On August 3, 2021 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, 2021 (Press release, Pain Therapeutics, AUG 3, 2021, View Source [SID1234585595]). Net loss for the second quarter ended June 30, 2021, was $5.1 million, or $0.13 per share, compared to a net loss of $1.1 million, or $0.05 per share, for the same period in 2020. Net cash used in operations was $7.4 million during the first six months of 2021. Net cash use for operations for full-year 2021 is expected to be approximately $20 to $25 million, consistent with previous financial guidance. Cash and cash equivalents were $278.3 million as of June 30, 2021, with no debt.

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Remi Barbier, President & CEO, and Eric Schoen, Chief Financial Officer, will host a conference call to review financial results and to preview the Company’s growth strategy.

The conference call is scheduled to begin at 9:00 am ET on Tuesday, August 3, 2021. Please dial in 15 minutes in advance to ensure a timely connection to the call.

Financial Highlights for Second Quarter 2021

At June 30, 2021, cash and cash equivalents were $278.3 million, compared to $93.5 million at December 31, 2020, with no debt.

Net cash used in operations during the six months ended June 30, 2021 was $7.4 million, net of reimbursements received from the National Institutes of Health (NIH) grant awards.

Research grant funding reimbursements of $0.9 million were received from NIH and recorded as a reduction in research and development (R&D) expenses. This compared to $1.1 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. 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 $3.9 million compared to $0.6 million for the same period in 2020. 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, costs of an on-going open-label study in simufilam, as well as increased personnel expenses.

General and administrative (G&A) expenses were $1.2 million compared to $0.8 million for the same period in 2020. This increase was due primarily to higher annual shareholder meeting and insurance costs 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. 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 August 3, 2021 Zymeworks Inc. (NYSE: ZYME), a clinical-stage biopharmaceutical company developing multifunctional biotherapeutics, reported that Janssen Biotech, Inc. (Janssen) dosed the first patient with JNJ-78278343, a bispecific antibody developed using Zymeworks’ Azymetric and EFECT therapeutic platforms (Press release, Zymeworks, AUG 3, 2021, View Source [SID1234585594]).

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"On behalf of everyone at Zymeworks, I’d like to congratulate the team at Janssen for advancing this bispecific candidate into the clinic," said Ali Tehrani Ph.D., Zymeworks’ President & CEO. "This represents our fourth pharmaceutical collaboration with programs reaching clinical development. We look to continue to demonstrate the value and strength of our platform technologies, which are an important part of our business model, complementing the value derived from our two lead clinical assets and preclinical pipeline."

Zymeworks will receive a payment in connection with this milestone under Zymeworks’ 2017 licensing agreement with Janssen. Under the terms of that agreement, Zymeworks provided Janssen with a worldwide, royalty-bearing license to research, develop and commercialize up to six bispecific antibodies directed to Janssen therapeutic targets using Zymeworks’ Azymetric and EFECT platforms. Janssen is responsible for all research, development and commercial activities under the licensing agreement. Zymeworks received an upfront payment of US$50 million and is eligible to potentially receive up to US$282 million in development and up to US$1.12 billion in commercial milestone payments, as well as tiered royalties on potential sales. Janssen also has the option to develop two additional bispecific programs under the agreement subject to a future option payment.

About the Azymetric Platform

The Azymetric platform enables the transformation of monospecific antibodies into bispecific and multispecific antibodies, allowing simultaneous binding to several different disease targets. This unique technology enables the development of multifunctional therapeutics that can block multiple signaling pathways, recruit immune cells to tumors, enhance receptor clustering and internalization and increase tumor-specific targeting. These features are designed to enhance efficacy while reducing toxicities and the potential for drug resistance. Azymetric therapeutics have been engineered to retain the desirable drug-like qualities of naturally occurring antibodies, including low immunogenicity, long half-life and high stability. In addition, they are compatible with standard manufacturing processes that deliver high yields and purity, potentially reducing drug development costs and timelines.

About the EFECT Platform

The EFECT platform is a library of antibody Fc modifications engineered to activate or suppress the antibody-mediated immune response. This platform, which is compatible with traditional monoclonal as well as Azymetric bispecific antibodies, further enables the customization and optimization of therapeutic responses for different diseases.

On August 3, 2021 Kiniksa Pharmaceuticals, Ltd. (Nasdaq: KNSA) ("Kiniksa"), a biopharmaceutical company with a portfolio of assets designed to modulate immunological pathways across a spectrum of diseases, reported second quarter 2021 financial results and recent corporate and portfolio activity (Press release, Kiniksa Pharmaceuticals, AUG 3, 2021, View Source [SID1234585593]).

