On February 16, 2023 Convergent Therapeutics Inc., a clinical stage biotechnology company, reported the titles of posters evaluating its lead asset, CONV01-α (225Ac−J591), a prostate-specific membrane antigen (PSMA)-targeted monoclonal antibody linked to Ac-225 (225Ac), that will be presented at the 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary (GU) Cancers Symposium happening in a hybrid mode at San Francisco, February 16-18 (Press release, Convergent Therapeutics, FEB 16, 2023, View Source [SID1234627343]).

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The complete list of abstract titles can be found here.

Poster presentations will focus on two ongoing clinical studies, a trials-in-progress update from NCT04506567, and a data update from NCT04946370, that are both currently investigating CONV01-α (225Ac−J591) in patients with progressive metastatic castration-resistant prostate cancer (mCRPC) and are led by Principal Investigator Dr. Scott T. Tagawa, Professor of Medicine in Urology at Weill Cornell Medicine.

Poster Details:
Abstract Number: TPS288
Abstract Title: A phase I/II dose-escalation study of fractionated 225Ac-J591 for progressive metastatic castration-resistant prostate cancer (mCRPC) in patients with prior treatment with 177Lu-PSMA.
Poster Session: Trials in Progress
Poster Bd #: Q10
Presenter: Jones T. Nauseef, MD, PhD

Abstract Number: 181
Abstract Title: Phase I results of a phase I/II study of pembrolizumab and AR signaling inhibitor (ARSI) with 225Ac-J591.
Poster Session A: Prostate Cancer
Poster Bd #: F18
Presenter: Michael Philip Sun, MD

On February 16, 2023 Promontory Therapeutics Inc., a clinical stage pharmaceutical company advancing small molecule immunotherapies in oncology, reported its Phase 2 clinical trial in progress of lead therapeutic candidate, PT-112, in patients with late-stage metastatic castration-resistant prostate cancer (mCRPC) (Press release, Promontory Therapeutics, FEB 16, 2023, View Source [SID1234627342]). The poster presentation took place at the 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary Cancers (ASCO GU) Symposium, in San Francisco.

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The poster titled, "A Phase 2 study of immunogenic cell death inducer PT-112 in metastatic castration-resistant prostate cancer patients" was presented by Alan H. Bryce, MD, Associate Professor of Medicine and Chair, Division of Hematology/Oncology, Department of Internal Medicine, Mayo Clinic Arizona. The goal of the ongoing study is to determine the optimal dosing level and schedule of PT-112, in line with the U.S. Food and Drug Administration’s Project Optimus. Additionally, this study is also assessing the safety and efficacy of PT-112 as per PCWG3 guidelines, as well as circulating tumor cell responses related to clinical benefit. Additional correlative research includes blood-based T cell receptor sequencing used to elucidate the underlying immune mechanism of PT-112 in humans.

"Prior clinical and non-clinical evidence provides strong rationale to further explore PT-112 as a treatment for patients with late-stage metastatic castration resistant prostate cancer, due to its immunogenicity and osteotropism," said Promontory Therapeutics co-founder and Chief Operating Officer Matthew Price. "What we believe makes this trial particularly informative is the incorporation of extensive biomarker and correlative research programs which we anticipate will provide supportive evidence of PT-112’s mechanism and help us understand patient populations for future studies."

Eligible patients must have received at least three intended life-prolonging therapies, including at least one AR-targeted therapy and at least one, but no more than two, prior lines of taxane. Patients must also have confirmed radiographic progression upon entering the trial. As of February 1, 2023, 41 patients have been treated in the study, with one of the three dosing schedules: 360 mg/m2 on days 1 and 15 of each cycle, 250 mg/m2 on day 1 and 15 of each cycle, or 360 mg/m2 on days 1 and 15 of cycle one and 250 mg/m2 on day 15 of each subsequent cycle.

"Patients with late-stage mCRPC currently have very limited treatment options that are both effective and durable," said Promontory Therapeutics Chief Medical Officer Johan Baeck, MD. "Although many trials are being conducted in prostate cancer, there are few agents with new mechanisms of action similar to PT-112. Pre-clinical research suggests that that PT-112 induces immunogenic cell death and osteotropism, and our prior dose escalation trials showed preliminary evidence of the safety, efficacy, and clinical benefit of PT-112 in mCRPC patients. We are looking forward to completing enrollment of this study in 2024 and sharing clinical and biomarker data upon completion of the study."

