On May 14, 2020 INOVIO (NASDAQ: INO) reported that 85 percent (44 out of 52) of patients newly diagnosed with the deadly brain cancer glioblastoma multiforme (GBM) who received the company’s DNA medicine INO-5401, in combination with INO-9012 and PD-1 inhibitor Libtayo (cemiplimab), were alive for at least 12 months or more (overall survival at 12 months: OS12) following treatment (Press release, Inovio, MAY 14, 2020, View Source [SID1234558053]). These data will be featured at an oral poster presentation at the ASCO (Free ASCO Whitepaper) 2020 Virtual Scientific Program, May 29-31, 2020.

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GBM is the most common and aggressive type of brain cancer. Currently, the median overall survival with standard of care therapy, which includes radiation and chemotherapy (temozolomide: TMZ), is approximately 15 to 22 months.

The Phase 1/2 clinical trial demonstrated that 84.4% percent (27 of 32) of patients with MGMT promoter unmethylated tumors, and 85% (17 of 20) of patients with MGMT promoter methylated tumors were alive at 12 months. This promising clinical result is coupled with a robust immunological response to all three cancer antigens in INO-5401, including human telomerase (hTERT), Wilms Tumor-1 (WT-1) and prostate specific membrane antigen (PSMA). Activated, cytotoxic T cells directed towards these cancer antigens commonly expressed on GBM tumors were detected in all patients tested to date and continue to support the immunogenic potential of INOVIO’s DNA medicines. Importantly, INO-5401 + INO-9012 was safe and well-tolerated when given not only with radiation and TMZ, but also with PD-1 inhibition with Libtayo, which is being jointly developed by Regeneron and Sanofi. These results are being presented in a virtual format at the 2020 Annual ASCO (Free ASCO Whitepaper) meeting (Abstract #2514).

Dr. David Reardon, Clinical Director, Center for Neuro-Oncology of Dana-Farber Cancer Institute and coordinating principal investigator of GBM-001 said, "Although these data are preliminary, and follow-up remains early, this novel combination of a cancer antigen-specific, T cell generating DNA medicine with a PD-1 inhibitor is exciting and may overcome more than 20 years of a standard of care that has proven sub-optimal for our patients with GBM. A tolerable, new combination of medicines utilizing a novel mechanism of action, such as that provided by INO-5401 and INO-9012 with cemiplimab, is very welcome for this hard-to-treat brain cancer, especially when shown to be tolerable with standards such as radiation and chemotherapy, and when demonstrating the immunogenicity seen in the GBM-001 study."

Dr. J. Joseph Kim, INOVIO’s President & CEO, said, "While we recognize these data are early, we are very excited to see robust immunogenicity and the potential for extending survival, coupled with a clear ability to be able to combine not only with the standard of care, but with a checkpoint inhibitor, Libtayo. Where others have failed with single-agent checkpoint inhibition in GBM, our DNA medicine combined with Libtayo and standard of care has demonstrated clear immunogenicity and the potential to extend overall survival."

In a previous announcement, INOVIO reported key interim data from the 52-patient clinical trial showed that 80% (16 of 20) of MGMT gene promoter methylated patients and 75% (24 of 32) unmethylated patients were progression-free at six months (PFS6) measured from the time of their first dose, exceeding historical standard-of-care data.

This immunotherapy combination with a PD-1 checkpoint inhibitor also exhibited supportive safety, tolerability, and immunogenicity data and suggested an acceptable safety profile consistent with that of Libtayo and INOVIO’s platform technology. The majority of patients tested had a T cell immune response to one or more tumor-associated antigens encoded by INO-5401. Immune responses to all three tumor-associated antigens were demonstrated in this study. INOVIO plans to report 18-month overall survival data later this year.

Study Design

The trial was designed to evaluate safety, immunogenicity and preliminary efficacy of INO-5401 and INO-9012 in combination with Libtayo, with radiation and chemotherapy, in subjects with newly-diagnosed glioblastoma (GBM). This is a Phase 1/2, open-label, multi-center trial conducted in 52 evaluable patients with GBM. There are two cohorts in this trial. Cohort A includes 32 participants with a tumor with an unmethylated O6-methylguanine-deoxyribonucleic acid (DNA) methyltransferase (MGMT) promoter. Cohort B includes 20 participants with a tumor with a MGMT methylated promoter. Both cohorts received INO-5401 and INO-9012 and Libtayo at the same doses and on the same dosing schedule, and both cohorts received radiation and temozolomide (TMZ). Interim data presented here and at SITC (Free SITC Whitepaper) was obtained as of October 2019 and overall survival data at 18 months is expected in Q4 2020. For more information of the clinical study, see www.clinicaltrials.gov, identifier NCT03491683.

