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AC Immune Reports Full Year 2022 Financial Results and Provides Corporate Update

On March 16, 2023 AC Immune SA (NASDAQ: ACIU), a clinical-stage biopharmaceutical company pioneering precision medicine for neurodegenerative diseases, reported results for the year ended December 31, 2022, and provided a corporate update (Press release, AC Immune, MAR 16, 2023, View Source [SID1234628886]).

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Dr. Andrea Pfeifer, CEO of AC Immune SA, commented: "Thanks to the strong progress by our experienced team over the past year, we now have highly innovative vaccines targeting neurotoxic species of Abeta, Tau or a-syn in mid- to late-stage development for Alzheimer’s and Parkinson’s disease, respectively. This progress was highlighted by ABATE’s successful interim readout in AD, which is enabling the quick and informed transition to this adaptive trial’s next cohorts. Importantly, ABATE remains on track for additional readouts on ACI-24.060’s safety and immunogenicity this year, and for interim Abeta PET analyses in 2024 that will provide an opportunity for early de-risking and potentially a rapid transition to a pivotal program."

"Together, our vaccines and the highly specific diagnostics we are developing against targets such as Tau and a-syn form the cornerstone of our strategy to enable precision medicine for neurodegenerative diseases. With this strategy, our long-term goal is to identify and treat the multifactorial pathologies of each patient at their earliest stages, so that we can minimize irreversible neuronal damage and enable disease prevention."

2022 and Subsequent Highlights

Pipeline Progress

Vaccine Programs

· Positive initial interim safety and immunogenicity data from the first AD cohort of the Phase 1b/2 ABATE trial of ACI-24.060, AC Immune’s wholly-owned anti-amyloid beta (Abeta) SupraAntigen vaccine candidate. Based on these data, ABATE has been expanded, as planned, to begin screening of individuals with Down syndrome (DS) for participation in part 2 of the trial and also to evaluate higher doses in patients with AD. Interim safety and immunogenicity data from AD and DS cohorts are expected in the second half of 2023.

· ACI-35.030, a potential first-in-class anti-pTau vaccine candidate, was selected for further development, based on clinical data from a Phase 1b/2 trial in AD presented at CTAD 2022. These data showed that vaccination with ACI-35.030 was well tolerated and elicited a strong, durable, and boost-able antibody response against pathological forms of Tau such as pTau and its aggregated form, enriched paired helical filaments. ACI-35.030, generated using AC Immune’s SupraAntigen technology platform, is being developed in collaboration with Janssen Pharmaceuticals, Inc. (Janssen), part of the Janssen Pharmaceutical Companies of Johnson & Johnson.

· ACI-24.060 preclinical data published in the peer-reviewed journal Brain Communications, showed it was well tolerated and generated a broad polyclonal immune response with high titers of antibodies against neurotoxic pyroglutamate Abeta (pyroGlu-Abeta), a major component of Abeta plaques. Additional preclinical data demonstrated ACI-24.060’s strong immunogenicity against another key neurotoxic Abeta species, oligomeric Abeta.

· A peer-reviewed publication in JAMA Neurology showed that the first generation formulation of ACI-24.060 was safe and elicited an immune response in the first-ever clinical study of an anti-Abeta vaccine in individuals with DS. Data from the Phase 1b study also provided evidence of target engagement by the polyclonal response to the studied vaccine.

Antibody Programs

· Detailed results from the Phase 2 Alzheimer’s Prevention Initiative (API) study evaluating the anti-Abeta monoclonal antibody crenezumab in autosomal dominant Alzheimer’s disease (ADAD) were presented at the 2022 Alzheimer’s Association International Conference (AAIC) by AC Immune’s partner Genentech, a member of the Roche group, and the Banner Alzheimer’s Institute. Numerical differences favoring crenezumab were observed across both co-primary endpoints, as well as multiple secondary and exploratory endpoints, though none were statistically significant. All participants in the study were offered up to one year of continued treatment (crenezumab for all carriers and placebo for all non-carrier) following the end of the double-blind period while primary results and additional analyses were pending. Final efficacy visits have begun.

