1stOncology™: Cancer Intelligence Service – Page 10144 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

Prothena Reports Fourth Quarter and Full Year 2020 Financial Results, and Provides Financial Guidance and R&D Update

On February 11, 2021 Prothena Corporation plc (NASDAQ:PRTA), a late-stage clinical company with expertise in protein dysregulation and a pipeline of investigational therapeutics for rare peripheral amyloid and neurodegenerative diseases, reported financial results for the fourth quarter and full year 2020 (Press release, Prothena, FEB 11, 2021, View Source [SID1234574889]). In addition, the Company provided 2021 financial guidance and an update on its R&D programs.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

Schedule Your 30 min Free Demo!

"We continue to see positive momentum in our pipeline with the recent announcement of the confirmatory, registration-enabling Phase 3 AFFIRM-AL study of birtamimab in AL amyloidosis and positive clinical findings reported in 2020 from both the Phase 2 study of prasinezumab in Parkinson’s disease and Phase 1 study of PRX004 in ATTR amyloidosis," said Gene Kinney, Ph.D., President and Chief Executive Officer of Prothena. "We are particularly pleased that our unique protein dysregulation platform has now resulted in the translation of our preclinical findings into clinical benefit for patients across multiple programs in our portfolio. We look forward to a number of significant milestones in 2021, with the planned initiation of 3 late-stage clinical studies, including the birtamimab AFFIRM-AL study, the prasinezumab Phase 2b study and the PRX004 Phase 2/3 study. In addition to these late-stage programs, we have also advanced the three targets under our global neuroscience collaboration with Bristol-Myers Squibb and expect to file an IND for PRX005 this year. Our strong cash position and potential partner payments provide a foundational capital position to fund the company through value-creating milestones as we transition to a fully integrated commercial company."

Full Year 2020 and Recent Developments:

Birtamimab, a potential treatment for AL amyloidosis, is a humanized monoclonal antibody designed to directly neutralize soluble toxic aggregates and promote clearance of amyloid that causes organ dysfunction and failure

Based on significant survival benefit observed in the previous VITAL study in Mayo Stage IV patients (HR=0.413, p=0.025, over 9 months) and multiple in-depth discussions with the U.S. Food and Drug Administration (FDA), Prothena announced plans in February 2021 to advance birtamimab into the confirmatory Phase 3 AFFIRM-AL study in Mayo Stage IV patients with AL amyloidosis. AFFIRM-AL is a global, registration-enabling Phase 3 study that will be conducted with a primary endpoint of all-cause mortality at p≤0.10 under a Special Protocol Assessment (SPA) agreement with FDA.
Prasinezumab, a potential treatment for Parkinson’s disease, is a humanized monoclonal antibody designed to target key epitopes within the C-terminus of alpha-synuclein and is the focus of the worldwide collaboration with Roche

Presented results from Phase 2 PASADENA study showing prasinezumab significantly slows progression on pre-specified clinical measures of Parkinson’s disease in September 2020 at the International Parkinson and Movement Disorder Society’s 2020 Congress. Prasinezumab is the first potentially disease-modifying, anti-alpha-synuclein antibody to demonstrate signals of efficacy on multiple pre-specified secondary and exploratory clinical endpoints in patients with early Parkinson’s disease.
Announced that Roche and Prothena will advance prasinezumab into a late-stage Phase 2b study in patients with early Parkinson’s disease. The study will be designed to further assess the efficacy of prasinezumab by expanding upon the patient population enrolled in PASADENA to include patients with early Parkinson’s disease on stable levodopa therapy. Prasinezumab is the first anti-alpha synuclein antibody to advance into late-stage development.
PRX004, a potential treatment for ATTR amyloidosis, is a humanized monoclonal antibody designed to deplete the pathogenic, non-native forms of the TTR protein

Reported results from the Phase 1 study of PRX004, the first anti-amyloid immunotherapy designed to deplete amyloid to demonstrate efficacy in ATTR amyloidosis. In the first report of clinical results with this depleter mechanism of action, PRX004 showed favorable results as demonstrated by slowing of neuropathy progression for all 7 evaluable patients at 9 months, including improvement in neuropathy in 3 of the 7 patients, and improved cardiac systolic function for all 7 patients. In this Phase 1 study, PRX004 was found to be generally safe and well tolerated across all dose levels.
PRX012, a potential treatment for Alzheimer’s disease, is monoclonal antibody targeting key epitopes within the N-terminus of Aβ

