On November 3, 2022 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 third quarter of 2022 (Press release, Pacira Pharmaceuticals, NOV 3, 2022, View Source;991.htm [SID1234623016]).

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Third Quarter 2022 Financial Highlights

Total revenues of $167.5 million
Net product sales of $132.6 million for EXPAREL, $26.5 million for ZILRETTA, and $4.5 million for iovera°
Net loss of $0.7 million, or $(0.02) per share (basic and diluted)
Adjusted EBITDA of $55.2 million
"In the third quarter, we continued to post increasing revenue and adjusted EBITDA, which underscores the success we are achieving in bringing our innovative non-opioid pain management solutions to patients," said Dave Stack, chairman and chief executive officer of Pacira BioSciences. "Moving forward, with an EXPAREL exclusivity runway extending into 2041 driving significant and durable operating cash flows, we are well-positioned to deliver near- and long-term value creation through a blend of new indications within our current commercial portfolio as well as new product development opportunities to support our growth in 2023 and beyond."

Recent Business Highlights

New EXPAREL Patent and Notice of Allowance. In October, the U.S. Patent and Trademark Office (USPTO) issued Patent Number 11,452,691. This patent is a chemical composition patent, with an expiration date of January 22, 2041. This patent is now listed in the U.S. Food and Drug Administration’s Approved Drug Products with Therapeutic Equivalents Evaluations (Orange Book) and is the 8th listed patent. The company also received a Notice of Allowance from the USPTO for a U.S. Patent Application that is a product by process patent for EXPAREL. After issuance, Pacira will submit this patent for listing in the Orange Book.

Positive Topline Data from Two Phase 3 Registration Studies of EXPAREL as a Lower Extremity Nerve Block. In September, the company announced positive topline results from two Phase 3 registration studies of EXPAREL as a single-dose nerve block for postsurgical regional analgesia in lower extremity surgeries. The first study evaluated EXPAREL as a femoral nerve block in the adductor canal for total knee arthroplasty and the second study evaluated EXPAREL as a sciatic nerve block in the popliteal fossa for bunionectomy. Both studies achieved statistical significance for the primary endpoint demonstrating a statistically significant reduction in cumulative pain scores from 0 to 96 hours (p<0.01) and the key secondary endpoint reduction in postsurgical opioid consumption through 96 hours (p<0.01) compared with bupivacaine HCl. In the bunionectomy study, EXPAREL also achieved statistical significance for the percentage of opioid-free subjects (p<0.001) through 96 hours compared with bupivacaine HCl. EXPAREL was well tolerated with a safety profile consistent with bupivacaine HCl.

Positive CHMP Opinion for EXPAREL for the Treatment of Postsurgical Pain in Children Aged 6 or Older. In September, the company announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending marketing authorization for an expanded indication of EXPAREL to include use in children aged 6 years and older as a field block for treatment of somatic post-operative pain from small- to medium-sized surgical wounds. The CHMP opinion was based on the results of the Phase 3 PLAY study of EXPAREL infiltration in pediatric patients undergoing spinal or cardiac surgeries. Overall findings were consistent with the pharmacokinetic and safety profiles for adult patients with no safety concerns identified at a dose of 4 mg/kg.

Positive Phase 1 Data for PCRX-201. The company recently finished analyzing data from its Phase 1 study of PCRX-201, a novel, intra-articular gene therapy product candidate that produces IL-1Ra for osteoarthritis. Based upon very compelling initial Phase 1 efficacy and safety data for PCRX-201, we are working with investigators and plan to request an FDA meeting to discuss the regulatory pathway forward for osteoarthritis of the knee—a very important and exciting addition to our durable non-opioid pain management pipeline.
Third Quarter 2022 Financial Results

