amounts of worldwide net product sales of Libtayo through 2023. The Company will also pay Sanofi a royalty on net product sales of Libtayo.
Also effective July 1, 2022, the Company will increase from 10% to 20% the share of its profits that are paid to Sanofi in connection with the development balance reimbursement under the antibody collaboration.
Second Quarter 2022 Financial Results

* Percentage not meaningful

** Effective July 1, 2022, the Company will record global net product sales of Libtayo.

Total revenues decreased by 44% to $2.857 billion in the second quarter of 2022, compared to $5.139 billion in the second quarter of 2021. Total revenues excluding REGEN-COV and Ronapreve(b) revenues for both periods increased by 20% to $2.849 billion in the second quarter of 2022, compared to the second quarter of 2021(a). There have been no sales of REGEN-COV in the United States during 2022 as the Company had completed its final deliveries of drug product under its agreements with the U.S. government as of December 31, 2021.

Sanofi collaboration revenue increased by 55% to $678 million in the second quarter of 2022, compared to the second quarter of 2021. This increase was primarily due to the Company’s share of profits from commercialization of antibodies, which were $497 million in the second quarter of 2022, compared to $328 million in the second quarter of 2021. The change in the Company’s share of profits from commercialization of antibodies was driven by higher Dupixent profits. Roche collaboration revenue decreased in the second quarter of 2022, compared to the second quarter of 2021, due to lower sales of Ronapreve.

* GAAP and non-GAAP amounts are equivalent as no non-GAAP adjustments have been recorded.

** Beginning with the first quarter of 2022, the Company added this new line item to its Statements of Operations, which includes IPR&D acquired in connection with asset acquisitions as well as up-front/opt-in payments related to license and collaboration agreements. Amounts recorded in this line would have historically been recorded to R&D. This change does not affect previously reported non-GAAP results for the three and six months ended June 30, 2021 as the Company recorded no such charges during either of these periods.

GAAP and non-GAAP R&D expenses increased in the second quarter of 2022, compared to the second quarter of 2021, primarily due to higher headcount and headcount-related costs and an increase in clinical manufacturing activities, partly offset by lower costs incurred in connection with REGEN-COV development activities.
Acquired IPR&D in the second quarter of 2022 included a $195 million charge related to the Company’s acquisition of Checkmate Pharmaceuticals.
The increase in GAAP and non-GAAP SG&A expenses in the second quarter of 2022, compared to the second quarter of 2021, was primarily due to higher headcount and headcount-related costs and an increase in commercialization-related expenses for EYLEA, partly offset by costs in 2021 for educational campaigns related to COVID-19 that did not recur in 2022.
GAAP and non-GAAP COGS decreased in the second quarter of 2022, compared to the second quarter of 2021, primarily due to the Company not recognizing any REGEN-COV net product sales in the United States during 2022.
Other Financial Information

GAAP other income (expense) included the recognition of net unrealized losses on equity securities of $164 million in the second quarter of 2022, compared to $409 million of net unrealized gains in the second quarter of 2021.

In the second quarter of 2022, the Company’s GAAP effective tax rate (ETR) was 11.5%, compared to 17.4% in the second quarter of 2021. The decrease in the GAAP ETR was primarily driven by the proportion of income earned in foreign jurisdictions with tax rates lower than the U.S. federal statutory rate, the impact of income earned in the United States during 2021 related to REGEN-COV, and, to a lesser extent, stock-based compensation. In the second quarter of 2022, the non-GAAP ETR was 13.6%, compared to 17.0% in the second quarter of 2021.

GAAP net income per diluted share was $7.47 in the second quarter of 2022, compared to $27.97 in the second quarter of 2021. Non-GAAP net income per diluted share was $9.77 in the second quarter of 2022, compared to $25.80 in the second quarter of 2021. A reconciliation of the Company’s GAAP to non-GAAP results is included in Table 3 of this press release.

During the second quarter of 2022, the Company repurchased shares of common stock under its share repurchase program, and recorded the cost of the shares received, or $394 million, as Treasury Stock. As of June 30, 2022, $2.099 billion remained available for share repurchases under the program.