· Further biofluid biomarker data from the Phase 2 Lauriet trial of the anti-Tau antibody semorinemab in mild-to-moderate AD were presented in a poster at CTAD 2022. Results featured in the poster showed statistically significant post-treatment reductions in pTau and total Tau in the cerebrospinal fluid with semorinemab versus placebo, supporting target engagement and modulation in the central nervous system. The Lauriet open label extension study is ongoing.

Diagnostic Programs

· The first live images of a-syn in human brains were shown using ACI-12589, an a-syn PET tracer discovered using AC Immune’s Morphomer platform. Analyses from a clinical study of the tracer presented at AD/PD 2022 showed enhanced contrast and a-syn target specificity in patients with multiple system atrophy (MSA), as well as increased tracer retention in brain areas affected by MSA disease processes, highlighting ACI-12589’s potential as the first non-invasive diagnostic for alpha-synucleinopathies (e.g. MSA).

· AC Immune’s partner, Life Molecular Imaging, imaged the first patient in the pivotal Phase 3 ADvance trial evaluating the Tau PET tracer, PI-2620, in AD. PI-2620 was discovered and developed using the Morphomer platform as part of a research collaboration between AC Immune and LMI.

Management Team

· Expanded executive team with the appointment of Howard Donovan as Chief HR Officer. Prior to joining AC Immune, Mr. Donovan led People Services at the World Economic Forum.

· Promoted Christopher Roberts to Interim Chief Financial Officer and Vice President, Finance. Mr. Roberts previously held the title of Associate Vice President, Finance, and prior to joining AC Immune worked as a Senior Manager for Ernst and Young.

· Promoted Julian Snow to Vice President, U.S. Finance & Corporate Development. Mr. Snow previously held the title of Associate Vice President, Financial Reporting and prior to joining AC Immune worked as a manager at BDO.

Thought Leadership and Collaborations

· Received a follow-on grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to support enhanced clinical studies of ACI-12589.

· Received new grants from MJFF and the Target ALS Foundation that collectively provide more than USD 500,000 in additional non-dilutive capital to support the advancement of diagnostic programs targeting TDP-43 (TAR DNA-binding protein 43).

· AC Immune Co-Founder and CEO Dr. Andrea Pfeifer received the prestigious Aenne Burda Award for Creative Leadership in recognition of her work in the field of neurodegenerative diseases.

Key Achieved and Anticipated 2023 Milestones

ACI-24.060

anti-Abeta vaccine

·      Reported interim Phase 1b safety and immunogenicity data from first AD cohort of Phase 1b/2 ABATE study

·      Initiation of first DS cohort of ABATE study expected in H1 2023

·      Submission of an Investigational New Drug (IND) application to enable expansion of ABATE study to the U.S. expected in H1 2023

·      Additional interim safety and immunogenicity data from AD cohort of ABATE study expected in H2 2023

·      Interim safety and immunogenicity data from DS cohort of ABATE study expected in H2 2023

ACI-7104
anti-a-syn vaccine ·        Update from Phase 2 VACSYN study expected in H2 2023
ACI-35.030
anti-pTau vaccine ·        Initiation of next trial in AD expected in H2 2023 (to be followed by milestone payment)
Semorinemab
anti-Tau antibody ·        Results from the open-label extension of the Phase 2 Lauriet trial in mild-to-moderate AD expected in H2 2023
Anti-TDP-43 antibody ·        Advancement of candidate into preclinical development (tox) expected in H2 2023
a-syn-PET tracer ·        Declaration of next clinical candidate for development in Parkinson’s disease expected in H2 2023
TDP-43-PET tracer ·        Clinical candidate declaration expected in H1 2023

Analysis of Financial Statements for the Year Ended December 31, 2022

· Cash Position: The Company had a total cash balance of CHF 122.6 million, composed of CHF 31.6 million in cash and cash equivalents and CHF 91.0 million in short-term financial assets. This compares to a total cash balance of CHF 198.2 million as of December 31, 2021. The Company’s cash balance provides sufficient capital resources to progress into at least Q3 2024 without consideration of potential income milestone payments.