Poster Details

Abstract/Poster 2514
Poster Discussion Session: Central Nervous System Tumors
The ASCO (Free ASCO Whitepaper) 2020 Virtual Scientific Program runs from May 29 -31.

About Glioblastoma Multiforme (GBM)

GBM is the most common and aggressive type of brain cancer and remains a devastating disease for both patients and caregivers. Its prognosis is extremely poor, despite a limited number of new therapies approved over the last 10 years. The median overall survival for patients receiving standard of care therapy is approximately 15 to 22 months and the median progression-free survival is approximately 7 months. In the U.S., the estimated annual incidence of GBM is 11,362 cases or 3.21 cases per 100,000 persons and the median age at diagnosis is 65 years.

About INO-5401 and INO-9012

INO-5401 encodes for INOVIO’s SynCon antigens for hTERT, WT1, and PSMA, and has the potential to be a powerful cancer immunotherapy in combination with checkpoint inhibitors. The National Cancer Institute previously highlighted hTERT, WT1, and PSMA among a list of important cancer antigens, designating them as high priorities for cancer immunotherapy development. These three antigens were reported to be over-expressed, and often mutated, in a variety of human cancers, and targeting these antigens may prove efficacious in the treatment of patients with cancer. INO-9012 encodes for IL-12, which is a T cell immune activator.

About INOVIO’s DNA Medicines Platform

INOVIO has 15 DNA medicine clinical programs currently in development focused on HPV-associated diseases, cancer, and infectious diseases, including coronaviruses associated with MERS and COVID-19 diseases being developed under grants from the Coalition for Epidemic Preparedness Innovations (CEPI). DNA medicines are composed of optimized DNA plasmids, which are small circles of double-stranded DNA that are synthesized or reorganized by a computer sequencing technology and designed to produce a specific immune response in the body.

INOVIO’s DNA medicines deliver optimized plasmids directly into cells intramuscularly or intradermally using INOVIO’s proprietary hand-held smart device called CELLECTRA. The CELLECTRA device uses a brief electrical pulse to reversibly open small pores in the cell to allow the plasmids to enter, overcoming a key limitation of other DNA and other nucleic acid approaches, such as mRNA. Once inside the cell, the DNA plasmids enable the cell to produce the targeted antigen. The antigen is processed naturally in the cell and triggers the desired T cell and antibody-mediated immune responses. Administration with the CELLECTRA device ensures that the DNA medicine is efficiently delivered directly into the body’s cells, where it can go to work to drive an immune response. INOVIO’s DNA medicines do not interfere with or change in any way an individual’s own DNA. The advantages of INOVIO’s DNA medicine platform are how fast DNA medicines can be designed and manufactured, the stability of the products which do not require freezing in storage and transport, and the robust immune response, safety profile, and tolerability that have been demonstrated in clinical trials.

With more than 2,000 patients receiving INOVIO investigational DNA medicines in more than 6,000 applications across a range of clinical trials, INOVIO has a strong track record of rapidly generating DNA medicine candidates with potential to meet urgent global health needs.

On May 14, 2020 Bavarian Nordic A/S (OMX: BAVA, OTC: BVNRY) reported its interim financial results and business progress for the first three months of 2020 (Press release, Bavarian Nordic, MAY 14, 2020, View Source [SID1234558052]).

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Paul Chaplin, President & Chief Executive Officer of Bavarian Nordic said: "The beginning of 2020 has been a productive period for Bavarian Nordic with continued progress on our strategic initiatives. Upon completion of the acquisition of Rabipur/RabAvert and Encepur from GSK, we entered the new year as a commercial vaccine company and are today reporting revenues of the acquired products for the first time. While we see increased uncertainties in certain markets and segments due to the COVID-19 situation, we are pleased to maintain our full-year guidance, supported by strong outlook in other parts of our business, including the recent large smallpox vaccine order from the U.S. government. With strong support as well as appreciation of our updated strategy from existing and new shareholders, we also completed the planned rights issue to finance the acquisition from GSK. As a company engaged in public health and preparedness, we feel the urgency to pursue a viable vaccine for COVID-19 and are extremely pleased to support AdaptVac and an international consortium in pursuing a capsid virus like particle-based vaccine, which we believe has the real possibility to meet the WHO criteria of a single shot vaccine to protect all populations from COVID-19."

