Alligator Bioscience: First Patient Dosed in Phase I study of ATOR-1015

On March 7, 2019 Alligator Bioscience (Nasdaq Stockholm: ATORX) reported that the first patient has successfully been dosed in its phase I study of ATOR-1015, its drug candidate in development for tumor-directed immunotherapy (Press release, Alligator Bioscience, MAR 7, 2019, View Source [SID1234538669]). ATOR-1015 is designed with properties that shall enable it to accumulate in the tumor area after an intravenous injection, and selectively exert its effect there.

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The study, being initiated in five different clinics across Sweden and Denmark, is a first-in-human dose-escalation study in up to 53 patients with advanced solid tumor disease. The primary aim of the study is to investigate the safety and tolerability of the drug and to identify the recommended dose for subsequent phase II studies. The results of the study are expected to read out in the second half of 2020.

"I state with satisfaction that ATOR-1015 is the first investigational tumor-localizing bispecific CTLA-4 antibody ever being tested in the clinic. With this, Alligator takes the lead in a very hot area of research. While immune activation through CTLA-4 has shown impressive efficacy in multiple cancers, it is coupled with severe toxicity. We believe that ATOR-1015 will be at least as effective as the approved monospecific CTLA-4 antibody Yervoy, and with less side effects," said Per Norlén, CEO of Alligator. "As the study progresses we look forward to learning more about the potential of this investigational medicine to improve the treatment of multiple cancers".

As previously communicated, Alligator has appointed Theradex Oncology, a global contract research organization with extensive expertise in oncology clinical development, to conduct the phase I study.

For further information, please contact:
Cecilia Hofvander, Director Investor Relations & Communications
Phone +46 46 540 82 06
E-mail: [email protected]

This information is such information as Alligator Bioscience AB (publ) is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out above, at 11:45 a.m. CET on March 7, 2019.

About ATOR-1015
ATOR-1015 is a next generation CTLA-4 bispecific antibody developed for tumor-directed immunotherapy with increased capability of regulatory T-cell depletion. It is wholly-owned by Alligator. ATOR-1015 binds to two different immune receptors: the checkpoint receptor CTLA-4 and the co-stimulatory receptor OX40. The immune activation is increased in areas where both target molecules are expressed at high levels, notably in the tumor microenvironment, which is believed to reduce adverse immune reactions.

About Yervoy
The active ingredient in Yervoy is ipilimumab, a protein that helps the body’s immune system to attack and destroy cancer cells. Yervoy is approved for the treatment of advanced melanoma (a type of skin cancer), and for combination treatment of advanced renal cell cancer (RCC) and colorectal cancer (MSIhigh CRC). The global total sales of Yervoy amounted to USD 1.2 billion in 2017. (Source: Fass.se, Cowen Therapeutics Outlook March 2018.)

Quanterix Corporation Releases Operating Results for Fourth Quarter and Full Year 2018
65% Q4 revenue growth; 65% full year growth

On March 7, 2019 Quanterix Corporation (NASDAQ:QTRX), a company digitizing biomarker analysis to advance the science of precision health, reported financial results for the three months and twelve months ended Dec. 31, 2018 (Press release, Quanterix, MAR 7, 2019, View Source [SID1234534085]).

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"This past quarter has been highly productive, capping off a strong year of accelerating growth, achieving all our key milestones, and positioning us very well for 2019," said Kevin Hrusovsky, Chief Executive Officer, President and Chairman, Quanterix. "Our Simoa technology is at the forefront of the biomarker revolution, which has gained considerable traction as biomarkers are increasingly being recognized for their promise to transform treatment options by accelerating the development of more effective and safer drugs for all disease categories, and monitoring patient response to treatment therapies to ensure their efficacy and safety. Longer term, we see the promise of Simoa biomarker assays being deployed as health screens to see disease very early in the disease cascade when treatments are most effective. We have been impressed with the acceleration of third-party, peer-reviewed publications and the resulting body of evidence showing the promise for Simoa blood based biomarker assays enabling early ‘pre-symptomatic’ detection of neurological diseases and cancers."

