On November 2, 2023 Be Biopharma, Inc. ("Be Bio"), a company pioneering the discovery and development of Engineered B Cell Medicines (BCMs) reported that it will present new preclinical data in an oral presentation at the 65th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) taking place December 9-12, 2023, in San Diego, California (Press release, Be Biopharma, NOV 2, 2023, View Source [SID1234636735]). The research will describe the development of an ex vivo precision-engineered B Cell Medicine (BCM) that produces active and sustained levels of factor IX (FIX). The abstract is now available online in a special issue of the ASH (Free ASH Whitepaper) official journal, Blood.

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"We look forward to presenting our research at the ASH (Free ASH Whitepaper) annual meeting, the pre-eminent global congress for scientific exchange in hematology," said Dr. Rick Morgan, Chief Scientific Officer, Be Bio. "Our team of dedicated researchers is inspired by the potential of this bold new class of medicines."

Details of the oral presentation are as follows:

Title: Development of an Ex Vivo Precision Gene Engineered B Cell Medicine That Produces Active and Sustained Levels of FIX for the Treatment of Hemophilia B
Session Name: 703 Cellular Immunotherapies: Basic and Translational: Novel Approaches for Next Generation Cellular Immunotherapies
Session Date: Sunday, December 10, 2023
Presentation Time: 9:30 AM PST
Room: San Diego Convention Center, Room 6A
Publication Number: 463

About Engineered B Cell Medicines – A New Class of Cellular Medicines
Imagine what could "Be?" In nature, a single B cell engrafts in the bone marrow and can produce thousands of proteins per second at constant levels over decades. B cells are nature’s exquisite medicine factories, manufacturing proteins to fight disease and maintain health. Unleashing the power of B cells is driving a new class of cellular medicines – Engineered B Cell Medicines (BCMs). BCMs have the potential to be durable, allogeneic, redosable and administered without toxic conditioning. The promise of BCMs could transform therapeutic biologics with broad application — across protein classes, patient populations and therapeutic areas.

On November 2, 2023 Bausch Health Companies Inc. (NYSE/TSX: BHC) ("Bausch Health" or the "Company" or "we" or "our") reported its third-quarter 2023 financial results and other key updates from the quarter (Press release, Bausch Health, NOV 2, 2023, View Source [SID1234636734]).

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"We are pleased with our solid third-quarter performance, as each of our business segments posted year-over-year revenue growth on both a reported and organic basis. We remain focused on advancing our R&D pipeline, strengthening our balance sheet and executing on our commercial strategies to drive global growth," said Thomas J. Appio, Chief Executive Officer, Bausch Health.

Bausch Health (excl. B+L) R&D Update
•RED-C: prevention and delay of first episode of hepatic encephalopathy
◦Enrollment of two global Phase 3 trials on track and expected to be completed in Q1 2024
•Amiselimod (S1P modulator): treatment of mild to moderate Ulcerative Colitis
◦Phase 2 study completed enrollment in July 2023 and induction portion of the study is expected to be completed in Q4 2023
•CABTREOTM: first triple combination product for the treatment of acne vulgaris
◦Received FDA approval on October 20, 2023
◦Commercial launch expected in Q1 2024
◦New Drug Submission was submitted to Health Canada on May 30, 2023
•Clear + Brilliant Touch: fractionated laser device for skin rejuvenation
◦Planned regulatory submissions on track for Europe, Canada, and Asia Pacific markets in 2024
•Next Generation Fraxel: fractionated laser device for skin resurfacing
◦FDA submission planned in Q1 2024 and approval is expected 1H 2024

Third Quarter 2023 Revenue Performance
Total reported revenues were $2.24 billion for the third quarter of 2023, compared with $2.05 billion in the third quarter of 2022, an increase of $192 million, or 9%. Excluding the impact of foreign exchange of $6 million and acquisitions, divestitures, and discontinuations of $19 million, revenue increased by 9% organically1 compared with the third quarter of 2022.

