On February 17, 2022 At the 2022 ASCO (Free ASCO Whitepaper) GU Cancers Symposium Bayer reported that results from the Phase III ARASENS trial which demonstrated that the use of the oral androgen receptor inhibitor (ARi) darolutamide plus androgen deprivation therapy (ADT) and docetaxel significantly increased overall survival (OS) in patients with metastatic hormone-sensitive prostate cancer (mHSPC) compared to ADT plus docetaxel. Darolutamide plus ADT and docetaxel significantly reduced the risk of death by 32.5% compared to ADT plus docetaxel (HR=0.68, 95% CI 0.57-0.80; P<0.001) (Press release, Bayer, FEB 17, 2022, View Source;ref=irrefndcd [SID1234608324]). At the data cutoff date for the primary analysis (October 25, 2021), the median treatment duration was longer for darolutamide plus ADT and docetaxel (41.0 months) versus ADT plus docetaxel (16.7 months).

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Amid these positive results Bayer raised peak sales expectation for Nubeqa (darolutamide) to exceed €3 billion.

"Subject to regulatory approval, the team at Bayer is excited to be able to offer even more patients suffering from prostate cancer an additional treatment option backed by strong clinical data," said Stefan Oelrich, Member of the Board of Management of Bayer and President of the Pharmaceuticals Division. "With the confirmation of darolutamide’s clinical profile and expansion into the metastatic setting as well as the investments that we are making in clinical trials in other potential indications, we feel that Nubeqa has the potential to generate peak sales of more than 3 billion euros".

Darolutamide is developed jointly by Bayer and Orion Corporation, a globally operating Finnish pharmaceutical company. Based on results from the pivotal Phase III ARAMIS trial, the compound is already approved for the treatment of patients with nmCRPC, who are at high risk of developing metastatic disease, in more than 60 markets worldwide. Results from the second Phase III ARASENS trial evaluating darolutamide plus androgen deprivation therapy (ADT) in combination with docetaxel in patients with metastatic hormone-sensitive prostate cancer (mHSPC) were presented yesterday as an oral presentation at the 2022 ASCO (Free ASCO Whitepaper) GU Cancer Symposium and simultaneously published in The New England Journal of Medicine. Bayer is already in discussions with health authorities worldwide regarding the submission for marketing authorization in this additional indication.

Darolutamide is being investigated in a broad development program with additional three ongoing or planned large clinical studies, to investigate its potential across prostate cancer patients from the early- to the late-stage of this disease. This includes another Phase III trial in mHSPC (ARANOTE) as well as an ANZUP-led international co-operative group Phase III trial, evaluating darolutamide as an adjuvant treatment for localized prostate cancer with very high risk of recurrence (DASL-HiCaP, ANZUP1801). Information about these trials can be found at www.clinicaltrials.gov. In addition, a study to explore the potential of darolutamide in the early setting for patients who have been treated with surgery or radiation and now see a rise in their prostate specific antigen (PSA) levels is also planned.

About Nubeqa (darolutamide)
Darolutamide is an oral androgen receptor inhibitor (ARi) with a distinct chemical structure that binds to the receptor with high affinity and exhibits strong antagonistic activity, thereby inhibiting the receptor function and the growth of prostate cancer cells. The low potential for blood-brain barrier penetration for darolutamide is supported by preclinical models and neuroimaging data in healthy humans. A low blood-brain barrier penetration would explain the overall low incidence of central nervous system (CNS)-related adverse events (AEs) compared to placebo as seen in the ARAMIS and ARASENS Phase III trials and the improved verbal learning and memory observed in the darolutamide arm of the Phase II ODENZA trial.

The product is approved under the brand name Nubeqa in more than 60 markets around the world, including the U.S., EU, Japan, China, for the treatment of patients with non-metastatic castration-resistant prostate cancer (nmCRPC), who are at high risk of developing metastatic disease. The compound is also currently being investigated in further studies across various stages of prostate cancer, including another Phase III trial in mHSPC (ARANOTE) as well as an ANZUP-led international co-operative group Phase III trial, evaluating darolutamide as an adjuvant treatment for localized prostate cancer with very high risk of recurrence (DASL-HiCaP, ANZUP1801). Information about these trials can be found at www.clinicaltrials.gov.

On February 17, 2022 Scandion Oncology (Scandion) reported its fourth quarter and year-end report for 2021 (Press release, Scandion Oncology, FEB 17, 2022, View Source,c3508355 [SID1234608203]). The following is taken from the report.