Fourth Quarter 2018 Financial Highlights
Key financial results for the fourth quarter are shown below:

Q4 revenue of $10.9M versus prior year Q4 of $6.6M, an increase of 65%.
Q4 product revenue was $7.5M versus prior year Q4 of $4.1M, an increase of 83%.
Q4 Service and Other revenue totaled $3.4M versus prior year Q4 of $2.2M, an increase of 52%.
Q4 gross margin percent totaled 48.2%, a 450 basis point improvement over the 43.7% in Q4 2017.
YTD 2018 Financial Highlights
Key financial results for 2018 YTD are shown below:

YTD revenue of $37.6M versus prior year $22.9M, an increase of 65%.
YTD product revenue of $23.4M versus prior year $14.1M, an increase of 65%.
YTD Service and Other revenue of $12.1M versus prior year $7.7M, an increase of 58%.
YTD Collaboration revenue of $2.15M versus prior year $1.07M, an increase of 100%.
YTD gross margin percent totaled 47.8%, a 410 basis point improvement over the 43.7% for full year 2017.
Excluding a one-time collaboration revenue item of $1.1M, total YTD revenue growth would have been 60%. See "Non-GAAP Financial Measures" below.

Fourth Quarter and Full Year 2018 Business Highlights

Gained unrestricted rights back for its Simoa technology in IVD markets with the termination of a license agreement with bioMérieux.
Third-party, peer-reviewed publications continued to increase with more than 40 new publications featuring Simoa technology in Q4 alone, and >200 during the full year, bringing the total to over 400.
Acquired Aushon BioSystems and successfully integrated the two companies. The acquisition included a CLIA certified laboratory, expanding services and accelerating entry into pharmaceutical drug trial services, as well as access to novel immunoassay technology to which Simoa sensitivity algorithms are being applied to deliver next generation Simoa capabilities.
Adoption of proprietary Neurofilament light chain (NfL) assay, a biomarker previously measured only in cerebral spinal fluid (CSF), but by using Simoa, researchers have shown it can be measured in blood and correlated to CSF results. This advance is enabling novel applications critical for advancing early detection, treatment and prevention of neurological diseases, including multiple sclerosis (MS); Parkinson’s disease; Alzheimer’s disease; brain cancer; and traumatic brain injuries (TBIs).
Invited by the FDA and presented on progress and the potential future for the NfL biomarker to agency officials.
Launched a test bed, early access program for the SP-X Imaging and Analysis System with a 10-plex Simoa cytokine panel for oncology research and drug development.
Increased employee headcount from 126 at Dec. 31, 2017 to 177 at Dec. 31, 2018 (>40%) and expanded the leadership team with the recent hiring of Jackson Streeter, M.D. to lead corporate development and strategy, formerly chief medical officer and chief executive officer of Banyan Biomarkers; and Mary-Ellen Cortizas to lead the Accelerator Lab, formerly founder and chief operating officer of Claritas Genomics.
Added to the Nasdaq Biotechnology Index (NBI) as part of the annual re-ranking, and attracted additional analyst coverage.
Participated in first EU Powering Precision Health Summit, which was a highly productive meeting of leading researchers, key opinion leaders, and other stakeholders in precision health.
Reinforced the Company’s digital biomarker position through presentations at PULSE The Atlantic Summit on Health Care, The Lake Nona Impact Forum, and MedCity Converge, as well as the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) Congress, where the Company had a record number of posters/presentations mentioning the use of Quanterix’ serum NfL assay for a wide range of clinical studies in MS disease progression and monitoring.
Conference Call
In conjunction with this announcement, Quanterix Corporation will host a conference call on March 7, 2019, at 4:30 p.m., EST to discuss the Company’s financial results and business outlook. To access this call, dial (833) 686-9351 for domestic callers, or (612) 979-9890 for international callers. Please reference the following conference ID: 3288482.

A live webcast will be accessible on the Investors section of Quanterix’ website: View Source The webcast will be available on the Company’s website for one year following completion of the call.

BioTime to Report Fourth Quarter and Full Year 2018 Financial Results on March 14th, 2019

On March 7, 2019 BioTime, Inc. (NYSE American and TASE: BTX), a clinical-stage biotechnology company focused on degenerative diseases, reported that it will report its fourth quarter and full year 2018 financial and operating results on Thursday, March 14th, 2019, following the close of the U.S. financial markets (Press release, BioTime, MAR 7, 2019, View Source;p=RssLanding&cat=news&id=2390475 [SID1234534111]). BioTime management will also host a conference call and webcast on Thursday, March 14th, 2019, at 4:30 p.m. Eastern Time/1:30 p.m. Pacific Time to discuss its fourth quarter and full year 2018 financial results and to provide a business update.