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"Our commercial team is doing a superb job with the launch of ARCALYST in recurrent pericarditis," said Sanj K. Patel, Chief Executive Officer and Chairman of the Board of Kiniksa. "Feedback received from physicians and patients highlights the transformational potential of ARCALYST for patients suffering from recurrent pericarditis. We have seen strong uptake and look forward to using this momentum to accelerate broader adoption across this underserved population."

"We are executing on the development of our clinical-stage pipeline," said John F. Paolini, MD, PhD, Chief Medical Officer of Kiniksa. "The potential broad utility of mavrilimumab across multiple indications is increasingly promising, and we expect data from our Phase 3 clinical trial in COVID-19-related ARDS in the first quarter of 2022. We are enrolling a Phase 2b clinical trial of vixarelimab in prurigo nodularis and believe vixarelimab has the potential to make a meaningful impact on these patients’ lives by addressing both the pruritus and the skin nodules associated with this devastating disease. KPL-404, our anti-CD40 program, has potential across a range of autoimmune diseases, and we plan to initiate a Phase 2 proof-of-concept clinical trial in rheumatoid arthritis in the fourth quarter of this year."

Portfolio Activity
ARCALYST (IL-1α and IL-1β cytokine trap)

ARCALYST net revenue in the second quarter of 2021 was $7.7 million.
The second quarter of 2021 represented Kiniksa’s first quarter of ARCALYST sales. ARCALYST became commercially available through Kiniksa on April 1st, 2021.
Greater than 100 physicians who did not participate in Phase 3 RHAPSODY prescribed ARCALYST to at least one recurrent pericarditis patient.
Greater than 65% of all recurrent pericarditis enrollments came from physicians practicing outside of the 12 Phase 3 RHAPSODY clinical trial sites in the U.S.
Greater than 90% of completed new patient enrollment cases were approved for coverage under the payer medical exceptions process.
Kiniksa continues to expect that the majority of payers will establish coverage policies within six months and that almost all payers will update their coverage policies within a year from launch.
Kiniksa OneConnectTM provided access, initiation and ongoing support for existing ARCALYST cryopyrin-associated periodic syndromes (CAPS) and deficiency of IL-1 receptor antagonist (DIRA) patients transitioning to Kiniksa product as well as new ARCALYST patients for all approved indications.
Mavrilimumab (monoclonal antibody inhibitor targeting GM-CSFRα)

Kiniksa’s interactions with the U.S. Food and Drug Administration (FDA) resulted in defined paths for Phase 3 development of mavrilimumab in COVID-19-related acute respiratory distress syndrome (ARDS) and in giant cell arteritis (GCA).
Kiniksa reported additional data from the Phase 2 portion of the Phase 2/3 clinical trial of mavrilimumab in patients with severe COVID-19-related ARDS.
Follow-up overall survival data from the cohort of non-mechanically ventilated patients through Day 90 demonstrated persistent clinical effect, confirming and extending the previously-reported Day 29 data.
Day 29 data from the cohort of mechanically-ventilated patients did not show a reduction in death.
Kiniksa continues to enroll non-mechanically ventilated patients in the Phase 3 clinical trial of mavrilimumab in COVID-19-related ARDS.
Kiniksa discontinued enrolling mechanically-ventilated patients in the Phase 3 trial.
Kiniksa expects data from the Phase 3 trial in non-mechanically-ventilated patients in the first quarter of 2022.
Vixarelimab (monoclonal antibody inhibitor of signaling through OSMRβ)

Kiniksa continues to enroll patients in a placebo-controlled Phase 2b clinical trial of vixarelimab in prurigo nodularis, evaluating a range of once-monthly dose regimens via subcutaneous (SC) injection.
The primary efficacy endpoint is the percent change from baseline in the weekly-average Worst-Itch Numeric Rating Scale at Week 16.
KPL-404 (monoclonal antibody inhibitor of the CD40 and CD154 interaction)

Kiniksa plans to initiate a placebo-controlled Phase 2 proof-of-concept clinical trial of KPL-404 in rheumatoid arthritis in the fourth quarter of 2021.
The planned trial is designed to provide safety data, pharmacokinetic characterization, and efficacy of chronic SC dosing over 12 weeks.
Rheumatoid arthritis was selected for demonstration of KPL-404 proof of concept as it is a well-characterized autoimmune disease with decades of published clinical data across diverse mechanistic classes, allowing for objective evaluation in established endpoints.
The pharmacokinetic lead-in of the planned trial supports characterization of chronic administration of KPL-404 in a patient population and provides optionality to evaluate the therapeutic potential of KPL-404 across a range of other autoimmune diseases with pathologies believed to be mediated by the CD40-CD154 pathway.
Upcoming Scientific Conference Presentations