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Bo Rode Hansen, President and CEO, comments

"The fourth quarter completed a busy and successful 2021 for Scandion in which we transformed the company on substantially all accounts, increasing its fundamental value. We have prepared for the future with continued focus on fundamental value creation and are poised to enter the right partnerships for Scandion when the data are ready."

Highlights during Q4 2021

On November 8, Scandion Oncology announced that the timeline for read-out of the dose-finding clinical Phase Ib study PANTAX will be extended, and read-out is expected in Q2-Q3 2022. The reasons are challenging patient recruitment and a staggered study design, as requested by the German authorities. Disregarding this postponement, the study is performing as well as the Company could have hoped for.
Highlights after the end of the period

On January 12, Scandion Oncology announced that Mads Kronborg, bringing more than a decade of corporate communication and investor relations experience in the global life-science industry, will now help plan and drive its external communication as Head of External Communication.
On January 18, Scandion Oncology announced that data with the Company’s lead compound SCO-101 as combination therapy in patients with metastatic colorectal cancer was accepted for poster presentation at the ASCO (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium.
On February 2, Scandion Oncology announced approval from the German and Spanish regulatory authorities to expand part 2 of the CORIST Phase II study to Germany and Spain.
The Q4 and year-end report 2021 is available on the Company’s website: www.scandiononcology.com.

Audiocast today, February 17 at 08:30 am CET

Today at 08:30, Scandion Oncology’s executive management will host a webcast and conference call presenting the results and a company update.

At the end of the presentation there will be a Q&A session.

Access to the event can be obtained as follows:

View Source

REPLAY access:

Webcast replay will be available at www.scandiononcology.com in the Investors section and at www.financialhearings.com

The information was provided by the contact person above for publication on February 17, 2022, at 07.30 CET.

On February 17, 2022 Celsion Corporation (NASDAQ: CLSN), a clinical-stage company focused on DNA-based immunotherapy and next-generation vaccines, reported that following a pre-planned interim safety review of 81 as treated patients randomized in the Phase I/II OVATION 2 Study with GEN-1 in advanced (Stage III/IV) ovarian cancer, the Data Safety Monitoring Board (DSMB) has unanimously recommended that the OVATION 2 Study continue treating patients with the dose of 100 mg/m2 (Press release, Celsion, FEB 17, 2022, View Source [SID1234608224]). The DSMB also determined that safety is satisfactory with an acceptable risk/benefit, and that patients tolerate up to 17 doses of GEN-1 during a course of treatment that lasts up to six months. No dose-limiting toxicities were reported.

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The OVATION 2 Study combines GEN-1, the Company’s IL-12 gene-mediated immunotherapy, with standard-of-care neoadjuvant chemotherapy (NACT) in patients newly diagnosed with Stage III/IV ovarian cancer. NACT is designed to shrink the cancer as much as possible for optimal surgical removal after three cycles of chemotherapy. Following NACT, patients undergo interval debulking surgery, followed by three additional cycles of chemotherapy to treat any residual tumor.

The OVATION 2 Study is designed with an 80% confidence interval for an observed Progression Free Survival (PFS) Hazard Ratio of 0.75, which would mean an approximate 33% improvement in risk for cancer progression when comparing the treatment arm (NACT + GEN-1) with the control arm (NACT only). GEN-1 is an immunotherapy that produces safe and durable local levels of IL-12, a pluripotent cytokine associated with the stimulation of innate and adaptive immune response against cancer. The GEN-1 nanoparticle comprises a DNA plasmid encoding IL-12 gene and a synthetic polymer facilitating plasmid delivery vector. Cell transfection is followed by persistent, local secretion of the IL-12 protein at therapeutic levels.

The Company also announced that more than 75% of the projected 110 patients have been enrolled in the OVATION 2 Study. Interim clinical data from the first 39 patients who have undergone interval debulking surgery showed that the GEN-1 treatment arm is showing a 27% improvement in R0 surgical resection rate over the control arm. A complete tumor resection (R0) is a microscopically margin-negative resection in which no gross or microscopic tumor remains in the tumor bed.

"Findings from our OVATION I and OVATION 2 studies show a consistent dose-dependent clinical response in both surgical outcome and tumor response, which is further supported by translational data of the tumor microenvironment," noted Nicholas Borys, M.D., Celsion’s executive vice president and chief medical officer. "We are encouraged by the current rate of patient recruitment and expect to complete enrollment by mid-2022. The primary endpoint for the study is progression-free survival (PFS) which we expect to report approximately 12 months after patient enrollment is completed."