2022 Financial Guidance(d)

The Company’s full year 2022 financial guidance consists of the following components (inclusive of updates made in connection with the Company’s purchase of Sanofi’s stake in Libtayo and acquisition of Checkmate Pharmaceuticals):

* GAAP and non-GAAP amounts are equivalent as no non-GAAP adjustments have been or are expected to be recorded.

** ETR guidance excludes the impact of the provision requiring capitalization and amortization of R&D expenses enacted as part of the Tax Cuts and Job Act (TCJA), as management’s current expectation is it will be deferred or repealed by Congress in 2022. If this provision of the TCJA is not deferred or repealed, the Company would expect its ETR to be lower than the guidance disclosed herein.

A reconciliation of full year 2022 GAAP to non-GAAP financial guidance is included below:

This press release uses non-GAAP R&D, non-GAAP SG&A, non-GAAP COGS, non-GAAP gross margin on net product sales, non-GAAP other income (expense), net, non-GAAP ETR, non-GAAP net income, non-GAAP net income per share, total revenues excluding REGEN-COV and Ronapreve, and free cash flow, which are financial measures that are not calculated in accordance with U.S. Generally Accepted Accounting Principles (GAAP). These non-GAAP financial measures are computed by excluding certain non-cash and/or other items from the related GAAP financial measure. The Company also includes a non-GAAP adjustment for the estimated income tax effect of reconciling items. A reconciliation of the Company’s GAAP to non-GAAP results is included in Table 3 of this press release.

The Company makes such adjustments for items the Company does not view as useful in evaluating its operating performance. For example, adjustments may be made for items that fluctuate from period to period based on factors that are not within the Company’s control (such as the Company’s stock price on the dates share-based grants are issued or changes in the fair value of the Company’s investments in equity securities) or items that are not associated with normal, recurring operations (such as restructuring- or integration-related expenses). Management uses these non-GAAP measures for planning, budgeting, forecasting, assessing historical performance, and making financial and operational decisions, and also provides forecasts to investors on this basis. With respect to free cash flows, the Company believes that this non-GAAP measure provides a further measure of the Company’s operations’ ability to generate cash flows. Additionally, such non-GAAP measures provide investors with an enhanced understanding of the financial performance of the Company’s core business operations. However, there are limitations in the use of these and other non-GAAP financial measures as they exclude certain expenses that are recurring in nature. Furthermore, the Company’s non-GAAP financial measures may not be comparable with non-GAAP information provided by other companies. Any non-GAAP financial measure presented by Regeneron should be considered supplemental to, and not a substitute for, measures of financial performance prepared in accordance with GAAP.

The casirivimab and imdevimab antibody cocktail is known as REGEN-COV in the United States and Ronapreve in other countries. The Company records net product sales of REGEN-COV in the United States and Roche records net product sales of Ronapreve outside the United States.

The Company’s collaborators provide it with estimates of the collaborators’ respective sales and the Company’s share of the profits or losses (if applicable) from commercialization of products for the most recent fiscal quarter. These estimates are revised, if necessary, in subsequent periods if the Company’s actual share of the profits or losses differ from those estimates.

The Company’s 2022 financial guidance does not assume the completion of any significant business development transactions not completed as of the date of this press release.

Gross margin on net product sales represents gross profit expressed as a percentage of total net product sales recorded by the Company. Gross profit is calculated as net product sales less cost of goods sold.

Corresponding reimbursements from collaborators and others for manufacturing of commercial supplies is recorded within revenues.

Conference Call Information

Regeneron will host a conference call and simultaneous webcast to discuss its second quarter 2022 financial and operating results on Wednesday, August 3, 2022, at 8:30 AM Eastern Time. Participants may access the conference call live via webcast, or register in advance and participate via telephone, on the "Investors and Media" page of Regeneron’s website at View Source Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call, including the dial-in number along with a unique passcode and registrant ID that can be used to access the call. A replay of the conference call and webcast will be archived on the Company’s website for at least 30 days.

