On February 28, 2023 Arcus Biosciences, Inc. (NYSE:RCUS), a clinical-stage, global biopharmaceutical company focused on developing differentiated molecules and combination therapies for people with cancer, reported financial results for the fourth-quarter and full-year ended December 31, 2022 and provided a pipeline update on its six clinical-stage investigational molecules – targeting TIGIT, the adenosine axis (CD73 and A2a/A2b receptors), HIF-2a and PD-1 – across multiple common cancers (Press release, Arcus Biosciences, FEB 28, 2023, View Source [SID1234627802]).

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"During 2022, Arcus established itself as a late-stage company and made tremendous progress in the advancement of its pipeline. This included the initiation of multiple registrational Phase 3 studies evaluating domvanalimab-based combinations versus standard of care, as well as the advancement of AB521, our HIF-2a inhibitor, into a Phase 1/1b study in patients," said Terry Rosen, Ph.D., chief executive officer of Arcus. "Importantly, we also presented an interim analysis of ARC-7 data in December, demonstrating the potential for our domvanalimab-based combination to be a best-in-class anti-TIGIT/anti-PD-1 combination. In 2023, we expect multiple Phase 1b and Phase 2 clinical readouts and advancement into the clinic of at least two new drug candidates. With $1.1 billion in cash and runway into 2026, we are well-positioned to establish Arcus as a leader in the development of innovative therapies for lung and GI cancers."

Pipeline Highlights:

Domvanalimab (Fc-silent anti-TIGIT monoclonal antibody)

Domvanalimab Updates:

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In 2022, Arcus and Gilead advanced their broad development strategy for domvanalimab, which now includes eight ongoing Phase 2 and Phase 3 studies evaluating domvanalimab-based combinations in non-small cell lung cancer (NSCLC) and upper gastrointestinal (GI) cancers.
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The companies now have four ongoing registrational Phase 3 studies: three of the studies are in multiple NSCLC settings (ARC-10, STAR-121 and Pacific-8) and one study is in upper GI cancer (STAR-221).
o
There are also four ongoing Phase 2 studies to evaluate domvanalimab-based combinations: three in NSCLC and one in upper GI cancer.

Interim data from ARC-7, a 150-patient, randomized Phase 2 study evaluating the safety and efficacy of zimberelimab alone vs. domvanalimab plus zimberelimab ("doublet") vs. domvanalimab plus zimberelimab and etrumadenant ("triplet") in 1L PD-L1 ≥50% metastatic NSCLC, were presented on December 20, 2022 at the ASCO (Free ASCO Whitepaper) Monthly Plenary Series.
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At the time of data cutoff, efficacy was evaluated in patients who started treatment at least 13 weeks prior and were therefore potentially eligible for at least two imaging scans (n=133).
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With a median follow-up time of approximately 12 months, a doubling of median progression-free survival (mPFS) was observed in each of the doublet and triplet arms, compared to zimberelimab monotherapy. The doublet and triplet arms demonstrated 12.0 months and 10.9 months mPFS, respectively, compared to 5.4 months observed in the zimberelimab monotherapy arm.
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The doublet and triplet combinations demonstrated, respectively, a 45% and 35% reduction in risk of progression or death compared to zimberelimab monotherapy.
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The domvanalimab-containing arms also demonstrated clinically meaningful improvements in six-month landmark PFS rates and objective response rate (ORR) compared to zimberelimab monotherapy.
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Safety was evaluated in all enrolled patients who received at least one dose (n=149). No unexpected safety signals were observed across the three study arms at the time of data cutoff. The domvanalimab-containing study arms appeared to be generally well tolerated and showed an overall safety profile consistent with the known safety profiles of each individual molecule to date.
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During the fourth quarter, Taiho Pharmaceutical, Arcus’s partner in Japan and other territories in Asia, opted to participate in two Phase 3 trials of domvanalimab combinations, STAR-121 in NSCLC and STAR-221 in upper GI cancer. Due to Taiho’s participation, Arcus expects to receive certain milestone payments from Taiho in 2023.
Upcoming Domvanalimab Milestones:

•
An updated analysis of the ARC-7 study, including efficacy evaluation for all 150 patients, is expected to be presented at the ASCO (Free ASCO Whitepaper) Annual Meeting in June 2023.
Etrumadenant (A2a/A2b adenosine receptor antagonist)

Etrumadenant Updates:

•
In the fourth quarter, Arcus and Gilead initiated VELOCITY-Lung, a Phase 2 platform study, operationalized by Gilead, which is evaluating domvanalimab-, etrumadenant-, and zimberelimab-based combinations in advanced NSCLC.
Upcoming Etrumadenant Milestones:

