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.

On November 2, 2023 Aptose Biosciences Inc. ("Aptose" or the "Company") (NASDAQ: APTO, TSX: APS), a clinical-stage precision oncology company developing highly differentiated targeted agents to treat hematologic malignancies, reported that clinical data for tuspetinib, a once daily oral therapy, has been selected for oral presentation at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition being held December 9-12, 2023, in San Diego, CA (Press release, Aptose Biosciences, NOV 2, 2023, View Source [SID1234636730]).

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Lead investigator Naval Daver, MD, Professor, Director Leukemia Research Alliance Program, Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX, will present data from Aptose’s ongoing APTIVATE trial of tuspetinib in relapsed/refractory patients with acute myeloid leukemia.

The abstract accepted for presentation is listed below and can be viewed online at the ASH (Free ASH Whitepaper) conference website. Note that the actual presentation will include more recent updates and additional data not found in the abstract.

Oral Presentation Details

Title: Tuspetinib Myeloid Kinase Inhibitor Safety and Efficacy As Monotherapy and Combined with Venetoclax in Phase 1/2 Trial of Patients with Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML)
Publication Number: 162
Oral Presentation Session Date & Time: Saturday, December 9, 2023, 3:15 PM PT
Session Name: 616. Acute Myeloid Leukemias: Investigational Therapies, Excluding Transplantation and Cellular Immunotherapies: Novel Uses of Approved Therapeutic Agents
Location: San Diego Convention Center, Room 6A

On November 2, 2023 AnaptysBio, Inc. (Nasdaq: ANAB), a clinical-stage biotechnology company focused on delivering innovative immunology therapeutics, reported operating results for the third quarter ended September 30, 2023 and provided a business update (Press release, AnaptysBio, NOV 2, 2023, View Source [SID1234636729]).

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"We’ve made strong progress this quarter executing against our multi-year plan to develop best-in-class immune cell modulators to drive differentiated clinical outcomes in heterogeneous, systemic autoimmune and inflammatory diseases," said Daniel Faga, president and chief executive officer of Anaptys. "Enrollment is ongoing in our global Phase 2b trials in atopic dermatitis for ANB032, our BTLA agonist, and rheumatoid arthritis for rosnilimab, our PD-1 agonist, while also initiating a global Phase 2 trial in ulcerative colitis for rosnilimab in Q4 2023."

Updates on Wholly Owned Immune Cell Modulator Pipeline

Rosnilimab (PD-1 agonist antibody)

Initiated in August a global Phase 2b trial in moderate-to-severe RA
420-patient placebo-controlled trial assessing three dose levels of subcutaneously administered rosnilimab (randomized 1:1:1:1) for a 12-week treatment duration on well-established endpoints, including DAS28-CRP, CDAI and ACR20/50/70
At Week 14, rosnilimab-treated patients who achieve low disease activity, defined as CDAI<=10, are eligible to be dosed for an additional 16-week all-active treatment period and then followed for a three-month off-drug follow-up period
Top-line Week 12 data anticipated by mid 2025
Anticipate initiation in Q4 2023 of a global Phase 2 trial in moderate-to-severe UC
130-patient placebo-controlled trial assessing two dose levels of subcutaneously administered rosnilimab (randomized 1:1:1) for a 12-week treatment duration on well-established endpoints, including clinical response on modified Mayo score (mMS), clinical remission on mMS and endoscopic remission
Rosnilimab and placebo-treated patients who achieved clinical response on mMS are eligible to continue on their assigned treatment for an additional 12 weeks, while patients on placebo who are non-responders will be crossed over to the high-dose rosnilimab treatment arm, in an all-active treatment period and then followed for a three-month off-drug follow-up period
Top-line Week 12 data anticipated by H1 2026
Hosted a virtual PD-1 Agonist (Rosnilimab) R&D Event in October 2023
Replay of the audio webcast is available here
Announcing two poster presentations at American College of Rheumatology (ACR) Convergence 2023 in San Diego, Nov. 10-15, 2023. Full preliminary program is available online on the ACR website –
Optimizing PD-1 Agonist Signaling with Membrane Proximal Binding of Rosnilimab, a Clinical Stage PD-1 Agonist IgG1 Antibody (abstract #0086)
Rosnilimab, a Novel PD-1 Agonist Monoclonal Antibody, Inhibits Peripheral T Cell Proliferation and Cytokine Secretion and Reduces Circulating PD-1 High Expressing T Cells: Results from a Phase 1 Healthy Volunteer Clinical Trial (abstract #0455)

ANB032 (BTLA agonist antibody)