On August 3, 2022 Propanc Biopharma, Inc. (OTCQB: PPCB) ("Propanc" or the "Company"), a biopharmaceutical company developing novel cancer treatments for patients suffering from recurring and metastatic cancer, reported that a Joint Research Collaboration Agreement has been established with the Universities of Jaén and Granada, Spain (Press release, Propanc, AUG 3, 2022, View Source [SID1234617390]). Since late 2020, Mrs. Belén Toledo Cutillas MSc, has been investigating an important experimental thesis on the effects of proenzyme therapy and the impact on the tumor microenvironment, which is key to the development, invasion, metastatic spread and recurrence of solid tumors. The work is being conducted at the laboratory of Professor Macarena Perán PhD, who is the lead researcher on the project and is the second Joint Research Collaboration Agreement currently in progress with the two Spanish Universities.

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To date, encouraging results demonstrates that proenzymes have specific effects on tumor cells and CSCs, but also other tumor elements in the tumor microenvironment. However, the most significant conclusion is that proenzymes cause a reversal of the malignant phenotype (observable characteristics) towards a normal, or benign state. This process of reversing the tumor phenotype, called differentiation, is how proenzymes exert anti-tumor, anti-cancer and anti-metastatic effects. Therefore, proenzyme treatment may be considered differentiation therapy, which exerts these effects on malignant cells, but leaves healthy cells alone. To achieve these results, Mrs. Cutillas used integrated 3-dimensional bio-impressions of tumor cells from patients with advanced solid tumors, developed at the Centre for Biomedical Research, University of Granada, led by Professor Juan Marchal MD.

Along with lead researcher, Professor Perán, and Mrs. Cutillas, additional research team members from the University of Jaén are assisting on the project, as well as Professor Marchal and Dr Maria Angel Garcia, from the University of Granada. Propanc will own any intellectual property developed during the research, and publishing rights, in exchange for any net future royalties from intellectual property rights.

Dr Julian Kenyon, Md, MB, ChB, Propanc’s Chief Scientific Officer said, "Evaluating the effects of proenzyme therapy in the tumor microenvironment is of scientific interest, as it demonstrates whether proenzymes penetrate into this target area and exert its effects. At the same time, it confirms the selectivity of the drug on solid tumors, by targeting cancer cells and leaving healthy cells alone. The clinical implications are also potentially significant, which may result in novel discoveries and lead to exciting new ways to treat cancer patients suffering from solid tumors without the toxicity normally associated with standard treatment regimens."

"Continuing to expand our research efforts in the use of proenzymes and cancer leading to the identification of further discoveries can provide additional patentable subject matter and expand our intellectual property portfolio, which covers the invention relating to our lead product candidate, PRP. I look forward to working closely with Professor Perán and her team on this exciting project," said Mr. James Nathanielsz, Propanc’s Chief Executive Officer.

PRP is a mixture of two proenzymes, trypsinogen and chymotrypsinogen from bovine pancreas administered by intravenous injection. A synergistic ratio of 1:6 inhibits growth of most tumor cells. Examples include kidney, ovarian, breast, brain, prostate, colorectal, lung, liver, uterine and skin cancers.

On August 3, 2022 Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage biopharmaceutical company utilizing proprietary genetic engineering platform technologies to create cell and gene therapeutics with the capacity to cure, reported it has entered into a broad strategic collaboration and license agreement with Roche, focused on developing allogeneic CAR-T therapies directed to hematologic malignancies (Press release, Poseida Therapeutics, AUG 3, 2022, View Source [SID1234617389]). The global collaboration covers the research and development of multiple existing and novel "off-the-shelf" cell therapies against targets in multiple myeloma, B-cell lymphomas and other hematologic indications.

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"We are excited to partner and collaborate with Roche, one of the world’s largest biotechnology companies, which has a successful track record in the discovery, development and commercialization of innovative medicines," said Mark Gergen, Chief Executive Officer of Poseida. "Roche is an ideal strategic partner for Poseida with its industry-leading R&D capabilities in oncology, complementary technologies and expertise, and global regulatory and commercial capabilities. Working together, we look forward to advancing novel allogeneic cell therapies based upon Poseida’s technologies for patients battling cancer."