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Interested parties may access the conference call by dialing (866) 888-8633 from the U.S. and Canada and (636) 812-6629 from elsewhere outside the U.S. and should request the "BioTime Inc. Call". A live webcast of the conference call will be available online in the Investors section of BioTime’s website. A replay of the webcast will be available on BioTime’s website for 30 days and a telephone replay will be available through March 21st, 2019, by dialing (855) 859-2056 from the U.S. and Canada and (404) 537-3406 from elsewhere outside the U.S. and entering conference ID number 1091719.

Arbutus Reports Fourth Quarter and Year-end 2018 Financial Results and Describes Recent Clinical Accomplishments and Key 2019 Objectives

On March 7, 2019 Arbutus Biopharma Corporation (Nasdaq: ABUS), an industry-leading Hepatitis B Virus (HBV) therapeutic solutions company, reported its fourth quarter and year-end 2018 financial results and provides a description of recent clinical accomplishments and key 2019 corporate objectives (Press release, Arbutus Biopharma, MAR 7, 2019, View Source [SID1234534131]).

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"Arbutus is committed to developing a cure for chronic Hepatitis B which we maintain can be best achieved by employing a combination of therapeutic agents with distinct, yet complementary mechanisms of action," said Dr. Mark J. Murray, President and Chief Executive Officer of Arbutus. "With multiple clinical trial initiations and data readouts expected throughout the year, 2019 promises to be an eventful and important year for Arbutus as we make progress toward our first novel combination regimen."

Recent Clinical Accomplishments and Key 2019 Objectives

AB-506

In a Phase 1a/1b clinical trial, AB-506, Arbutus’ oral capsid inhibitor, successfully progressed through the healthy volunteer portion and is now being evaluated in HBV patients in the 28-day multiple dose Phase 1b portion of the trial. Top-line results of this Phase 1a/1b clinical trial are expected late in the second quarter of 2019.

A Phase 2 dose-finding and long-term safety trial of AB-506 in combination with an approved nucleoside analogue (NA) is expected to be initiated late in the second half of the year to support AB-506 use in future combination trials.

AB-506 inhibits HBV capsid assembly which inhibits HBV replication, a mode of action complementary to NAs; its use in patients is expected to reduce the levels of HBV DNA in the blood.
AB-729

AB-729 is currently completing IND-enabling studies and is expected to begin a Phase 1a/1b clinical trial in the second quarter of 2019 and progress into HBV patients in the second half of the year. AB-729 is an RNAi agent which blocks HBsAg expression and can be administered subcutaneously and we anticipate will be dosed monthly.

A Phase 2 clinical trial combining AB-729, AB-506 and an approved NA is expected to initiate in the first half of 2020.
AB-452 and RNA Destabilizer Program

Arbutus is developing oral RNA-destabilizers that have shown compelling anti-viral effects in multiple preclinical models. As a result of a nonclinical safety finding with our lead RNA-destabilizer, AB-452, we are conducting a series of in vitro and in vivo studies to further characterize the compound, its mechanism of action and pharmacokinetic profile before deciding to initiate clinical trials. A go/no go decision is expected in the second half of the 2019.

In parallel, the Company is also advancing a number of follow on compounds with distinct chemical scaffolds into the lead optimization stage.
Dr. Michael J. Sofia, Arbutus’ Chief Scientific Officer, stated, "We believe our RNA destabilizer program is amongst the most advanced programs of its kind in the HBV space and we remain confident that this mechanism represents a very relevant and important therapeutic target; success here could be very meaningful for patients and for Arbutus."

ARB-1467

The Company has discontinued development of ARB-1467. Results from the ARB-1467 clinical trials confirmed the potential therapeutic value of an RNAi agent and informed the development of our next-generation RNAi agent, AB-729.
Early R&D Programs

The Company continues a robust discovery effort focused on back-up compounds for its current pipeline as well as discovery efforts focused on reawakening HBV patient’s immune response and on novel HBV-specific targets. These programs include orally available compounds targeting PD-L1 and HBV cccDNA.
Cash Position and 2019 Cash Guidance

The Company ended the year with approximately $125 million in cash, cash equivalents and short-term investments which we believe is sufficient to fund operations into 2020. The Company expects to use approximately $70 to $75 million in cash in 2019.
ONPATTRO Royalty Entitlement

ONPATTRO is an RNAi therapeutic that has been developed for the treatment of hereditary ATTR (hATTR) amyloidosis, and has been approved by the FDA and the EMA. Arbutus has a royalty entitlement on global sales of ONPATTRO for the LNP technology licensed by Arbutus to Alnylam for this product. The Company began recognizing royalty income in 2018. The royalty rate is tiered, based on product sales, and in the low to mid-single digits.