•
Data from the randomized cohort of ARC-6, a Phase 1b/2 study evaluating etrumadenant plus zimberelimab and docetaxel versus docetaxel in metastatic castrate-resistant prostate cancer (CRPC), are expected in 2023.
•
Data from ARC-9, a Phase 1b/2 study evaluating etrumadenant-based combinations in 2L and third-line (3L) metastatic colorectal cancer (mCRC), are expected in 2023.
Quemliclustat (small-molecule CD73 inhibitor)

Quemliclustat Updates:

•
In the fourth quarter, Arcus and Gilead initiated EDGE-Lung, a Phase 2 platform study to evaluate domvanalimab-, quemliclustat-, and zimberelimab-based combinations in advanced NSCLC.
Upcoming Quemliclustat Milestones:

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In the first half of 2023, Arcus and Gilead expect PFS and OS data from all 90 patients in the ongoing Phase 1/1b ARC-8 trial evaluating quemliclustat plus chemotherapy with or without zimberelimab in first-line pancreatic cancer.
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Arcus and Gilead expect to initiate one or more cohorts with quemliclustat-based combinations in GI cancers in the ongoing Phase 2 ARC-21 study.

AB521 (HIF-2a inhibitor)

Upcoming AB521 Milestones:

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During the fourth quarter, Arcus completed Cohort 1 of ARC-20, a Phase 1/1b study of AB521 in cancer patients; at a dose level of 20 mg daily, deep reductions in serum erythropoietin were observed, which was consistent with observations from the ARC-14 study, evaluating AB521 in healthy volunteers.
o
Arcus is enrolling Cohort 2, evaluating a 50 mg daily dose, which Arcus believes may achieve greater drug levels than the marketed competitor.
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Initial data from ARC-20 are anticipated in late 2023 or early 2024.
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A Phase 2 study evaluating AB521 in combination with other agents is anticipated for the second half of 2023.
Discovery Programs

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Arcus is on track to initiate a Phase 1 trial in cancer patients for AB598, its anti-CD39 antibody, in the first half of 2023.
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Arcus plans to initiate a Phase 1 trial for AB801, a potent and highly selective Axl inhibitor, in 2023. The early development plan is expected to focus on treatment-resistant tumor types, such as STK11-mutant NSCLC.
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Arcus expects to advance its first candidate against an inflammation target, AB375, a highly selective KIT inhibitor, into the clinic in early 2024.
Financial Results for the Fourth-Quarter and Full-Year 2022

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Cash, cash equivalents and marketable securities were $1.1 billion as of December 31, 2022, compared to $681 million as of December 31, 2021. The increase was primarily due to the receipt of $725 million from Gilead in January 2022. Arcus expects cash utilization for 2023 to be between $300 million and $350 million. Its cash, cash equivalents and marketable securities on-hand is expected to be sufficient to fund operations into 2026.
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Revenues were $34 million for the fourth-quarter 2022, compared to $355 million for the same period in 2021. In the fourth-quarter 2022, Arcus recognized $26 million in license and development service revenues for programs optioned by Gilead. Arcus further recognized $8 million in collaboration revenue related to Gilead’s ongoing rights to access Arcus’s research and development pipeline in accordance with the Gilead collaboration agreement. Revenues were $112 million for the full-year 2022, compared to $383 million for the same period in 2021.
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Research and Development (R&D) Expenses were $80 million for the fourth-quarter 2022, compared to $51 million for the same period in 2021. Arcus’s expanding clinical and development activities increased costs by $60 million partially offset by $31 million in higher reimbursements for shared expenses from Arcus’s collaborations, primarily the Gilead collaboration, which was expanded in December 2021. The $29 million increase in R&D costs net of reimbursements was driven by Arcus’s expanding clinical and development activities as Arcus enrolled more patients in its existing and new studies, which drove increases of $12 million in net clinical costs and $9 million in net manufacturing costs. Arcus’s growing headcount drove a $5 million increase in net employee compensation costs. For fourth-quarter 2022 and 2021, Arcus recognized gross reimbursements of $49 million and $18 million, respectively, for shared expenses from its collaborations, primarily the Gilead collaboration. For the full-year 2022 and 2021, R&D expenses were $288 million and $257 million, respectively. This includes gross reimbursements of $161 million and $25 million, respectively, for shared expenses from Arcus’s collaborations, primarily the Gilead collaboration, which was expanded in December 2021.
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General and Administrative (G&A) Expenses were $28 million for the fourth-quarter 2022, compared to $23 million for the same period in 2021. The increase was driven by the increased complexity of supporting Arcus’s expanding clinical pipeline and partnership obligations. Arcus’s growing headcount and 2022 stock awards drove a $4 million increase in employee compensation costs, including a $2 million increase in non-cash stock-based compensation, as well as increases in office facilities due to the expansion of office space to support the higher headcount. G&A expenses were $104 million for the full-year 2022, compared to $72 million for the same period in 2021.
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Net Income (Loss): Net loss was $67 million for the fourth-quarter 2022, compared to a net income of $280 million for the same period in 2021. Net loss was $267 million for the full-year 2022, compared to a net income of $53 million for the same period in 2021.