Under the agreement, Roche will receive from Poseida either exclusive rights or options to develop and commercialize a number of allogeneic CAR-T programs in Poseida’s portfolio that are directed to hematologic malignancies, including P-BCMA-ALLO1, an allogeneic CAR-T for the treatment of multiple myeloma and for which a Phase 1 study is underway, and P-CD19CD20-ALLO1, an allogeneic dual CAR-T for the treatment of B-cell malignancies with an IND expected in 2023. Building on complementary expertise and capabilities, the parties will also collaborate in a research program to create and develop next-generation features and improvements for allogeneic CAR-T therapies, from which they would jointly develop additional allogeneic CAR-T product candidates directed to existing and new hematologic targets. For a subset of both the Poseida portfolio programs licensed or optioned to Roche and the parties’ future collaboration programs, Poseida will conduct the Phase 1 studies and manufacture clinical materials before transitioning the programs to Roche for further development and commercialization. Roche will be solely responsible for the late-stage clinical development and global commercialization of all products that are subject to the collaboration.

"We are excited to partner with Poseida to further explore the potential of allogeneic cell therapies to transform cancer care by developing off-the-shelf products that can address high unmet medical needs for a broad patient population," said James Sabry, Global Head of Pharma Partnering at Roche. "Poseida’s differentiated platform technologies complement our ongoing internal efforts and partnerships to discover and develop cell therapies as a next generation of medicines for patients."

Under the agreement, Poseida will receive $110 million upfront and could receive up to $110 million in near-term milestones and other payments in the next several years. In addition, Poseida is eligible to receive research, development, launch, and net sales milestones and other payments potentially up to $6 billion in aggregate value, as well as tiered net sales royalties into the low double digits, across multiple programs.

"We are thrilled that Roche has embraced the opportunity to partner with us and use Poseida’s unique allogeneic approach to develop CAR-T product candidates," said Devon J. Shedlock, Ph.D., Chief Scientific Officer, Cell Therapy at Poseida. "Using our proprietary technologies and manufacturing process including our booster molecule, we have the potential to develop and manufacture a product with high levels of stem cell memory T cells, which are correlated with potent antitumor efficacy in the clinic, at a scale that can potentially reach more patients and enable broad commercial use."

The effectiveness of the agreement is subject to clearance under the Hart-Scott-Rodino Antitrust Improvements Act (HSR Act).

Poseida Therapeutics Conference Call and Webcast Information

Wednesday, August 3, 2022 at 8:30 a.m. ET

Poseida’s management team will host a conference call and webcast today at 8:30 a.m. ET to discuss the collaboration and Poseida’s novel approach to allogeneic cell therapy. The dial-in numbers for domestic and international callers are 800-267-6316 and 203-518-9814, respectively. The conference ID number for the call is PSTX0803.

Participants may access the live webcast on the Investors & Media Section of the Poseida website, www.poseida.com. An archived replay of the webcast will be available for approximately 30 days following the event.

On August 3, 2022 Personalis, Inc. (Nasdaq: PSNL), a leader in advanced genomics for precision oncology, reported financial results for the second quarter ended June 30, 2022 (Press release, Personalis, AUG 3, 2022, View Source [SID1234617388]).

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Second Quarter and Recent Highlights

Reported quarterly revenue of $18.2 million in the second quarter of 2022 compared with $21.7 million in the second quarter of 2021
Revenue from biopharma and other customers, excluding the VA MVP (as defined below), of $14.2 million in the second quarter of 2022 compared with $8.2 million in the second quarter of 2021, a 74% increase; revenue from biopharma and other customers includes revenue of $6.8 million from Natera in the second quarter of 2022; revenue from biopharma customers excluding Natera of $7.4 million for the second quarter of 2022
Revenue from the U.S. Department of Veterans Affairs Million Veteran Program (VA MVP) of $4.0 million in the second quarter of 2022 compared with $13.5 million in the second quarter of 2021
Added another patent family to its molecular residual disease (MRD)-related IP portfolio, with priority to January 2013; the new patent describes the detection of MRD and recurrence by using whole genome sequencing of a patient’s tumor to identify variants for a personalized liquid biopsy assay
Appointed Lonnie Shoff to the Personalis Board of Directors effective August 2, 2022; Ms. Shoff brings extensive clinical diagnostics experience including executive roles at Thermo Fisher and Roche Diagnostics
Cash, cash equivalents, and short-term investments were $233.5 million as of June 30, 2022
"We are pleased with the pace of our oncology revenue growth compared with last year, as well as the continued adoption of our NeXT Platform, which is driven by the differentiation and strength of our offerings," said John West, Chief Executive Officer of Personalis. "We are making solid progress on our strategic priorities, with the goal of becoming the leading provider of the most comprehensive and actionable cancer genomic tests. Ultimately, we aim to enable a new standard of oncology care to help patients live better and longer lives."

