On June 26, 2018 BerGenBio ASA (OSE:BGBIO) reported that on a top-line, preliminary basis, the first efficacy endpoint has been met in its Phase II clinical trial (BGBC008) evaluating bemcentinib, a first-in-class oral selective AXL inhibitor, in combination with the Merck & Co., Inc., Kenilworth, N.J., USA anti-PD-1 therapy KEYTRUDA (pembrolizumab) as a potential new treatment regimen for advanced non-small cell lung cancer (NSCLC)(Press release, BerGenBio, JUN 26, 2018, View Source [SID1234527463]) . The primary efficacy endpoint requires at least four patients (out of the first 22 treated patients) to achieve clinical responses when treated with the novel drug combination, defined as either complete or partial response, as measured by Response Evaluation Criteria in Solid Tumors (RECIST).

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Richard Godfrey, Chief Executive Officer of BerGenBio, commented: "Immunotherapy has become a major component of the treatment of many cancers – patients who respond to immune checkpoint inhibitors like KEYTRUDA enjoy long-term disease control with excellent quality of life. Unfortunately, only a minority of lung cancer patients receiving KEYTRUDA monotherapy in second-line respond to treatment. The BGBC008 combination trial of bemcentinib with KEYTRUDA evaluates whether the addition of our selective AXL inhibitor will improve the outcome of immunotherapy.

"Clearing the first efficacy threshold in this ongoing Phase II trial is very encouraging and we intend to begin enrolment for Stage 2 of this study in which 24 further patients will be enrolled under the same protocol. Thus far, we are delighted to see activity in a number of patients receiving this novel treatment regimen. A particularly encouraging finding is that we see responses in patients who are negative for the PD-L1 biomarker, for whom KEYTRUDA monotherapy is not indicated. The second stage of the trial is intended to confirm activity and biomarker correlation in a larger group of patients – comprehensive analysis of the Phase II data will continue and will be presented at a future scientific conference.

"Successfully completing this important milestone further supports our belief in the potential of bemcentinib to become a cornerstone of cancer therapy. We look forward to sharing more details from our Phase II clinical programme during major clinical conferences in the coming months."

About the BGBC008 trial combining bemcentinib with KEYTRUDA (pembrolizumab) conducted in collaboration with Merck & Co., Inc.
Design
The BGBC008 trial is a Phase II multi-centre open-label study of bemcentinib in combination with KEYTRUDA (pembrolizumab) in previously treated, immunotherapy naïve, patients with advanced adenocarcinoma of the lung, the most common form of non-small cell lung cancer (NSCLC). The objective of the trial is to determine the anti-tumour activity of this novel drug combination and responses will be correlated with biomarker status (including AXL kinase and PD-L1 expression).

A pre-defined efficacy endpoint was set at four or more responses observed in the first 22 patients based on previously reported response rates to KEYTRUDA monotherapy in the second line setting in NSCLC.

Status June 2018
To date, 4 responses (partial responses as per RECIST v1.1) have been observed in the first 22 patients. A number of patients remain ongoing and are awaiting the confirmation of their best response.

Patients generally tolerated the novel drug combination well – no new safety events were reported from the combination of bemcentinib with KEYTRUDA at full dose.

A preliminary interim analysis of the trial (from 15 patients evaluable for response) was presented at ASCO (Free ASCO Whitepaper) 2018, where tumour shrinkage was observed in about half of the patients analysed to date. Results looked particularly promising in patients who did not express the PD-L1 biomarker, i.e. representing 1/3 of NSCLC patients, a group for whom KEYTRUDA monotherapy as a second line is not indicated.

For more information please access trial NCT03184571 at www.clinicaltrials.gov.

On June 25, 2018 Nordic Nanovector ASA (OSE: NANO) reported that Eduardo Bravo has been appointed as its Chief Executive Officer (Press release, Nordic Nanovector, JUN 25, 2018, View Source [SID1234553497]).