Total revenues were $167.5 million in the third quarter of 2022, versus the $127.7 million reported for the third quarter of 2021.
EXPAREL net product sales were $132.6 million in the third quarter of 2022, versus the $121.9 million reported for the third quarter of 2021.
ZILRETTA net product sales were $26.5 million in the third quarter of 2022. The company began recognizing ZILRETTA sales upon completing its acquisition of Flexion Therapeutics, Inc. in November 2021.
Third quarter 2022 iovera° net product sales were $4.5 million, versus the $4.2 million reported for the third quarter of 2021.
Sales of bupivacaine liposome injectable suspension to a third-party licensee for use in veterinary practice were $3.0 million in the third quarter of 2022, versus the $0.7 million reported for the third quarter of 2021.
Third quarter royalty and collaborative licensing and milestone revenues were $0.9 million in both 2022 and 2021.
Total operating expenses were $146.2 million in the third quarter of 2022, versus the $96.3 million reported for the third quarter of 2021.
Research and development (R&D) expenses were $19.4 million in the third quarter of 2022, compared to $11.6 million in the third quarter of 2021. R&D expenses included $7.2 million and $4.7 million of product development and manufacturing capacity expansion costs in the third quarters of 2022 and 2021, respectively.
Selling, general and administrative (SG&A) expenses were $61.3 million in the third quarter of 2022, compared to $47.9 million in the third quarter of 2021.
GAAP net loss was $0.7 million, or $(0.02) per share (basic and diluted) in the third quarter of 2022, compared to GAAP net income of $17.7 million, or $0.40 per share (basic) and $0.39 per share (diluted), in the third quarter of 2021.
Non-GAAP net income was $29.9 million, or $0.65 per share (basic) and $0.64 per share (diluted) in the third quarter of 2022, compared to $32.5 million, or $0.73 per share (basic) and $0.72 per share (diluted), in the third quarter of 2021.
Adjusted EBITDA was $55.2 million in the third quarter of 2022, compared to $48.1 million in the third quarter of 2021.
Pacira ended the third quarter of 2022 with cash, cash equivalents and available-for-sale investments ("cash") of $346.1 million. Cash provided by operations was $42.7 million in the third quarter of 2022, compared to $60.3 million in the third quarter of 2021.
Pacira had 45.8 million basic and diluted weighted average shares of common stock outstanding in the third quarter of 2022.
See "Non-GAAP Financial Information" below.

Financial Guidance

Since early 2020, the company’s revenues have been impacted by COVID-19 and pandemic-related challenges that included the significant postponement or suspension in the scheduling of elective surgical procedures due to public health guidance and government directives. While the degree of impact has diminished during the course of the pandemic due to the introduction of vaccines and the lessening of elective surgery restrictions, certain pandemic-related operational challenges persist. It remains unclear how long it will take the elective surgery market to normalize or if restrictions on elective procedures will recur due to future COVID-19 variants or otherwise. Given the continued uncertainty around labor shortages, COVID-19 and the pace of recovery for the elective surgery market, the company is currently not providing revenue or gross margin guidance. To provide greater transparency, Pacira is reporting monthly intra-quarter unaudited net product sales for EXPAREL, ZILRETTA, and iovera° until it has gained enough visibility around the impacts of COVID-19. Pacira is also providing weekly EXPAREL utilization and elective surgery data within its investor presentation, which is accessible at investor.pacira.com

Today the company is reiterating its full-year 2022 operating expense guidance as follows:

Non-GAAP R&D expense of $75 million to $85 million; and
Non-GAAP SG&A expense of $220 million to $230 million.
Stock-based compensation of $47 million to $50 million.
See "Non-GAAP Financial Information" below.

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, Thursday, November 3, 2022, at 8:30 a.m. ET. For listeners who wish to participate in the question and answer session via telephone, please pre-register at investor.pacira.com/upcoming-events. All registrants will receive dial-in information and a PIN allowing them to access the live call. 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 of the webcast will be available on the Pacira website for approximately two weeks following the call.

Non-GAAP Financial Information

This press release contains financial measures that do not comply with U.S. generally accepted accounting principles (GAAP), such as non-GAAP net income, non-GAAP net income per common share, non-GAAP weighted average common shares outstanding-diluted, non-GAAP cost of goods sold, non-GAAP research and development (R&D) expense, non-GAAP selling, general and administrative (SG&A) expense, and adjusted EBITDA (as defined below), because these non-GAAP financial measures exclude the impact of items that management believes affect comparability or underlying business trends.

These measures supplement the company’s financial results prepared in accordance with GAAP. Pacira management uses these measures to better analyze its financial results, estimate its future cost of goods sold, R&D expense and SG&A expense outlook for 2022 and to help make managerial decisions. In management’s opinion, these non-GAAP measures are useful to investors and other users of the Company’s financial statements by providing greater transparency into the operating performance of Pacira and its future outlook. Such measures should not be deemed to be an alternative to GAAP requirements or a measure of liquidity for Pacira. Non-GAAP measures are also unlikely to be comparable with non-GAAP disclosures released by other companies. See the tables below for a reconciliation of GAAP to non-GAAP measures.

On November 3, 2022 Akoya Biosciences, Inc. (Nasdaq: AKYA) ("Akoya"), The Spatial Biology Company, reported that it will be participating in three upcoming investor conferences (Press release, Akoya Biosciences, NOV 3, 2022, View Source [SID1234623015]).

