On May 9, 2018 Horizon Pharma plc (NASDAQ:HZNP) reported its first-quarter 2018 financial results and increased its full-year 2018 net sales and adjusted EBITDA guidance (Press release, Horizon Pharma, MAY 9, 2018, View Source [SID1234526308]). The Company also announced that, effective in the second-quarter 2018, the Company is realigning its operating structure and will report financial results as two separate operating segments: its strategic growth business, orphan and rheumatology; and primary care. The new operating structure reflects the evolution of the Company’s strategy and vision of transitioning Horizon Pharma to a biopharmaceutical company focused on rare disease medicines.

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"Our orphan and rheumatology medicines represented approximately 77 percent of the Company’s first-quarter net sales and generated double-digit growth driven by 48 percent growth of KRYSTEXXA," said Timothy P. Walbert, chairman, president and chief executive officer, Horizon Pharma plc. "Our decision to operate orphan and rheumatology separately from primary care marks a pivotal next step in our ongoing strategic transformation to a company focused on rare disease medicines. We made significant advancements during the first quarter toward our goal, including progress ahead of our expectations in the Phase 3 clinical trial for our key rare disease medicine in development, teprotumumab, which is now 50 percent enrolled."

First-Quarter and Recent Company Highlights

New Head of R&D and Chief Scientific Officer: Shao-Lee Lin, M.D., Ph.D., joined the Company in January 2018 as executive vice president, head of research and development (R&D) and chief scientific officer. Dr. Lin is an accomplished pharmaceutical executive, physician and scientist with more than 20 years of academic and clinical research experience and will accelerate the development of a robust pipeline to drive the Company’s next phase of growth.

"We are committed to establishing Horizon Pharma as a leader in the rare disease space, and one of our goals to support that objective is to enhance the capabilities of our R&D organization," said Lin. "We are well on our way, having made several important additions to the organization that expand our development capabilities, support our business development team in evaluating and identifying development-stage opportunities and lead our therapeutic areas from a clinical development strategy and portfolio management perspective. Enhancing our R&D organization will enable us to maximize our on-market medicines and develop new medicines for patients with unmet needs – and in the case of rare diseases, some of the most significantly underserved patients."
Intellectual Property Update: The Company recently received two Notices of Allowance from the U.S. Patent and Trademark Office for U.S. patent application numbers 15/457,643 and 15/687,132, both entitled "Methods of Therapeutic Monitoring of Nitrogen Scavenging Drugs" that cover RAVICTI. The U.S. patents scheduled to issue from these applications will expire on Sept. 22, 2030. After issuance, the Company plans to list the U.S. patents in the FDA’s Approved Drug Products with Therapeutic Equivalence Evaluations, or Orange Book.
Research and Development
Orphan Candidates and Programs:

Teprotumumab: The Phase 3 clinical trial for teprotumumab, the Company’s fully human monoclonal antibody IGF-1R-inhibitor in development for the treatment of thyroid eye disease (TED), a rare eye disease, is 50 percent enrolled and is on track for enrollment completion by year end, or earlier. The pivotal confirmatory study is evaluating teprotumumab for the treatment of moderate-to-severe active TED, which has no FDA-approved treatments. The Company estimates peak annual U.S. net sales of more than $750 million for teprotumumab, assuming U.S. FDA approval.
Rheumatology Pipeline Candidates and Programs:

Immunomodulation Studies: The evaluation of the use of immunomodulation therapies to enhance the response rate to KRYSTEXXA is being studied in two investigator-initiated trials, using two different immunomodulators, both of which are commonly used by rheumatologists. REduCing Immunogenicity to PegloticasE (RECIPE) is a double-blind, placebo controlled trial to evaluate the impact of a 12-week course of immunomodulating therapy with daily doses of mycophenolate mofetil (MMF). Tolerization Reduces Intolerance to Pegloticase and Prolongs the Urate Lowering Effect (TRIPLE) is an exploratory, open-label adaptive trial with multiple patient cohorts, including a cohort to evaluate the impact of adding daily doses of the immunomodulator azathioprine for a two-week run-in period, followed by KRYSTEXXA every two weeks for a total of 13 doses along with daily doses of azathioprine.