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Mr Bravo brings more than 25 years’ experience in the biopharmaceutical industry and a strong track record in leading and growing international biotech and pharmaceutical organisations. Since 2011, as CEO of TiGenix, a dual-listed (Euronext Brussels and NASDAQ) biopharmaceutical company developing novel stem cell therapies, he successfully developed the company through several financing rounds, led its IPO on NASDAQ, and secured European marketing approval of its lead asset. In January 2018, Takeda Pharmaceutical Co. Ltd announced it was acquiring TiGenix for €520 million.

Prior to joining TiGenix’ predecessor, Cellerix, in 2005, Mr Bravo held several senior management positions at Sanofi-Aventis and SmithKline Beecham. He is currently Chairman of Vivet Therapeutics. He holds a degree in Business Administration and an MBA (INSEAD).

Mr Bravo will take up the CEO position in Nordic Nanovector on 2 July and will be based in London, UK.

Ludvik Sandnes, Chairman of Nordic Nanovector’s Board of Directors, said: "I am very pleased that we have attracted someone of Eduardo’s calibre to become the CEO of Nordic Nanovector. He has extensive experience in corporate and product development, which is central to Nordic Nanovector’s future success, and will provide excellent leadership under which the Company can continue to realise its significant potential with Betalutin. I would also like to thank Tone Kvåle for supporting the company as Interim CEO over the past few months."

Eduardo Bravo added: "I am looking forward to this exciting new role as the CEO of Nordic Nanovector. It is clear that the Company has an exciting future based on the significant potential of Betalutin in follicular lymphoma and NHL more broadly. I am joining an excellent board and management team and am confident that with my complementary experience and networks, we can succeed with Betalutin to provide physicians with another treatment option to improve the lives of patients who are in dire need of effective therapies to control their disease and build significant value in the company."

On joining Nordic Nanovector, Mr Bravo will be granted 250,000 PSUs (performance share units) and the company’s Board of Directors has undertaken to grant him a further 50,000 PSUs as part of the company’s annual grant of PSUs in the first quarter of 2019. For further information about the PSUs and the related warrants, see pages 66-68 in the company’s annual report for 2017. In addition, on joining Nordic Nanovector, Mr Bravo will receive a sign-on bonus of EUR 50,000 and has undertaken to invest, at minimum, the net amount after tax in Nordic Nanovector shares and will do so following this announcement.

On June 25, 2018 Stemline Therapeutics, Inc. (Nasdaq:STML), a clinical-stage biopharmaceutical company developing novel oncology therapeutics, reported that it has completed submission of a rolling Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for ELZONRISTM (tagraxofusp; SL-401), a potential treatment for patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN), a CD123+ malignancy of unmet medical need for which the agent was awarded Breakthrough Therapy Designation (BTD) (Press release, Stemline Therapeutics, JUN 25, 2018, View Source [SID1234532234]).

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Survival probability in first-line BPDCN patients who received ELZONRIS (12mcg/kg/day) in Stages 1, 2 and 3

Ivan Bergstein, M.D., Stemline’s CEO, commented, "The completion of our rolling BLA submission is a major milestone for Stemline and the overall BPDCN patient community. We want to recognize the hard work of our dedicated investigators as well as the entire Stemline team in completing this submission. We also want to especially thank all of the patients and their families who participated in the clinical development program. We are committed to bringing this promising agent to BPDCN patients as rapidly as possible."

BPDCN Efficacy – Stages 1, 2, and 3, ELZONRIS (12mcg/kg/day) (n=42)
The trial of investigational agent, ELZONRIS, in patients with BPDCN was comprised of 3 stages, with Stage 3 serving as the pivotal cohort for confirmation of efficacy. To ensure ongoing access to ELZONRIS, patients with BPDCN are being enrolled in an additional cohort, Stage 4. Stage 3 met its primary endpoint with a CR+CRc (complete response + clinical complete response) rate of 54% (95% CI: 25.1, 80.8). A summary of efficacy results is shown below.

Summary table: Efficacy of ELZONRIS (12mcg/kg/day) in patients with BPDCN (Stages 1, 2 and 3 [n=42])

Line of Therapy First-line Relapsed / Refractory
n 29 13
ORR, n (%) 26 (90%) 9 (69%)
CR+CRc+CRi, n (%) 21 (72%) 5 (38%)
CR 12 1
CRc 7 3
CRi 2 1
PR, n (%) 5 (17%) 4 (31%)
Bridged to SCT, n (%) 13 (45%) 1 (8%)
Abbreviations: ORR=overall response rate; CR=complete response; CRc=clinical CR (CR with minimal residual skin abnormality); CRi=CR with incomplete hematologic recovery; PR=partial response; SCT=stem cell transplant.