Reported revenues by segment were as follows:

Three Months Ended September 30, Reported Change
Change at Constant Currency1
(Non-GAAP)
Change in Organic Revenue1
(Non-GAAP)
(in millions) 2023 2022 Amount Pct.
Total Bausch Health Revenues $2,238 $2,046 $192 9 % 9 % 9 %
Bausch Health (excl. B+L) $1,231 $1,104 $127 12 % 10 % 10 %
Salix segment $614 $544 $70 13 % 13 % 13 %
International segment $275 $250 $25 10 % 3 % 4 %
Solta Medical segment $83 $72 $11 15 % 17 % 17 %
Diversified segment $259 $238 $21 9 % 9 % 9 %
Bausch + Lomb segment $1,007 $942 $65 7 % 8 % 7 %

Salix Segment
Salix segment reported and organic1 revenues were $614 million for the third quarter of 2023, compared with $544 million for the third quarter of 2022, an increase of $70 million, or 13%. Sales growth was driven by Xifaxan, Relistor, and Trulance.

International Segment
International segment reported revenues were $275 million for the third quarter of 2023, compared with $250 million for the third quarter of 2022, an increase of $25 million, or 10%. Excluding the impact of foreign exchange of $17 million and divestitures and discontinuations of $1 million, segment revenues increased organically1 by 4% compared with the third quarter of 2022, led by strong performances in Latin America and Poland.

Solta Medical Segment
Solta Medical segment reported revenues were $83 million for the third quarter of 2023, compared with $72 million in the third quarter of 2022, an increase of $11 million, or 15%, which was driven by growth in the Asia Pacific region. Excluding the impact of foreign exchange of $1 million, segment revenues increased organically1 by 17% compared with the third quarter of 2022.

Diversified Segment
Diversified segment reported revenues were $259 million for the third quarter of 2023, compared with $238 million for the third quarter of 2022, an increase of $21 million, or 9% on both a reported and organic1 basis, primarily attributable to increases in sales in Generics and Neurology.

Bausch + Lomb Segment
Bausch + Lomb segment reported revenues were $1,007 million for the third quarter of 2023, compared with $942 million for the third quarter of 2022, an increase of $65 million, or 7%. Excluding the impact of foreign exchange of $10 million, acquisitions of $15 million and divestitures and discontinuations of $3 million, the Bausch + Lomb segment revenue increased organically1 by 7%, compared with the third quarter of 2022, driven by increases across all business units.

Consolidated Operating Income
Consolidated operating income was $14 million for the third quarter of 2023, compared with operating income of $244 million for the third quarter of 2022, a decrease of $230 million. The change is primarily due to an increase in goodwill impairments, higher selling, general and administrative expenses, and investments in research and development, which were partially offset by higher revenues and associated gross profit, and lower amortization of intangible assets.

Net (Loss) Income Attributable to Bausch Health
Net loss attributable to Bausch Health for the third quarter of 2023 was $378 million, compared with net income attributable to Bausch Health of $399 million for the third quarter of 2022, a decrease of $777 million, primarily due to the decrease in Operating Income and a gain on extinguishment of debt of $570 million recorded in the third quarter of 2022.

Adjusted net income attributable to Bausch Health (non-GAAP)1 for the third quarter of 2023 was $377 million, compared with $277 million for the third quarter of 2022, an increase of $100 million primarily due to higher revenues and gross profit, partially offset by higher selling, general and administrative expenses and investments in research and development.

(Loss) Earnings Per Share Attributable to Bausch Health
GAAP loss per share attributable to Bausch Health for the third quarter of 2023 was $1.03, compared with earnings per share of $1.10 for the third quarter of 2022.

Adjusted EBITDA Attributable to Bausch Health (non-GAAP)1
Adjusted EBITDA attributable to Bausch Health (non-GAAP)1 was $830 million for the third quarter of 2023, as compared to $766 million for the third quarter of 2022, an increase of $64 million.

Cash Provided by (Used in) Operating Activities
The Company generated $281 million of cash from operating activities in the third quarter of 2023 compared with cash used of $1,263 million in the third quarter of 2022. The increase in cash flow reflects improved operating results as well as the impact in 2022 of a reduction of $1.2 billion from restricted cash in connection with the settlement of legacy U.S. securities litigation.

Balance Sheet Highlights as of September 30, 2023:
•Cash and cash equivalents of $780 million.
•Bausch Health (excl. B+L) had availability under its 2027 revolving credit facility of $952 million and Bausch + Lomb had availability of approximately $300 million under its revolving credit facility.
•Bausch Health (excl. B+L) has an accounts receivable credit facility which provides for up to $600 million of availability, $350 million of which was drawn as of September 30, 2023.