New Rheumatology Programs: In January 2018, the Company announced two development programs for next-generation biologics for uncontrolled gout (chronic gout that is refractory to conventional therapies) to support and sustain the Company’s market leadership in uncontrolled gout: HZN-003 and PASylated uricase technology. HZN-003 is a pre-clinical, genetically engineered uricase derivative with optimized uricase and optimized PEGylation technology. PASylated uricase technology may improve the half-life of uricase, and the Company is collaborating with a third party to identify a lead candidate that could use the technology to construct a next-generation gout biologic. The Company also announced the addition of HZN-002, a pre-clinical, novel dexamethasone conjugate with the potential to address inflammatory diseases through its targeted delivery technology.
New Operating Structure Aligned with Long-term Strategy

Given the Company’s focus on rare disease medicines, effective in the second quarter of 2018, the Company is realigning its structure to operate its strategic growth business, orphan and rheumatology, separate from its primary care business. The new structure allows the Company to more efficiently allocate its resources to address unmet treatment needs for patients with rare diseases.

As part of the new operating structure, the Company has realigned its commercial operations under a new leadership position, executive vice president and chief commercial officer, and recently promoted Vikram Karnani to that role. Karnani was most recently senior vice president, rheumatology business unit, leading the successful growth to date of KRYSTEXXA. In addition, aligned with the new operating structure, the Company is adding critical R&D leadership roles to support the orphan and rheumatology segment, including recently hired clinical development heads for both of these therapeutic areas.

As a result of these changes, in the second quarter of 2018, the Company will begin reporting its financial results as two separate operating segments: the orphan and rheumatology segment, the Company’s strategic rare disease-focused business and the primary care segment, reporting net sales and operating income for each segment.

First-Quarter 2018 Total Company Financial Results

Note: For additional detail and reconciliation of non-GAAP financial measures to the most directly comparable GAAP financial measures, please refer to the tables at the end of this release.

Total Net Sales: First-quarter net sales were $223.9 million, an increase of 1.4 percent, driven by continued strong growth of the Company’s orphan and rheumatology medicines. Net sales of $220.9 million in the first quarter of 2017 included PROCYSBI and QUINSAIR net sales of $4.9 million in Europe, the Middle East and Africa (EMEA) regions. The EMEA marketing rights to PROCYSBI and QUINSAIR were divested in June 2017. Excluding the 2017 EMEA net sales of PROCYSBI and QUINSAIR, year-over-year growth would have been 3.7 percent.

Gross Profit: Under U.S. GAAP in the first quarter of 2018, the gross profit ratio was 48.1 percent compared to 37.0 percent in the first quarter of 2017. The non-GAAP gross profit ratio in the first quarter of 2018 was 87.0 percent compared to 88.5 percent in the first quarter of 2017.

Operating Expenses: R&D expenses were 7.9 percent of net sales; and selling, general and administrative (SG&A) expenses were 80.2 percent of net sales. Non-GAAP R&D expenses were 6.8 percent of net sales, and non-GAAP SG&A expenses were 65.2 percent of net sales.
Income Tax Rate: The income tax rate in the first quarter of 2018 on a GAAP basis was 0.2 percent and on a non-GAAP basis was 44.5 percent.

Net (Loss) Income: On a GAAP basis in the first quarter of 2018, net loss was $157.3 million. First-quarter 2018 non-GAAP net income was $4.8 million.

Adjusted EBITDA: In the first quarter of 2018, adjusted EBITDA was $33.6 million.

Earnings (Loss) per Share: On a GAAP basis in the first quarter of 2018, diluted loss per share was $0.96; in the first quarter of 2017, diluted loss per share was $0.56. Non-GAAP diluted earnings per share in the first quarter of 2018 and 2017 were $0.03 and $0.21, respectively. Weighted average shares outstanding used for calculating GAAP diluted loss per share and non-GAAP diluted earnings per share in the first quarter of 2018 were 164.5 million and 167.8 million, respectively.

(1) On June 23, 2017, Horizon Pharma completed the divestiture of a European subsidiary that owned the marketing rights to PROCYSBI and QUINSAIR in Europe, the Middle East and Africa to Chiesi Farmaceutici S.p.A. Horizon Pharma retains marketing rights for the two medicines in the United States, Canada, Latin America and Asia.
Combined first-quarter 2018 net sales of orphan and rheumatology medicines of $172.2 million increased 11 percent, driven by continued strong KRYSTEXXA vial growth, as well as growth of RAVICTI and PROCYSBI. Combined first-quarter 2017 net sales of orphan and rheumatology medicines of $155.3 million included EMEA net sales of PROCYSBI and QUINSAIR, which were divested in June 2017, of $4.9 million. Excluding the 2017 EMEA net sales of PROCYSBI and QUINSAIR from combined orphan and rheumatology net sales, year-over-year growth would have been 15 percent.