Presented data from next generation anti-Aβ antibodies for subcutaneous administration to improve patient access at the 13th Clinical Trials on Alzheimer’s Disease (CTAD) Conference in November 2020
Multi-immunogen vaccine, for the potential prevention and treatment of Alzheimer’s disease, is a multi-immunogen Aβ-tau vaccine

Presented data on a multi-immunogen vaccine that targets key Aβ and tau epitopes, the two main pathological proteins involved in the cause and progression of Alzheimer’s disease, at the CTAD Conference in November 2020
Corporate

Appointed Brandon Smith as Chief Business Officer, responsible for leading Prothena’s business development activities, portfolio strategic planning and alliance management activities. Mr. Smith joined Prothena after serving as Chief Operating Officer at Iconic Therapeutics
Upcoming Milestones:

Birtamimab

Phase 3 AFFIRM-AL study initiation expected mid-2021
VITAL study 9-month results expected to be presented at medical conference in 2021
Prasinezumab

New pre-specified exploratory subgroup analyses from Part 1 of the Phase 2 PASADENA study to be presented at the 15th International Conference for Alzheimer’s and Parkinson’s Diseases in March 2021 (ADPD 2021)
$60 million clinical milestone payment to be achieved upon first patient dosed in late-stage Phase 2b study in patients with early Parkinson’s disease; further details expected in 2Q 2021
Results from Part 2 of the PASADENA study expected to be presented at an upcoming medical conference
PRX004

Phase 1 study results expected to be presented at medical conference in 2021
Phase 2/3 study in patients with ATTR-cardiomyopathy expected to initiate 4Q 2021
PRX005

Preclinical data to be presented in March at ADPD 2021
IND filing expected 3Q 2021
$80 million potential payment from Bristol-Myers Squibb upon exercising their US license option in 2021
PRX012

IND filing expected in 1Q 2022
Upcoming Investor Conferences

Members of the senior management team will present and participate in investor meetings at the following upcoming investor conferences:

H.C. Wainwright Global Life Sciences Conference on Tuesday and Wednesday, March 9-10, 2021, virtual presentations will be available on demand both days
Oppenheimer 31st Annual Healthcare Conference on Wednesday March 17, 2021 at 10:00 AM ET
Stifel’s 3rd Annual CNS Day on Thursday April 1, 2021 at 8:00 AM ET
Fourth Quarter and Full Year of 2020 Financial Results and 2021 Financial Guidance

For the fourth quarter and full year of 2020, Prothena reported a net loss of $30.7 million and $111.1 million, respectively, as compared to a net loss of $21.6 million and $77.7 million for the fourth quarter and full year of 2019, respectively. Net loss per share for the fourth quarter and full year of 2020 was $0.77 and $2.78, respectively, as compared to a net loss per share of $0.54 and $1.95 for the fourth quarter and full year of 2019, respectively.

Prothena reported total revenue of $0.4 million and $0.9 million for the fourth quarter and full year of 2020, respectively, primarily due to license revenue in the fourth quarter and collaboration revenue for the full year as compared to total revenue of $0.3 million and $0.8 million for the fourth quarter and full year of 2019, respectively, primarily due to Roche collaboration revenue.

Research and development (R&D) expenses totaled $20.8 million and $74.9 million for the fourth quarter and full year of 2020, respectively, as compared to $15.5 million and $50.8 million for the fourth quarter and full year of 2019, respectively. The increase in R&D expense for the fourth quarter and full year of 2020 compared to the same periods in the prior year was primarily due to higher manufacturing costs primarily related to our PRX005, birtamimab and PRX012 programs and to a lesser extent PRX004, higher collaboration expense with Roche related to the prasinezumab program and higher R&D consulting expense. R&D expenses included non-cash share-based compensation expense of $2.1 million and $8.2 million for the fourth quarter and full year of 2020, respectively, as compared to $2.0 million and $8.1 million for the fourth quarter and full year of 2019, respectively.

General and administrative (G&A) expenses totaled $9.9 million and $38.7 million for the fourth quarter and full year of 2020, respectively, as compared to $8.1 million and $35.7 million for the fourth quarter and full year of 2019, respectively. The increase in G&A expenses for the fourth quarter and full year of 2020 compared to the same periods in the prior year was primarily related to higher costs for our director and officer insurance premiums. G&A expenses included non-cash share-based compensation expense of $3.2 million and $13.8 million for the fourth quarter and full year of 2020, respectively, as compared to $3.3 million and $15.5 million for the fourth quarter and full year of 2019, respectively.

