On February 16, 2023 RedHill Biopharma Ltd. (Nasdaq: RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company, reported that following a positive pre-MAA meeting it plans to submit a Marketing Authorisation Application (MAA) to the UK Medicines & Healthcare products Regulatory Agency (MHRA) seeking approval for RHB-102 (Bekinda)[1] for oncology support (management of nausea and vomiting induced by cytotoxic chemotherapy and radiotherapy, also referred to as CINV and RINV) in adults and children over the age of 12 (Press release, RedHill Biopharma, FEB 16, 2023, View Source [SID1234627344]).

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"This green light for submission from the UK’s MHRA is a major milestone towards potential approval and commercialization of RHB-102 in the UK for treating chemotherapy and radiotherapy induced nausea and vomiting. The MHRA pre-MAA scientific advice meeting evaluated the available RHB-102 clinical and pharmacokinetic data generated to date," said Gilead Raday, RedHill’s Chief Operating Officer and Head of R&D. "Following the positive input from the MHRA pre-MAA review team we aim to complete our submission of for marketing authorization application of RHB-102 to the UK regulatory authorities in the second half of this year."

RHB-102 is a proprietary 24-hr bimodal release, once-daily oral tablet formulation of ondansetron, a 5-HT3 antagonist considered the gold standard in the treatment and prevention of CINV/RINV. Between 70-80% of patients undergoing chemotherapy or radiotherapy will experience nausea and/or vomiting. The global CINV/RINV market is estimated to be worth over $10 billion by 2031, with the UK market expected to grow at 6.4% CAGR, accounting for around 20% of the European market[2].

Data to support the submission was generated from several clinical studies including the successful U.S. Phase III GUARD study with RHB-102 24 mg for acute gastroenteritis and gastritis[3].

About RHB-102 (BEKINDA):

RHB-102 is a proprietary, bimodal release, once-daily oral pill formulation of the antiemetic drug ondansetron, targeting several gastrointestinal indications. RHB-102 24 mg is intended to provide patients with relief from nausea and vomiting symptoms for a full 24-hour period with a single oral tablet. If approved for marketing by the MHRA, RHB-102 24 mg could become the first oral 24hr extended-release 5-HT3 antiemetic drug in the UK indicated for the treatment of CINV/RINV.

The RHB-102 Phase III GUARD gastroenteritis study results were published in JAMA Network Open3. The RHB-102 Phase II IBS-D study results were published in The American Journal of Gastroenterology[4].

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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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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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