In February 2021, the Company announced that GEN-1 received FDA Fast Track Designation in advanced ovarian cancer. Celsion plans to request FDA Breakthrough Therapy Designation for GEN-1 based on the encouraging clinical data.

"We thank the DSMB members for their work and advice," said Michael H. Tardugno, Celsion’s chairman, president and chief executive officer. "FDA Fast Track and Orphan Drug Designations for GEN-1 in advanced ovarian cancer are important for our future commercialization efforts. In addition, under the Biologics Price Competition and Innovation Act of 2009, sponsors of new, licensed biological products like GEN-1 that are approved through a Biologics License Application receive 12 years of market exclusivity. The FDA cannot license any 351(k) application for a biosimilar or interchangeable product that relies on the previously approved product as a reference for biosimilarity during this 12-year period."

About GEN-1 Immunotherapy

GEN-1, designed using Celsion’s proprietary TheraPlas platform technology, is an IL-12 DNA plasmid vector encased in a nanoparticle delivery system, which enables cell transfection followed by persistent, local secretion of the IL-12 protein. IL-12 is one of the most active cytokines for the induction of potent anti-cancer immunity acting through the induction of T-lymphocyte and natural killer (NK) cell proliferation. The Company previously reported positive safety and encouraging Phase I results with GEN-1 given as monotherapy or a combination therapy in patients with advanced peritoneally metastasized primary or recurrent ovarian cancer, and recently completed a Phase Ib dose-escalation trial (OVATION 1 Study) of GEN-1 in combination with carboplatin and paclitaxel in patients with newly diagnosed ovarian cancer.

About Epithelial Ovarian Cancer

Epithelial ovarian cancer (EOC) is the fifth deadliest malignancy among women in the United States. There are approximately 22,000 new cases of ovarian cancer every year and the majority (approximately 70%) are diagnosed in advanced stages III and IV. EOC is characterized by dissemination of tumor in the peritoneal cavity with a high risk of recurrence (75%, stage III and IV) after surgery and chemotherapy. Since the five-year survival rates of patients with stages III and IV disease at diagnosis are poor (41% and 20%, respectively), there remains a need for a therapy that not only reduces the recurrence rate but also improves overall survival. The peritoneal cavity of advanced ovarian cancer patients contains the primary tumor environment and is an attractive target for regional approach to immune modulation.

On February 17, 2022 Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the "Company" or "Quoin"), a specialty pharmaceutical company focused on rare and orphan diseases, reported that its wholly-owned subsidiary, Quoin Pharmaceuticals, Inc., has entered into an exclusive Distribution Agreement with Neopharm Medical Supplies, an exclusive distributor for leading medical and pharma manufacturers in Israel, for QRX003, the Company’s investigational treatment for Netherton Syndrome, a rare and devastating genetic disease for which there is currently no available treatment or cure (Press release, NeoPharm, FEB 17, 2022, View Source [SID1234608241]).

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Under the terms of the revenue sharing agreement, Neopharm gains exclusive rights to commercialize QRX003 in Israel. Quoin will be the exclusive supplier of QRX003 to Neopharm.

Dr. Michael Myers, Chief Executive Officer of Quoin, commented, "This is Quoin’s sixth distribution agreement in the last four months for QRX003 and we’re excited to add Neopharm, who is a leading distributor in Israel. We now have 54 countries covered by our partnership agreements for QRX003, encompassing many regions of the globe. Establishing a broad distribution network is a key part of our commitment to ensure that every patient, everywhere will have access to this product once it has been approved for this devastating disease."

About Netherton Syndrome

Netherton Syndrome, a form of Ichthyosis, is a rare, hereditary skin disorder caused by a mutation in the SPINK5 gene (serine protease inhibitor, Kazal Type 5) that leads to severe skin barrier defects and recurring infections, as well as a pronounced predisposition to allergies, asthma, and eczema. Patients also often suffer from severe dehydration, chronic skin inflammation and stunted growth.

Currently, there is no cure for Netherton Syndrome, nor are there any approved therapeutic treatments.

On February 17, 2022 Prothena Corporation plc (NASDAQ:PRTA), a late-stage clinical biotechnology company with a robust pipeline of investigational therapeutics built on protein dysregulation expertise, reported financial results for the fourth quarter and full year 2021 (Press release, Prothena, FEB 17, 2022, View Source [SID1234608258]). In addition, the Company provided 2022 financial guidance and an update on business highlights.