First-quarter 2018 net sales of primary care medicines were $51.7 million, negatively impacted by seasonality, to a somewhat greater degree than originally anticipated. First-quarter net sales also included a negative $14 million impact from an additional accrual for medicines in the wholesale and retail channel following the Company’s price action. Excluding the additional accrual, which did not occur in first-quarter 2017, first-quarter 2018 primary care net sales on a pro-forma basis were similar to first-quarter 2017, reflecting the stability of this business.
Cash Flow Statement and Balance Sheet Highlights

On a GAAP basis in the first quarter of 2018, operating cash flow was negative $60.8 million. Non-GAAP operating cash flow was negative $52.7 million in the first quarter of 2018, as expected. GAAP and non-GAAP operating cash flow in the first quarter of 2017 included a significant one-time working capital benefit associated with the implementation of managed care contracts for certain primary care medicines.
The Company had cash and cash equivalents of $674.3 million as of March 31, 2018.

As of March 31, 2018, the total principal amount of debt outstanding was $2.019 billion, which comprised $844 million in senior secured term loans due 2024; $300 million senior notes due 2024; $475 million senior notes due 2023; and $400 million exchangeable senior notes due 2022. As of March 31, 2018, net debt was $1.344 billion.
Full-Year 2018 Guidance

The Company now expects full-year 2018 net sales guidance of $1.170 billion to $1.200 billion, an increase from the previous range of $1.150 billion to $1.180 billion. Full-year 2018 adjusted EBITDA is now expected to be $390 million to $415 million, an increase from the previous range of $370 million to $395 million. Company guidance now assumes a delay in the implementation of a U.S. government rule related to 340B entity drug pricing to July 1, 2019, following the U.S. Department of Health and Human Services’ proposal to delay the effective date to that date. As a result, the Company now expects full-year 2018 net sales growth for KRYSTEXXA of more than 65 percent.

Webcast

At 8 a.m. EST / 1 p.m. IST today, the Company will host a live webcast to review its financial and operating results and provide a general business update. The live webcast and a replay may be accessed at View Source Please connect to the Company’s website at least 15 minutes prior to the live webcast to ensure adequate time for any software download that may be needed to access the webcast. A replay of the webcast will be available approximately two hours after the live webcast.

On May 9, 2018 BioSpecifics Technologies Corp. (NASDAQ: BSTC), a biopharmaceutical company that originated and continues to develop collagenase based therapies with a first in class collagenase-based product marketed as XIAFLEX in the U.S. and Xiapex in Europe, reported its financial results for the first quarter ended March 31, 2018 and provided a corporate update (Press release, BioSpecifics Technologies, MAY 9, 2018, View Source [SID1234526362]).

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"We are enthusiastic about the sustained year-over-year revenue growth of our CCH product, XIAFLEX, in its marketed indications, Dupuytren’s Contracture and Peyronie’s Disease, and by the potential progress of the broader CCH development pipeline. During the first quarter of 2018, XIAFLEX became commercially available in Canada for Peyronie’s Disease and our partner Endo reported updated data from the Phase 2b clinical trial of CCH for the treatment of cellulite at the American Society for Aesthetic Plastic Surgery," said Thomas L. Wegman, President of BioSpecifics. "We also remain focused on identifying and developing CCH for medically necessary indications, and we remain on track to report preliminary data later this year for our ongoing Phase 1 clinical trial in uterine fibroids, a painful condition of the reproductive tract in which benign tumors contain large amounts of collagen and we are excited by the potential of CCH to alleviate patients’ symptoms and ultimately avoid invasive surgery."

First Quarter 2018 Financial Results

BioSpecifics reported net income of $4.0 million for the first quarter ended March 31, 2018, or $0.55 per basic share and $0.54 per share on a fully diluted basis, compared to net income of $3.3 million, or $0.47 per basic share and $0.46 per share on a fully diluted basis, for the same period in 2017.