2023 Financial Outlook
The Company updated its full-year revenue and Adjusted EBITDA (non-GAAP)1 guidance:

Previous Guidance (as of Aug. 3, 2023) Current Guidance (as of Nov. 2, 2023)
BHC BHC
(excl. B+L) B+L BHC BHC
(excl. B+L) B+L
Revenues (in Billions) $8.45 – $8.65 $4.50 – $4.65 $3.95 – $4.00 $8.585 – $8.710 $4.550 – $4.625 $4.035 – $4.085
Organic1 growth vs. Prior Year
2%-5% 4%-6%
Adjusted EBITDA1 (in Billions)
$3.00 – $3.15 $2.30 – $2.40 $0.70 – $0.75 $3.01 – $3.11 $2.30 – $2.35 $0.71 – $0.76

Other than with respect to GAAP revenues, the Company only provides guidance on a non-GAAP basis. The Company does not provide a reconciliation of forward-looking Adjusted EBITDA (non-GAAP)1 to GAAP net income (loss), due to the inherent difficulty in forecasting and quantifying certain amounts that are necessary for such reconciliation. Because deductions (such as restructuring, gain or loss on extinguishment of debt and litigation and other matters) used to calculate projected net income (loss) vary dramatically based on actual events, the Company is not able to forecast on a GAAP basis with reasonable certainty all deductions needed in order to provide a GAAP calculation of projected net income (loss) at this time. The amount of these deductions may be material and, therefore, could result in projected GAAP net income (loss) being materially less than projected Adjusted EBITDA (non-GAAP)1. These statements represent forward-looking information and may represent a financial outlook, and actual results may vary. Please see the risks and assumptions referred to in the "Forward-looking Statements" section of this news release. The guidance in this news release is only effective as of the date it is given and will not be updated or affirmed unless and until the Company publicly announces updated or affirmed guidance.

Conference Call Details
Date:
Thursday, November 2, 2023
Time: 8:00 a.m. ET
Webcast: View Source

A replay of the conference call will be available on the investor relations website.

On November 2, 2023 Aurinia Pharmaceuticals Inc. (NASDAQ: AUPH) (Aurinia or the Company) reported its financial results for the three and nine months ended September 30, 2023 (Press release, Aurinia Pharmaceuticals, NOV 2, 2023, View Source [SID1234636733]). Amounts are expressed in U.S. dollars.

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Net product revenue was $40.8 million for the three months ended September 30, 2023 and $25.5 million for the same period in 2022, representing growth of approximately 60%. Net product revenue was $116.2 million for the nine months ended September 30, 2023 and $75.1 million for the same period ended 2022, representing growth of approximately 55%.

Total net revenue was $54.5 million for the three months ended September 30, 2023 and $55.8 million for the same period in 2022. Total net revenue was $130.4 million for the nine months ended September 30, 2023 and $105.6 million for the same period in 2022.

"We are very pleased with our overall results for the first nine months of the year. Reporting another strong quarter of results reinforces our ability to execute and deliver against key metrics. Our team continues to focus on business fundamentals and steady performance," said Peter Greenleaf, President, and Chief Executive Officer of Aurinia. "We continue to deliver new data on LUPKYNIS and grow the overall LN market. In addition, we received a $10.0 million milestone from our collaboration partner outside the U.S. as a result of securing pricing and reimbursement approvals in three of the five major European markets."

For the fiscal year 2023, the Company is narrowing its net product revenue guidance to a range of $155 – $160 million for net product sales of LUPKYNIS. This guidance range is based on assumptions regarding PSF run rates, consistent conversion rates, time to convert, persistency and pricing.

Third Quarter 2023 and Recent Highlights

Full results from AURORA 2 (the long-term extension study of the Phase 3 AURORA trial) were published in Arthritis & Rheumatology, the official peer-reviewed journal of the American College of Rheumatology, demonstrating kidney preservation over the 3-year study period as measured by eGFR (estimated glomerular filtration rate) along with additional efficacy, safety, and tolerability of LUPKYNIS over the study duration.
A total of 14 LUPKYNIS clinical abstracts were accepted for presentation at the upcoming American Society of Nephrology and the American College of Rheumatology being held in November 2023. Led by several leading experts in nephrology and rheumatology, these presentations reinforce the long-term safety and efficacy profile of LUPKYNIS for the treatment of adults with active lupus nephritis (LN), a serious complication of systemic lupus erythematosus (SLE). The robust set of data demonstrates Aurinia’s deep commitment to sustained research in autoimmune diseases, including lupus.
Received notification that the pricing and reimbursement milestone was secured. As a result, this triggered a $10 million milestone from Otsuka Pharmaceutical Co. Ltd (Otsuka). Additionally, LUPKYNIS received marketing acceptance in Scotland by the Scottish Medicines Consortium.
Appointed three new directors to the Board of Directors – Dr. Karen Smith, Jeffrey Bailey, and Dr. Robert Foster.
LUPKYNIS Product Performance Highlights