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Stephens Annual Investment Conference (Nashville, TN)
Fireside Chat on Wednesday, November 16th at 2:00 PM CT
Canaccord Genuity Annual MedTech, Diagnostics and Digital Health & Services Forum (New York, NY)
Multi-omics Panel on Thursday, November 17th at 1:00 PM ET
Piper-Sandler Annual Healthcare Conference (New York, NY)
Fireside Chat on Thursday, December 1st at 11:30 AM ET
Live and archived webcasts of the events will be available on the "Investors" section of the Akoya website at View Source

On November 3, 2022 Madrigal Pharmaceuticals, Inc. (NASDAQ:MDGL), a clinical-stage biopharmaceutical company pursuing novel therapeutics for nonalcoholic steatohepatitis (NASH), reported an overview of upcoming resmetirom Phase 3 data presentations and reports third quarter 2022 financial results (Press release, Synta Pharmaceuticals, NOV 3, 2022, View Source [SID1234623014]).

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Paul Friedman, M.D., Chief Executive Officer of Madrigal, stated, "The Madrigal team is focused on delivering topline data from the pivotal MAESTRO-NASH biopsy study in Q4 2022. Positive results from the study would allow us to finalize our new drug application for resmetirom, with the goal of filing for Subpart H accelerated approval in the first half of 2023. The efficacy and safety data we are generating across four Phase 3 MAESTRO studies put Madrigal in a strong position to navigate this accelerated approval pathway and potentially address the unmet needs of patients who currently have no approved therapy to treat NASH with significant fibrosis."

Becky Taub, M.D., Chief Medical Officer and President of Research & Development of Madrigal, stated, "The MAESTRO program continues to generate data that reinforce our conviction in the potential of resmetirom to become a foundational therapy for patients with NASH. At the upcoming American Association for the Study of Liver Diseases (AASLD) Liver Meeting, we’ll be presenting results from the compensated NASH cirrhosis cohort studied in MAESTRO-NAFLD-1. The safety and efficacy data in this more advanced patient population helped support our decision to initiate the Phase 3 MAESTRO-NASH Outcomes study, a noninvasive clinical endpoint study that assesses the rate of progression from early well-compensated NASH cirrhosis to decompensated NASH cirrhosis."

Dr. Taub continued, "A second oral presentation at AASLD will examine the ability of several noninvasive strategies to identify NASH patients with significant liver fibrosis using screening data from the MAESTRO-NASH biopsy study. Given the comprehensive diagnostic strategies included in MAESTRO studies, Madrigal has a unique opportunity to define noninvasive measures to diagnose and manage patients with NASH in real world clinical practice."

Presentations at the AASLD Liver Meeting

The following abstracts have been accepted for presentation at the AASLD Liver Meeting, taking place November 4-8 in Washington, DC:

Oral Presentation (abstract 100): Sunday, November 6th

A 52-week Phase 3 clinical trial of resmetirom in 180 patients with well-compensated NASH cirrhosis. Presenter: Stephen Harrison

In patients with well-compensated cirrhosis included in an open-label active resmetirom treatment arm of the Phase 3 MAESTRO-NAFLD-1 safety study, resmetirom lowered markers of cardiovascular risk and NASH fibrosis. Following 52 weeks of treatment with resmetirom, patients achieved reductions in magnetic resonance imaging proton density fat fraction (MRI-PDFF), FibroScan controlled attenuation parameter (CAP), FibroScan vibration-controlled transient elastography (VCTE), magnetic resonance elastography (MRE), liver and spleen volume, ALT, AST, GGT, LDL-C, triglycerides, ApoB, and lipoprotein (a). Resmetirom appeared safe and was well-tolerated during 52 weeks of treatment.

Oral Presentation (abstract 102): Sunday, November 6th

Utility of FIB-4, MRE, MRI and FibroScan to identify patients with at-risk F2-F3 NASH based on screening data from a 2000 patient biopsy confirmed cohort of the resmetirom Phase 3 clinical trial, MAESTRO-NASH. Presenter: Rohit Loomba

FIB-4 of ≥1.3 is frequently used to identify potential at-risk patients with NASH, but an analysis of screening data from the MAESTRO-NASH biopsy study found this threshold lacked the sensitivity to accurately identify patients with NASH with significant fibrosis (F2-F3). The authors concluded the influence of age on FIB-4 may require an age adjustment to ensure younger patients are not removed from consideration for therapy. MRE, MAST (MRI-AST) and FAST (FibroScan-AST) showed reasonable accuracy for identifying patients with NASH with significant fibrosis.