The Company adopted Accounting Standard Codification (ASC) 606, Revenue from Contracts with Customers, as of January 1, 2018, applying the modified-retrospective method, changed the timing of its recognition of royalty and mark-up on cost of goods sold revenue. Beginning in 2018, BioSpecifics recorded these royalty revenues based on estimates of the net sales and mark-up on cost of goods sold that occurred during the relevant period thereby eliminating the previously utilized one quarter lag. Previously, these amounts were not recognized until they were fixed and determinable. In addition, deferred revenue associated with the pre-payment of foreign mark-up on cost of goods sold revenue will no longer be recognized over time based on sales by non-affiliated sub-licensees of Endo outside of the U.S. and, under ASC 606, would have been recognized when the Company amended its License Agreement in 2016. Prior period amounts have not been adjusted and the Company recognized a cumulative-effect adjustment to retained earnings on January 1, 2018 of $10.3 million.

Total revenue for the first quarter ended March 31, 2018 was $7.1 million, compared to $7.7 million for the same period in 2017. The decrease in total revenues for the quarterly period was primarily due to lower mark-up on cost of goods sold revenue in the U.S and prepaid foreign mark-up on cost of goods sold revenue recognized under new revenue standard ASC 606, as of January 1, 2018, which was partially offset by an increase in royalties associated with slightly higher overall net sales of XIAFLEX.

Licensing revenue consists of licensing fees, sublicensing fees and milestones. BioSpecifics recognized licensing revenue for the first quarter ended March 31, 2018 and 2017 of approximately $4,409 in each period.

Research and development (R&D) expenses for the first quarter ended March 31, 2018 were $0.2 million compared to $0.3 million for the same period in 2017.

General and administrative expenses for the first quarter ended March 31, 2018 were $2.1 million, compared to $2.4 million for the same period in 2017.

Provision for income taxes for the first quarter ended March 31, 2017 were $1.1 million, compared to $1.8 million for the same period in 2017.

As of March 31, 2018, BioSpecifics had cash and cash equivalents and investments of $64.2 million, compared to $65.1 million as of December 31, 2017. The Company’s cash and cash equivalents and investments were slightly lower due to the timing of Endo’s payment of its quarterly XIAFLEX royalties, which was received in April 2018.

CCH Pipeline Updates and Anticipated Upcoming Milestones

BioSpecifics manages the development of collagenase clostridium histolyticum (CCH) for the treatment of uterine fibroids and has the right to initiate the development of any new potential indication not licensed by Endo. Endo’s licensed indications include Dupuytren’s Contracture and Peyronie’s Disease, both approved and marketed; in addition to cellulite, adhesive capsulitis, human and canine lipoma, lateral hip fat and plantar fibromatosis.

In April 2018, Endo presented clinical data from the Phase 2b study of CCH for the treatment of cellulite during the Premier Global Hot Topics session at the annual meeting of the American Society for Aesthetic Plastic Surgery (ASAPS). A statistically significant proportion of CCH subjects reported being "Satisfied" or "Very Satisfied" with their cellulite treatment compared to placebo subjects. CCH was also well-tolerated by all dose groups. Most adverse events (AEs) were mild to moderate and primarily limited to the local injection area such as injection site bruising (approximately 75 percent) and injection site pain (approximately 59 percent) with 92 percent of all related AEs were mild to moderate in the CCH group compared to 96 percent in the placebo group.
In April 2018, Paladin Labs, a subsidiary of Endo, announced the commercial availability of XIAFLEX in Canada for the treatment of Peyronie’s Disease.
In February 2018, Endo initiated two Phase 3 clinical trials of CCH for the treatment of cellulite. Top-line results are expected in the first quarter of 2019.
BioSpecifics is conducting an ongoing Phase 1 clinical trial of CCH for the treatment of uterine fibroids with data expected later this year. This Phase 1 open-label dose escalation study is being conducted at the Department of Gynecology & Obstetrics at Johns Hopkins University and is designed to enroll 15 female subjects treated prior to hysterectomy. The trial will assess the safety and tolerability of a single injection of CCH directly into the uterine fibroid under transvaginal ultrasound guidance. The secondary endpoints will assess symptoms of pain and bleeding, quality of life throughout the study in addition to size, collagen content and rate of apoptosis of CCH treated fibroids.