Total non-cash share-based compensation expense was $5.2 million and $22.0 million for the fourth quarter and full year of 2020, respectively, as compared to $5.3 million and $23.6 million for the fourth quarter and full year of 2019, respectively.

As of December 31, 2020, Prothena had $298.1 million in cash, cash equivalents and restricted cash and no debt.

As of February 5, 2021, Prothena had approximately 39.9 million ordinary shares outstanding.

The Company expects the full year 2021 net cash used in operating and investing activities to be $51 to $74 million, which includes an expected $60 million milestone payment from Roche upon first patient dosed in the late-stage Phase 2b study of prasinezumab and expects to end the year with approximately $235 million in cash, cash equivalents and restricted cash (midpoint). The estimated full year 2021 net cash used in operating and investing activities is primarily driven by an estimated net loss of $79 to $111 million, which includes an estimated $20 million of non-cash share-based compensation expense.

Conference Call Details

Prothena management will discuss these results and its 2021 financial guidance during a live audio conference call today, Thursday, February 11, 2021, at 8:30 AM ET. The conference call will be made available on the Company’s website at www.prothena.com under the Investors tab in the Events and Presentations section. Following the live audio webcast, a replay will be available on the Company’s website for at least 90 days.

To access the call via dial-in, please dial (877) 887-5215 (U.S. and Canada toll free) or (315) 625-3069 (international) five minutes prior to the start time and refer to conference ID number 5677514. A replay of the call will be available until February 25, 2021 via dial-in at (855) 859-2056 (U.S. toll free) or (404) 537-3406 (international), Conference ID Number 5677514.

Clovis Oncology Presents Data in Advanced Prostate Cancer at ASCO 2021 Genitourinary Cancers Symposium Virtual Meeting

On February 11, 2021 Clovis Oncology, Inc. (NASDAQ: CLVS) reported Rubraca data being presented at the American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary Cancers Virtual Symposium 2021 (Press release, Clovis Oncology, FEB 11, 2021, View Source [SID1234574888]). These include data from the Phase 1b RAMP study evaluating Rubraca in combination with Xtandi, exploratory analyses from the pivotal TRITON2 study, and an analysis evaluating the rates of adverse events for different metastatic castration-resistant prostate cancer (mCRPC) treatments in a population of insured patients in the United States.

"We are pleased to share these data with the medical and scientific community to inform choices related to mCRPC treatment. The research into co-occurring alterations in mCRPC patients with a mutation of BRCA underscores the importance of genomic testing in men with mCRPC," said Patrick J. Mahaffy, President and CEO of Clovis Oncology. "Additionally, we are encouraged by the results from the RAMP study, which lay the groundwork for the Phase 3 CASPAR clinical trial sponsored by the Alliance for Clinical Trials in Oncology evaluating Rubraca and Xtandi as a novel combination therapy in men with first-line metastatic castration-resistant prostate cancer."

Data from the RAMP study are presented by Arpit Rao, MD, Assistant Professor, Hematology, Oncology and Transplantation at the University of Minnesota. This Phase 1b study is investigating the combination of Rubraca and Xtandi in biomarker-unselected (including BRCA1/2 mutation negative) patients with mCRPC to assess the pharmacokinetics (PK) and safety of the combination. Treatment with rucaparib and enzalutamide had no clinically significant effect on the PK profiles of either drug, and the safety profile of the combination was consistent with that associated with each drug as monotherapy. The results presented at ASCO (Free ASCO Whitepaper) GU show that this combination is well-tolerated and without any significant drug-drug interactions. Data from the RAMP study support the randomized, placebo-controlled Phase 3 CASPAR study (Alliance A031902; NCT04455750) that is studying Rubraca and Xtandi, and is the subject of a Trial in Progress (TiP) poster being presented at the ASCO (Free ASCO Whitepaper) GU meeting. It is expected to begin enrolling mCRPC patients shortly.

The CASPAR study is sponsored by the Alliance for Clinical Trials in Oncology and will enroll approximately 1,000 patients in the United States. It is expected to open at hundreds of National Clinical Trials Network (NCTN) sites nationally. This is the only combination trial of a PARP inhibitor and novel anti-androgen with an overall survival co-primary endpoint. Patients who have received prior abiraterone/apalutamide in a non-mCRPC setting are allowed to enroll to maximize applicability in the era of rapidly changing standards-of-care. The Alliance is part of the NCTN sponsored by the National Cancer Institute (NCI).

