On May 8, 2019 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported financial results for the first quarter ended March 31, 2019 and provided a corporate update (Press release, BioCryst Pharmaceuticals, MAY 8, 2019, View Source [SID1234535902]).

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"In a year with many milestones across our multiple advancing programs of oral medicines for rare diseases, BioCryst has achieved significant progress in the first quarter and we look forward to reporting data from our APeX-2 trial in the second quarter and filing a new drug application by the end of the year," said Jon Stonehouse, president and chief executive officer of BioCryst.

"We believe that oral BCX7353 could be transformative for many HAE patients and provide them with the opportunity for a normal life without the burden and discomfort of frequent injections and infusions," Stonehouse added.

First Quarter 2019 Corporate Developments

The company dosed the first patients in its APeX-J trial in Japan, designed to support potential Japanese approval of BCX7353 for the prevention of HAE attacks.

On March 4, 2019, the company announced that it is advancing BCX9930, an oral Factor D inhibitor, into Phase 1 clinical development in the second quarter of 2019 for the treatment of complement-mediated diseases.

On February 23, 2019, the company announced data from the completed ZENITH-1 trial (including the 250 mg and 500 mg dose cohorts) of BCX7353 for the acute treatment of HAE attacks at the annual meeting of the American Academy of Allergy, Asthma & Immunology. The company plans to commence a Phase 3 trial, ZENITH-2, in the summer of 2019.

On February 6, 2019, the company announced it had entered into a $100 million secured credit facility with MidCap Financial Trust pursuant to the terms and conditions of an amended and restated credit and security agreement.

On January 4, 2019, the company announced it had appointed Steve Aselage to its board of directors.

On January 2, 2019, the company announced the dosing of the first subject in a randomized, placebo-controlled Phase 1 clinical trial to evaluate intravenous galidesivir, its investigational broad-spectrum antiviral drug, in healthy volunteers.
Upcoming Key Milestones

HAE Program – BCX7353

Report 24-week safety and efficacy results from the Phase 3 APeX-2 clinical trial (Q2 2019)

Begin ZENITH-2, a Phase 3 clinical trial of oral BCX7353 (750 mg) for the acute treatment of HAE (Summer 2019)

File a new drug application for oral BCX7353 for the prevention of HAE attacks with the U.S. Food and Drug Administration (FDA) (Q4 2019)

File a marketing authorization application for oral BCX7353 for the prevention of HAE attacks with the European Medicines Agency (EMA) (Q1 2020)
Complement Factor D Inhibitor Program – BCX9930

Begin a Phase 1 trial of BCX9930, an oral Factor D inhibitor for treatment of complement-mediated diseases, in healthy subjects (Q2 2019)

Report Phase 1 results (Q4 2019)
ALK-2 Inhibitor Program – BCX9250

Begin a Phase 1 clinical trial of BCX9250, an oral ALK-2 kinase inhibitor for treatment of FOP, in healthy subjects (2H 2019)
First Quarter 2019 Financial Results

For the three months ended March 31, 2019, total revenues were $5.9 million, compared to $4.0 million in the first quarter of 2018. The increase was primarily due to the recognition of $1.7 million of peramivir product sales to Green Cross, the company’s commercial partner in Korea, and an increase in revenue from galidesivir development under U.S. government contracts, and partially offset by lower royalty revenue.

Research and development (R&D) expenses for the first quarter of 2019 increased to $27.5 million from $18.4 million in the first quarter of 2018, primarily due to increased spending on the HAE, preclinical and galidesivir programs.

General and administrative (G&A) expenses for the first quarter of 2019 decreased to $6.2 million, compared to $7.6 million in the first quarter of 2018. The decrease was primarily due to approximately $4.7 million of merger-related costs that were incurred in the first quarter of 2018 but did not recur in 2019, offset by an overall increase in G&A expenses as the company prepares for the commercial launch of BCX7353.

Interest expense was $2.7 million in the first quarter of 2019, compared to $2.2 million in the first quarter of 2018 and was primarily associated with enhancements to the company’s secured credit facility in July 2018 and February 2019.

Net loss for the first quarter of 2019 was $31.1 million, or $0.28 per share, compared to a net loss of $25.8 million, or $0.26 per share, for the first quarter of 2018.