Financial highlights from the first quarter

Revenues of DKK 365 million comprised of DKK 321 million from sale of the new products, Rabipur/RabAvert and Encepur and DKK 44 million from contract work
Other operating income of DKK 628 million from sale of Priority Review Voucher
EBITDA of DKK 641 million
Cash flow from financing activities was positive with DKK 1,363 million following completion of rights issue in March generating DKK 2,824 million in gross proceeds, partly offset by repayment of DKK 1,382 million bridge financing for upfront payment to GSK (including amortized costs of DKK 9 million)
Strong cash position of DKK 2,205 million at end of the quarter, excluding unutilized credit facilities of DKK 244 million
Outlook for the year maintained with revenues of approximately DKK 1,900 million and EBITDA of approximately DKK 675 million.
DKK million Q1 2020 Q1 2019 2020 Guidance
Revenue 365 127 1,900
EBITDA 641 (90) 675
Securities, cash and cash equivalents 2,205 1,928 1,350
Events after the reporting date

In April, the Company was awarded a new USD 202 million order for JYNNEOS smallpox vaccine from the U.S. government with manufacturing and deliveries occurring in 2020 and 2021. This in addition to the USD 299 million order for delivery of freeze-dried doses and brings the total value of current orders and options beyond USD 500 million.
In May, the Company signed an exclusive heads of agreement with AdaptVac to license their proprietary capsid virus like particle (VLP) based SARS-CoV-2 subunit (COVID-19) vaccine. A final license agreement is expected to be in place by end of first half 2020. As part of the agreement, Bavarian Nordic will be responsible for the clinical development and global commercialization of the vaccine.
Conference call and webcast
The management of Bavarian Nordic will host a conference call today at 2 pm CEST (8 am EDT) to present the interim results followed by a Q&A session. A listen-only version of the call can be accessed via View Source To join the Q&A session, use one of the following dial-in numbers: Denmark: +45 32 72 80 42, UK: +44 (0) 844 571 8892, USA: +1 631-510-7495. Participant code is 5654328.

On May 14, 2020 argenx (Euronext & Nasdaq: ARGX), a clinical-stage biotechnology company developing a deep pipeline of differentiated antibody-based therapies for the treatment of severe autoimmune diseases and cancer, reported its financial results for the first quarter ended March 31, 2020 and provided a business update (Press release, argenx, MAY 14, 2020, View Source [SID1234558051]).

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"We are very excited to be approaching our first pivotal Phase 3 data readout with topline data from the ADAPT trial still on track for mid-year. This is an important milestone as it will precipitate our transition from a late-stage development company towards an integrated commercial organization. We have been investing in the expansion of our commercial infrastructure and are preparing for a 2021 launch of efgartigimod in gMG in the U.S., if approved," stated Tim Van Hauwermeiren, CEO of argenx.

"Over the last several months, the COVID-19 global pandemic has presented an unprecedented challenge, and as we face uncertainty in the coming months, we are grateful to have strong fundamentals across our business and the ability to fund our deep antibody pipeline. I am most grateful, however, for the continued support of our employees who have demonstrated exceptional focus and an unwavering dedication to our mission of developing therapies for the treatment of severe autoimmune diseases and cancer. This commitment is underscored by our decision to evaluate the potential of ARGX-117 to attenuate complement activation in severe respiratory illness associated with COVID-19 while obtaining key metrics of our drug candidate in this first-in-human trial."

FIRST QUARTER 2020 AND RECENT HIGHLIGHTS

argenx commitment to its people, patients and business
Despite the challenges of the COVID-19 pandemic, argenx remains focused on executing on its "argenx 2021" vision to become a fully integrated, global immunology company. In order to minimize impact on employees, patients and their communities, physicians and ongoing business priorities, argenx has implemented measures across its organization and in the operation of its globally run clinical trials.