There were approximately 1,939 patients on LUPKYNIS therapy at September 30, 2023, compared with 1,354 at September 30, 2022, representing an increase of approximately 43% year over year.
Aurinia added 436 patient start forms (PSFs) during the three months ended September 30, 2023, compared to 374 during the three months ended September 30, 2022, representing an increase of approximately 17% over the same period last year.
Through the end of October 2023, the Company recorded approximately 1,510 PSFs since January 1, 2023.
Conversion rates remain consistent with approximately 90% of PSFs converted to patients on therapy.
Time to convert has improved to an all-time high with the large majority (64%) of patients on therapy by 20 days.
Adherence improved from 84% at September 30, 2022 to 87% at September 30, 2023.
Persistency at 12 months has maintained at 54%; and remained stable at further months on therapy: 48% at 15 months and 43% at 18 months.
Financial Results for the Three and Nine Months Ended September 30, 2023

Total net revenue was $54.5 million and $55.8 million for the three months ended September 30, 2023 and September 30, 2022, respectively. Total net revenue was $130.4 million and $105.6 million for the nine months ended September 30, 2023 and September 30, 2022, respectively.

Net product revenue was $40.8 million and $25.5 million for the three months ended September 30, 2023 and September 30, 2022, respectively. Net product revenue was $116.2 million and $75.1 million for the nine months ended September 30, 2023 and September 30, 2022, respectively. The increase for both periods is primarily due to an increase in product sales to our two main customers for LUPKYNIS, driven predominantly by further penetration of the LN market.

License, royalty and collaboration revenue was $13.7 million and $30.3 million for the three months ended September 30, 2023 and September 30, 2022, respectively. License, royalty and collaboration revenue was $14.2 million and $30.5 million for the nine months ended September 30, 2023 and September 30, 2022, respectively. The decrease for both periods is due to the recognition of a $30.0 million regulatory milestone from Otsuka following the EC marketing authorization of LUPKYNIS in September 2022 partially offset by the recognition of a $10.0 million pricing and reimbursement milestone as well as recognition of collaboration revenue from Otsuka in the quarter ended September 2023.

Total cost of sales and operating expenses were $70.8 million and $65.3 million for the three months ended September 30, 2023 and September 30, 2022, respectively. Total cost of sales and operating expenses were $192.4 million and $189.0 million for the nine months ended September 30, 2023 and September 30, 2022, respectively. Further breakdown of cost of sales and operating expense drivers and fluctuations are highlighted in the following paragraphs.

Cost of sales were $6.8 million and $2.4 million for the three months ended September 30, 2023 and September 30, 2022, respectively. The increase is primarily due to increased sales of LUPKYNIS, coupled with the amortization of the monoplant finance right of use asset, which was placed into service in late June 2023.

Cost of sales were $8.8 million and $4.3 million for the nine months ended September 30, 2023 and September 30, 2022, respectively. The increase is primarily due to increased sales of LUPKYNIS coupled with the amortization of the monoplant finance right of use asset, partially offset by higher inventory reserves in 2022 due to the write-down of FDA validation batches.

Gross margin for the three months ended September 30, 2023 and September 30, 2022 was approximately 88% and 96%, respectively. Gross margin for the nine months ended September 30, 2023 and September 30, 2022 was approximately 93% and 96% respectively.

Selling, general and administrative (SG&A) expenses, inclusive of share-based compensation, were $47.8 million and $52.2 million for the three months ended September 30, 2023 and September 30, 2022, respectively. The decrease is primarily due to a decrease in professional fees and services (including legal fees with respect to litigation matters that occurred during the three months ended September 30, 2022), partially offset by an increase in share-based compensation expense.

SG&A expenses, inclusive of share-based compensation, were $145.0 million and $148.9 million for the nine months ended September 30, 2023 and September 30, 2022, respectively. The decrease was primarily due to a decrease in professional fees and services (including legal fees) and other corporate costs (including rent and insurance), partially offset by an increase in share-based compensation expense.