Financial Results

Cash, Cash Equivalents and Investments

As of December 31, 2018, Arbutus had cash, cash equivalents and short-term investments totaling $124.6 million, as compared to $139.0 million in cash and cash equivalents, short-term investments, and restricted investments at December 31, 2017.

Net Loss

For the year ended December 31, 2018, net loss attributable to common shares was $67.2 million ($1.21 basic and diluted loss per common share) as compared to $85.3 million ($1.56 basic and diluted loss per common share) for 2017.

Revenue

Revenue was $5.9 million in 2018 compared to $10.7 million in 2017. The decrease was related primarily to a $7.5 million non-recurring, upfront payment in 2017 from Alexion Pharmaceuticals, Inc. Revenue in 2018 includes $4.3 million pursuant to our license agreement with Gritstone Oncology, Inc.

Research and Development

Research and development expenses were $57.9 million, including $2.7 million of non-cash stock based compensation in 2018 compared to $62.7 million in 2017, including $9.2 million of non-cash stock based compensation. Excluding the decrease in non-cash stock based compensation expense, which was due to the expiry of certain share repurchase rights in 2017, R&D expenses in 2018 have increased as Arbutus’ pipeline expands and advances into the clinic.

General and Administrative

General and administrative expenses were $16.0 million in 2018, including $3.3 million of non-cash stock based compensation compared to $16.1 million in 2017, including $5.9 million of non-cash stock based compensation.

Site Consolidation

Site consolidation expenses were $4.8 million in 2018.

In the first half of 2018, Arbutus substantially completed a site consolidation and organizational restructuring to better align its HBV business in Warminster, PA, by reducing the Company’s global workforce and closing its facility in Burnaby, Canada. We expect related total cash expenditures will be approximately $5.6 million upon completion, of which approximately $4.8 million has been incurred to date.

Impairment of intangible assets

In 2018, the Company recorded a $14.8 million ($10.5 million net of tax benefit) non-cash expense for the impairment of intangible assets related to the indefinite deferral of further development of its AB-423 capsid inhibitor program, due to the successful progression of its AB-506 capsid inhibitor program.

Decrease in fair value of contingent consideration

In 2018 the Company recorded a non-cash decrease in contingent consideration of $7.3 million compared to a $1.4 million increase in 2017.

The decrease in 2018 was due primarily to the Company’s decision to indefinitely defer clinical development of AB-423 thereby reducing the probability of achieving future development milestones, as well as a recalibration in the expected timing of future sales milestones, resulting in a reduction in the estimated fair value of the liability.

Equity investment loss

The Company recorded a gain of $24.9 million on its initial investment in Genevant, a jointly owned company with Roivant Sciences Ltd., and equity losses of $5.6 million for its proportionate share of the Genevant’s net loss. Financial results of Genevant are recorded on a one-quarter lag basis. The Company currently owns approximately 40% of the common equity of Genevant as of December 31, 2018.

Outstanding Shares

The Company had 55.5 million common shares issued and outstanding at December 31, 2018. In addition, the Company had 6.8 million options outstanding and 1.164 million Preferred Shares outstanding, which (including the annual 8.75% coupon) will be mandatorily convertible into 22.6 million common shares on October 18, 2021. Assuming the outstanding options and convertible preferred shares were fully converted, the Company would have had 84.9 million common shares outstanding at December 31, 2018.

Conference Call Today

Arbutus will hold a conference call and webcast today, Thursday, March 7, 2019 at 4:30 PM Eastern Time (1:30 PM Pacific Time) to provide a corporate update. You can access a live webcast of the call through the Investors section of Arbutus’ website at www.arbutusbio.com. Alternatively, you can dial 1-866-393-1607 or 1-914- 495-8556 and reference conference ID 1942769.

An archived webcast will be available on the Arbutus website after the event. Alternatively, you may access a replay of the conference call by calling 1-855-859-2056 or 1-404-537-3406, and reference conference ID1942769.

Zymeworks Reports 2018 Year-End Financial Results

On March 6, 2019 Zymeworks Inc. (NYSE/TSX: ZYME), a clinical-stage biopharmaceutical company developing multifunctional therapeutics, reported financial results for the year ended December 31, 2018 (Press release, Zymeworks, MAR 6, 2019, View Source [SID1234534014]).