For more information about PT-112 and Promontory Therapeutics’ clinical pipeline visit www.PromontoryTx.com. Further details on the PT-112 clinical trial in prostate cancer (NCT02266745) can be found at clinicaltrials.gov.

About PT-112

PT-112 is the first small-molecule conjugate of pyrophosphate in oncology, and possesses a unique pleiotropic mechanism of action that promotes immunogenic cell death (ICD), through the release of damage associated molecular patterns (DAMPs) that bind to dendritic cells and lead to downstream immune effector cell recruitment in the tumor microenvironment. PT-112 represents a highly potent inducer of this immunological form of cancer cell death. Further, PT-112 harbors a property known as osteotropism, or the propensity of the drug to reach its highest concentrations in certain areas of the bone, making it a candidate for treatment of patients with cancers that originate in, or metastasize to, the bone. The first in-human study of PT-112 demonstrated an attractive safety profile and evidence of long-lasting responses among heavily pre-treated patients and won "Best Poster" within the Developmental Therapeutics category at the ESMO (Free ESMO Whitepaper) 2018 Annual Congress. The combination Phase 1b dose escalation study of PT-112 with PD-L1 checkpoint inhibitor avelumab in solid tumors was reported in an oral presentation at the ESMO (Free ESMO Whitepaper) 2020 Virtual Congress and the Phase 2a dose confirmation cohort in non-small cell lung cancer (NSCLC) patients was reported at ESMO (Free ESMO Whitepaper) I-O 2022. The Phase 1 study in patients with relapsed or refractory multiple myeloma presented at ASH (Free ASH Whitepaper) 2020 is the third completed Phase 1 study of PT-112. Monotherapy Phase 2 development is ongoing in mCRPC, and includes the Phase 2 proof of concept study in thymic epithelial tumors under the company’s formal CRADA with the NCI.

On February 16, 2023 Mallinckrodt plc (NYSE American: MNK), a global specialty pharmaceutical company, reported that Siggi Olafsson, President and Chief Executive Officer, will present at Cowen’s 43rd Annual Health Care Conference in Boston, MA on Tuesday, March 7, 2023 at 2:50 p.m. Eastern Time (Press release, Mallinckrodt, FEB 16, 2023, https://www.prnewswire.com/news-releases/mallinckrodt-to-present-at-cowens-43rd-annual-health-care-conference-301748832.html [SID1234627341]).

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Individuals who cannot attend the meeting in person can find webcast information at: https://wsw.com/webcast/cowen132/mnk/1849260. A replay will also be available following the meeting.

On February 16, 2023 PacBio (NASDAQ: PACB) reported financial results for the quarter and fiscal year ended December 31, 2022 (Press release, Pacific Biosciences, FEB 16, 2023, View Source [SID1234627340]).

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Fourth quarter results

Received record orders in the fourth quarter, including orders for 96 instruments, which included orders for 76 Revio systems from 43 customers across 13 countries and representing a broad set of applications.
Revenue of $27.4 million, a 24% decrease compared with $36.0 million in the prior year period.
Instrument revenue of $6.1 million, compared with $16.2 million in the prior-year period.
Consumables revenue of $16.7 million compared with $15.0 million in the prior year period. Sequel II and IIe consumables represented approximately 94% of our total consumable revenue in the fourth quarter of 2022, with the rest from older systems and other consumables.
Service and other revenue of $4.6 million compared with $4.8 million in the prior year period.
Delivered 18 Sequel IIe systems, compared with 48 Sequel II/IIe systems in the prior year period.
Installed base of 512 Sequel II/IIe systems as of December 31, 2022, compared with 374 as of December 31, 2021.
Gross profit for the fourth quarter of 2022 was $5.1 million, representing a 69% decrease compared with $16.8 million for the fourth quarter of 2021 and a gross margin of 19% in the fourth quarter of 2022 compared to 47% for the fourth quarter of 2021. Non-GAAP gross profit for the fourth quarter of 2022 was $5.3 million and represented a non-GAAP gross margin of 19% in the fourth quarter of 2022, compared to 47% for the fourth quarter of 2021 (see accompanying tables for reconciliations of GAAP and non-GAAP measures).

Operating expenses totaled $92.2 million for the fourth quarter of 2022, compared to $81.4 million for the fourth quarter of 2021. Non-GAAP operating expenses totaled $87.6 million for the fourth quarter of 2022, compared to $79.9 million for the fourth quarter of 2021. Operating expenses for the fourth quarter of 2022 and the fourth quarter of 2021 included non-cash share-based compensation of $16.8 million and $17.5 million, respectively.