Kiniksa plans to present additional data from RHAPSODY, the pivotal Phase 3 trial of rilonacept, as well as the study design of the RESONANCE Registry, at the European Society of Cardiology virtual congress, which will be held August 27, 2021 through August 30, 2021. Details of the poster presentations are as follows:
Antonio Brucato, MD, Department of Biomedical and Clinical Science, University of Milan, Fatebenefratelli Hospital, Milan, will present a poster entitled, Health-Related Quality of Life in Patients with Recurrent Pericarditis: Results from RHAPSODY, a Phase 3 Study of Rilonacept.
Alison Reid, PhD, Kiniksa Pharmaceuticals Corp., will present a poster entitled, RESONANCE Registry: Rationale and Design of the Retrospective and Prospective Longitudinal, Observational Registry in Pediatric and Adult Patients with Recurrent Pericarditis.
Financial Results

Total revenue in the second quarter of 2021 was $7.7 million. Second quarter 2021 revenue is related to product sales for ARCALYST, which became commercially available through Kiniksa on April 1, 2021. Kiniksa did not generate product revenue in the second quarter of 2020.
Net loss for the second quarter of 2021 was $41.6 million, compared to a net loss of $37.5 million for the second quarter of 2020.
Total operating expenses for the second quarter of 2021 were $48.3 million, compared to $31.9 million for the second quarter of 2020.
Non-cash, share-based compensation expense for the second quarter of 2021 was $5.7 million, compared to $4.9 million for the second quarter of 2020.
As of June 30, 2021, the company had cash, cash equivalents and short-term investments of $225.9 million and no debt.
Financial Guidance

Kiniksa expects ARCALYST net revenue for the third quarter of 2021 to be between $9.0 million and $10.0 million.
Kiniksa expects that its cash, cash equivalents and short-term investments will fund its current operating plan into 2023.
Conference Call Information
Kiniksa will host a conference call and webcast at 8:30 a.m. Eastern Time on Tuesday, August 3, 2021 to discuss second quarter 2021 financial results and recent corporate and portfolio activity. Individuals interested in participating in the call should dial (866) 614-0636 (U.S. and Canada) or (409) 231-2053 (international) using conference ID number 1485623. To access the webcast, please visit the Investors and Media section of Kiniksa’s website at www.kiniksa.com. The archived webcast will be available on Kiniksa’s website for 14 days beginning approximately one hour after the call has completed.

On August 3, 2021 TRACON Pharmaceuticals (NASDAQ: TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted cancer therapeutics and utilizing a cost efficient, CRO-independent product development platform to partner with ex-U.S. companies to develop and commercialize innovative products in the U.S., reported that Charles Theuer, M.D., Ph.D., President and Chief Executive Officer, will present a corporate overview at the BTIG Virtual Biotechnology Conference on August 10, 2021 at 12:00pm Eastern Time (Press release, Tracon Pharmaceuticals, AUG 3, 2021, View Source [SID1234585592]).

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To access a live webcast of the presentation, please visit the "Events and Presentations" page within the "Investors" section of the TRACON Pharmaceuticals website at www.traconpharma.com.

On August 3, 2021 Pacira BioSciences, Inc. (Nasdaq: PCRX), the industry leader in its commitment to non-opioid pain management and regenerative health solutions, reported financial results for the second quarter of 2021 (Press release, Pacira Pharmaceuticals, AUG 3, 2021, View Source;991.htm [SID1234585591]).

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Second Quarter 2021 Financial Highlights

Total revenues of $135.6 million
GAAP net income of $19.1 million, or $0.43 per share (basic) and $0.42 (diluted)
Non-GAAP Adjusted EBITDA of $50.3 million
"We made terrific progress in the first half of 2021 with strong sales momentum in the second quarter and continuing in recent weeks," said Dave Stack, chairman and chief executive officer of Pacira BioSciences. "Record revenue in the second quarter significantly ramped our bottom line, allowing us to achieve our highest-ever adjusted EBITDA for a quarter. Market dynamics remain highly favorable and leave us confident that we will deliver robust topline growth, improved margins, and accelerating earnings for the remainder of 2021, leaving us well-positioned to achieve the goals laid out within our five-year plan."