Overall Safety
148 patients received ELZONRIS (12mcg/kg/day) across all Stemline-sponsored trials, including in BPDCN, myeloproliferative neoplasms, and acute myeloid leukemia. A summary of safety results is shown below.

Summary table: Safety and tolerability of ELZONRIS (12mcg/kg/day) in all Stemline-sponsored clinical trials (n=148)

Most Common Adverse Events (AEs) (>15% treatment-related AEs, TRAEs)
Preferred Term All Grades, n (%) TRAEs, n (%)
TRAEs All AEs Gr 1-2 Gr 3 Gr 4 Gr 5
ALT increased 65 (43.9%) 80 (54.1%) 31 (20.9%) 34 (23.0%) 0 (0.0%) 0 (0.0%)
AST increased 65 (43.9%) 74 (50.0%) 30 (20.3%) 31 (20.9%) 4 (2.7%) 0 (0.0%)
Hypoalbuminaemia 65 (43.9%) 73 (49.3%) 64 (43.2%) 1 (0.7%) 0 (0.0%) 0 (0.0%)
Thrombocytopenia 39 (26.4%) 48 (32.4%) 7 (4.7%) 8 (5.4%) 24 (16.2%) 0 (0.0%)
Nausea 38 (25.7%) 70 (47.3%) 37 (25.0%) 1 (0.7%) 0 (0.0%) 0 (0.0%)
Pyrexia 33 (22.3%) 60 (40.5%) 33 (22.3%) 0 (0.0%) 0 (0.0%) 0 (0.0%)
Fatigue 30 (20.3%) 67 (45.3%) 26 (17.6%) 4 (2.7%) 0 (0.0%) 0 (0.0%)
Weight increased 28 (18.9%) 42 (28.4%) 28 (18.9%) 0 (0.0%) 0 (0.0%) 0 (0.0%)
Chills 26 (17.6%) 40 (27.0%) 25 (16.9%) 1 (0.7%) 0 (0.0%) 0 (0.0%)
Capillary leak syndrome (CLS)a 25 (16.9%) 25 (16.9%) 16 (10.8%) 5 (3.4%) 3 (2.0%) 1 (0.7%)
Hypotension 23 (15.5%) 36 (24.3%) 17 (11.5%) 5 (3.4%) 1 (0.7%) 0 (0.0%)
Oedema peripheral 22 (14.9%) 57 (38.5%) 21 (14.2%) 1 (0.7%) 0 (0.0%) 0 (0.0%)
a0.7% (1/148) for all trials (12mcg/kg/day) and 1.6% (3/182) for all trials (all doses) were grade 5. A myocardial infarction, grade 5, was also reported in a patient who experienced a grade 4 CLS.

Overall Survival (OS)
In first-line BPDCN patients who received ELZONRIS (12mcg/kg/day) in Stages 1, 2 and 3, the median OS has not been reached. Median follow up was 13.8 months (range: 0.2-37.4+ months).

About ELZONRISTM (tagraxofusp; SL-401)
ELZONRISTM (tagraxofusp; SL-401) is a novel targeted investigational therapy directed to CD123, a cell surface receptor expressed on a range of malignancies. ELZONRIS has successfully completed a pivotal trial in patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN), an indication for which it was granted Breakthrough Therapy Designation (BTD). A rolling Biologics License Application (BLA) submission has been completed. ELZONRIS is also being evaluated in clinical trials in additional indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF), acute myeloid leukemia (AML), and myeloma.

On June 25, 2018 Andarix Pharmaceuticals, a clinical stage company aimed at developing a targeted peptide therapies for hard to treat cancers, reported that it will present a poster at the Metals in Medicine Gordon Research Conference (Press release, Andarix Pharmaceuticals, JUN 25 2018, View Source [SID1234527792]) .The conference will take place June 25-29 in Andover, NH.