A work-from-home mandate continues for all employees in the U.S., Belgium and Japan, excluding those providing essential services such as laboratory staff; additionally, all work-related global and domestic travel are suspended.
In order to enable patients in its clinical trials to receive study drug with continuity, argenx is implementing telehealth, remote monitoring activities and more flexible dosing schedules in its protocols where possible.
argenx conducts clinical trials globally, including in areas impacted by COVID-19 in North America, Europe and Japan. Enrollment is expected to be delayed in ongoing trials conducted by argenx, but the extent of the full impact is not quantifiable until the trajectory of the pandemic is better understood. The Company will continue to monitor the impact of COVID-19 on all ongoing clinical trials and will implement changes as necessary.

Efgartigimod trials remain open with additional registrational trials expected to launch this year

Efgartigimod is currently being evaluated in four targeted indications where IgG autoantibodies are directly pathogenic. A fifth indication is expected to be announced this year.

Generalized Myasthenia Gravis (gMG)
Topline results from the pivotal Phase 3 ADAPT clinical trial expected by mid-2020
All patients have completed primary 26-week trial; patients continue to be dosed in the ADAPT+ one-year, open-label extension study
If ADAPT data are positive, a Biologics License Application (BLA) submission is expected to be filed by end of 2020, with commercial launch planned in the U.S. in 2021
Plans remain on track to engage with the U.S. Food and Drug Administration (FDA) in 2020 on potential bridging strategy for subcutaneous (SC) ENHANZE-efgartigimod
·Well-established alliance with Lonza supports robust and flexible manufacturing capabilities and supply chain remains on track to be commercial-ready by end of 2020

Primary Immune Thrombocytopenia (ITP)
Phase 3 ADVANCE trial ongoing and expected to enroll approximately 150 primary ITP patients dosed with 10mg/kg IV efgartigimod
Confirmatory trial of IV efgartigimod expected to initiate in the first half of 2020
ADVANCE SC trial expected to initiate in the second half of 2020 evaluating 10mg/kg IV efgartigimod for induction of platelet response and 2mL fixed dose of SC efgartigimod for maintenance
Pemphigus Vulgaris (PV)
Phase 3 registrational trial expected to start in second half of 2020
Detailed proof-of-concept data from adaptive Phase 2 trial presented at Society for Investigative Dermatology (SID) Virtual Annual Meeting; presentation currently available on SID and argenx websites.
Presentation includes updated data from 31 evaluable patients treated with 10mg/kg or 25mg/kg of IV efgartigimod (data as of cutoff date of March 25, 2020)
90% (28/31) achieved rapid disease control; median time to disease control for monotherapy and combination therapy is 15 and 22 days
Complete clinical remission observed in 70% (7/10) of patients receiving optimized dosing regimen determined to be efgartigimod dosed at least every two weeks in combination with oral prednisone (0.25-0.5mg/kg)
73% (11/15) of patients receiving 25mg/kg efgartigimod achieved end of consolidation, including patients who preferred to taper steroid dose
11 patients currently still on study
Tolerability profile shown to be favorable and consistent with data from previous efgartigimod studies
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Phase 2 ADHERE trial ongoing with SC ENHANZE-efgartigimod

Janssen and LEO Pharma have paused enrollment of clinical trials of cusatuzumab and LP0145 (ARGX-112)
Enrollment is paused in two ongoing clinical trials initiated under the global cusatuzumab collaboration and licensing agreement with Cilag GmbH International, an affiliate of the Janssen Pharmaceutical Companies of Johnson & Johnson. Trials that have paused enrollment under the collaboration include:

Pivotal Phase 2 CULMINATE study evaluating cusatuzumab in combination with azacitidine for the treatment of newly diagnosed elderly acute myeloid leukemia (AML) patients who are unfit for intensive chemotherapy;
Phase 1b platform trial evaluating cusatuzumab in combination with venetoclax and azacitidine
Additionally, LEO Pharma has paused enrollment of the ongoing trial of LP0145 for the treatment of atopic dermatitis.