Arcus Ongoing and Announced Clinical Studies

Trial Name

Arms

Setting

Status

NCT No.

Lung Cancer

ARC-7

zim vs. dom + zim vs. etruma + dom + zim

1L NSCLC (PD-L1 ≥ 50%)

Ongoing Randomized Phase 2

NCT04262856

PACIFIC-8

(Operationalized by AZ)

dom + durva vs. durva

Curative-Intent Stage 3 NSCLC

Ongoing Registrational Phase 3

NCT05211895

ARC-10

dom + zim vs. pembro

1L NSCLC (PD-L1 ≥ 50%)

Ongoing Registrational Phase 3

NCT04736173

STAR-121

(Operationalized by GILD)

dom + zim + chemo vs. pembro + chemo

1L NSCLC (PD-L1 all-comers)

Ongoing Registrational Phase 3

NCT05502237

EDGE-Lung

dom +/- zim +/- quemli +/- chemo

1L/2L NSCLC (lung cancer platform study)

Ongoing Randomized Phase 2

NCT05676931

VELOCITY-Lung

(Operationalized by GILD)

dom +/- zim +/- etruma +/- sacituzumab govitecan-hziy or other combos

1L/2L NSCLC (lung cancer platform study)

Ongoing Randomized Phase 2

NCT05633667

Gastrointestinal Cancers

ARC-9

etruma + zim + mFOLFOX vs. SOC

2L/3L/3L+ CRC

Ongoing

Randomized Phase 2

NCT04660812

EDGE-Gastric

(ARC-21)

dom + zim ± chemo

1L/2L Upper GI Malignancies

Ongoing

Randomized Phase 2

NCT05329766

STAR-221

dom + zim + chemo vs. nivo + chemo

1L Gastric, Gastroesophageal Junction (GEJ), and Esophageal Adenocarcinoma (EAC)

Ongoing Registrational Phase 3

NCT05568095

Pancreatic Cancer

ARC-8

quemli + zim + gem/nab-pac vs. quemli + gem/nab-pac

1L, 2L PDAC

Ongoing Randomized Phase 1/1b

NCT04104672

Prostate Cancer

ARC-6

etruma + zim + SOC vs. SOC (also enrolling sacituzumab govitecan-hziy combination cohorts)

2L/3L CRPC

Ongoing Randomized Phase 2

NCT04381832

Various

ARC-12

AB308 + zim

Advanced Malignancies

Ongoing

Phase 1/1b

NCT04772989

ARC-14

AB521

Healthy Volunteers

Ongoing

NCT05117554

ARC-20

AB521

Cancer Patients / ccRCC

Ongoing Phase 1/1b

NCT05536141

dom: domvanalimab; durva: durvalumab; etruma: etrumadenant; gem/nab-pac: gemcitabine/nab-paclitaxel; nivo: nivolumab; pembro: pembrolizumab; quemli: quemliclustat; SOC: standard-of-care; zim: zimberelimab

ccRCC: clear-cell renal cell carcinoma; CRC: colorectal cancer; CRPC: castrate-resistant prostate cancer; GI: gastrointestinal; NSCLC: non-small cell lung cancer; PDAC: pancreatic ductal adenocarcinoma

About the Gilead Collaboration

In May 2020, Gilead and Arcus entered into a 10-year collaboration that provided Gilead immediate rights to zimberelimab and the right to opt into all other Arcus programs arising during the collaboration term. In November 2021, Gilead and Arcus

amended the collaboration in connection with Gilead’s option exercise for three of Arcus’s then-clinical stage programs. For all other programs that are in clinical development or new programs that enter clinical development thereafter, the opt-in payments are $150 million per program. Gilead’s option, on a program-by-program basis, expires after a specified period of time following the achievement of a development milestone for such program and Arcus’s delivery to Gilead of the requisite qualifying data package. Concurrent with the May 2020 collaboration agreement, Gilead and Arcus entered into a stock purchase agreement under which Gilead made a $200 million equity investment in Arcus. That stock purchase agreement was amended and restated in February 2021 in connection with Gilead’s increased equity stake in Arcus from 13% to 19.5%, with an additional $220 million investment.