Financial Results for the Nine Months Ended September 30, 2022

As of September 30, 2022, Madrigal had cash, cash equivalents and marketable securities of $153.2 million, compared to $270.3 million at December 31, 2021. This decrease in cash and marketable securities resulted primarily from cash used in operations for the nine months ended September 30, 2022 of $166.3 million, partially offset by the net proceeds ($49 million) from the Loan Facility ("Loan Facility") with Hercules Capital, Inc. ("Hercules").

Operating expenses were $80.4 million and $208.3 million for the three month and nine month periods ended September 30, 2022, compared to $63.2 million and $177.9 million in the comparable prior year periods.

Research and development expenses for the three and nine month periods ended September 30, 2022 were $68.3 million and $174.7 million, compared to $54.9 million and $152.3 million in the comparable prior year periods. The increase is attributable primarily to additional activities related to the Phase 3 clinical trials, and an increase in head count.

General and administrative expenses for the three and nine month periods ended September 30, 2022 were $12.1 million and $33.6 million, compared to $8.3 million and $25.6 million in the comparable prior year periods. The increase is due primarily to increases in commercial preparation activities, including an increase in headcount and an increase in non-cash stock compensation.

Interest income for the three and nine month periods ended September 30, 2022 was $0.7 million and $1.1 million, compared to $0.1 million and $0.3 million in the comparable prior year periods. These increases in interest income were due primarily to higher average interest rates in 2022.

Interest expense for the three and nine month periods ended September 30, 2022 was $1.5 million and $2.3 million, compared to $0 million and $0 million in the comparable prior year periods. The increase in interest expense was as a result of the Loan Facility with Hercules, which we closed in May of 2022.

About the Resmetirom Phase 3 Registration Program for the Treatment of NASH

Madrigal is currently conducting four Phase 3 clinical trials to demonstrate the safety and efficacy of resmetirom for the treatment of NASH: MAESTRO-NASH, MAESTRO-NAFLD-1, MAESTRO-NAFLD-OLE, and MAESTRO-NASH Outcomes.

MAESTRO-NASH is a multicenter, randomized, double-blind, placebo-controlled Phase 3 study of resmetirom in patients with liver biopsy-confirmed NASH and was initiated in March 2019. The study enrolled more than 1,000 patients with biopsy-proven NASH (at least half with F3 (advanced) fibrosis, the remainder F2 or F1B (moderate fibrosis, with a few earlier F1 patients), randomized 1:1:1 to receive once-daily resmetirom 80 mg, resmetirom 100 mg, or placebo. After 52 weeks of treatment, a second biopsy is performed. The dual primary surrogate endpoints on biopsy are NASH resolution with ≥2-point reduction in NAS (NAFLD Activity Score), and with no worsening of fibrosis OR a 1-point decrease in fibrosis with no worsening of NASH. Achievement of either primary endpoint is considered a successful trial outcome. A key secondary endpoint is lowering of LDL-C. The planned target enrollment was announced as completed on June 30, 2021.

All patients enrolled in the MAESTRO-NASH study (up to 2,000 in total) continue on therapy after the initial 52-week treatment period for up to 54 months to accrue and measure hepatic clinical outcome events including progression to cirrhosis on biopsy (52 weeks and 54 months) and hepatic decompensation events.

MAESTRO-NAFLD-1 was initiated in December 2019 and the 52-week multicenter, randomized, placebo-controlled Phase 3 study of resmetirom in over 1,200 patients with NAFLD, presumed

NASH, has completed the double-blind arms and an open-label 100 mg arm. An additional open-label active treatment arm in patients with early (well-compensated) NASH cirrhosis is ongoing. The primary endpoint was to evaluate the safety and tolerability of resmetirom. A separate 52-week Phase 3 clinical trial, an open-label extension study of MAESTRO-NAFLD-1 (MAESTRO-NAFLD-OLE) is ongoing.

Patients in the 52-week Phase 3 MAESTRO-NAFLD-1 study were randomized 1:1:1:1 to receive once-daily resmetirom 80 mg, resmetirom 100 mg, placebo in double-blind arms, or resmetirom 100 mg in an open-label arm. MAESTRO-NAFLD-1 (unlike MAESTRO-NASH) did not include a liver biopsy and represents a "real-life" NASH study. Patients with 3 metabolic risk factors were documented with NASH or NAFLD by historical liver biopsy or noninvasive techniques. Using noninvasive measures, MAESTRO-NAFLD-1 was designed to provide incremental safety information to support the NASH indication as well as provide additional data regarding clinically relevant key secondary efficacy endpoints to better characterize the potential clinical benefits of resmetirom on cardiovascular- and liver-related endpoints. The primary safety endpoint and several key secondary endpoints were met, including LDL-C, apolipoprotein B, and triglyceride lowering and reduction of liver fat as determined by MRI-PDFF. Additional secondary and exploratory endpoints were assessed, including reduction in liver enzymes, FibroScan, and MRE scores, and other NASH biomarkers.