· Contract Revenues: The Company recorded CHF 3.9 million in contract revenues for the year end December 31, 2022 compared with no contract revenues in the prior year. The increase relates to the progression of PI-2620 into Phase 3 development in AD.

· R&D Expenditures: R&D expenses decreased by CHF 1.9 million for the year ended December 31, 2022 to CHF 60.3 million, predominantly due to:

o Discovery and preclinical expenses (- CHF 3.1 million): The Company decreased expenditures across a variety of its discovery and preclinical programs. This decrease was predominantly due to the advancement of the Company’s ACI-24.060 vaccine into clinical studies.

o Clinical expenses (- CHF 1.8 million): The Company had a net decrease in clinical expenditures largely due to the timing of activities across various cohorts and the completion of certain R&D cost sharing activities for our ACI-35.030 vaccine. We increased expenditures in other clinical programs, notably for the clinical development of ACI-7104.056 and the initiation of our Phase 1b/2a ABATE study for ACI-24.060.

o Salary- and benefit-related costs (+ CHF 1.2 million): Personnel expenses increased due to the net increase in FTEs in 2022 along with the annualization of 2021 hires.

· G&A Expenditures: G&A expenses decreased by CHF 2.1 million for the year ended December 31, 2022 to CHF 15.8 million. This decrease is related to prior year transaction costs incurred to complete the asset acquisition of Affiris’ a-syn portfolio and the reduction in personnel expenses.

· Other Operating Income: The Company recorded CHF 1.3 million in grant income for R&D activities performed under our grants for the year ended December 31, 2022, an increase of CHF 0.1 million compared to the prior period.

· IFRS Loss for the Period: The Company reported a net loss after taxes of CHF 70.8 million for the year ended December 31, 2022, compared with a net loss of CHF 73.0 million for the prior period.

2023 Financial Guidance

· AC Immune anticipates that its total cash burn will be in the range of CHF 65 to CHF 75 million for the full year 2023. The Company defines cash burn as operating expenditures adjusted to include capital expenditures and offset by significant non-cash items (including share-based compensation and depreciation expense).

2seventy bio Reports Fourth Quarter and Full Year 2022 Financial Results and Recent Operational Progress

On March 16, 2023 2seventy bio, Inc. (Nasdaq: TSVT), a leading immuno-oncology cell therapy company, reported financial results and recent highlights for the fourth quarter and full year ended December 31, 2022 (Press release, 2seventy bio, MAR 16, 2023, View Source [SID1234628885]).

"In our first full year of operations, 2seventy made tremendous strides across all aspects of our business: from advancing early-stage research, building out manufacturing capabilities, initiating clinical studies, and delivering to patients in the commercial setting with Abecma," said Nick Leschly, chief kairos officer. "Bolstered by strong growth in the fourth quarter, we delivered Abecma to hundreds of patients with multiple myeloma in the U.S. and expect continued strong growth in 2023. With our partners at BMS, we continue to develop this product into earlier lines of multiple myeloma treatment on the back of positive data from the KarMMa-3 study and the planned KarMMa-9 study and continue to grow our manufacturing capabilities to support a potential peak sales opportunity of $2-3 billion in the U.S. With our recent financing and the growth of Abecma, we are in a strong position to continue to prudently advance our clinical and preclinical pipeline, driving towards delivering proof-of-concept data this year for our bbT369 and SC-DARIC33 programs and advancement of MUC and MAGE solid tumor programs to IND by the end of the year. I couldn’t be more optimistic for what lies ahead in 2023 and the position we are in today, and that is thanks to the hard work of our employees, the patients and physicians who participate in our studies, and our shareholders who share in our mission to deliver more time to patients with cancer."