Non-cash SG&A share-based compensation expense included within SG&A expenses was $9.6 million and $6.6 million for the three months ended September 30, 2023 and September 30, 2022, respectively. Non-cash SG&A share-based compensation expense included within SG&A expenses, was $27.0 million and $21.5 million for the nine months ended September 30, 2023 and September 30, 2022, respectively.

Research and development (R&D) expenses, inclusive of share-based compensation, were $13.6 million and $11.0 million for the three months ended September 30, 2023 and September 30, 2022, respectively. The primary drivers for the increase were due to an increase in CRO and developmental costs as the Company advances its preclinical assets.

R&D expenses, inclusive of share-based compensation expense, were $39.4 million and $35.1 million for the nine months ended September 30, 2023 and September 30, 2022, respectively. The increase was primarily due to an increase in costs to advance the Company’s preclinical assets coupled with an increase in share-based compensation expense partially offset by the decrease in costs associated with the completion of the AURORA 2 continuation study and drug interaction study, which were substantially completed in 2022.

Non-cash R&D share-based compensation expense included with R&D expense was $2.0 million and $1.5 million for the three months ended September 30, 2023 and September 30, 2022, respectively. Non-cash R&D share-based compensation expense included with R&D expenses was $5.7 million and $3.5 million for the nine months ended September 30, 2023 and September 30, 2022, respectively.

Other (income) expense, net was $2.6 million and $(0.3) million for the three months ended September 30, 2023 and September 30, 2022, respectively. The change is primarily related to expenses incurred for shareholder matters partially offset by foreign exchange gain related to the revaluation of the monoplant finance lease liability.

Other (income) expense, net was $(0.7) million and $0.6 million for the nine months ended September 30, 2023 and September 30, 2022, respectively. The change is primarily related to change in fair value assumptions driven predominantly by rising interest rates related to our deferred compensation liability and foreign exchange gain on revaluation of the monoplant finance lease liability, partially offset by expenses incurred for shareholder matters.

Interest income was $4.5 million and $1.5 million for the three months ended September 30, 2023 and September 30, 2022, respectively. Interest income was $12.4 million and $2.2 million for the nine months ended September 30, 2023 and September 30, 2022, respectively. The increase for both periods is due to higher yields on our investments as a result of increased interest rates.

For the three months ended September 30, 2023, Aurinia recorded a net loss of $13.4 million or $0.09 net loss per common share, as compared to a net loss of $9.0 million or $0.06 net loss per common share for the three months ended September 30, 2022. For the nine months ended September 30, 2023, Aurinia recorded a net loss of $51.1 million or $0.36 net loss per common share, as compared to a net loss of $82.1 million or $0.58 net loss per common share for the nine months ended September 30, 2022.

Financial Liquidity at September 30, 2023

As of September 30, 2023, Aurinia had cash, cash equivalents and restricted cash and investments of $338.5 million compared to $389.4 million at December 31, 2022. The decrease is primarily related to the continued investment in commercialization activities and post approval commitments of our approved drug, LUPKYNIS, inventory purchases, advancement of our pipeline and monoplant payments, partially offset by an increase in cash receipts from sales of LUPKYNIS.

Aurinia believes that it has sufficient financial resources to fund its operations, which include funding commercial activities, such as FDA related post approval commitments, manufacturing and packaging of commercial drug supply, funding its supporting commercial infrastructure, advancing its R&D programs and funding its working capital obligations for at least the next few years.

This press release is intended to be read in conjunction with the Company’s unaudited condensed consolidated financial statements and Management’s Discussion and Analysis for the quarter ended September 30, 2023 in the Company’s Quarterly Report on Form 10-Q and the Company’s Annual Report on Form 10-K for the year ended December 31, 2022, including risk factors disclosed therein, which will be accessible on Aurinia’s website at www.auriniapharma.com, on SEDAR at www.sedarplus.ca or on EDGAR at www.sec.gov/edgar.

Conference Call Details

Aurinia will host a conference call and webcast to discuss the quarter ended September 30, 2023 financial results today, Thursday, November 2, 2023 at 8:30 a.m. ET. The audio webcast can be accessed under "News/Events" through the "Investors" section of the Aurinia corporate website at www.auriniapharma.com. In order to participate in the conference call, please dial the corrected call-in number for participants +1 (877) 407-9170 / + 1 201-493-6756 (Toll-free U.S. & Canada). An audio webcast can be accessed under "News/Events" through the Investors section of the Aurinia corporate website at www.auriniapharma.com. A replay of the webcast will be available on Aurinia’s website.