Pursuant to the collaboration, Gilead and Arcus are currently co-developing and equally sharing global development costs for five clinical candidates, including: domvanalimab, an Fc-silent anti-TIGIT antibody; etrumadenant, a dual adenosine A2a/A2b receptor antagonist; quemliclustat, a small molecule inhibitor of CD73; and zimberelimab, an anti-PD1 antibody.

On February 28, 2023 Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T) products for cancer, reported a corporate update and reported financial results for the quarter and year ended December 31, 2022 (Filing, 3 mnth, DEC 31, Allogene, 2022, FEB 28, 2023, View Source [SID1234627801]).

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"We are very proud that data from our pipeline candidates continues to break new ground in the field of allogeneic cell therapy. From our CD19 program for non-Hodgkin lymphoma to our BCMA program for multiple myeloma, we have established that our AlloCAR T technology can induce deep, clinically meaningful responses in patients," said David Chang, M.D., Ph.D., President, Chief Executive Officer and Co-Founder of Allogene. "We continue to hear from physicians that gaining access to autologous CAR T therapy and the inevitable wait times associated with manufacturing remain a critical factor for patients. We believe the future of CAR T rests on the ability for an off-the-shelf option to address both time and access. The data we presented at our R&D Showcase in late 2022 indicates Allogene is making large strides toward giving patients back precious time."

Pipeline Updates

ALLO-501A: Anti-CD19 AlloCAR T Program

In October 2022, Allogene initiated the industry’s first potentially pivotal Phase 2 allogeneic CAR T clinical trial with ALLO-501A. The single-arm trial is enrolling patients with relapsed/refractory (r/r) large B cell lymphoma (LBCL) and utilizes a single dose of ALLO-501A (120 million CAR+ cells) with the FCA90 (fludarabine, 30mg/m2, cyclophosphamide 300 mg/m2 and ALLO-647 30 mg, daily for 3 days) lymphodepletion regimen. The ALPHA2 trial will enroll approximately 100 patients who have received at least two prior lines of therapy and have not received prior anti-CD19 therapy. The primary endpoint of this trial is overall response rate (ORR), and the key secondary endpoint is duration of response (DoR). Patients may receive treatment as an outpatient at the investigator’s discretion. The Company expects to complete enrollment in the Phase 2 ALPHA2 trial in 1H 2024.

In November 2022, Phase 1 data from the ALPHA trial with ALLO-501 and ALPHA2 trial with ALLO-501A for the treatment of r/r LBCL was presented at the Company’s R&D Showcase. Data from the Phase 1 trials of ALLO-501 and ALLO-501A support the ability of a single administration of CAR T cells to generate deep and durable responses comparable to those with approved autologous CAR T therapies. Highlights included:
• As of the October 25, 2022 data cutoff, the ORR and Complete Response (CR) rate was 67% and 58%, respectively, among the 12 patients treated with the Single Dose FCA90 regimen using Alloy process material. The median duration of response was 23.1 months.
• Of patients who received single dose FCA90 and evaluable at six months, the ongoing CR rate was 50% and all CRs at six months were durable at 12 months. The longest CR ongoing at 26+ months.

• Phase 1 trials demonstrated a manageable safety profile with no observed dose limiting toxicities (DLTs), graft-vs-host disease (GvHD) or severe immune effector cell-associated neurotoxicity syndrome (ICANS).

• Among patients treated with Single Dose FCA90, there was no Grade 3+ cytokine release syndrome (CRS). One patient (8%) experienced a Grade 3+ infection and two (17%) experienced prolonged Grade 3+ cytopenia.

• 92% of all enrolled patients received product with 100% of infused product manufactured and released per product specifications. Patients were able to initiate treatment within two days of enrollment.

The Company is preparing for a Phase 3 study in earlier line LBCL targeting trial initiation in 1H 2024.

The Company is developing ALLO-647, its proprietary anti-CD52 monoclonal antibody intended to enable expansion and persistence of AlloCAR T product candidates, including ALLO-501A. The EXPAND trial, which is intended to demonstrate the contribution of ALLO-647 to the lymphodepletion regimen, will be open to enrollment early in the second quarter.