Timing to restart enrollment of all trials will depend on the trajectory of COVID-19 infection rates

ARGX-117 being evaluated as potential treatment for ARDS in COVID-19 patients

ARGX-117 is a potentially first-in-class complement-targeting antibody against C2 with potential therapeutic applications in severe autoimmune diseases.

argenx is sponsoring a Phase 1 trial in collaboration with Ghent University Hospital to evaluate ARGX-117 as a potential treatment for acute respiratory distress syndrome (ARDS), a frequent and serious complication associated with COVID-19.
Both classical and lectin pathways of complement system are active in ARDS; by targeting C2, an important component of both pathways, ARGX-117 could inhibit downstream inflammatory responses associated with ARDS
Collaboration leverages longstanding Immunology Innovation Program relationships with Erik Hack, M.D., Ph.D., Professor Emeritus, UMC Utrecht, and Bart Lambrecht, M.D., Ph.D., Director of Inflammation at VIB, UZGent and Belgian National Commissioner on the COVID-19 pandemic
Key pharmacokinetic (PK), pharmacodynamic (PD), dose finding and tolerability data to be gained during first-in-human trial
Phase 1 trial in healthy volunteers to start by end of 2020
Following analysis of Phase 1 data, argenx plans to launch Phase 2 proof-of-concept trial in multifocal motor neuropathy (MMN) within its neuromuscular franchise, and to develop in additional indications

argenx continues to expand its early-stage pipeline

Cash, cash equivalents and current financial assets totaled €1,305.5 million on March 31, 2020, compared to €1,335.8 million on December 31, 2019 and €961.6 million on March 31, 2019.

Operating income amounted to €23.4 million for the three months ended March 31, 2020, compared to €40.0 million for the three months ended March 31, 2019. The decrease in the first three months of 2020 was primarily explained by the revenue recognized in the first quarter of 2019, following a $30.0 million development milestone payment received under the AbbVie collaboration agreement.

Research and development expenses increased by €60.1 million during the three months ended March 31, 2020 to reach €94.9 million, compared to €34.8 million for the three months ended March 31, 2019. This planned increase was mainly the result of (i) increased external research and development expenses reflecting higher clinical trial costs and manufacturing expenses related to the development of the argenx product candidate portfolio and (ii) higher personnel expenses as a result of increased costs of the share-based payment compensation plans related to the grant of stock options to argenx research and development employees and increased costs associated with additional research and development employees.

Selling, general and administrative expenses totaled €25.0 million and €11.3 million for the three months ended March 31, 2020 and 2019, respectively. The increase of €13.7 million was principally linked to an increase of personnel expense, resulting from (i) higher costs of the share-based payment compensation plans related to the grant of stock options to its selling, general and administrative employees and (ii) increased costs associated with additional employees recruited to strengthen its selling, general and administrative activities, notably in preparation of the potential commercial launch of efgartigimod in the U.S., if approved.

For the three months ended March 31, 2020, financial income, which primarily relate to interests received on its cash and cash equivalents and current financial assets, amounted to €1.7 million compared to €3.5 million for the same period in 2019. Financial expense amounted to €5.0 million for the three months ended March 31, 2020 and corresponded mainly to a decrease in net asset value on its current financial assets following the impact of the COVID-19 outbreak on the financial markets.

Exchange gains totaled €20.8 million for the three months ended March 31, 2020 compared to €9.5 million for the three months ended March 31, 2019 and were mainly attributable to unrealized exchange rate gains on cash, cash equivalents and current financial assets position in U.S. dollars due to the favorable fluctuation of the EUR/USD exchange rate.

The total comprehensive loss for the three months ended March 31, 2020 was €80.0 million compared to a total comprehensive profit, which was principally due to the milestone payment received from AbbVie as indicated above, of €6.7 million for the three months ended March 31, 2019.

EXPECTED 2020 FINANCIAL CALENDAR:

July 30, 2020: HY 2020 financial results & business update
October 22, 2020: Q3 financial results & business update
CONFERENCE CALL DETAILS
The first quarter 2020 results will be discussed during a conference call and webcast presentation today at 3 pm CET/9 am ET. To participate in the conference call, please select your phone number below and use the confirmation code 6736269. The webcast may be accessed on the homepage of the argenx website at www.argenx.com or by clicking here.

On May 14, 2020 Orion Corporation reported new ARAMIS Phase III data to be presented at ASCO (Free ASCO Whitepaper) 2020: Nubeqa (darolutamide) significantly improved overall survival with a favorable safety profile in men with non-metastatic prostate cancer (Press release, Orion , MAY 14, 2020, View Source [SID1234558050]).