Enrollment ongoing for global Phase 2b trial in moderate-to-severe atopic dermatitis (AD)
160-patient placebo-controlled trial assessing three dose levels of subcutaneously administered ANB032 (randomized 1:1:1:1) for a 14-week treatment duration and then followed for a six-month off-drug follow-up period on well-established endpoints, including EASI75 and IGA 0/1
Top-line Week 14 data anticipated by year-end 2024
Presented poster on ANB032’s previously reported healthy volunteer Phase 1 data and a trial-in-progress poster presentation on ANB032’s Phase 2b study in moderate-to-severe AD at the 32 European Academy of Dermatology and Venerology (EADV) Congress in October 2023
Poster presentations are available here

ANB033 (anti-CD122 antagonist antibody)

Plan to submit an Investigational New Drug (IND) application in H1 2024

Updates on Legacy Clinical-Stage Cytokine Antagonist Programs Available for Out-Licensing

Announced positive top-line Phase 3 clinical trial results of imsidolimab (IL-36R) in generalized pustular psoriasis (GPP)
53.3% of patients who received a single dose of 750mg IV imsidolimab achieved GPPPGA 0/1 (clear or almost clear) at Week 4 (primary endpoint), compared to 13.3% of patients on placebo (p=0.0131)
Demonstrated favorable safety and tolerability with no SAEs, low incidence and no increase of infections vs. placebo and no cases of DRESS or Guillain-Barre in imsidolimab-treated patients
Only one of 30 (3.3%) imsidolimab-treated patients had detectable ADA, which were non-neutralizing
Intend to out-license imsidolimab in 2024

Updates on GSK Immuno-Oncology Financial Collaboration

GSK anticipates top-line data in H2 2024 from COSTAR Lung Phase 3 trial comparing cobolimab, a TIM-3 antagonist, plus dostarlimab, a PD-1 antagonist, plus docetaxel to dostarlimab plus docetaxel to docetaxel alone in patients with advanced NSCLC who have progressed on prior anti-PD-(L)1 therapy and chemotherapy
GSK anticipates top-line data in H1 2024 from the FIRST Phase 3 trial for platinum-based therapy with dostarlimab and niraparib versus platinum-based therapy as first-line treatment of Stage III or IV nonmucinous epithelial ovarian cancer
Anaptys has regained full global rights to GSK4074386, a Phase 2 ready LAG-3 antagonist antibody, from GSK

Year-End Cash Guidance

Reiterating cash runway through year-end 2026 with updated expected year-end 2023 cash and investments of $400 to $410 million

Third Quarter Financial Results

Cash, cash equivalents and investments totaled $453.3 million as of September 30, 2023, compared to $584.2 million as of December 31, 2022, for a decrease of $130.9 million. The decrease relates to cash used for the $50 million stock repurchase program and operating activities.
Collaboration revenue was $3.3 million and $8.2 million for the three and nine months ended September 30, 2023, compared to $1.3 million and $3.5 million for the three and nine months ended September 30, 2022. The change is due primarily to increased royalties recognized for sales of Jemperli.
Research and development expenses were $30.9 million and $98.8 million for the three and nine months ended September 30, 2023, compared to $22.1 million and $65.4 million for the three and nine months ended September 30, 2022. The increase was due primarily to manufacturing and development costs for rosnilimab, ANB032 and ANB033. The R&D non-cash, stock-based compensation expense was $2.2 million and $7.7 million for the three and nine months ended September 30, 2023 as compared to $1.5 million and $5.0 million in the same period in 2022.
General and administrative expenses were $10.2 million and $31.7 million for the three and nine months ended September 30, 2023, compared to $8.9 million and $27.2 million for the three and nine months ended September 30, 2022. The G&A non-cash, stock-based compensation expense was $5.6 million and $17.4 million for the three and nine months ended September 30, 2023 as compared to $4.7 million and $15.7 million in the same period in 2022.
Net loss was $37.3 million and $121.4 million for the three and nine months ended September 30, 2023, or a net loss per share of $1.41 and $4.49, compared to a net loss of $33.5 million and $102.3 million for the three and nine months ended September 30, 2022, or a net loss per share of $1.18 and $3.64.

On November 2, 2023 Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, reported its consolidated financial results for the third quarter ended September 30, 2023 and reviewed recent business highlights (Press release, Alnylam, NOV 2, 2023, View Source [SID1234636728]).