ALLO-715: Anti-BCMA AlloCAR T Programs

Data from the Phase 1 UNIVERSAL trial with ALLO-715 for the treatment of r/r multiple myeloma (MM) was also presented at the Company’s R&D Showcase and subsequently published in Nature Medicine, accompanied by an editorial. The UNIVERSAL trial is the first allogeneic anti-BCMA CAR T to demonstrate proof-of-concept in MM with response rates that are similar to an approved autologous CAR T therapy. Highlights include:
• Dose expansion cohorts demonstrated substantial and durable responses.
• Through a median follow-up of 14.8 months as of the October 11, 2022 data cutoff, the ORR was 67% in the FCA60 cohort and the very good partial response or better rate (VGPR+) was 42%. All VGPR+ were minimal residual disease (MRD) negative.

• The median DoR was 9.2 months, with the longest response ongoing at 24 months.
• 92% of all enrolled patients received product with 100% of infused product manufactured and released as per product specifications. None of the patients received bridging therapy and patients were able to initiate treatment immediately following enrollment. Median time from enrollment to lymphodepletion was 5 days.

• Safety profile was manageable with low-grade and reversible neurotoxicity and no GvHD. Eight patients (29%) experienced Grade 3+ infections and eight patients experienced prolonged Grade 3+ cytopenias.

The Company is evaluating manufacturing processes improvements across its BCMA candidates to achieve optimal performance.

ALLO-316: Anti-CD70 AlloCAR T Program

ALLO-316, the Company’s first AlloCAR T candidate for solid tumors, targets CD70, an antigen expressed on clear cell renal cell carcinoma (RCC) and other malignancies. At the Company’s R&D Showcase, the Company presented initial data demonstrating promising anti-cancer activity in the subset of nine patients with confirmed CD70-positive RCC from the ongoing Phase 1 TRAVERSE trial. Highlights include:

• As of the data cutoff date of November 17, 2022, the disease control rate (DCR) in patients who were CD70+ was 100% including three patients who achieved a partial response (PR) (two confirmed and one unconfirmed with the longest response lasting until month eight).

• Cell expansion in patients with CD70 positive tumor was robust, and there was a trend toward greater tumor shrinkage in patients with high CD70 expression.

• Across all patients treated in the trial, ALLO-316 has demonstrated a generally manageable safety profile with no GvHD. One dose limiting toxicity of auto-immune hepatitis occurred in the second dose level. Grade 3+ prolonged cytopenia was observed in three patients (18%). Grade 3 CRS was observed in one patient. Neurotoxicity was low grade, reversible and seen in only three patients (18%).

The Company is deploying a new investigational in vitro companion diagnostic (IVD) assay designed to prospectively assess CD70 expression levels to enhance patient selection. TRAVERSE will continue to explore varying cell dose and lymphodepletion regimens, including FC and FCA. Subject to ongoing results in the TRAVERSE trial, the Company intends to complete planned dose exploration and initiate expansion cohort enrollment in 2023. The Company may also investigate ALLO-316 for other CD70 expressing solid tumors and hematologic indications, or in combination with other anticancer therapies such as immune checkpoint inhibitors.

Next Generation Platform Technology

Allogene has pursued an integrated strategy within Research and Development aimed at matching technology with insights obtained from the clinic to create solutions designed to advance patient outcomes. One of these is Dagger, a proprietary technology designed to control rejection of AlloCAR T cells by the host immune cells. This technology deploys a CD70 CAR on AlloCAR T cells in an effort to recognize and deplete CD70 positive alloreactive host T cells. Preclinical data indicate that CD70 Dagger CARs can be combined with other anti-tumor CARs in a single cell, providing both protection from allorejection and dual specificity killing capability, thus offering a differentiated next generation product candidate profile.

Fourth Quarter and Year-End Financial Results

• Research and development expenses were $75.4 million for the fourth quarter of 2022, which includes $7.4 million of non-cash stock-based compensation expense. For the full year of 2022, research and development expenses were

$256.4 million. Research and development expense for the year includes $42.5 million of non-cash stock-based compensation expense.

• General and administrative expenses were $21.0 million for the fourth quarter of 2022, which includes $9.8 million of non-cash stock-based compensation expense. For the full year of 2022, general and administrative expenses were $79.3 million, which includes $41.1 million of non-cash stock-based compensation expense.
• Net loss for the fourth quarter of 2022 was $94.8 million, or $0.66 per share, including non-cash stock-based compensation expense of $17.2 million. For the full year of 2022, net loss was $332.6 million, or $2.32 per share, including non-cash stock-based compensation expense of $83.6 million.
• The Company had $576.5 million in cash, cash equivalents, and investments as of December 31, 2022.