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Darolutamide significantly reduced risk of death by 31 percent (HR=0.69, 95% CI 0.53-0.88; p=0.003) in men with non-metastatic castration-resistant prostate cancer (nmCRPC)
Darolutamide significantly delayed the time to pain progression, time to first initiation of cytotoxic chemotherapy, and time to first symptomatic skeletal event (SSE)
Darolutamide continues to demonstrate a favorable safety profile, with no new safety signals observed, even with a longer treatment duration, allowing men with nmCRPC to maintain their active lifestyle
ARAMIS data will be presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2020 Virtual Scientific Program on Friday, May 29, and will be available on the ASCO (Free ASCO Whitepaper) website (View Source)
Abstract 5514
Darolutamide (Nubeqa) is shown to significantly improve overall survival (OS) and significantly delay the onset of cancer-associated symptoms, while minimizing the toxicity associated with treating men with non-metastatic castration-resistant prostate cancer (nmCRPC). These data from the pre-specified final OS analysis of the Phase III ARAMIS trial will be presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2020 Virtual Scientific Program, which takes place from May 29-31, 2020.

Previously published results from the ARAMIS trial demonstrated a highly significant improvement in the primary efficacy endpoint of metastasis-free survival (MFS), with a median of 40.4 months for darolutamide plus androgen deprivation therapy (ADT) compared to 18.4 months for placebo plus ADT (p<0.001); however, OS data were not yet mature at the time of the MFS analysis.

"Men with nmCRPC typically do not have cancer symptoms. In selecting a treatment for these patients, my goal as a clinician is to improve their overall survival while limiting side effects and drug interactions," said Karim Fizazi, M.D., Ph.D., Professor of Medicine at the Institut Gustave Roussy, Villejuif, France. "These data add to the growing evidence for darolutamide as an effective treatment option with a favorable safety profile that extends patients’ lives and delays cancer symptoms and morbidities, without disrupting their daily activities."

Final OS Analysis Presented at ASCO (Free ASCO Whitepaper) Virtual Scientific Program
Men receiving darolutamide plus ADT demonstrated a significant improvement in OS compared to placebo plus ADT, with a 31 percent reduction in risk of death (HR=0.69, 95% CI 0.53-0.88; p=0.003).

Darolutamide has a distinct chemical structure and inhibits the growth of prostate cancer cells while limiting the burden of side effects on patients’ everyday lives. With extended follow-up, darolutamide’s safety profile remains favorable, allowing men with nmCRPC to continue their daily lives without disruption. Consistent with the previously reported primary analysis results, darolutamide plus ADT showed a favorable tolerability confirmed by a longer-term safety analysis compared to ADT alone, without clinically relevant increases in rates of hypertension, falls or central nervous system (CNS) effects. In the follow-up analysis of secondary endpoints, all secondary endpoints were statistically significant. Darolutamide plus ADT significantly delayed time to pain progression, time to first initiation of cytotoxic chemotherapy and time to first symptomatic skeletal event (SSE) versus placebo plus ADT.

Under the brand name Nubeqa, darolutamide is developed jointly by Orion and Bayer, and indicated for the treatment of men with nmCRPC, who are at high risk of developing metastatic disease. The approvals of Nubeqa in the European Union (EU), U.S., Australia, Brazil, Canada, and Japan are based on the pivotal Phase III ARAMIS trial data evaluating the efficacy and safety of darolutamide plus ADT compared to placebo plus ADT.

About the ARAMIS trial
The ARAMIS trial is a randomized, Phase III, multi-center, double-blind, placebo-controlled trial evaluating the safety and efficacy of oral darolutamide in patients with nmCRPC who are currently being treated with ADT and are at high risk for developing metastatic disease. In the clinical study, 1,509 patients were randomized in a 2:1 ratio to receive 600 mg of darolutamide orally twice daily or placebo along with ADT. Patients with a history of seizure were allowed in the study.

About Nubeqa (darolutamide)
Darolutamide was approved in March 2020 in the European Union (EU) under the brand name Nubeqa for the treatment of men with non-metastatic castration-resistant prostate cancer (nmCRPC), who are at high risk of developing metastatic disease. Nubeqa has also received regulatory approval in the U.S., Australia, Brazil, Canada as well as Japan, and filings in other regions are underway or planned by Bayer.