Cash, cash equivalents and investments totaled $121.6 million at March 31, 2019, and reflect a decrease from $128.4 million at December 31, 2018. Cash and investments reflect the proceeds from an enhancement to our secured credit facility in February 2019 and were partially offset by normal operating expenses. Operating cash use for the first quarter of 2019 was $27.1 million.

In February 2019, the company entered into a $100 million secured credit facility with MidCap Financial Trust which further enhanced the company’s cash position with $20 million of immediate additional non-dilutive capital and also provided additional financial flexibility through the ability to draw another $50 million of milestone-based non-dilutive capital.

Financial Outlook for 2019

BioCryst continues to expect net operating cash use to be in the range of $105 to $130 million, and its 2019 operating expenses to be in the range of $120 to $145 million. The company’s operating expense range excludes equity-based compensation expense due to the difficulty in reliably projecting this expense, as it is impacted by the volatility and price of the company’s stock, as well as by the vesting of the company’s outstanding performance-based stock options.

Conference Call and Webcast

BioCryst management will host a conference call and webcast at 8:30 a.m. ET today to discuss the financial results and provide a corporate update. The live call may be accessed by dialing 877-303-8027 for domestic callers and 760-536-5165 for international callers and using conference ID # 1777029. A live webcast of the call and any slides will be available online at the investors section of the company website at www.biocryst.com. A telephone replay of the call will be available by dialing 855-859-2056 for domestic callers or 404-537-3406 for international callers and entering the conference ID # 1777029.

On May 8, 2019 BerGenBio ASA (OSE: BGBIO) reported that it has completed enrolment into the second stage of its Phase II trial (BGBC008, Cohort A NCT03184571) evaluating the Company’s selective AXL inhibitor bemcentinib in combination with MSD’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab), in patients with previously treated advanced adenocarcinoma of the lung (non-small cell lung cancer, NSCLC) whose disease is progressing on or after prior systemic chemotherapy (Press release, BerGenBio, MAY 8, 2019, View Source [SID1234535901]).

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The trial has previously met the efficacy endpoint in the first stage and reported preliminary efficacy including a 40% ORR and 5.9 months Progression-free survival (PFS) in AXL positive patients (n=10). The second stage of the trial enrolling a further 24 patients for a total of 48 is designed to confirm the safety, clinical efficacy and biomarker correlation of the combination.

Comprehensive exploratory studies will continue to evaluate biomarkers in tumour and blood indicative of AXL expression and immune modulation. Results from the trial are expected during 2019.

The trial, which began in October 2017, is being conducted under a clinical collaboration with Merck & Co., Inc., Kenilworth, N.J., USA, through a subsidiary, and is taking place at sites in the US, UK, Norway and Spain.

Richard Godfrey, Chief Executive Officer of BerGenBio, commented: "A majority of NSCLC patients now receive anti-PD(L)-1 therapies like KEYTRUDA as a first- or second-line treatment for their advanced disease. Enhancing responses to these novel agents, particularly in patients with no or limited expression of PD-L1, the established biomarker for these drugs, is a significant unmet need. I am encouraged by the efficacy signals seen during stage 1 of the trial, particularly in AXL positive, and often PD-L1 negative or low, patients and look forward providing data from an extended dataset during the coming months together with further details on our late stage strategy in this indication."

– End –

About NSCLC
It is estimated that more than 230,000 new cases of lung cancer have been diagnosed in the US in 2018 and it is the leading cause of cancer deaths. 65% of NSCLCs are of adenocarcinoma pathology. Although various treatments exist for NSCLC, they are often curtailed by acquired resistance to therapy and immune evasion. Novel treatments overcoming these mechanisms in NSCLC are urgently required.

About the BGBC008 trial
The BGBC008 trial is a Phase II open-label study of bemcentinib in combination with KEYTRUDA (pembrolizumab) in previously treated patients with advanced adenocarcinoma of the lung run at centres in the US, UK, Spain and Norway. 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).

Patients eligible for participation in Cohort A must have progressed on or after prior therapy excluding immunotherapy whereas patients in Cohort B will be actively progressing on a therapy regimen containing an anti-PD(L)-1 therapy.