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"Prothena made meaningful progress in multiple therapeutic indications across our portfolio in 2021 with the advancement of three clinical stage programs. We announced the initiation of the confirmatory Phase 3 AFFIRM-AL study of birtamimab, Phase 2b PADOVA study of prasinezumab, and Phase 1 study of PRX005. Additionally, we presented positive preclinical findings for our anti-Aβ PRX012 and our dual Aβ/tau vaccine at AAIC in June last year," said Gene Kinney, Ph.D., President and Chief Executive Officer of Prothena. "In 2021, we also received $200 million from strategic partnerships with leading pharmaceutical companies and bolstered our cash position with $175 million raised through equity offerings. In 2022, we look forward to multiple scientific congresses starting with the presentation of additional preclinical data at AD/PD in March. Our strong capital position funds Prothena through multiple value-creating milestones as we transition into a fully integrated commercial company."

2021 Business Highlights and Upcoming Milestones

Neurodegenerative Diseases Portfolio

Alzheimer’s Disease (AD)

PRX012, a potential best-in-class treatment for AD, is an investigational monoclonal antibody targeting a key epitope at the N-terminus of amyloid beta (Aβ) with high binding potency

•Presented preclinical results at the Alzheimer’s Association International Conference in 2021 (AAIC) demonstrating that PRX012 significantly cleared both pyroglutamate-modified and -unmodified Aβ plaque in post-mortem brain tissue of late-stage AD patients
•Investigational New Drug (IND) application filing expected 1Q 2022

PRX005, a potential best-in-class treatment for AD, is an investigational antibody that specifically targets a key epitope within the microtubule binding region (MTBR) of tau, a protein implicated in diseases including AD, frontotemporal dementia (FTD), progressive supranuclear palsy (PSP), chronic traumatic encephalopathy (CTE), and other tauopathies. PRX005 is part of the global neuroscience research and development collaboration with Bristol Myers Squibb

•Received $80 million option payment from Bristol Myers Squibb for execution of U.S. license agreement in 2021
•Phase 1 study initiated in 2021
•Topline Phase 1 data expected in 2022

Dual Aβ/tau vaccine, a potential first-in-class treatment and prevention for AD, is a dual-target vaccine targeting key epitopes within the Aβ and tau proteins to promote amyloid clearance and blockade of pathogenic tau

•AAIC presentation in 2021 showcased preclinical data demonstrating that Prothena’s dual Aβ/tau vaccine generated appropriate and balanced antibody titers promoting both phagocytosis of Aβ plaque and blockade of tau transmission in vitro
•Presentation of preclinical data at the International Conference on Alzheimer’s and Parkinson’s Diseases (AD/PD) expected in March 2022
•IND filing expected in 2023

Parkinson’s Disease (PD)

Prasinezumab, a potential first-in-class treatment for PD, 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
•Earned $60 million clinical milestone payment in 2021 upon dosing of the first patient in the global Phase 2b PADOVA study for prasinezumab (NCT#04777331)
•Presentation of additional data by Roche at AD/PD expected in March 2022
•Phase 2b PADOVA study results expected in 2024

Rare Peripheral Amyloid Diseases Portfolio

AL Amyloidosis (AL)

Birtamimab, a potential best-in-class amyloid depleter treatment for AL, is an investigational humanized monoclonal antibody designed to directly neutralize soluble toxic aggregates and promote clearance of amyloid that causes organ dysfunction and failure

•Reached Special Protocol Assessment (SPA) agreement with FDA at p≤0.10 and initiated confirmatory Phase 3 AFFIRM-AL study of birtamimab in Mayo Stage IV patients with AL amyloidosis in 2021 (NCT#04973137)
•Confirmatory Phase 3 AFFIRM-AL study results expected in 2024

ATTR Amyloidosis (ATTR)

PRX004, a potential first-in-class treatment for ATTR, is a humanized monoclonal antibody designed to deplete the pathogenic, non-native forms of the TTR protein, and is being developed by Novo Nordisk for the treatment of ATTR cardiomyopathy

•Announced Novo Nordisk acquisition of ATTR business for a total aggregate of up to $1.23 billion and Prothena received $60 million up front payment in 2021
•Novo Nordisk expected to initiate a Phase 2 trial in 1H 2022 with PRX004 for the treatment of ATTR cardiomyopathy

2021 Organizational Highlights

•Sanjiv Patel, MBBS, MA, MBA, appointed to the Board of Directors
•Hideki Garren, M.D., Ph.D., appointed to Chief Medical Officer
•Tran Nguyen, Chief Financial Officer, appointed to the additional, newly created role of Chief Strategy Officer
•Brandon Smith promoted from Chief Business Officer to Chief Operating Officer