On November 2, 2023 Arvinas, Inc. (Nasdaq: ARVN), a clinical-stage biotechnology company creating a new class of drugs based on targeted protein degradation, reported that management will participate in five upcoming investor conferences (Press release, Arvinas, NOV 2, 2023, View Source [SID1234636732]):

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Truist Securities BioPharma Symposium on Wednesday, November 8.
Sean Cassidy, chief financial officer, and Ron Peck, M.D., chief medical officer will be available to participate in one-on-one meetings.

Stifel Healthcare Conference on Tuesday, November 14.
John Houston, Ph.D., chairperson, chief executive officer, and president, and Ron Peck, M.D., chief medical officer, will be available to participate in one-on-one meetings and will participate in a fireside chat. A live audio webcast of the presentation will be on the Events + Presentations section of the Company’s website.

Jefferies London Healthcare Conference on Thursday, November 16.
John Houston, Ph.D., chairperson, chief executive officer, and president, and Sean Cassidy, chief financial officer will be available to participate in one-on-one meetings.

Piper Sandler 35th Annual Healthcare Conference on Tuesday, November 28.
Ian Taylor, Ph.D., chief scientific officer, and Randy Teel, Ph.D., senior vice president, corporate and business development, will be available to participate in one-on-one meetings and will participate in a fireside chat. A live audio webcast of the presentation will be on the Events + Presentations section of the Company’s website.

6th Annual Evercore ISI HealthCONx Conference on Thursday, November 30.

Ron Peck, M.D., chief medical officer and Jeff Boyle, vice president, investor relations, will be available to participate in one-on-one meetings and will participate in a fireside chat. A live audio webcast of the presentation will be on the Events + Presentations section of the Company’s website.

On November 2, 2023 Arcellx, Inc. (NASDAQ: ACLX), a biotechnology company reimagining cell therapy through the development of innovative immunotherapies for patients with cancer and other incurable diseases, reported that new clinical data from its Phase 1 study of CART-ddBCMA in patients with relapsed or refractory multiple myeloma will be presented at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition taking place December 9-12, 2023 in San Diego, California (Press release, Arcellx, NOV 2, 2023, View Source [SID1234636731]). The data in the ASH (Free ASH Whitepaper) abstract published today is from a June 2, 2023 data cut. The oral presentation at ASH (Free ASH Whitepaper) will be on Monday, December 11, 2023, at 5 p.m. PT and will include new data with a median follow-up of 26.5 months. The company will also have a medical affairs booth (#748) in Hall E of the San Diego Convention Center.

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As detailed in the abstract (#1023), 38 patients were evaluable for efficacy and safety analysis as of the June 2, 2023 cutoff date, based on a median follow-up of 22 months following treatment. These evaluable patients comprised the dose escalation cohorts for the first dose level (100 (+/- 20) million CAR+ T cells, n=6) and the second dose level (300 (+/- 20) million CAR+ T cells, n=6), and a dose expansion cohort at 100 (+/- 20) million CAR+ T cells (n=26). The median dose administered to patients in the first dose level and dose expansion cohorts was 115 million CAR+ T cells. All patients evaluable for this analysis have poor prognostic factors with 38 of 38 (100%) patients triple-refractory, 26 of 38 (68%) penta-refractory, and 34 of 38 (89%) refractory to last-line of treatment by International Myeloma Working Group (IMWG) criteria. Additionally, 9 of 38 patients (24%) patients had high tumor burden with >60% bone marrow plasma (BMPC) cells, 13 of 38 patients (34%) patients had extramedullary disease (EMD), and 11 of 38 (29%) patients had high-risk cytogenetics (Del 17p, t(14;16), t(4;14)) at baseline. At baseline, 24 of 38 (63%) had at least one high risk clinical feature, defined as presence of EMD, BMPC >60% or B2M >5.5. All 38 patients had at least three prior lines of therapy.

The interim CART-ddBCMA Phase 1 clinical results (June 2, 2023 cutoff date) demonstrate deep and durable responses in patients with poor prognostic factors.