Data from the 52-week first 1,000 patient portion of MAESTRO-NASH, together with data from MAESTRO-NAFLD-1 and other data, including safety parameters, will form the basis for a potential subpart H submission to FDA for accelerated approval of resmetirom for treatment of NASH.

In August 2022, Madrigal initiated MAESTRO-NASH Outcomes, a randomized double-blind placebo-controlled study in approximately 700 patients with early NASH cirrhosis to allow for noninvasive monitoring of progression to liver decompensation events. A positive outcome is expected to support the full approval of resmetirom for noncirrhotic NASH, potentially accelerating the timeline to full approval. In addition, this study has the potential to support an additional indication for resmetirom in patients with well-compensated NASH cirrhosis.

On November 3, 2022 Exelixis, Inc. (Nasdaq: EXEL) and Catalent, Inc. (NYSE: CTLT) reported a new license agreement under which Catalent’s Redwood Bioscience subsidiary will grant Exelixis an exclusive license to three target programs with lead antibody and/or ADC candidates (Press release, Exelixis, NOV 3, 2022, View Source [SID1234623013]). The ADC candidates have been developed using Catalent’s proprietary SMARTag technology, and each of the licensed antibodies has potential for development as an ADC or other biologic therapy using a variety of technologies to which Exelixis has access through its partner network. In September 2020, Exelixis and Catalent entered into a separate agreement under which Catalent is applying its SMARTag bioconjugation platform to build ADCs using monoclonal antibodies from Exelixis’ growing preclinical pipeline.

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Under the terms of the new agreement, Exelixis will make an upfront payment to Catalent of $30 million in exchange for rights to the three biologics programs. Exelixis will fund the development work conducted by Catalent until development candidate selection is complete, after which Exelixis will assume responsibility for conducting preclinical, clinical, and commercial activities. Catalent will be eligible for development and sales milestone payments, as well as sales-based royalties.

"Exelixis is committed to building a robust biologics pipeline, and this agreement expands our portfolio to include three highly promising targets with broad potential in multiple solid tumor indications, including bladder, breast, lung, ovarian, pancreatic, and other cancers," said Peter Lamb, Ph.D., Executive Vice President, Scientific Strategy and Chief Scientific Officer, Exelixis. "We believe that the antibodies, ADCs and data Catalent has generated to date will enable rapid advancement of these programs, enabling an acceleration of our in-house biotherapeutics pipeline. We are excited for this new opportunity to continue our work together to advance additional programs that have the potential to provide patients with new therapeutic options."

"Catalent Biologics is a leader in applying precision protein engineering and novel chemistries to develop optimized therapeutics," said Mike Riley, President, Division Head for BioProduct Delivery, Catalent Biologics. "Our versatile SMARTag platform and growing portfolio of preclinical ADCs against promising targets create multiple opportunities for Catalent to partner with leading biopharmaceutical companies, such as Exelixis. We expect the new license agreement with Exelixis will yield innovative and differentiated therapies with the potential to improve care and outcomes for cancer patients."

The SMARTag technology platform was developed by Redwood Bioscience based on discoveries in the lab of Carolyn Bertozzi, recent winner of the Nobel Prize in Chemistry and Chair of Redwood’s scientific advisory board. The platform provides optimized site-specific protein-modification, as well as linker and payload technologies for ADCs and other bioconjugates. The SMARTag platform overcomes the limitations associated with traditional protein chemistries that produce heterogeneous products with variable conjugate potency, toxicity, and stability, and enables the development of ADCs with a wider therapeutic window and improved manufacturability.

On November 3, 2022 BeiGene, (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global biotechnology company, reported clinical and real-world data accepted at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, scheduled for December 10-14, 2022, in New Orleans (Press release, BeiGene, NOV 3, 2022, View Source [SID1234623012]). BeiGene is focused on developing innovative and affordable oncology medicines to improve treatment outcomes and access for patients worldwide.

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Key presentations

Phase 1 trial results demonstrating proof of concept for BGB-11417, a novel B-cell lymphoma 2 (Bcl-2) inhibitor in chronic lymphocytic leukemia/small lymphocytic leukemia;
Long-term efficacy and safety data from the MAGNOLIA trial of BRUKINSA (zanubrutinib) in patients with relapsed/refractory (R/R) marginal zone lymphoma; and
Updated results from a Phase 2 trial showing tolerability and efficacy of zanubrutinib in patients with B-cell malignancies who were intolerant to acalabrutinib.
"BeiGene has established a solid foundation in both the research and development of innovative medicines for hematologic malignancies," said Mehrdad Mobasher, M.D., M.P.H., Chief Medical Officer, Hematology at BeiGene. "At this year’s ASH (Free ASH Whitepaper) meeting, we look forward to sharing a wealth of data from our broad, global development programs. We will showcase our deep expertise and drive to innovate — from the Phase 1 data for our novel Bcl2 inhibitor, BGB-11417 and through numerous presentations demonstrating meaningful clinical efficacy and a consistent safety profile for BRUKINSA across several B-cell malignancies."