2023 Financial Guidance

• The Company expects a decrease in cash, cash equivalents, and investments of approximately $250 million in 2023. Based on current expectation, the Company expects the cash runway to be sufficient to fund operations into 2025. GAAP Operating Expenses are expected to be approximately $350 million, including estimated non-cash stock-based compensation expense of approximately $90 million. These estimates exclude any impact from potential business development activities.

Conference Call and Webcast Details

Allogene will host a live conference call and webcast today at 2:00 p.m. Pacific Time / 5:00 p.m. Eastern Time to discuss financial results and provide a business update. If you would like the option to ask a question on the conference call, please use this link to register. Upon registering for the conference call, you will receive a personal PIN to access the call, which will identify you as the participant and allow you the option to ask a question. The listen-only webcast will be made available on the Company’s website at www.allogene.com under the Investors tab in the News and Events section. Following the live audio webcast, a replay will be available on the Company’s website for approximately 30 days

On February 28, 2023 AIM ImmunoTech Inc. (NYSE American: AIM) ("AIM" or the "Company"), an immuno-pharma company focused on the research and development of therapeutics to treat multiple types of cancers, immune disorders, and viral diseases – including COVID-19, the disease caused by the SARS-CoV-2 virus – reported that the AMP-270 Phase 2 study of Ampligen as a therapy for locally advanced pancreatic cancer ("LAPC") has opened its first clinical site and has begun recruiting patients (Press release, AIM ImmunoTech, FEB 28, 2023, View Source [SID1234627800]).

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The AMP-270 clinical trial is a randomized, open-label, controlled, parallel-arm study with the primary objective of comparing the efficacy of Ampligen versus a no treatment control group following FOLFIRINOX for subjects with locally advanced pancreatic adenocarcinoma. Secondary objectives include comparing safety and tolerability.

Thomas K. Equels, M.S., J.D., Chief Executive Officer of AIM ImmunoTech, commented, "We continue to believe that Ampligen and the AMP-270 study have the potential to establish a much-needed new standard of care for LAPC where there remains a significant unmet need. Our team continues to execute on advancing this important study forward and we are pleased that the first of our sites is open for recruitment. We are working closely with preeminent cancer institutions across the United States and Europe to build momentum toward the commencement of patient enrollment and dosing and look forward to achieving those milestones as soon as possible."

AMP-270 is expected to enroll approximately 90 subjects in up to 30 centers across the United States and Europe. The site at Gabrail Cancer & Research Center in Canton, Ohio, has opened, with additional clinical sites at other premier cancer centers across the U.S. and Europe expected to open and enroll patients in the near future.

For more information about the AMP-270 please visit ClinicalTrials.gov and reference identifier NCT05494697.

About Ampligen

Ampligen is AIM’s dsRNA product candidate being developed for globally important cancers, viral diseases and disorders of the immune system. Ampligen has demonstrated in the clinic the potential for standalone efficacy in a number of solid tumors. Additionally, Ampligen has shown success in increasing survival rates and efficacy in the treatment of animal tumors when used in combination with checkpoint blockade therapies.

Ampligen is currently being evaluated as a combinational therapy for the treatment of a variety of solid tumor types in multiple clinical trials – both underway and planned – at major cancer research centers around the country. Ampligen is being used to treat pancreatic cancer patients in an Early Access Program approved by the Inspectorate of Healthcare in the Netherlands at Erasmus Medical Center. Additionally, Ampligen is also approved in Argentina for the treatment of severe chronic fatigue syndrome and is currently being evaluated in SARS-CoV-2/COVID-19, myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS) and Post COVID Conditions.

On February 28, 2023 ADC Therapeutics SA (NYSE: ADCT) reported financial results for the fourth quarter and full year ended December 31, 2022, and provided business updates (Press release, ADC Therapeutics, FEB 28, 2023, View Source [SID1234627799]).

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Recent Highlights and Developments

ZYNLONTA (loncastuximab tesirine-lpyl)

• Revenue from the net sales of ZYNLONTA in the fourth quarter of 2022 increased to $19.8 million, a 16.5% increase over the same quarter in 2021, and $74.9 million for the full year 2022, partially offset by higher gross-to-net sales deductions. The sequential third quarter to fourth quarter slight decline of 7.0% in net sales was due to higher gross-to-net and individual ordering account fluctuations.

• The European Commission (EC) and the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) granted conditional marketing authorization for the use of ZYNLONTA (loncastuximab tesirine) for the treatment of relapsed or refractory diffuse large B-cell lymphoma (DLBCL).

• The Company entered into a collaboration and clinical supply agreement with Roche to evaluate ZYNLONTA in combination with glofitamab and mosunetuzumab in addition to polatuzumab in the Phase 1 LOTIS-7 trial.