On May 9, 2018 MEI Pharma, Inc. (NASDAQ: MEIP), a pharmaceutical company focused on leveraging its extensive development and oncology expertise to identify and advance new therapies for cancer, reported results for its third quarter ended March 31, 2018 (Press release, MEI Pharma, MAY 9, 2018, View Source [SID1234526378]).

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"Over the past quarter we continued making key advances in each of our four clinical programs, as we look forward to important data readouts before mid-year in three of the four programs," said Daniel P. Gold, Ph.D., president and chief executive officer of MEI Pharma. "In particular, we will be presenting data at ASCO (Free ASCO Whitepaper) 2018 from our study evaluating ME-401, an orally administered PI3K delta inhibitor demonstrating potential class leading efficacy in follicular lymphoma. Based on the data in this program, we anticipate progressing into a single-agent registration study later in 2018. In addition, we will present data at ASCO (Free ASCO Whitepaper) 2018 from an investigator-initiated study of ME-344 in HER2-negative breast cancer."

Dr. Gold added: "We also look forward to initiating our Phase 1 study of voruciclib, a selective and orally administered CDK inhibitor differentiated by potent inhibition of CDK9, in relapsed/refractory B lymphocyte malignancies. Finally, we are looking forward to an interim analysis this quarter from the Phase 2 pracinostat study in MDS and the potential to continue advancing the study together with our partner Helsinn."

Recent Program Highlights and Upcoming Milestones

ME-401

An abstract with results from a Phase 1b study in relapsed/refractory CLL and FL has been accepted and will be presented at the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting to be held June 1-5 in Chicago, IL.
Pracinostat (partnered with Helsinn Healthcare, SA)

In January 2018, the European Medicines Agency granted Orphan Drug Designation to pracinostat, currently in a Phase 3 study in combination with azacitidine for the treatment of acute myeloid leukemia (AML) in adult patients unfit for induction chemotherapy.
The Company anticipates results from stage 1 of a Phase 2 dose-optimization study in myelodysplastic syndrome (MDS) in the first half of 2018.
Voruciclib

In January 2018, the U.S. Food and Drug Administration cleared the Company’s Investigational New Drug Application (IND) for voruciclib.
The Company expects to initiate a Phase 1 single-agent study in relapsed/refractory B cell malignancies in the first half of 2018.
ME-344

In February 2018, the Company announced that a planned interim review of data supports the continuation of its multicenter, investigator-initiated, study evaluating ME-344 in patients with HER2-negative breast cancer. The interim study data show that ME-344 was generally well-tolerated and, consistent with previous preclinical data, demonstrate the potential to reverse resistance to antiangiogenic therapy.
An abstract featuring interim results from the Phase 1 study in HER2 negative breast cancer in combination with bevacizumab (marketed as Avastin) has been accepted and will be presented at the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting to be held June 1-5 in Chicago, IL.
Corporate

In February 2018, the Company announced the appointment of industry veteran Frederick W. Driscoll to its board of directors, who will also serve on the audit committee.
Financial Highlights

As of March 31, 2018, the Company had $36.2 million in cash, cash equivalents and short-term investments, with no outstanding debt. The Company believes its cash position will be sufficient to fund operations into through the first half of calendar year 2019.
Research and development expenses, including cost of research and development revenue, were $4.0 million for the three months ended March 31, 2018, compared to $3.0 million for the three months ended March 31, 2017. The increase was primarily due to increased drug manufacturing expenses for ME-401, expenses related to voruciclib, and increased costs related to salaries, share-based compensation, and other internal costs.
General and administrative expenses were $2.5 million for the three months ended March 31, 2018, compared to $2.2 million for the three months ended March 31, 2017. The increase was primarily due to professional services expenses and share-based compensation.
Revenues were $0.4 million for the three months ended March 31, 2018, compared to $4.5 million for the three months ended March 31, 2017. The decrease was related to lower levels of research and development activities associated with the Helsinn license agreement and $3.8 of upfront license fee revenue recognized for the three months ended March 31, 2017.
Net loss was $5.9 million, or $0.16 per share, for the three months ended March 31, 2018, compared to net loss of $0.6 million, or $0.02 per share for the three months ended March 31, 2017.