Net loss for the fourth quarter of 2022 was $84.4 million, compared to a net loss of $69.3 million for the fourth quarter of 2021. Non-GAAP net loss was $79.6 million for the fourth quarter of 2022, compared to $66.4 million for the prior-year period.

Net loss per share for the fourth quarter of 2022 was $0.37 compared to net loss per share of $0.31 for the fourth quarter of 2021. Non-GAAP net loss per share for the fourth quarter of 2022 was $0.35 compared to $0.30 for the fourth quarter of 2021.

GAAP and non-GAAP gross profit, net loss, and net loss per share in the fourth quarter reflect loss on purchase commitments and adjustments for excess inventory totaling approximately $7.1 million primarily related to a faster-than-expected ramp in Revio demand, which resulted in a faster-than-expected decline in Sequel II/IIe demand upon the launch of Revio.

Cash, cash equivalents, and investments, excluding short- and long-term restricted cash, at December 31, 2022, totaled $772.3 million, compared to $1,044.4 million at December 31, 2021.

Fiscal year 2022 results

Revenue of $128.3 million, a 2% decrease compared with $130.5 million in 2021.
Placed 138 Sequel II/IIe systems during the year compared to 171 Sequel II/IIe systems placed in 2021.
Instrument revenue of $48.7 million compared with $61.3 million in 2021.
Consumables revenue of $60.0 million compared with $52.2 million in 2021.
Service and other revenue of $19.6 million compared with $17.0 million in 2021.
Gross profit for 2022 was $49.0 million, representing a 17% decrease compared with $58.9 million for 2021 and gross margin of 38% compared to 45% for 2021. Non-GAAP gross profit for 2022 was $49.8 million and represented a non-GAAP gross margin of 39%, compared to 45% for 2021.

Operating expenses totaled $356.2 million, compared to $269.3 million for 2021. Non-GAAP operating expenses totaled $353.7 million, compared to $236.9 million for 2021. Operating expenses for 2022 and 2021 included non-cash share-based compensation of $73.8 million and $67.2 million, respectively. Excluding merger-related expenses, non-GAAP operating expenses included non-cash share-based compensation of $73.8 million in 2022 compared to $55.7 million in 2021.

Net loss for 2022 was $314.2 million, compared to a net loss of $181.2 million for 2021. Non-GAAP net loss was $311.0 million, compared to $190.0 million for 2021.

Net loss per share for 2022 was $1.40, compared to net loss per share of $0.89 for 2021. Non-GAAP net loss per share for 2022 was $1.38 compared to $0.93 for 2021.

Updates since our last earnings release

Celebrated long-read sequencing being named Nature Methods’ "Method of the Year 2022," recognizing the impact of long-read sequencing in various groundbreaking studies, including those leveraging PacBio.
Shipped our first early access Revio system to the Broad Institute and expect to commence commercial Revio shipments in March 2023.
Delivered Onso to three beta customers, including the Broad Institute, Corteva Agriscience, and Weill Cornell Medical College, and remain on track to begin commercialization of Onso in the second quarter of 2023.
Announced plans to expand MAS-Seq technology to 16S rRNA and bulk RNA-Seq solutions, which can expand throughput in these applications by up to 16-fold through short fragment concatenation.
Introduced the PacBio Compatible program designed to make PacBio sequencing more accessible, which includes partners across all ends of the sequencing workflow, from automation and sample and library prep to secondary and tertiary analysis tools.
Announced a collaboration with the University of Tokyo Graduate School of Medicine to study the use of long-read sequencing and novel bioinformatics methods in the hopes of better understanding the genetic causes of certain rare diseases in individuals and cohorts within the Japanese population.
Announced HiFi sequencing technology will be used in a pilot project for the Children’s Rare Disease Cohorts Initiative (CRDC) at Boston Children’s Hospital to investigate genetic and epigenetic variants associated with rare pediatric diseases.
Raised approximately $201 million in gross proceeds in an upsized public offering of common stock in January 2023.
"The year is off to an excellent start with a strong Revio order book and continued customer enthusiasm for the new product," said Christian Henry, President, and Chief Executive Officer. "Momentum is clearly building for highly accurate, long-read solutions, with Nature Methods naming long-read sequencing its ‘Method of the Year’ and studies like the preprint from All of Us researchers concluding that we should continue developing population-scale cohorts sequenced with long-reads only."