Abecma Commercial Summary
Our partner, Bristol Myers Squibb (BMS), reported total U.S. Abecma (idecabtagene vicleucel; ide-cel) fourth quarter revenues of $94 million, which represents 25% growth over the prior quarter, and 40% growth over the fourth quarter of 2021, and full year revenues of $297 million, which is in line with our stated 2022 U.S. Abecma revenue guidance of $250-300 million, in each case such revenue is shared equally with BMS. Assuming continued strong demand for Abecma and achievement of planned increases in manufacturing capacity throughout the year, we anticipate topline 2023 U.S. revenues of $470-570 million.

Assuming continued increases in manufacturing capacity and growth in the addressable patient population based on an anticipated approval in 3L+ multiple myeloma, we expect Abecma to generate $200-300 million in operating income for 2seventy bio in the 2024-25 period.

We reported Abecma-related collaborative arrangement revenue of $8.7 million for the fourth quarter of 2022 and collaborative arrangement revenue net of share of collaboration loss of $3.1 million for the year.

Financial Guidance
At the end of February 2023, 2seventy bio raised approximately $117 million in net proceeds through a public equity offering. Based on our current operating plans, which includes these net proceeds and the ongoing commercialization of Abecma, we believe our current cash position will be sufficient to fund operations into 2026.

"The financial outlook for 2seventy continues to strengthen, and the recent equity financing puts us in a privileged position, with cash runway now into 2026," said Chip Baird, chief financial officer. "We expect the cash flow from the Abecma collaboration to grow significantly over the 2023-25 time period, which will continue to reduce our annual net cash spend and our need for future capital infusions. Our pipeline programs have the potential to deliver meaningful benefit for cancer patients and we will balance an entrepreneurial mindset with a disciplined approach to capital allocation."

RECENT HIGHLIGHTS
KARMMA-3 DATA IN NEJM – On February 10, 2023, 2seventy announced the publication of our KarMMa-3 data in the New England Journal of Medicine. The positive results of this pivotal Phase 3 showed at median follow up of 18.6 months, treatment with Abecma (n=254) demonstrated a clinically meaningful and statistically significant improvement in the primary endpoint of progression-free survival (PFS) compared with standard regimens (n=132), with a median PFS of 13.3 months (95% CI: 11.8-16.1) vs. 4.4 months (95% CI: 3.4-5.9), respectively (HR:0.49; p<0.0001). This represents a 51% reduction in risk of disease progression or death with Abecma. Based on results from KarMMa-3, Abecma is the first and only chimeric antigen receptor (CAR) T cell therapy to demonstrate superiority over standard regimens in triple-class exposed relapsed and refractory multiple myeloma in a randomized, controlled Phase 3 trial. 2seventy and BMS anticipate submitting an sBLA to the FDA in the first quarter of 2023 to seek approval in the third line setting.

REGENERON COLLABORATION AMENDMENT – In January, 2seventy bio announced an expanded translational collaboration with Regeneron to facilitate the acceleration of novel cell therapy-based combinations for solid tumors. The collaboration will leverage 2seventy bio’s unique cell therapy engineering and early-stage development capabilities, including the newly built in-house clinical cell therapy manufacturing facility, with Regeneron’s differentiated antibodies and bispecifics. To support this expanded clinical development plan Regeneron made an approximately $20 million equity investment in 2seventy bio at a 50% premium and has committed to another approximately $20 million in near-term pre-clinical and clinical milestones. The parties will continue sharing costs for these activities in a manner largely consistent with the existing agreement, with Regeneron covering 75% of certain preclinical costs necessary to study combinations and 100% of the costs for the arms of the clinical studies that include Regeneron agents through regulatory approval. For other programs, cost-sharing will follow the existing 50/50 cost sharing agreement.

BOARD APPOINTMENT – Earlier this month, 2seventy bio announced the appointment of Wei Lin, M.D. to the company’s Board of Directors.