Additional presentations highlight combinability and strength of pipeline

Preliminary safety and efficacy of zanubrutinib in combination with lenalidomide in patients with R/R diffuse large B-cell lymphoma;
Multiple posters for Phase 1 studies with BGB-11417 as monotherapy or in combination, showing promising efficacy in B-cell and myeloid malignancies, along with a manageable safety profile; and
Safety and efficacy of zanubrutinib in combination with zandelisib in patients with R/R follicular lymphoma or mantle cell lymphoma.
Investor event

BeiGene will host an ancillary event in New Orleans on December 11 at 8:00 pm CST for investors and analysts attending ASH (Free ASH Whitepaper). BeiGene senior management will review highlights of the presented data, and special guests will join them for a Q&A panel. The event will be webcast live and it can be accessed from the investors section of BeiGene’s website at View Source, View Source or View Source An archived replay will be posted for 90 days following the event.

Hematologic

Malignancy

Title

Presentation details

Oral

R/R Follicular Lymphoma

R/R Mantle Cell Lymphoma

Safety and Efficacy of the PI3Kδ Inhibitor Zandelisib in Combination with the BTK Inhibitor Zanubrutinib in Patients with Relapsed/Refractory (R/R) Follicular Lymphoma (FL) or Mantle Cell Lymphoma (MCL)

#78

Date: 12/10/2022

Time: 10:45 AM

R/R Marginal Zone Lymphoma

Long-Term Efficacy and Safety of Zanubrutinib in Patients With Relapsed/Refractory (R/R) Marginal Zone Lymphoma (MZL): Final Analysis of the MAGNOLIA (BGB-3111-214) Trial

#234

Date: 12/10/2022

Time: 3:15 PM

Chronic Lymphocytic Leukemia

Small Lymphocytic Leukemia

A Phase 1 Study With The Novel B-Cell Lymphoma 2 (Bcl-2) Inhibitor BGB-11417 As

Monotherapy or in Combination With Zanubrutinib (ZANU) in Patients (Pts) With CLL/SLL: Preliminary Data

#962

Date: 12/12/2022

Time: 4:45 PM

Poster

B-Cell Malignancies

Efficacy and Safety of Zanubrutinib in Japanese Patients With Mature B-Cell Malignancies

#1590

Date: 12/10/2022

Time: 5:30 – 7:30 PM

B-Cell Malignancies

Zanubrutinib in Acalabrutinib-Intolerant Patients (Pts) with B-Cell Malignancies

#1587

Date: 12/10/2022

Time: 5:30 – 7:30 PM

R/R Diffuse Large B-Cell Lymphoma

Preliminary Safety and Efficacy of Zanubrutinib in Combination With Lenalidomide in Patients With Relapsed/Refractory Diffuse Large B-Cell Lymphoma

#1627

Date: 12/10/2022

Time: 5:30 – 7:30 PM

R/R B-Cell Malignancies

Biomarker Analysis of Zanubrutinib and Tislelizumab Combination Therapy in Patients with Relapsed/Refractory B-Cell Malignancies

#1529

Date: 12/10/2022

Time: 5:30 – 7:30 PM

Acute Myeloid Leukemia

Preliminary Safety and Efficacy of BGB-11417, a Novel BCL2 Inhibitor, in Combination With Azacitidine in Patients With Acute Myeloid Leukemia (AML)

#1443

Date: 12/10/2022

Time: 5:30 – 7:30 PM

Mature B-cell Malignancies

A Phase 1 Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Preliminary Antitumor Activity of BCL2 Inhibitor BGB-11417 in Adult Patients With Mature B-cell Malignancies: Preliminary Data

#2989

Date: 12/11/2022

Time: 6:00 – 8:00 PM

Multiple Myeloma

Preliminary Safety and Efficacy of a BCL-2 Inhibitor, BGB-11417, in Patients With Relapsed/Refractory Multiple Myeloma Harboring t(11,14): A Non-randomized, Open-label, Phase 1b/2 Study