Nubeqa 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 compound is also being investigated in a Phase III study in metastatic hormone-sensitive prostate cancer (ARASENS). Information about these trials can be found at www.clinicaltrials.gov.

About castration-resistant prostate cancer (CRPC)
Prostate cancer is the second most commonly diagnosed malignancy in men worldwide. In 2018, an estimated 1.2 million men were diagnosed with prostate cancer, and about 358,000 died from the disease worldwide. Prostate cancer is the fifth leading cause of death from cancer in men. Prostate cancer results from the abnormal proliferation of cells within the prostate gland, which is part of a man’s reproductive system. It mainly affects men over the age of 50, and the risk increases with age.

Treatment options range from surgery to radiation treatment to therapy using hormone-receptor antagonists, i.e., substances that stop the formation of testosterone or prevent its effect at the target location. However, in nearly all cases, the cancer eventually becomes resistant to conventional hormone therapy.

Prostate cancer that is confined to the prostate region which is treated with ADT but keeps progressing without showing metastases, even when the amount of testosterone is reduced to very low levels in the body, is known as non-metastatic castration-resistant prostate cancer (nmCRPC). In men with progressive nmCRPC, a rapid prostate specific antigen (PSA) doubling time has been consistently associated with reduced time to first metastasis and death. About one-third of men with nmCRPC go on to develop metastases within two years.

On May 14, 2020 Xenetic Biosciences, Inc. (NASDAQ:XBIO) ("Xenetic" or the "Company"), a biopharmaceutical company focused on advancing XCART, a personalized CAR T platform technology engineered to target patient- and tumor-specific neoantigens, reported its financial results for the quarter ended March 31, 2020 and provided a corporate update (Press release, Xenetic Biosciences, MAY 14, 2020, View Source [SID1234558049]).

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"Over the course of the first quarter, we continued to make encouraging progress towards securing academic collaborations, the next major milestone for us in advancing the XCART platform," commented Jeffrey Eisenberg, Chief Executive Officer of Xenetic. "Additionally, despite the fact that the COVID-19 health pandemic carries on, we have been fortunate to not have experienced any significant operational impacts and have maintained a cash runway expected to fund our projected operational and development efforts through mid-2021. We are excited for the year ahead and to further unlock the value that the XCART technology platform holds."

XCART Platform Technology Overview: Significantly differentiated, proprietary approach to personalized CAR T therapy for the treatment of multiple tumor types of B-cell Non-Hodgkin lymphomas, an area of significant unmet need, with the potential to address an initial global market opportunity of over $5 billion annually.1 Xenetic believes XCART has the potential to transform CAR T therapy.

The Company announced in February 2020 the appointments of Greg MacMichael, Ph.D. and Maksim Mamonkin, Ph.D. to its Scientific Advisory Board, each of whom brings with them extensive knowledge in cell therapy engineering and design, cell therapy manufacturing, and CMC expertise and capabilities. Both Dr. MacMichael and Dr. Mamonkin are actively engaged with the Company to advance the development of the XCART technology platform.

Xenetic is actively engaged in ongoing discussions to advance the development of XCART through one or more collaborations with academic or development partners.

Expected 2020 Milestones

Enter into academic site collaborations
INTERACT meeting with the United States Food and Drug Administration ("FDA")
Advance IND-enabling studies
Explore opportunities for Orphan Drug designation
PolyXen Platform Technology: Patent-protected platform technology designed for protein or peptide therapeutics, enabling next-generation biological drugs by prolonging a drug’s circulating half-life and potentially improving other pharmacological properties.

Program Highlights:

Exclusive License Agreement with Takeda Pharmaceuticals Co. Ltd. ("Takeda") in the field of coagulation disorders. Takeda currently has one active development program underway utilizing the PolyXen platform technology.
Royalty payments expected to continue to grow as the relevant product launch continues to be rolled out by the sublicensee.
Summary of Financial Results for First Quarter 2020

Net loss for the three months ended March 31, 2020 was approximately $1.2 million compared to a net loss of approximately $1.3 million for the same period in 2019. As of March 31, 2020, working capital was $9.2 million compared to $9.7 million as of December 31, 2019. The decrease in working capital was primarily due to the Company’s net loss for the three months ended March 31, 2020. The Company ended the quarter with approximately $9.4 million of cash.