UPCOMING ANTICIPATED MILESTONES
ABECMA

Initiation of KarMMa-9 study in patients with newly-diagnosed multiple myeloma by end of 2023
Potential FDA approval of sBLA in 3L+ multiple myeloma by the end of 2023
PIPELINE

Data update from Phase I CRC-403 study of bbT369 in patients with relapsed and/or refractory B cell non-Hodgkin lymphoma (B-NHL) anticipated by the end of 2023
Data update from Phase I PLAT-08 study of SC-DARIC33 in patients with acute myeloid leukemia by the end of 2023
Submission of an IND for MUC-16 program in ovarian cancer, being developed in partnership with Regeneron by end of 2023
Led by JW Therapeutics, initiation of an investigator-initiated study in China of 2seventy bio’s potency enhanced MAGE-A4 TCR program in solid tumors by end of 2023
FINANCIAL GUIDANCE

Topline U.S. Abecma revenue of $470-570 million in 2023
Net cash spend of $180-220 million in 2023
Cash runway into 2026
SELECT FOURTH QUARTER AND FULL YEAR 2022 FINANCIAL RESULTS

Our partner, BMS, reported total U.S. revenues of $94 million and $297 million for Abecma for the three and twelve months ended December 31, 2022, respectively. 2seventy bio and BMS share equally in all profits and losses related to development, manufacture, and commercialization of Abecma in the U.S. We reported collaborative arrangement revenue of $8.7 million for the three months ended December 31, 2022 and collaborative arrangement revenue net of share of collaboration loss of $3.1 million for the twelve months ended December 31, 2022.
Total 2seventy bio revenues were $56.2 million for the three months ended December 31, 2022 compared to $16.0 million for the three months ended December 31, 2021. Total revenues were $91.5 million for the twelve months ended December 31, 2022 compared to $54.5 million for the twelve months ended December 31, 2021. The increase for both the three and twelve-month periods was primarily due to an increase in service revenue in the fourth quarter of 2022, driven by the release of deferred revenue relating to bb21217, the development of which was discontinued in 2022.
Research and development expenses were $60.1 million for the three months ended December 31, 2022 compared to $57.2 million for the three months ended December 31, 2021. This increase was primarily driven by increased costs for manufacturing activities of suspension lentiviral vector for ide-cel and MAGE-A4 development, partially offset by a decrease in costs related to our share of research and development expenses under our collaboration with BMS. Research and development expenses were $248.7 million for the twelve months ended December 31, 2022, compared to $252.6 million for the twelve months ended December 31, 2021. This decrease was primarily driven by a decrease in costs related to our share of research and development expenses under our collaboration with BMS, partially offset by increases in costs for manufacturing activities of suspension lentiviral vector.
Selling, general and administrative expenses were $18.7 million for the three months ended December 31, 2022, compared to $24.5 million for the three months ended December 31, 2021. Selling, general and administrative expenses were $79.5 million for the twelve months ended December 31, 2022, compared to $93.5 million for the twelve months ended December 31, 2021. The decrease for both the three and twelve-month periods was primarily driven by decreased employee compensation expenses, reflective of efforts to streamline 2seventy bio’s operating model and a decrease in IT and other facility-related costs due to a lower allocation of these costs to selling, general and administrative expense based on headcount and facility square footage.
Net loss was $23.1 million for the three months ended December 31, 2022, compared to $61.0 million for the three months December 31, 2021. Net loss was $254.2 million for the twelve months ended December 31, 2022, compared to $292.2 million for the twelve months ended December 31, 2021.
2seventy bio ended 2022 with cash, cash equivalents and marketable securities of $267.7 million.

Sporos BioDiscovery to Present Preclinical Findings on SPR1, a Next-Generation TEAD Inhibitor, at the American Association for Cancer Research (AACR) Annual Meeting 2023

On March 15, 2023 — Sporos BioDiscovery, Inc. (a wholly owned affiliate of Sporos Bioventures, "Sporos" or the "Company"), a precision oncology company developing a diversified pipeline of small molecule therapeutic programs targeting cancer vulnerabilities in the tumor and tumor microenvironment, reported that it will present a poster highlighting preclinical data from the company’s novel, isoform selective TEAD inhibitor, SPR1, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2023 taking place April 14-19, 2023, in Orlando, FL.