#3235

Date: 12/11/2022

Time: 6:00 – 8:00 PM

Relapsed/Refractory B-Cell Malignancies

Genomic Characterization of Patients in Phase 2 Study of Zanubrutinib in BTK Inhibitor-Intolerant Patients With Relapsed/Refractory B-Cell Malignancies

#4176

Date: 12/12/2022

Time: 6:00 – 8:00 PM

Non-Hodgkin’s Lymphoma

Waldenström Macroglobulinemia

A Phase 1 Study With the Novel B-Cell Lymphoma 2 (Bcl-2) Inhibitor BGB-11417 As Monotherapy or in Combination with Zanubrutinib (ZANU) in Patients (Pts) With Non-Hodgkin’s Lymphoma (NHL) or Waldenström macroglobulinemia (WM): Preliminary Data

#4201

Date: 12/12/2022

Time: 6:00 – 8:00 PM

R/R Mantle Cell Lymphoma

Long-Term Outcomes of Second-Line vs Later-Line Zanubrutinib Treatment in Patients With Relapsed/Refractory MCL: An Updated Pooled Analysis

#2894

Date: 12/11/2022
Time: 6:00 – 8:00 PM

Online Only

RWE

Real-World Evidence (RWE) Studies Supported By Hematologic Oncology FDA Approval: What Do They Look Like

RWE

Development of Hypertension and Atrial Fibrillation Following Diagnosis of B-Cell Malignancies – A Retrospective Analysis of US MarketScan Insurance Claims Database

About BRUKINSA

BRUKINSA (zanubrutinib) is a small-molecule inhibitor of Bruton’s tyrosine kinase (BTK) discovered by BeiGene scientists that is currently being evaluated globally in a broad clinical program as a monotherapy and in combination with other therapies to treat various B-cell malignancies. BRUKINSA was specifically designed to deliver targeted and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared to other approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease-relevant tissues.

BRUKINSA is supported by a broad clinical program which includes more than 4,500 subjects in 35 trials across 28 markets. To date, BRUKINSA has received approvals covering more than 55 countries and regions, including the United States, China, the EU, Switzerland, Great Britain, Canada, Australia, and additional international markets.

About BGB-11417

BGB-11417 is a highly potent and selective Bcl-2 inhibitor designed to produce deeper and more sustained target inhibition.

Compared with venetoclax, BGB-11417 exhibited greater potency (>10-fold) and higher target selectivity and showed signs of overcoming treatment resistance in pre-clinical studies and tumor models i

BRUKINSA IMPORTANT SAFETY INFORMATION AND U.S. INDICATIONS

Warnings and Precautions

Hemorrhage

Fatal and serious hemorrhagic events have occurred in patients with hematological malignancies treated with BRUKINSA monotherapy. Grade 3 or higher hemorrhage events including intracranial and gastrointestinal hemorrhage, hematuria and hemothorax have been reported in 3.4% of patients treated with BRUKINSA monotherapy. Hemorrhage events of any grade occurred in 35% of patients treated with BRUKINSA monotherapy.

Bleeding events have occurred in patients with and without concomitant antiplatelet or anticoagulation therapy. Co-administration of BRUKINSA with antiplatelet or anticoagulant medications may further increase the risk of hemorrhage.

Monitor for signs and symptoms of bleeding. Discontinue BRUKINSA if intracranial hemorrhage of any grade occurs. Consider the benefit-risk of withholding BRUKINSA for 3-7 days pre- and post-surgery depending upon the type of surgery and the risk of bleeding.

Infections

Fatal and serious infections (including bacterial, viral, or fungal) and opportunistic infections have occurred in patients with hematological malignancies treated with BRUKINSA monotherapy. Grade 3 or higher infections occurred in 27% of patients, most commonly pneumonia. Infections due to hepatitis B virus (HBV) reactivation have occurred.

Consider prophylaxis for herpes simplex virus, pneumocystis jiroveci pneumonia and other infections according to standard of care in patients who are at increased risk for infections. Monitor and evaluate patients for fever or other signs and symptoms of infection and treat appropriately.

Cytopenias

Grade 3 or 4 cytopenias, including neutropenia (26%), thrombocytopenia (11%) and anemia (8%) based on laboratory measurements, were reported in patients treated with BRUKINSA monotherapy. Grade 4 neutropenia occurred in 13% of patients, and Grade 4 thrombocytopenia occurred in 3.6% of patients.

Monitor complete blood counts regularly during treatment and interrupt treatment, reduce the dose, or discontinue treatment as warranted. Treat using growth factor or transfusions, as needed.