All Patients:

Of the 38 evaluable patients with a median follow-up of 22 months

•
100% overall response rate (ORR) achieved in all patients per IMWG criteria

•
29 of 38 evaluable patients achieved a complete response (CR) or a stringent complete response (sCR) (> CR rate, 76%)

•
35 patients achieved a very good partial response or higher (>VGPR rate, 92%)

Median duration of response, progression free survival (PFS), and overall survival were not reached at the time of the June 2, 2023 data cut because 25 of 38 (66%) evaluable patients had ongoing responses.

The Kaplan-Meier method estimated PFS rates for 6, 12, and 18 months were 92%, 74%, and 67% respectively. Durable responses were also observed in patients with high-risk features (EMD, BMPC ≥ 60%, or B2M ≥ 5.5 mg/L at baseline) and high-risk cytogenetics.

PFS rates at 6-, 12-, and 18 months by Kaplan-Meier method were:

PFS Rates (%)
6-month 12-month 18-month
All dosed (n=38)

92.1 74.3 67.5
Age ≥65 years (n=20)

95.0 84.4 78.4
Complete responders (n=29)

96.4 88.8 84.6
High Risk Features* (n=24)

91.7 73.3 68.1
Extramedullary Disease (n=13)

92.3 64.6 64.6
High Risk Cytogenetics (n=11)

81.8 70.1 70.1

*
High risk features defined as presence of EMD, BMPC ≥ 60, or B2M ≥5.5 mg/L. Note: increased from prior presentation from 22 to 24 subjects as a result of database update based on monitoring and query resolution.

CART-ddBCMA dosed at RP2D (115 million (+/- 10) CAR+ T cells) continues to be well-tolerated at the time of the data cut:

•
Adverse events with CART-ddBCMA, including CRS and ICANS, were manageable

•
No tissue-targeted toxicities were observed

•
No cases of delayed neurotoxicity events or parkinsonian symptoms were observed

ASH Presentation Details:

Title: Phase 1 Study of CART-ddBCMA for the Treatment of Patients with Relapsed and/or Refractory Multiple Myeloma: Results from at Least 1-Year Follow-up in All Patients

Speaker: Matthew J. Frigault, M.D., Clinical Director of the Cellular Therapy Service at Massachusetts General Cancer Center, and Assistant Professor at Harvard Medical School

Session Name: 704. Cellular Immunotherapies: Early Phase and Investigational Therapies: CAR-T Cell Therapies for Multiple Myeloma and B Cell Lymphomas

Session Date: Monday, December 11, 2023

Session Time: 4:30 – 6:00 p.m. PT (CART-ddBCMA oral presentation will be at 5 p.m. PT)

Location: San Diego Convention Center, Room 6A, San Diego, California

Publication Number: 1023

Webcast Event:

Arcellx will host a live webcast event with an expert panel of clinicians to discuss the clinical results on Monday, December 11, 2023 at 8 p.m. PT. The event will be accessible from Arcellx’s website at www.arcellx.com in the Investors section. A replay of the webcast will be archived and available for 30 days following the event.

About Multiple Myeloma

Multiple Myeloma (MM) is a type of hematological cancer in which diseased plasma cells proliferate and accumulate in the bone marrow, crowding out healthy blood cells and causing bone lesions, loss of bone density, and bone fractures. These abnormal plasma cells also produce excessive quantities of an abnormal immunoglobulin fragment, called a myeloma protein (M protein), causing kidney damage and impairing the patient’s immune function. Multiple myeloma is the third most common hematological malignancy in the United States and Europe, representing approximately 10% of all hematological cancer cases and 20% of deaths due to hematological malignancies. The median age of patients at diagnosis is 69 years with one-third of patients diagnosed at an age of at least 75 years. Because MM tends to afflict patients at an advanced stage of life, patients often have multiple co-morbidities and toxicities that can quickly escalate and become life-endangering.

About CART-ddBCMA

CART-ddBCMA is Arcellx’s BCMA-specific CAR-modified T-cell therapy utilizing the company’s novel BCMA-targeting binding domain for the treatment of patients with relapsed or refractory multiple myeloma. CART-ddBCMA is currently in a Phase 2 study. Arcellx’s proprietary binding domains are novel synthetic proteins designed to bind specific therapeutic targets. CART-ddBCMA has been granted Fast Track, Orphan Drug, and Regenerative Medicine Advanced Therapy Designations by the U.S. Food and Drug Administration.