On May 9, 2018 Array BioPharma Inc., (Nasdaq: ARRY) reported results for its third quarter of fiscal 2018 and provided an update on the progress of its key clinical development programs (Press release, Array BioPharma, MAY 9, 2018, View Source [SID1234526313]).

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"Preparations for the anticipated U.S. launch of encorafenib and binimetinib in BRAF-mutant melanoma are well underway," said Ron Squarer, Chief Executive Officer. "We are pleased to have our entire commercial leadership and infrastructure in place and are poised for an exciting 2018, as we look ahead to commercialization and additional data updates from our encorafenib and binimetinib clinical trials."

COLUMBUS PHASE 3 TRIAL
Regulatory
Array’s New Drug Applications (NDAs) to support use of the encorafenib and binimetinib combination for the treatment of patients with BRAF-mutant advanced, unresectable or metastatic melanoma remain under review by the FDA with a target action date under Prescription Drug User Fee Act (PDUFA) of June 30, 2018.

The European Medicines Agency (EMA), as well as the Swiss Medicines Agency (Swissmedic) and the Australian Therapeutic Goods Administration (TGA), are reviewing the Marketing Authorization Applications (MAAs) submitted by Pierre Fabre and Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) has accepted the Manufacturing and Marketing Approval (MMA) applications submitted by Ono Pharmaceutical Co, Ltd.

The regulatory submissions were based on findings from the pivotal Phase 3 COLUMBUS trial.

COLUMBUS Median Overall Survival Results
Array will announce additional results from the Phase 3 COLUMBUS trial in an oral presentation (Abstract #223875) at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2018 Annual Meeting on June 4.

Array previously announced that treatment with the combination of encorafenib 450 mg daily and binimetinib 45 mg twice daily (COMBO450) reduced the risk of death compared to treatment with vemurafenib 960 mg daily [hazard ratio (HR) of 0.61, (95% CI 0.47, 0.79, p<0.001)] in patients with BRAF-mutant melanoma in the Phase 3 COLUMBUS trial.

•
The Phase 3 trial showed mOS of 33.6 months for patients treated with COMBO450, compared to 16.9 months for patients treated with vemurafenib as a monotherapy.

Exhibit 99.1

•
As previously reported, the combination of encorafenib and binimetinib was generally well-tolerated. Grade 3/4 adverse events (AEs) that occurred in more than 5% of patients receiving the combination were increased gamma-glutamyltransferase (GGT) (9%), increased blood creatine phosphokinase (CK) (7%) and hypertension (6%). The incidence of selected any grade AEs of special interest, defined based on toxicities commonly associated with commercially available BRAF+MEK-inhibitor treatments for patients receiving the combination of encorafenib and binimetinib included: rash (22%), pyrexia (18%), serous retinopathy including retinal pigment epithelial detachment (20%) and photosensitivity (5%). Full safety results of COLUMBUS Part 1 were published in The Lancet Oncology.

The Lancet Oncology Publication
Detailed results of the pivotal Phase 3 COLUMBUS trial for the treatment of patients with BRAF-mutant advanced, unresectable or metastatic melanoma were published online on March 21, 2018 and in the May 2018 print edition of The Lancet Oncology.

BEACON CRC PHASE 3 TRIAL
Array will present updated results from the 30 patient safety lead-in of the Phase 3 BEACON CRC trial at the ESMO (Free ESMO Whitepaper) 20th World Congress on Gastrointestinal Cancer June 20-23, 2018.

Array will present updated results from the 30 patient safety lead-in of the Phase 3 BEACON CRC trial evaluating the triplet combination of encorafenib, binimetinib and cetuximab, an EGFR antagonist, in patients with BRAF-mutant CRC whose disease has progressed after one or two prior regimens at the ASCO (Free ASCO Whitepaper) 2018 Gastrointestinal Cancers Symposium.

•
The estimated mPFS at the time of analysis was 8 months in 29 patients with BRAFV600E-mutant CRC.
•
The confirmed overall response rate (ORR) was 48% with 3 complete responses in patients with BRAFV600E-mutant CRC. Further, the ORR was 62% in the 16 patients who received only one prior line of therapy.
•
These data represent improvements compared to several approved standard of care benchmarks for this population which range between 4% to 8% ORR and 1.8 and 2.5 months mPFS. [1-4]
•
The triplet combination was generally well-tolerated. Two patients discontinued treatment due to AEs with only one of these considered related to treatment. The most common grade 3 or 4 AEs seen in at least 10% of patients were fatigue, urinary tract infection, increased aspartate aminotransferase (AST) and increased blood CK.
•
Enrollment in the randomized portion of BEACON CRC is ongoing. BRAF mutations are estimated to occur in 10% to 15% of patients with CRC and represent a poor prognosis for these patients.