Upcoming Investor Conference

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

•Oppenheimer 32nd Annual Healthcare Conference, March 15, 2022, at 1:20 PM ET

Fourth Quarter and Full Year of 2021 Financial Results
For the fourth quarter and full year of 2021, Prothena reported a net loss of $33.2 million and net income of $67.0 million, respectively, as compared to a net loss of $30.7 million and $111.1 million for the fourth quarter and full year of 2020, respectively. Net loss per share for the fourth quarter of 2021 was $0.71 and net income per share on a diluted basis for the full year of 2021 was $1.38, as compared to net loss per share of $0.77 and $2.78 for the fourth quarter and full year of 2020, respectively.
Prothena reported total revenue of $1.2 million and $200.6 million for the fourth quarter and full year of 2021, respectively. Revenue for the fourth quarter of 2021 related to $1.2 million from Bristol Myers Squibb. Revenue for the full year of 2021, included $79.7 million from Bristol Myers Squibb for PRX005 U.S. License and U.S. Development Services and $60.7 million from the sale of the intellectual property and related rights to the Company’s ATTR amyloidosis business and pipeline to Novo Nordisk. In addition, the full year revenue included $60.0 million in clinical milestone payment from Roche related to the global Phase 2b PADOVA study for prasinezumab and a nominal amount of license revenue from Roche. This compares to total revenue of $0.4 million and $0.9 million for the fourth quarter and full year of 2020, primarily from collaboration revenue from Roche.

Research and development (R&D) expenses totaled $22.1 million and $82.3 million for the fourth quarter and full year of 2021, respectively, as compared to $20.8 million and $74.9 million for the fourth quarter and full year of 2020, respectively. The increase in R&D expense for the fourth quarter and full year of 2021 compared to the same periods in the prior year was primarily due to higher personnel expenses, higher clinical trial expenses primarily related to the birtamimab and PRX005 programs (offset in part by lower PRX004 clinical trial expense); offset in part by lower collaboration expenses related to the prasinezumab program with Roche as a result of the cost share opt-out exercised in May 2021 and lower manufacturing costs primarily related to PRX005 and birtamimab programs (offset in part by higher PRX012 preclinical expense). R&D expenses included non-cash share-based compensation expense of $2.9 million and $9.5 million for the fourth quarter and full year of 2021, respectively, as compared to $2.1 million and $8.2 million for the fourth quarter and full year of 2020, respectively.
General and administrative (G&A) expenses totaled $12.2 million and $46.3 million for the fourth quarter and full year of 2021, respectively, as compared to $9.9 million and $38.7 million for the fourth quarter and full year of 2020, respectively. The increase in G&A expenses for the fourth quarter and full year of 2021 compared to the same periods in the prior year was primarily related to higher personnel expenses, legal expenses, consulting and expense for our director and officer insurance premium. G&A expenses included non-cash share-based compensation expense of $4.0 million and $15.1 million for the fourth quarter and full year of 2021, respectively, as compared to $3.2 million and $13.8 million for the fourth quarter and full year of 2020, respectively.
Total non-cash share-based compensation expense was $6.9 million and $24.7 million for the fourth quarter and full year of 2021, respectively, as compared to $5.2 million and $22.0 million for the fourth quarter and full year of 2020.

As of December 31, 2021, Prothena had $580.4 million in cash, cash equivalents and restricted cash, and no debt. This includes net proceeds raised of $175 million raised through equity offerings and a total of $200 million in payments from partners Bristol Myers Squibb, Novo Nordisk and Roche.
As of February 11, 2022, Prothena had approximately 46.7 million ordinary shares outstanding.

2022 Financial Guidance

The Company expects the full year 2022 net cash used in operating and investing activities to be $120 to $132 million, which includes an expected $40 million clinical milestone payment from Novo Nordisk and expects to end the year with approximately $454 million in cash, cash equivalents and restricted cash (midpoint). The estimated full year 2022 net cash used in operating and investing activities is primarily driven by an estimated net loss of $154 to $170 million, which includes an estimated $32 million of non-cash share-based compensation expense.

Conference Call Details

Prothena management will discuss these results and its 2022 financial guidance during a live audio conference call today, Thursday, February 17, 2022, at 4:30 PM 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 (888) 440-6385 (U.S. and Canada toll free) or +00 1 646 960-0180 (international) five minutes prior to the start time and refer to conference ID number 92750. A

replay of the call will be available until March 3, 2022, via dial-in at (800) 770-2030 (U.S. toll free) or +00 1 647 362-9199 (international), Conference ID Number 92750.