Second Primary Malignancies

Second primary malignancies, including non-skin carcinoma, have occurred in 14% of patients treated with BRUKINSA monotherapy. The most frequent second primary malignancy was non-melanoma skin cancer, reported in 8% of patients. Other second primary malignancies included malignant solid tumors (4.0%), melanoma (1.7%) and hematologic malignancies (1.2%). Advise patients to use sun protection and monitor patients for the development of second primary malignancies.

Cardiac Arrhythmias

Atrial fibrillation and atrial flutter were reported in 3.2% of patients treated with BRUKINSA monotherapy. Patients with cardiac risk factors, hypertension, and acute infections may be at increased risk. Grade 3 or higher events were reported in 1.1% of patients treated with BRUKINSA monotherapy. Monitor signs and symptoms for atrial fibrillation and atrial flutter and manage as appropriate.

Embryo-Fetal Toxicity

Based on findings in animals, BRUKINSA can cause fetal harm when administered to a pregnant woman. Administration of zanubrutinib to pregnant rats during the period of organogenesis caused embryo-fetal toxicity including malformations at exposures that were 5 times higher than those reported in patients at the recommended dose of 160 mg twice daily. Advise women to avoid becoming pregnant while taking BRUKINSA and for 1 week after the last dose. Advise men to avoid fathering a child during treatment and for 1 week after the last dose.

If this drug is used during pregnancy, or if the patient becomes pregnant while taking this drug, the patient should be apprised of the potential hazard to a fetus.

Adverse reactions

The most common adverse reactions, including laboratory abnormalities, in ≥ 30% of patients who received BRUKINSA (N = 847) included decreased neutrophil count (54%), upper respiratory tract infection (47%), decreased platelet count (41%), hemorrhage (35%), decreased lymphocyte count (31%), rash (31%) and musculoskeletal pain (30%).

Drug Interactions

CYP3A Inhibitors: When BRUKINSA is co-administered with a strong CYP3A inhibitor, reduce BRUKINSA dose to 80 mg once daily. For coadministration with a moderate CYP3A inhibitor, reduce BRUKINSA dose to 80 mg twice daily.

CYP3A Inducers: Avoid coadministration with moderate or strong CYP3A inducers.

Specific Populations

Hepatic Impairment: The recommended dose of BRUKINSA for patients with severe hepatic impairment is 80 mg orally twice daily.

INDICATIONS

BRUKINSA is a kinase inhibitor indicated for the treatment of adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy.
This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
BRUKINSA is indicated for the treatment of adult patients with Waldenström’s macroglobulinemia (WM).
BRUKINSA is indicated for the treatment of adult patients with relapsed or refractory marginal zone lymphoma (MZL) who have received at least one anti-CD20-based regimen.
This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
Please see full U.S. Prescribing Information at www.beigene.com/PDF/BRUKINSAUSPI.pdf and Patient Information at www.beigene.com/PDF/BRUKINSAUSPPI.pdf

BeiGene Oncology

BeiGene is committed to advancing best- and first-in-class clinical candidates internally or with like-minded partners to develop impactful and affordable medicines for patients across the globe. We have a growing R&D and medical affairs team of approximately 3,300 colleagues dedicated to advancing more than 100 clinical trials that have involved more than 16,000 subjects. Our expansive portfolio is directed predominantly by our internal colleagues supporting clinical trials in more than 45 countries and regions. Hematology-oncology, and solid tumor targeted therapies, and immuno-oncology are key focus areas for the Company, with both monotherapies and combination therapies prioritized in our research and development. BeiGene currently has three licensed medicines discovered and developed in our own labs: BTK inhibitor BRUKINSA in the U.S., China, the European Union, Switzerland, Great Britain, Canada, Australia, and additional international markets; and the non-FC-gamma receptor binding anti-PD-1 antibody tislelizumab as well as the poly adenosine diphosphate-ribose polymerase (PARP) inhibitor pamiparib in China.

BeiGene also partners with innovative companies who share our goal of developing therapies to address global health needs. We commercialize a range of oncology medicines in China licensed from Amgen, Bristol Myers Squibb, EUSA Pharma, and Bio-Thera. We also plan to address greater areas of unmet need globally through our other collaborations including Mirati Therapeutics, Seagen, and Zymeworks.

In January 2021 BeiGene and Novartis announced a collaboration granting Novartis rights to co-develop, manufacture, and commercialize BeiGene’s anti-PD-1 antibody tislelizumab in North America, Europe, and Japan. Building upon this productive collaboration, BeiGene and Novartis announced an option, collaboration, and license agreement in December 2021 for BeiGene’s TIGIT inhibitor ociperlimab that is in Phase 3 development. Novartis and BeiGene also entered into a strategic commercial agreement through which BeiGene will promote five approved Novartis oncology products across designated regions of China.