Data from the TRITON2 clinical trial are presented by Wassim Abida MD, PhD at Memorial Sloan Kettering Cancer Center. These data underscore the antitumor activity of Rubraca among men with BRCA-mutated mCRPC and commonly co-occurring genomic alterations. Alterations in tumor suppressor genes, including TP53, PTEN and RB1, are associated with poor prognosis in patients with prostate cancer. Results from TRITON2 showed antitumor activity for Rubraca in patients with BRCA-mutated mCRPC associated with or without co-occurring alterations in these genes. There was no clear difference in radiographic and PSA response rates for patients with or without co-occurring TP53, PTEN, or RB1 alterations. Based on these results, researchers concluded that patients with mCRPC associated with a BRCA alteration should be considered for treatment with rucaparib irrespective of the presence of co-occurring alterations in these tumor suppressor genes.

In addition, data from an analysis of clinically significant events (CSEs) associated with mCRPC treatments were presented by Kelvin A. Moses, MD, PhD, Associate Professor of Urology at Vanderbilt University Medical Center. Researchers designed the analysis to better understand the association between mCRPC treatments and development of CSEs in a population of insured patients in the United States. Using an administrative claims database for the period from January 2008 to March 2019, the analysis found that among available mCRPC treatments, chemotherapy-based regimens had the highest CSE rates per treatment year. These data indicate the burden of treatment for patients and can inform treatment decisions.

Clovis Oncology-sponsored e-posters are available online at www.clovisoncology.com/pipeline/scientific-presentations.

About Rubraca (rucaparib)

Rucaparib is an oral, small molecule inhibitor of PARP1, PARP2 and PARP3 being developed in multiple tumor types, including ovarian and metastatic castration-resistant prostate cancers, as monotherapy, and in combination with other anti-cancer agents. Exploratory studies in other tumor types are also underway. Rubraca is an unlicensed medical product outside of the U.S. and Europe

Rubraca U.S. FDA Approved mCRPC Indication

Rubraca is indicated for the treatment of adult patients with a deleterious BRCA mutation (germline and/or somatic)-associated metastatic castration-resistant prostate cancer (mCRPC) who have been treated with androgen receptor-directed therapy and a taxane-based chemotherapy. Select patients for therapy based on an FDA-approved companion diagnostic for Rubraca.

This indication is approved under accelerated approval based on objective response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Select Important Safety Information

Myelodysplastic Syndrome (MDS)/Acute Myeloid Leukemia (AML) has occurred in patients treated with Rubraca, and are potentially fatal adverse reactions. In 1146 treated patients, MDS/AML occurred in 20 patients (1.7%), including those in long term follow-up. Of these, 8 occurred during treatment or during the 28 day safety follow-up (0.7%). The duration of Rubraca treatment prior to the diagnosis of MDS/AML ranged from 1 month to approximately 53 months. The cases were typical of secondary MDS/cancer therapy-related AML; in all cases, patients had received previous platinum-containing regimens and/or other DNA damaging agents. In TRITON2, MDS/AML was not observed in patients with mCRPC (n=209) regardless of homologous recombination deficiency (HRD) mutation.

Do not start Rubraca until patients have recovered from hematological toxicity caused by previous chemotherapy (≤ Grade 1). Monitor complete blood counts for cytopenia at baseline and monthly thereafter for clinically significant changes during treatment. For prolonged hematological toxicities (> 4 weeks), interrupt Rubraca or reduce dose and monitor blood counts weekly until recovery. If the levels have not recovered to Grade 1 or less after 4 weeks or if MDS/AML is suspected, refer the patient to a hematologist for further investigations, including bone marrow analysis and blood sample for cytogenetics. If MDS/AML is confirmed, discontinue Rubraca.

Based on findings in genetic toxicity and animal reproduction studies, advise male patients with female partners of reproductive potential or who are pregnant to use effective methods of contraception during treatment and for 3 months following last dose of Rubraca. Advise male patients not to donate sperm during therapy and for 3 months following the last dose of Rubraca.

Most common adverse reactions in TRITON2 (≥ 20%; Grade 1-4) were fatigue/asthenia (62%), nausea (52%), anemia (43%), AST/ALT elevation (33%), decreased appetite (28%), rash (27%), constipation (27%), thrombocytopenia (25%), vomiting (22%), and diarrhea (20%).

Co-administration of rucaparib can increase the systemic exposure of CYP1A2, CYP3A, CYP2C9, or CYP2C19 substrates, which may increase the risk of toxicities of these drugs. Adjust dosage of CYP1A2, CYP3A, CYP2C9, or CYP2C19 substrates, if clinically indicated. If co-administration with warfarin (a CYP2C9 substrate) cannot be avoided, consider increasing frequency of international normalized ratio (INR) monitoring.