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"We continued to deliver strong commercial results in the third quarter, with the successful launch of AMVUTTRA contributing to 35% year-over-year growth in net product revenues," said Yvonne Greenstreet, MBChB, Chief Executive Officer of Alnylam. "While we were disappointed by the recent Complete Response Letter for ONPATTRO for the treatment of the cardiomyopathy of ATTR amyloidosis, we remain confident in the HELIOS-B Phase 3 study of vutrisiran, which is on track to deliver topline results in early 2024. We’re also excited about advancements across our broad pipeline of RNAi therapeutics, including recent positive topline results from the KARDIA-1 Phase 2 study of zilebesiran, as well as upcoming Phase 1 results for ALN-TTRsc04 and ALN-KHK by year-end. With this continued execution we remain on track to meet our Alnylam P5x25 goals of becoming a top-tier biotech company delivering sustained innovation and exceptional financial results."

Third Quarter 2023 and Recent Significant Corporate Highlights

Commercial Performance

Total TTR: ONPATTRO (patisiran) & AMVUTTRA (vutrisiran)

Achieved global net product revenues for ONPATTRO and AMVUTTRA for the third quarter of $82 million and $149 million, respectively, representing 35% total TTR reported year-over-year growth compared to Q3 2022.
Attained over 3,790 hATTR amyloidosis patients with polyneuropathy worldwide on commercial treatment with ONPATTRO or AMVUTTRA as of September 30, 2023.
Total Ultra-Rare: GIVLAARI (givosiran) & OXLUMO (lumasiran)

Achieved global net product revenues for GIVLAARI and OXLUMO for the third quarter of $54 million and $29 million, respectively, representing 33% total Ultra-Rare reported year-over-year growth compared to Q3 2022.
Attained over 625 patients worldwide on commercial GIVLAARI treatment as of September 30, 2023.
Attained over 375 patients worldwide on commercial OXLUMO treatment as of September 30, 2023.
Leqvio (inclisiran)

Leqvio launch in the U.S. and other markets is ongoing, with focus on patient on-boarding, removing access hurdles and enhancing medical education. In the U.S., Leqvio is covered at or near label for 76% of patients. More than 55% of Leqvio source of business in the U.S. is now through "Buy and Bill" acquisition mode. FDA expanded the label to include primary hyperlipidemia (patients at increased risk of ASCVD) and the removal of four adverse reactions from the safety section as well as Limitations of Use. In Q3 2023, Leqvio was approved in China and in Japan and is now approved in 93 countries.
R&D Highlights

Reported positive topline results from the KARDIA-1 Phase 2 dose-ranging study of zilebesiran, an investigational RNAi therapeutic in development to treat hypertension in patients at high cardiovascular risk.

Published results from Phase 1 study of zilebesiran in the New England Journal of Medicine.

Reported updated positive interim results for the ongoing single ascending dose portion of the Phase 1 study of ALN-APP in patients with early-onset Alzheimer’s disease at the 2023 Alzheimer’s Association International Conference and at the 16th Clinical Trials on Alzheimer’s Disease conference.

Based on the achievement of specified clinical proof-of-principle criteria for the Phase 1 ALN-APP program, Alnylam received a $100 million milestone payment from Regeneron.
Presented new 24-month results from an interim analysis of the open-label extension period of the APOLLO-B Phase 3 study of patisiran in patients with the cardiomyopathy of transthyretin-mediated amyloidosis at the Heart Failure Society of America Annual Scientific Meeting 2023.

Published results from Phase 3 APOLLO-B study of patisiran in the New England Journal of Medicine.

Sanofi reported positive data for fitusiran, in development for the treatment of hemophilia A or B, with or without inhibitors, from the Phase 3 open-label extension study (ATLAS-OLE).

Sanofi is currently in discussions with the FDA regarding filing in 2024.
Additional Business Updates

Entered into a global strategic collaboration with Roche for the co-development and co-commercialization of zilebesiran.
Recognized by Science magazine as a Top Employer for the fifth consecutive year.
Upcoming Events

Alnylam announces today that it will present a review of its RNAi platform and pipeline progress at a virtual R&D Day on December 13, 2023.

Alnylam announces today that results from the KARDIA-1 Phase 2 dose-ranging study of zilebesiran will be presented as a Late-Breaker at the American Heart Association Scientific Sessions 2023 on November 11, 2023, in Philadelphia, Pennsylvania.

In addition, in late 2023:

Alnylam intends to report topline results from the Phase 1 study of ALN-TTRsc04.
Alnylam intends to report topline results from the Phase 1 study of ALN-KHK.
Vir is conducting multiple trials evaluating the potential for ALN-HBV02 (VIR-2218) and VIR-3434 to achieve a functional cure for chronic hepatitis B. Phase 2 data readouts are on track for Q4 2023.
Vir also announced that initial Phase 2 data readouts for the SOLSTICE trial evaluating ALN-HBV02 (VIR-2218) and VIR-3434 as monotherapy and in combination for the treatment of people living with chronic hepatitis delta, the most aggressive form of viral hepatitis, are expected in Q4 2023.