Both cohorts follow a two-stage design, Cohort A has previously successfully progressed into the second stage after meeting its first efficacy endpoint. Cohort B will evaluate advancement into stage 2 after 13 patients have been assessed for response.

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

About AXL
AXL kinase is a cell membrane receptor and an essential mediator of the biological mechanisms that drive aggressive and life-threatening diseases. In cancer, AXL drives tumour survival, treatment resistance and spread, as well as suppressing the body’s immune response to tumours. AXL expression has been established as a negative prognostic factor in many cancers. AXL inhibitors, therefore, have potential value at the centre of cancer combination therapy, addressing significant unmet medical needs and multiple high-value market opportunities.

On May 8, 2019 BerGenBio ASA (OSE:BGBIO), a clinical-stage biopharmaceutical company developing novel, selective AXL kinase inhibitors for multiple cancer indications, reported its results for the first quarter 2019 (Press release, BerGenBio, MAY 8, 2019, View Source [SID1234535900]).

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A presentation and live webcast by the Company’s management will take place today at 10.00 am CET in Oslo, please see below for details.

Richard Godfrey, Chief Executive Officer of BerGenBio, commented: "We are pleased to see the positive strategic and clinical momentum generated in the last year continue into 2019. We have continued to see promising initial data emerging from the clinical development programme with bemcentinib, particularly in AML and NSCLC. We believe that this data provides us with sufficient evidence to begin the initiation of late stage clinical development in these indications during 2019. We are looking forward to providing an update on these data at the forthcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) meeting in June."

Operational Highlights – First Quarter 2019
Lead candidate bemcentinib meets efficacy endpoint in combination with cytarabine in AML patients unfit for intensive therapy

Phase II trial evaluating bemcentinib in combination with low-intensity chemo-therapy in AML patients unfit for intensive therapy fully recruited
Bemcentinib in combination with Low-dose Cytarabine (LDAC) meets efficacy endpoint: Three out of 10 evaluable LDAC patients (30%) had CR/CRi
Combos warrant further expansion, meanwhile, company prepares for late stage bemcentinib monotherapy trial in later line elderly AML patients unfit for intensive chemotherapy
Phase II trial with bemcentinib and KEYTRUDA in NSCLC made important progress

Additional cohort (Cohort B) added under collaboration agreement with Merck & Co extending eligibility to patients who have disease progression on prior immune checkpoint inhibitors
Start of Phase II Investigator-Initiated Trial Evaluating Selective AXL Inhibitor bemcentinib in high-risk MDS

Up to 43 patients to be enrolled at leading MDS centres across Europe
Start of Phase I trial evaluating first-in-class anti-AXL antibody BGB149

BGB149, a wholly owned asset, is the first therapeutic anti-AXL monoclonal antibody to enter clinical development
Phase I study will investigate safety and pharmacokinetics in healthy volunteers
Start of phase I trial evaluating ADCT-601, a novel anti-AXL antibody drug conjugate (ADC), in patients with advanced solid tumours

ADCT-601 uses a proprietary AXL antibody developed by BerGenBio and licensed to ADC Therapeutics for ADC development
Phase I dose escalation and expansion trial will evaluate ADCT-601 in 75 cancer patients
Trial managed and sponsored by ADC Therapeutics
First clinical milestone met and milestone payment received
Preclinical data presented at AACR (Free AACR Whitepaper) reinforces bemcentinib’s potential to reverse tumour immunosuppression and therapy resistance

Extensive data in pre-clinical models of non-small cell lung cancer (NSCLC) and pancreatic cancer demonstrating AXL’s role in reversing tumour-mediated immunosuppression and therapy resistance presented at AACR (Free AACR Whitepaper)
bemcentinib shown to reverse AXL’s effects, thus acting synergistically with immune cells and anti-cancer therapies
Key appointments to executive team and Board to prepare organisation for next phase of development

Key appointments to executive team and Board to prepare organisation for next phase of development; Board of Directors strengthened. Grunde Eriksen, Debra Barker and Pamela Trail elected as new board members.
Dr Dominic Smethurst appointed as permanent Chief Medical Officer (CMO)
Favourable outcome of arbitration brought by BerGenBio against Rigel Pharmaceuticals clarifying the extent of Rigel’s entitlement to receive compensation in the event of a sale of BerGenBio, its assets, or a license to Bemcentinib
R&D grant of up to NOK 11 m awarded by Research Council of Norway
Financial highlights