Second Quarter 2022 Financial Results

Revenue was $18.2 million in the three months ended June 30, 2022
Gross margin was 23.5% in the three months ended June 30, 2022
Operating expenses were $32.2 million in the three months ended June 30, 2022
Net loss was $27.5 million in the three months ended June 30, 2022 and net loss per share was $0.60 based on a weighted-average basic and diluted share count of 45.6 million
Cash, cash equivalents, and short-term investments were $233.5 million as of June 30, 2022
Full Year 2022 Outlook

Personalis expects the following for the full year of 2022:

Total company revenue is expected to be in the range of $62.0 million to $67.0 million
Revenue from biopharma and all other customers, excluding the VA MVP, is expected to be in the range of $55.0 million to $60.0 million
Net loss is expected to be in the range of $110.0 million to $115.0 million
Webcast and Conference Call Information

Personalis will host a conference call to discuss the second quarter 2022 financial results after market close on Wednesday, August 3, 2022 at 2:00 p.m. Pacific Time / 5:00 p.m. Eastern Time. To access the live call via telephone, please register in advance using the link here. Upon registering, each participant will receive an email confirmation with dial-in numbers and a unique personal PIN that can be used to join the call. The live webinar can be accessed at View Source A replay of the webinar will be available shortly after the conclusion of the call and will be archived on the company’s website.

On August 3, 2022 Cassava Sciences, Inc. (Nasdaq: SAVA), a clinical-stage biotechnology company focused on Alzheimer’s disease, reported financial results for the second quarter ended June 30, 2022, a mid-year corporate update, and interim clinical results of an open-label study with simufilam (Press release, Pain Therapeutics, AUG 3, 2022, View Source [SID1234617387]). Simufilam is Cassava Sciences’ lead drug candidate for the proposed treatment of Alzheimer’s disease. SavaDx is Cassava Sciences’ investigational diagnostic candidate to detect Alzheimer’s disease from a simple blood draw.

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"I’m pleased with our operating performance in the first half of 2022," said Remi Barbier, President & CEO. "I’m also humbled by the hard work of our team members and clinical partners. Persistence and a focus on performance are key when you’re trying to beat Alzheimer’s disease."

Update on Patient Enrollment for Phase 3 Program
A total of over 400 patients are now enrolled in our on-going Phase 3 program of simufilam in Alzheimer’s disease. Enrollment is almost evenly split between the two Phase 3 studies.

Overview of Phase 3 Program with Simufilam in Alzheimer’s Disease
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. Both studies have the same co-primary efficacy endpoints: ADAS-Cog12 (a cognitive scale) and ADCS-ADL (a functional scale). A secondary efficacy endpoint for both studies is iADRS, a clinical tool that combines cognitive and functional scores from ADAS-Cog & ADCS-ADL. Patients are now being screened in clinical trial sites in the U.S., Canada, Puerto Rico and Australia.

RETHINK-ALZ is the trade name of our 52-week Phase 3 study. This randomized, double-blind, placebo-controlled study is designed to evaluate the safety & efficacy of oral simufilam 100 mg or placebo, twice daily, over 52 weeks in approximately 750 patients with Alzheimer’s disease.

REFOCUS-ALZ is the trade name of our 76-week Phase 3 study. This randomized, double-blind, placebo-controlled study is designed to evaluate the safety & efficacy of oral simufilam 100 mg, 50 mg or placebo, twice daily, over 76 weeks in approximately 1,000 patients with Alzheimer’s.