Quarterly Conference Call Information

Management will host a quarterly conference call to discuss its fourth quarter ended December 31, 2022, results today at 4:30 p.m. Eastern Time. Investors may listen to the call by dialing 866-652-5200, or if outside the U.S., by dialing 412-317-6060, and request to join the "PacBio Q4 Earnings Call." The call will be webcast live and will be available for replay at PacBio’s website at View Source

On February 16, 2023 Cue Biopharma, Inc. (Nasdaq: CUE), a clinical-stage biopharmaceutical company developing a novel class of injectable biologics to selectively engage and modulate disease-specific T cells directly within the patient’s body, reported that the company’s therapeutic Immuno-STAT (Selective Targeting and Alteration of T cells) platform and biologics will be featured in a poster at the 67th Biophysical Society Annual Meeting, taking place February 18-22, 2023 in San Diego, CA (Press release, Cue Biopharma, FEB 16, 2023, View Source [SID1234627339]). The research is part of a strategic research collaboration that Cue Biopharma initiated with Dr. Michael Dustin’s laboratory at the University of Oxford in May 2020 to determine the molecular mechanisms underlying the activity of the company’s interleukin 2 (IL-2) based CUE-100 series of biologics.

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"The data continue to support the differentiated mechanism of action of Cue Biopharma’s Immuno-STAT CUE-100 series of biologics, by uniquely mimicking the natural process of immune synapse formation," said Jesusa Capera-Aragones, Ph.D., post-doctoral researcher, Dustin laboratory of the Kennedy Institute at the University of Oxford. "By simultaneously presenting a targeted antigen and the immunostimulatory molecule IL-2, Cue Biopharma’s biologics enable the specific and simultaneous engagement of IL-2 and T cell receptors, as it would occur in nature. This promotes the selective activation of disease-specific "killer" T cells, while maximizing T cell activation and minimizing off-target binding, potentially translating in a larger therapeutic window reducing toxicities seen with other IL-2-based therapies.

Dr. Anish Suri, Ph.D., president and chief scientific officer of Cue Biopharma, added "We are very pleased with the research and collaboration with Dr. Dustin’s laboratory of the Kennedy Institute at the University of Oxford. The data continue to validate the platform design and support the results we are seeing as part of our Phase 1 clinical study of our lead CUE-100 candidate, CUE-101, in human papilloma virus (HPV)+ head and neck cancer, that shows activation of HPV+ specific "killer" T cells in patients. We look forward to sharing clinical progress and continue to provide clinical validation of the platform, with potential to treat a large number of cancers."

Presentation Details

Abstract title: Understanding the Spatial and Functional Link Between the IL-2R and TCR at the Immunological Synapse
Presenter: Jesusa Capera-Aragones, Ph.D., from the Kennedy Institute at the University of Oxford
Poster Session: Membrane Receptors and Signal Transduction Track A: Novel Engineered Cell Therapies and Concepts
Poster Board Number: B231
Date and Time: February 22, 2023, 10:30 a.m. PST
Location: Exhibit Hall BC
Program Number: 2481-Pos

About the CUE-100 Series
The CUE-100 series consists of Fc-fusion biologics that incorporate peptide-MHC (pMHC) molecules along with rationally engineered IL-2 molecules. This singular biologic is anticipated to selectively target, activate and expand a robust repertoire of tumor-specific T cells directly in the patient’s body. The binding affinity of IL-2 for its receptor has been deliberately attenuated to achieve preferential selective activation of tumor-specific effector T cells while reducing the potential for effects on regulatory T cells (Tregs) or broad systemic activation, potentially mitigating the dose-limiting toxicities associated with current IL-2-based therapies.

About Immuno-STAT
The company’s Immuno-STAT (Selective Targeting and Alteration of T cells) platform biologics are designed for targeted modulation of disease-associated T cells in the areas of immuno-oncology and autoimmune disease. Each of our biologic drugs is designed using our proprietary scaffold comprising: 1) a peptide-major histocompatibility complex (pMHC) to provide selectivity through interaction with the T cell receptor (TCR), and 2) a unique co-stimulatory signaling molecule to modulate the activity of the target T cells. The simultaneous engagement of co-regulatory molecules and pMHC binding mimics the signals delivered by antigen presenting cells (APCs) to T cells during a natural immune response. This design enables Immuno-STAT biologics to engage with the T cell population of interest, resulting in selective T cell modulation. Because our drug candidates are delivered directly in the patient’s body (in vivo), they are fundamentally different from other T cell therapeutic approaches that require the patients’ T cells to be extracted, modified outside the body (ex vivo) and reinfused.