Total operating expenses for the first quarter were NOK 54.5 million (Q1 2018 NOK 54.8 million)
Research and development expenses accounted for 70% of total operating expenses in Q1
Comprehensive loss for the first quarter amounted to NOK 44.3 million (Q1 2018 a loss of NOK 53.8 million)
Cash and cash equivalents amounted to NOK 306.7 million at the end of March 2019
Presentation and Webcast Details
A presentation by BerGenBio’s senior management team will take place today at 10:00 am CET at:

Felix Konferansesenter,
Bryggetorget 3,
0125 Oslo.

The presentation will webcast live and the link will be available at www.bergenbio.com in the section Investors/Financial Reports. A recording will be available shortly after the webcast has finished.

The results report and presentation will be available at www.bergenbio.com in the section: Investors/Financial Reports from 7:00 am CET the same day.

On May 8, 2019 Arvinas, Inc. (Nasdaq: ARVN), a biopharmaceutical company creating a new class of therapies that degrades disease-causing proteins, reported financial results for the first quarter of 2019 and provided a corporate update (Press release, Arvinas, MAY 8, 2019, View Source [SID1234535898]).

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"The first quarter of 2019 was Arvinas’ first quarter as a clinical-stage company and we remain laser-focused on moving our clinical and preclinical programs ahead," said John Houston, Ph.D., Chief Executive Officer at Arvinas. "We are excited that our Phase 1 clinical trial of ARV-110 for the treatment of men with metastatic castration-resistant prostate cancer is underway and look forward to sharing preliminary clinical data regarding the trial in the second half of 2019."

Ian Taylor, Ph.D., Chief Scientific Officer at Arvinas, added, "The ARV-110 clinical trial is a milestone for patients affected by diseases that may be addressable by targeted protein degradation. Our second PROTAC protein degrader, ARV-471, remains on-track to enter the clinic in the third quarter as a potential treatment for patients with locally-advanced or metastatic breast cancer. Our recent progress has excited us further about what we can achieve with our PROTAC protein degrader platform and its potential to significantly improve the lives of patients."

Business Highlights and Recent Developments

Began dosing patients in our first clinical trial, a Phase 1 clinical trial of ARV-110, which will evaluate the safety and tolerability of ARV-110 in patients with metastatic castration-resistant prostate cancer (mCRPC) who have progressed on standard of care therapies. We anticipate preliminary data from the study in the second half of 2019 and believe ARV-110 is the first in a new class of targeted protein degraders to enter human clinical trials.
Completed our initial public offering (IPO) in October 2018, issuing an aggregate of 7,700,482 shares of common stock, including 200,482 additional shares of common stock upon the exercise, in part by the underwriters, of their option to purchase additional shares at a public offering price of $16.00 per share. The company received aggregate gross proceeds of approximately $123.2 million.
Anticipated Milestones and Expectations

Present preliminary safety, tolerability and pharmacokinetic data from the Phase 1 clinical trial of ARV-110 in the second half of 2019.
Initiate a Phase 1 clinical trial of ARV-471 in patients with locally advanced or metastatic ER-positive / HER2-negative breast cancer in the third quarter of 2019 and share preliminary clinical data in 2020.
Discuss our neuroscience strategy and present preclinical data from our tau program in the second half of 2019.
Financial Guidance

Based on its current operating plan, Arvinas expects its cash, cash equivalents, and marketable securities as of March 31, 2019 will be sufficient to fund its operating expenses and capital expenditure requirements into the first half of 2021.

First Quarter Financial Highlights

Cash, Cash Equivalents, and Marketable Securities Position: As of March 31, 2019, cash, cash equivalents, and marketable securities were $175.0 million as compared to $187.8 million as of December 31, 2018. The decrease related to cash used to fund operations of $15.5 million offset by cash received from a collaborator of $2.7 million.

Research and Development Expenses: Research and development expenses were $14.2 million for the quarter ended March 31, 2019, as compared to $7.1 million for the quarter ended March 31, 2018. The increase in research and development expenses for the quarter primarily related to expenses associated with the initiation of our Phase 1 clinical trial of ARV-110 and IND-enabling expenses associated with ARV-471 as well as increased personnel and other expenses related to our platform research and exploratory programs research.