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"Over the past year, we have made significant progress advancing our own pipeline while our partners continued developing their assets using our technology," said Ali Tehrani, Ph.D., Zymeworks’ President & CEO. "Our lead program, ZW25, continued to generate impressive clinical data and our second product candidate, ZW49, recently began a Phase 1 clinical trial. We also established our seventh and eighth pharmaceutical partnerships while our long-term partner, Eli Lilly, advanced two of their Azymetric bispecifics into the clinic."

Dr. Tehrani continued, "We entered 2019 with $200 million on our balance sheet, which will enable us to aggressively expand our clinical activities, and we look forward to providing updates throughout the year."

2018 Business Highlights and Recent Developments

ZW49 Investigational New Drug Application Accepted; Phase 1 Trial Underway
Zymeworks’ second product candidate, ZW49, a novel biparatopic HER2-targeted antibody drug conjugate (ADC), received clearance for a Phase 1 first-in-human clinical trial, which has begun enrollment in the United States.
Expanded Global Clinical Development for ZW25 and ZW49
Zymeworks expanded the ZW25 clinical trial into South Korea and also entered into a licensing agreement and collaboration with BeiGene to leverage their resources and expertise to accelerate the development of ZW25 and ZW49 in the Asia Pacific region (Zymeworks continues to own all rights in Japan).
Partner’s Programs Progress into the Clinic
Eli Lilly advanced two Azymetric immune-oncology bispecifics into the clinic triggering milestone payments to Zymeworks. Zymeworks currently has eight active collaborations that offer up to US$7.6 billion in potential milestone payments plus royalties.
Financial Results for the Year Ended December 31, 2018

Revenue in 2018 was $53.0 million as compared to $51.8 million in 2017. Revenue for both years was primarily comprised of non-recurring upfront fees, expansion payments and milestone payments from the Company’s licensing and collaboration agreements. Revenue for 2018 included: recognition of $23.5 million out of the $60.0 million upfront fee from BeiGene associated with a new licensing agreement; an $18.0 million upfront fee related to a second licensing agreement with Daiichi Sankyo; a $5.0 million upfront fee related to a new licensing agreement with LEO Pharma; a $4.0 million research program expansion fee from Celgene; a $2.0 million development milestone payment from Eli Lilly, and $0.5 million in research support payments from various collaborators. Revenue for 2017 included a $50.0 million upfront fee from Janssen and a $1.0 million milestone payment from Daiichi Sankyo, as well as $0.8 million in research support payments.

For the year ended December 31, 2018, research and development expenses were $56.7 million as compared to $41.7 million in the prior year. The change was primarily due to an increase in activities related to ZW25 clinical studies and associated manufacturing costs, as well as development activities for ZW49. There were also additional research and development activities, resulting in incremental headcount-related expense and non-cash stock-based compensation expense. Research and development expenses included non-cash stock-based compensation expense of $2.2 million from equity classified equity awards and a $2.0 million expense related to the non-cash mark-to-market revaluation of certain historical liability classified equity awards.

For the year ended December 31, 2018, general and administrative expenses were $29.5 million as compared to $18.6 million in the prior year. The change was primarily due to an increase in non-cash liability classified equity adjustments and stock-based compensation, as well as other increases in compensation and professional fees associated with year-on-year corporate growth. General and administrative expense included non-cash stock-based compensation expense of $3.7 million from equity classified equity awards and $5.4 million expense related to the non-cash mark-to-market revaluation of certain historical liability classified equity awards.

The net loss for the year ended December 31, 2018, was $36.6 million as compared to $10.4 million in 2017, primarily due to an increase in research and development expenses associated with our lead therapeutic candidates and other programs, general and administrative expenses, and the accounting impact of an increase of $6.9 million related to the non-cash mark-to-market revaluation of certain historical liability classified equity awards.

Zymeworks expects research and development expenditures to increase over time due to the ongoing development of product candidates and other clinical, preclinical, and regulatory activities. Additionally, Zymeworks anticipates continuing to receive revenue from its existing and future strategic partnerships, including technology access fees and milestone-based payments. However, Zymeworks’ ability to receive these payments is dependent upon either Zymeworks or its collaborators successfully completing specified research and development activities.

As of December 31, 2018, Zymeworks had $200.2 million in cash and cash equivalents and short-term investments.