Future Open-label Extension Study for the Phase 3 Program
In the second half of 2022, we expect to initiate an open-label extension study for our Phase 3 program. This new study is designed to provide no-cost access to simufilam to patients with Alzheimer’s disease who have successfully completed a Phase 3 study of simufilam.

On-going Open-label Study with Simufilam in Alzheimer’s Disease
In March 2020, we initiated a long-term, open-label study to evaluate simufilam 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 over 12 months or more. This study has reached its target enrollment of approximately 200 patients. We expect all patients for this study will complete drug treatment in Q4 2022. We expect to announce top-line clinical results for this study approximately year-end 2022.

On-going Open-label Study – Interim Analysis on 100 Subjects at 12 Months
An interim analysis was conducted on the first 100 evaluable patients who completed at least 12 months of open-label treatment with simufilam 100 mg twice daily. Top-line results of this interim analysis show that from baseline to month-12:

Drug appears safe and well tolerated.
Overall ADAS-Cog11 scores improved an average of 1.5 points (S.D. ± 6.6; P<0.05)
63% of the 100 patients showed an improvement in ADAS-Cog11 scores, and this group of patients improved an average of 5.6 points (S.D. ± 3.8).
An additional 21% of the 100 patients declined less than 5 points on ADAS-Cog11, and this group of patients declined an average of 2.7 points (S.D. ± 1.4).
The 11-item Alzheimer’s Disease Assessment Scale–Cognitive subscale (ADAS-Cog) was originally developed by the research community to measure cognitive impairment in patients with Alzheimer’s disease. ADAS-Cog is often used in clinical studies of patients with Alzheimer’s because it can help determine incremental improvements or declines in cognition over time.

Standard deviation ("S.D.") is a measure of how dispersed the data is in relation to the average. A low standard deviation generally shows the data are closely clustered around the average. A high standard deviation generally shows that the data is widely spread.

All clinical data from our open-label study are inherently exploratory in nature and, as with all open-label data, should be interpreted with caution. Data results from our open-label study does not constitute, and should not be interpreted as, evidence of therapeutic benefit for simufilam.

Cognition Maintenance Study (CMS) with Simufilam in Alzheimer’s Disease
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. 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. To enroll in the CMS, patients must have previously completed 12 months or more of open-label treatment with simufilam.

The target enrollment for the CMS is 100 or more patients. Over 50 patients have now completed this study. Our goal is to complete patient enrollment for the CMS in Q4 2022. We expect to announce clinical results of the CMS approximately third-quarter 2023.

SavaDx
Our investigational product candidate, called SavaDx, is an early-stage program focused on detecting the presence of Alzheimer’s disease from a simple blood draw. SavaDx is currently designed as an antibody-based detection system for altered filamin A (FLNA) protein. Working with third parties, we continue to evaluate an innovative method to detect FLNA without the use of antibodies. For business, technical and personnel reasons, we continue to prioritize the development of simufilam, our novel drug candidate, over SavaDx.

Financial Update
Net loss for the second quarter 2022 was $19.3 million, or $0.48 per share, compared to a net loss of $5.1 million, or $0.13 per share, for the same period in 2021. Net cash used in operations was $34.6 million during the first six months of 2022. Net cash use for operations for full-year 2022 is expected to be approximately $80 to $90 million. Cash and cash equivalents were $197.2 million as of June 30, 2022, with no debt.

Financial Results for Second Quarter 2022

At June 30, 2022, cash and cash equivalents were $197.2 million, with no debt.
Net loss was $19.3 million, or $0.48 per share. This compares to a net loss of $5.1 million, or $0.13 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 for a Phase 3 program of simufilam in Alzheimer’s disease.
Net cash used in operations was $34.6 million during the first six months of 2022.
Net cash use in operations for full year 2022 is expected to be approximately $80 to $90 million.
Research and development (R&D) expenses were $16.9 million. This compared to $3.9 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 and pre-clinical studies and support functions and personnel expenses.
General and administrative (G&A) expenses were $3.0 million. This compared to $1.2 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 as well as depreciation and amortization.
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.