Cami (camidanlumab tesirine)

• Based on the positive pivotal Phase 2 data, the Company is actively seeking a partner to progress this program.

Pipeline

• ADCT-901 (targeting KAAG1): Dose escalation in the Phase 1 single-agent trial is progressing. The Company is amending the protocol to explore different dosing schedules to optimize the potential clinical outcomes for patients and in preparation for regulatory interactions.

• ADCT-601 (targeting AXL): Dose escalation in the Phase 1b trial is progressing and the IHC assay is under final validation. The study is comprised of a monotherapy arm including patients with sarcoma, non-small cell lung cancer and those with AXL gene amplification and a combination arm with gemcitabine in patients with sarcoma.

• ADCT-602 (targeting CD22): Initial data showing encouraging clinical activity from the Phase 1 study of ADCT-602 for patients with relapsed or refractory acute lymphoblastic leukemia were released in an oral presentation at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting by The University of Texas MD Anderson Cancer Center.

Corporate Update

• Mohamed Zaki, MD, PhD, was appointed the Company’s new Chief Medical Officer (CMO), effective January 3, 2023. Dr. Zaki is a pharmaceutical industry veteran with over 20 years of experience in oncology and hematology drug development. Most recently, he served as Vice President & Global Head of Oncology Development at AbbVie. Dr. Zaki worked on ibrutinib, venetoclax, lenalidomide and pomalidomide, among many other notable therapies.

• Jose "Pepe" Carmona was appointed the Company’s new Chief Financial Officer (CFO), effective December 19, 2022. Mr. Carmona is a seasoned CFO with over 20 years of financial leadership optimizing capital formation, developing and implementing capital allocation strategies, managing multibillion-dollar international commercial businesses and executing partnerships in the pharmaceutical and biotech industry, both in the U.S. and internationally.

Upcoming Expected Milestones

ZYNLONTA

• European phased launch by partner Sobi in 2Q 2023

• Complete enrollment of the LOTIS-5 study in 2024

• Preliminary safety and efficacy data from the LOTIS-9 study in 2024

• Preliminary safety and efficacy data from the LOTIS-7 study in 2024

Pipeline

ADCT-901 (targeting KAAG1)

• Preliminary data from Phase 1 dose escalation study in 1H 2024

ADCT-601 (targeting AXL)

• Preliminary data from Phase 1 dose escalation/expansion study in 1H 2024

ADCT-212 (targeting PSMA)

• Initiate Phase 1 study in 1H 2024

ADCT-602 (targeting CD22)

• Complete Phase 1 dose expansion study in 1H 2024

ADCT-701 (targeting DLK-1)

• Initiate Phase 1 study in 2H 2023

Fourth Quarter and Full Year Financial Results

Cash and Cash Equivalents

Cash and cash equivalents were $326.4 million as of December 31, 2022, compared to $380.9 million as of September 30, 2022. This does not include the $50.0 million milestone received in February 2023 from Sobi, triggered by European regulatory approval of ZYNLONTA in third-line DLBCL. Based on the Company’s business plan and expected $75.0 million milestone from HealthCare Royalty Partners, triggered by the first EU commercial sale, the Company expects its cash runway now extends into the middle of 2025.

Product Revenue

Net product revenue was $19.8 million for the fourth quarter and $74.9 million for the full year ended 2022, compared to $17.0 million for the same quarter and $33.9 million for the full year ended 2021. Net product revenues are for U.S. sales of ZYNLONTA which commenced in May 2021. The 120.9% year-over-year increase was principally due to a full year of sales activity in 2022 as compared to a partial year in 2021. The increase in sales volume was partially offset with higher gross-to-net deductions.

License Revenue

License revenue was $50.0 million for the fourth quarter of 2022. The Company recognized $50.0 million from Sobi for the European regulatory approval of ZYNLONTA in third-line DLBCL. License revenue was $135.0 million for the full year 2022 and included the $50.0 million European regulatory approval milestone from Sobi, as well as $85.0 million in upfront payments from our partners, Sobi and Mitsubishi Tanabe Pharma Corporation.

Cost of Product Sales

Cost of product sales was $0.5 million for the fourth quarter and $4.6 million for the full year 2022, compared to $0.8 million for the same quarter and $1.4 million for the year full 2021. The increase of $3.2 million for the year was primarily associated with $2.5 million of impairment charges for product intermediates and antibodies that did not meet the Company’s specifications. The specification issues did not, and are not expected to, impact the Company’s ability to supply commercial product. In addition, cost of product sales increased due to a full year of sales activity in 2022 as compared to 2021 due to the commencement of ZYNLONTA sales in May 2021.