AbClone announces the excellence of its CAR-T pipeline at the European CAR-TCR conference

On February 10, 2021 AbClon reported that the company will participate in the "CAR-TCR Europe Summit" (Press release, AbClon, FEB 10, 2021, View Source;wr_id=131&page=4 [SID1234638628]).

AT101 CD19 targets disease protein B As a treatment for cell-derived blood cancer, existing CD19 CAR-T shows anticancer effect AT101 For the model)Yescata, etc., , AT101. is seeking differentiation by developing new humanized antibodies with completely different action sites, while FMC63Cell therapy uses mouse-derived antibodies.

AT501is HER2 an ovarian cancer treatment targeting disease protein, This is a material applied with AbClone’s original technology Switchable CAR-T (zCAR-T) . By controlling the concentration of the switch substance administered in the body CAR-T is a mechanism that regulates the activity of cells. CAR-T Disadvantage of cell therapy CRS (Cytokine Release Syndrome) It is possible to structurally overcome. In addition, , its platform nature has been strengthened as cross-application is possible for various cancer types by changing the switch material. Innovation CAR-T is a cell therapy.

PCI Biotech – release of the RNA Therapeutics Virtual Conference presentation

On February 10, 2021 PCI Biotech (OSE: PCIB), a clinical-stage biopharma company developing innovative therapeutics that address significant unmet medical needs in cancer reported the fimaNAc focused presentation for the 12th Annual RNA Therapeutics Virtual Conference, a UK based online event taking place February 10-11, 2021 (Press release, PCI Biotech, FEB 10, 2021, View Source [SID1234585153]). The 2021 conference is set to explore the latest developments in RNA delivery agents and RNA-based therapeutics with the latest case studies on advanced mRNA technologies, oligonucleotide delivery, therapeutic applications and future trends and innovations. PCI Biotech is also a sponsor of the event.

Today, Wednesday February 10 2021, at 13:10pm (CET) Dr. Anders Høgset, CSO, will present an overview of PCI Biotech’s proprietary platform technology, focusing on the delivery of RNA molecules, including the most recent data on the use of the fimaNAc delivery technology in the exciting field of RNA based therapies.

Per Walday, CEO of PCI Biotech, comments: Encouraging collaborative preclinical results have recently been achieved with the fimaNAc technology and we are very pleased to be able to share these data now. The rapid development progress of mRNA-based vaccines against Covid-19 has generated much attention to the potential of nucleic acid therapeutics generally, and mRNA particularly. The results with fimaNAc suggests that this technology provides an appealing intracellular delivery solution for certain applications within this class of therapeutics.

An online recording of the full presentation held by Dr Høgset will be made available on PCI Biotech’s website (www.pcibiotech.com) under "Scientific publications & presentations" after the event.

Sensei Biotherapeutics Announces Closing of Initial Public Offering

On February 10, 2021 Sensei Biotherapeutics, Inc., a clinical-stage immunotherapy company focused on the discovery and development of next generation therapeutics for cancer, reported the closing of its initial public offering of 7,000,052 shares of its common stock on February 8, 2021, as well as the issuance of an additional 1,030,243 shares pursuant to the exercise by the underwriters of their option to purchase additional shares on February 10, 2021, at a public offering price of $19.00 per share (Press release, Sensei Biotherapeutics, FEB 10, 2021, View Source [SID1234575166]). The gross proceeds to Sensei from the two closings, before deducting underwriting discounts and commissions and other offering expenses payable by Sensei, were approximately $152.6 million. All of the shares in the offering were offered by Sensei. Sensei’s common stock is listed on the Nasdaq Global Market under the ticker symbol "SNSE."

Citigroup, Piper Sandler & Co. and Berenberg acted as joint book-running managers for the offering. Oppenheimer & Co. acted as the lead manager for the proposed offering.

A registration statement relating to the securities was declared effective by the Securities and Exchange Commission on February 3, 2021. The offering of these securities is being made only by means of a prospectus. A copy of the final prospectus may be obtained from Citigroup Global Markets Inc., c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, or by telephone at 800-831-9146 or by email at [email protected]; Piper Sandler & Co., Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, or by telephone at 800-747-3924 or by email at [email protected]; or Berenberg Capital Markets LLC, Attention: Investment Banking, 1251 Avenue of the Americas, 53rd Floor, New York, NY 10020, or by telephone at 646-949-9000 or by email at [email protected].

This press release shall not constitute an offer to sell, or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation, or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.