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The theme of the conference is: The Indispensable Role of Metals in Medical Diagnostics, Therapeutics and Beyond. Andarix will be presenting clinical findings pertaining to Tozaride, a Rhenium metal-based drug developed by the company. The conference is an opportunity for Andarix to present its innovative technology to world
leading scientists and international metal-science experts.

About Tozaride
Tozaride is a novel, best-in-class therapeutic for lung and other cancers, which is based on a radio-labeled somatostatin peptide. Early clinical studies demonstrated that Tozaride is well tolerated and that treatment can promote disease stabilization and improve overall survival in heavily pre-treated advanced lung cancer patients. Tozaride targeted radiotherapy represents a new treatment paradigm which is expected to yield
significant clinical benefits for both small cell lung cancer (SCLC), and non-small cell lung cancer (NSCLC) patients. This therapeutic stands to provide an additional treatment option for patients who are not eligible for, or who have not responded to current therapies.

On June 25, 2018 Verseon (AIM:VSN), a technology-based pharmaceutical company employing a computer-driven platform to develop a diverse drug pipeline, reported its Final Results for the year ended December 31, 2017 (Press release, Verseon, JUN 25, 2018, View Source [SID1234527517]). The report and accounts are available for download from the Company’s website (www.verseon.com).

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Adityo Prakash, CEO of Verseon Corporation, commented: "We have made significant progress across our pipeline over the past year. Most notably, our first PROAC (precision oral anticoagulant), VE-1902, completed regulatory toxicology and safety pharmacology testing and is now about to enter clinical trials. We also announced a new rare-disease program in which we are developing oral drugs for hereditary angioedema, a potentially life-threatening genetic disorder. In addition, we have demonstrated efficacy in multiple in vivo models for our orally dosed diabetic macular edema candidates and have shown that our novel anticancer agents hold promise for the treatment of multidrug resistant cancers."

"We have worked diligently to build a strong foundation for our platform that can roll out a steady stream of drug candidates. We look forward to sending VE-1902 into clinical trials, the first of many future clinical candidates across our pipeline."

Highlights
Finance

Results for the year ended December 31, 2017:

Total assets on the balance sheet stood at $54.2 million, compared to $69.6 million at the end of 2016.
Cash, cash equivalents, and short-term investments stood at $11.6 million, compared to $46.9 million at the end of 2016.
Property, equipment, buildings and land totaled $40.7 million, compared to $22.3 million at the end of 2016.
Research and development expenses were $15.1 million, compared to $11.5 million in 2016, primarily attributable to an acceleration of our drug programs and preparation for clinical trials.
General and administrative expenses were $6.3 million, compared to $5.8 million in 2016.
Non-cash expenses include stock-based compensation of $0.9 million, compared to $0.8 million in 2016, and also a currency exchange gain of $0.6 million, compared to a loss of $2.6 million in 2016.
Net loss was $20.4 million or $0.13 per basic share, compared to a net loss of $19.5 million or $0.13 per basic share in 2016.
Post-period events:

Closed $22.7M mortgage for our research and development facility, realizing a portion of the value created through the buildout.
Currently evaluating a range of non-dilutive funding options linked to future revenues. This will enable us to accelerate the development of our programs through clinical studies to market, capturing their significant long-term value.
Anticoagulation

Developing novel class of precision oral anticoagulants (PROACs) for long-term anticoagulant-antiplatelet combination therapy.
First PROAC, VE-1902, successfully completed regulatory toxicology studies and is about to enter clinical trials.
Second PROAC, VE-2851, is in preliminary toxicology studies and is expected to enter clinical trials in 2019.
Diabetic macular edema

Developing oral DME drugs with the potential to complement or replace current eye injections.
Candidates show efficacy in multiple in vivo models when administered orally.
Hereditary angioedema

Developing oral drugs for this rare, potentially life-threatening disease.
Candidates show efficacy in a well-established preclinical model with oral dosing.
Oncology

Developing new anticancer agents for the treatment of multidrug resistant cancers.
Candidates show potency against a variety of cancer cell lines and are largely unaffected by common modes of drug resistance.
Facilities development

Occupying purpose-built research and development facility.
Closed PACE funding for energy-related improvements.