General and Administrative Expenses: General and administrative expenses were $5.6 million for the quarter ended March 31, 2019, as compared to $1.2 million for the quarter ended March 31, 2018. The increase in general and administrative expenses for the quarter was primarily related to increased employee expenses and other compliance costs associated with becoming a public company in the fourth quarter of 2018.

Revenues: Revenue was $4.0 million for the quarter ended March 31, 2019, as compared to $4.1 million for the quarter ended March 31, 2018. Revenues are generated primarily from the license and rights to technology fees and research and development activities related to the collaboration and license agreement with Pfizer that was initiated in January 2018 and the amended and restated option, license and collaboration agreement with Genentech that was initiated in November 2017.

Net Loss: Net loss was $14.4 million for the quarter ended March 31, 2019, as compared to $4.2 million for the quarter ended March 31, 2018. The increase in net loss for the quarter ended March 31, 2019 was primarily due to increased research and development expenses and increased general and administrative expenses.

About ARV-110
ARV-110 is an orally-bioavailable PROTAC protein degrader designed to selectively target and degrade androgen receptor protein (AR). ARV-110 is being developed as a potential treatment for men with metastatic castration-resistant prostate cancer (mCRPC). ARV-110 has demonstrated activity in preclinical models of AR mutation or overexpression, both common mechanisms of resistance to currently available AR-targeted therapies. Arvinas believes the differentiated pharmacology of ARV-110, including its iterative activity, has the potential to translate into improved clinical outcomes for patients.

About ARV-471
ARV-471 is an orally-bioavailable PROTAC protein degrader designed to target the estrogen receptor (ER) for the treatment of patients with locally-advanced or metastatic ER-positive / HER2-negative breast cancer. A Phase 1 clinical trial for ARV-471 in patients with locally-advanced or metastatic ER-positive / HER2-negative breast cancer is expected to begin in the third quarter of 2019 and preliminary clinical data is expected in 2020.

On May 8, 2019 Clarity Pharmaceuticals, a radiopharmaceutical company focused on the treatment of serious disease, is reported that it has completed its anticipated capital raising of $10,000,005.50 (Press release, Clarity Pharmaceuticals, MAY 8, 2019, View Source [SID1234535879]). This includes the previously announced strategic investment of $5,000,002.75 from GenesisCare, the largest private provider of cancer treatment in Australia and Europe, an additional $3,965,957.00 from the undertaking of a Rights Issue to Clarity’s existing shareholders, and now the remaining amount of $1,034,045.75 from new shareholders.

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The funds will enable Clarity to progress the development of its pipeline of radiopharmaceuticals, including Cu-64 SARTATE and Cu-67 SARTATE, which are next generation, highly targeted, theranostic (diagnostic and therapy) pharmaceuticals with increased specificity and in vivo stability. Clarity’s first theranostic trial of SARTATE in a serious brain cancer called meningioma commenced in July 2018 with the study halfway complete. Clarity will also progress clinical trials with SARTATE in neuroendocrine tumours (NETs) and neuroblastoma in collaboration with leading institutions in the US and Australia. In addition to the development of SARTATE, the capital raising will also enable the clinical development of two other products in Clarity’s pipeline, a prostate specific membrane antigen (PSMA) targeting product for the diagnosis and treatment of prostate cancer; and a bombesin product that targets the gastrin-releasing peptide receptor, which is present in a number of serious cancers including lung, ovarian, prostate, and breast.

Dr Alan Taylor, Executive Chairman of Clarity Pharmaceuticals, commented, "We are very pleased to have completed our capital raising which included a strategic investment from our partner GenesisCare, strong support from our current shareholder base as well as great demand from new investors. This capital raising also coincides with recent additions and changes to the team, including ex-Algeta CEO and CMO, Dr Thomas Ramdahl and Dr Gillies O’Bryan-Tear respectively, joining our Board, as well as former Algeta executive, Dr Colin Biggin, moving to acting CEO. Our growing team is dedicated to continuing our important work of developing safer and more effective treatments for children and adults with cancer together with a number of our collaborators and world leading experts in the field."