"We look forward to presenting preclinical data on our TEAD inhibitor, which we believe has best-in-class potential based on its optimized TEAD isoform selectivity profile and resultant preclinical activity superior to that of other compounds in development," said Stephen Rubino, Ph.D., Sporos’ Chief Executive Officer. "SPR1 inhibitors show single-agent activity against multiple TEAD-dependent cell lines in vitro and in vivo, including a range of non-NF2 mutation cell lines and those cell lines that are simply YAP-hyperactive. In addition, our differentiated TEAD inhibitors have demonstrated potent synergistic activity in combination with inhibitors of the MAPK pathway, creating the potential for broad applicability across a number of cancers with serious unmet medical need."

Presentation Details:

Title: A next generation TEAD inhibitor with refined isoform specificity for superior safety & efficacy
Session Category: Experimental and Molecular Therapeutics
Session Title: New Drug Targets
Session Date and Time: Sunday Apr 16, 2023, 1:30 PM – 5:00 PM
Abstract Number: 445

(Press release, Sporos BioDiscovery, MAR 15, 2023, View Source [SID1234662123])

Aimed-GeneQuantum reveals ‘FGFR3-ADC’ preclinical data at World ADC

On March 15, 2023 AimedBio and Genequantum healthcare reported the preclinical study result of co-developing FGFR3-ADC ‘AMB302/GQ1011’ will be presented at the "World ADC".

– They have confirmed AMB302/GQ1011’s anti-tumor effect in both bladder cancer and GBM cancer models, and plan to submit IND and start phase 1 clinical trial in 1H 2024.

(Press release, AimedBio, MAR 15, 2023, View Source;s_keyword=&s_where=&start=20 [SID1234656921])

ImmuneOnco’s Phase Ib/II clinical study of IMM27M combined with PD-1 antibody in patients with advanced solid tumors has been approved by the National Medical Products Administration (NMPA)

On March 15, 2023 ImmuneOnco Biopharmaceuticals (Shanghai) Inc. (hereinafter referred to as "ImmuneOnco") reported that the Phase Ib/II clinical study of the Company’s self-developed ADCC-enhanced CTLA-4 antibody drug (project number: IMM27M) jointly with Tislelizumab targeting PD-1 in patients with advanced solid tumors was approved by the National Medical Products Administration (NMPA), which is another milestone in the Company’s development (Press release, ImmuneOnco Biopharma, MAR 15, 2023, View Source [SID1234655685]).

IMM27M is an IgG1 antibody against the CTLA-4 target. It has been genetically engineered to significantly enhance the ADCC activity. Compared with the same drug Ipilimumab, the drug effect in animals is significantly better than Ipilimumab at the same dose. At a lower dose (0.3mg/ kg) can completely remove the tumor.

The phase I dose-escalation clinical trial of the IMM27M project for solid tumors is progressing smoothly. The first patient has been administered in June 2022, and the 5mg/kg dose group has been achieved so far. Preliminary clinical data of the previous dose (3mg/kg) show that IMM27M is well tolerated, and there has been no dose-limiting toxicity (DLT) so far, and it has shown positive effects on some patients with advanced solid tumors (such as melanoma, breast cancer). The curative effect signal, after one to two cycles of treatment, the tumor shrunk by 22.8% and 28.7%, respectively.

Dr. Tian Wenzhi, Founder and Chairman of ImmuneOnco, said,

We are very pleased that our new generation of CTLA-4 antibody project IMM27M combined with tislelizumab has been approved by the NMPA for Phase Ib/II clinical trials in patients with advanced solid tumors. Repeated in vivo studies have demonstrated that IMM27M has a strong anti-tumor effect It is active and can be combined with multiple drugs in the company’s pipeline for clinical research. The dual-immune combination therapy of CTLA4 combined with PD1/PD-L1 has proven to have a clear clinical synergy. BMS’s ipilimumab combined with O. The indications have been approved by the FDA for marketing, covering multiple clinical indications such as melanoma, colorectal cancer, renal cell carcinoma, hepatocellular carcinoma, non-small cell lung cancer, etc. We believe that IMM27M will have great clinical development value. We will continue to advance the research of the IMM27M project and bring good news to the majority of cancer patients.