Research and Development (R&D) Expenses

R&D expenses were $48.7 million for the fourth quarter and $187.9 million for the full year 2022, compared to $42.5 million for the same quarter and $158.0 million for the full year 2021. R&D expenses increased as a result of continued investments in the pipeline.

Selling and Marketing (S&M) Expenses

S&M expenses were $16.2 million for the fourth quarter and $69.1 million for the full year 2022, as compared to $18.6 million for the same quarter and $64.8 million for the full year 2021. The decrease in S&M expenses for the quarter was primarily due to lower share-based compensation expense. The increase in S&M expenses during the year is primarily due to increased professional expenses relating to the commercial launch of ZYNLONTA during the year partially offset by lower share-based compensation expense.

General and Administrative (G&A) Expenses

G&A expenses were $15.1 million for the fourth quarter and $72.0 million for the full year 2022, compared to $17.9 million for the same quarter and $71.5 million for the full year. G&A expenses decreased during the fourth quarter primarily due to lower share-based compensation expense. G&A expenses for the full year increased primarily due to higher professional fees and costs associated with the CEO transition, partially offset by lower share-based compensation expense.

Net Loss and Adjusted Net Loss

Net loss was $24.2 million, or a net loss of $0.30 per basic and diluted share for the fourth quarter and $155.8 million, or a net loss of $1.99 per basic and diluted share for the year ended December 31, 2022. This compares to a net loss of $34.4 million, or a net loss of $0.45 per basic and diluted share, for the same quarter and $230.0 million, or a net loss of $3.00 per basic and diluted share, for the year ended December 31, 2021.

Adjusted net loss, which is a non-IFRS financial measure, was $7.9 million, or an adjusted net loss of $0.10 per basic and diluted share for the fourth quarter and $81.7 million, or an adjusted net loss of $1.05 per basic and diluted share for the full year ended December 31, 2022. This compares to an adjusted net loss of $30.0 million, or an adjusted net loss of $0.39 per basic and diluted share, for the same quarter and $186.1 million, or an adjusted net loss of $2.42 per basic and diluted share, for the year ended December 31, 2021.

The decrease in net loss and adjusted net loss for the quarter and year ended December 31, 2022, as compared to the same period in 2021, was primarily due to higher product and license revenue, partially offset by the increase in operating expenses. The decrease in net loss for the quarter and year-ended December 31, 2022 was also attributable to lower share-based compensation expense and

income arising from changes in the fair value of our convertible loan derivatives and warrant obligations, offset by the extinguishment of our convertible loans and derivatives.

About ZYNLONTA (loncastuximab tesirine-lpyl)

ZYNLONTA is a CD19-directed antibody drug conjugate (ADC). Once bound to a CD19-expressing cell, ZYNLONTA is internalized by the cell, where enzymes release a pyrrolobenzodiazepine (PBD) payload. The potent payload binds to DNA minor groove with little distortion, remaining less visible to DNA repair mechanisms. This ultimately results in cell cycle arrest and tumor cell death.

The U.S. Food and Drug Administration (FDA) has approved ZYNLONTA (loncastuximab tesirine-lpyl) for the treatment of adult patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified (NOS), DLBCL arising from low-grade lymphoma and also high-grade B-cell lymphoma. The trial included a broad spectrum of heavily pre-treated patients (median three prior lines of therapy) with difficult-to-treat disease, including patients who did not respond to first-line therapy, patients refractory to all prior lines of therapy, patients with double/triple hit genetics and patients who had stem cell transplant and CAR-T therapy prior to their treatment with ZYNLONTA. This indication is approved by the FDA under accelerated approval based on overall response rate and continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

ZYNLONTA is also being evaluated as a therapeutic option in combination studies in other B-cell malignancies and earlier lines of therapy.

On February 28, 2023 AbbVie (NYSE: ABBV) reported that it will participate in the Cowen 43rd Annual Health Care Conference on Tuesday, March 7, 2023 (Press release, AbbVie, FEB 28, 2023, https://news.abbvie.com/news/press-releases/abbvie-to-present-at-cowen-43rd-annual-health-care-conference.htm [SID1234627798]). Robert A. Michael, vice chairman and president, Scott Reents, executive vice president, chief financial officer, Jeffrey R. Stewart, executive vice president, chief commercial officer and Roopal Thakkar, senior vice president, development and regulatory affairs and chief medical officer, will present at 8:10 a.m. Central time.

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A live audio webcast of the presentation will be accessible through AbbVie’s Investor Relations website at investors.abbvie.com. An archived edition of the session will be available later that day.