Second Quarter 2021 Financial Results

Total revenues were $135.6 million in the second quarter of 2021, versus the $75.5 million reported for the second quarter of 2020.
EXPAREL net product sales were $130.1 million in the second quarter of 2021, versus the $73.0 million reported for the second quarter of 2020.
Second quarter 2021 iovera° net product sales were $3.8 million, versus the $1.4 million reported for the second quarter of 2020.
Sales of bupivacaine liposome injectable suspension to a third-party licensee for use in veterinary practice were $1.0 million in the second quarter of 2021, versus the $0.8 million in the second quarter of 2020.
Second quarter 2021 royalty and collaborative licensing and milestone revenues were $0.7 million, versus the $0.3 million in the second quarter of 2020.
Total operating expenses were $100.7 million in the second quarter of 2021, compared to $82.7 million in the second quarter of 2020.
Research and development (R&D) expenses were $12.6 million in the second quarter of 2021, compared to $13.6 million in the second quarter of 2020. R&D expenses included $4.6 million and $6.1 million of product development and manufacturing capacity expansion costs in the second quarters of 2021 and 2020, respectively.
Selling, general and administrative (SG&A) expenses were $50.8 million in the second quarter of 2021, compared to $43.3 million in the second quarter of 2020.
GAAP net income was $19.1 million, or $0.43 per share (basic) and $0.42 (diluted), in the second quarter of 2021, compared to a GAAP net loss of $7.3 million, or $0.17 per share (basic and diluted), in the second quarter of 2020.
Non-GAAP net income was $35.3 million, or $0.80 per share (basic) and $0.77 (diluted), in the second quarter of 2021, compared to non-GAAP net income of $5.0 million, or $0.12 per share (basic and diluted), in the second quarter of 2020.
Adjusted EBITDA was $50.3 million in the second quarter of 2021, compared to $8.5 million in the second quarter of 2020.
Pacira ended the second quarter of 2021 with cash, cash equivalents and short-term investments ("cash") of $646.6 million. Cash provided by operations was $30.1 million in the second quarter of 2021, compared to cash used in operations of $15.6 million in the second quarter of 2020.
Pacira had 44.1 million basic and 45.6 million diluted weighted average shares of common stock outstanding in the second quarter of 2021.
See "Non-GAAP Financial Information" below.

Recent Business Highlights

FDA approval of enhanced EXPAREL manufacturing process. In July 2021, the U.S. Food and Drug Administration (FDA) approved the company’s enhanced manufacturing process for EXPAREL, which is housed at a custom facility in Swindon, England under a partnership with Thermo Fisher Scientific Pharma Services. The company expects to start selling commercial product manufactured in this 200-liter suite later this year.

Patent granted for EXPAREL. In June 2021, the United States Patent and Trademark Office issued U.S. Patent No. 11,033,495 related to EXPAREL. The patent, "Manufacturing of Bupivacaine Multivesicular Liposomes," claims composition of EXPAREL prepared by an improved manufacturing process and will have an expiration date of January 22, 2041. Pacira submitted this patent for listing in the FDA’s "Approved Drug Products with Therapeutic Equivalence Evaluations" (the Orange Book) in July 2021.

Distribution agreement with Eurofarma in Latin America. In June 2021, Pacira announced a distribution agreement with Eurofarma Laboratories S.A. (Eurofarma) for the development and commercialization of EXPAREL in Latin America. Under the terms of the agreement, Eurofarma obtained the exclusive right to market and distribute EXPAREL in 19 countries including Argentina, Brazil, Colombia, and Mexico.
Financial Guidance

The company’s net product sales were negatively impacted by the COVID-19 pandemic in 2020 due to the significant postponement or suspension in the scheduling of elective surgical procedures resulting from public health guidance and government directives. Elective surgery restrictions began to lift on a state-by-state basis in April 2020, allowing EXPAREL sales to return to year-over-year growth in June 2020. However, while many restrictions have since eased and COVID-19 vaccines become more widely available and administered to the general public, it is still unclear how long it will take the elective surgery market to normalize, or if restrictions on elective procedures will recur due to COVID-19 variant strains or otherwise. In order to provide greater transparency, the company is reporting monthly intra-quarter unaudited net product sales until it has gained enough visibility around the impacts of COVID-19. The company reports average daily growth rates for EXPAREL to normalize for differences in the number of selling days per reporting period. The company is also providing weekly EXPAREL utilization and elective surgery data within its investor presentation, which is accessible at investor.pacira.com.

Today’s Conference Call and Webcast Reminder

The Pacira management team will host a conference call to discuss the company’s financial results and recent developments today, Tuesday, August 3, 2021, at 8:30 a.m. ET. To participate in the conference call, dial 1-877-845-0779 and provide the passcode 9991311. International callers may dial 1-720-545-0035 and use the same passcode. In addition, a live audio of the conference call will be available as a webcast. Interested parties can access the event through the "Events" page on the Pacira website at investor.pacira.com.

For those unable to participate in the live call, a replay will be available at 1-855-859-2056 (domestic) or 1-404-537-3406 (international) using the passcode 9991311. The replay of the call will be available for one week from the date of the live call. The webcast will be available on the Pacira website for approximately two weeks following the call.