Encorafenib and binimetinib are investigational medicines and are not currently approved in any country.

IMMUNO-ONCOLOGY COLLABORATIONS: TRIALS ADVANCING WITH BRISTOL-MYERS SQUIBB AND MERCK; TRIAL WITH PFIZER EXPECTED TO START THIRD QUARTER OF 2018
Array is developing binimetinib in combination with PD-1/ PD-L1 checkpoint inhibitors and has announced separate, strategic collaborations with Bristol-Myers Squibb, Merck and Pfizer, but in each case, are pursuing a unique trial design to explore different clinical approaches.

Bristol-Myers Squibb
•
The clinical trial continues to advance and is designed to investigate the safety, tolerability and efficacy of binimetinib in combination with nivolumab (anti-PD-1 therapy), with and without ipilimumab (CTLA-4 antibody), in patients with advanced metastatic microsatellite stable (MSS) CRC and the presence of a RAS mutation who have received one or two prior regimens.

The trial is jointly supported by Array and Bristol-Myers Squibb and sponsored by Array.

Merck

The clinical trial continues to advance and is designed to investigate the safety, tolerability and efficacy of binimetinib in combination with pembrolizumab (anti-PD-1 therapy), with and without FOLFOX or FOLFIRI (chemotherapy) in patients with CRC whose tumors are not microsatellite instability-high (MSI-H).

The trial is sponsored and funded by Merck, with Array providing binimetinib supply.

Pfizer

The clinical trial is designed to investigate the safety, tolerability and efficacy of several novel anti-cancer combinations, including binimetinib, avelumab (anti-PD-L1 therapy) and talazoparib (PARP inhibitor) across various tumor types and is expected to begin during the third quarter of 2018.

Exhibit 99.1

Initially, the focus will be in non-small cell lung cancer (NSCLC) and pancreatic cancer, with additional indications being explored at a later stage.

The trial will be sponsored and funded by Pfizer, with Array providing binimetinib supply.

FINANCIAL HIGHLIGHTS
Novartis Financial Commitment
Novartis continues to substantially fund all ongoing trials with encorafenib and binimetinib that were active or planned as of the close of the Novartis Agreements in 2015, including the COLUMBUS Phase 3 trial. Reimbursement revenue from Novartis was approximately $87 million for the 12 months ended March 31, 2018, of which $24.8 million was recorded in the quarter ended March 31, 2018. Total revenue and upfront payment collected from Novartis since the start of the 2015 agreement is $373.5 million.

Third Quarter of Fiscal 2018 Compared to Second Quarter of Fiscal 2018 (Sequential Quarters Comparison)
· Revenue for the third quarter of fiscal 2018 was $66.4 million, compared to $42.2 million for the prior quarter. The increase was primarily due to an upfront license fee from ASLAN Pharmaceuticals as well as higher Novartis reimbursement revenue.
· Cost of partnered programs for the third quarter of fiscal 2018 was $17.7 million, compared to $13.7 million for the prior quarter. The increase was primarily due to higher costs incurred for the BEACON CRC trial as it continues to advance, as well as additional resources engaged on collaborations.
· Research and development expense for proprietary programs was $53.6 million, compared to $42.6 million in the prior quarter. The increase was driven by higher activity on the Novartis transitioned studies and pre-commercial manufacturing costs for encorafenib and binimetinib.
· Loss from Operations for the quarter was $21.8 million, compared to a loss from operations of $25.7 million in the previous quarter. The decrease in net loss was primarily due to increased revenue, which was partially offset by increased research and development expense.
· Net loss for the second quarter was $22.9 million, or ($0.11) per share, compared to $34.1 million, or ($0.17) per share, in the prior quarter.
· Cash, Cash Equivalents and Marketable Securities as of March 31, 2018 were $440 million.

Third Quarter of Fiscal 2018 Compared to Third Quarter of Fiscal 2017 (Prior Year Comparison)
· Revenue for the third quarter of fiscal 2018 increased by $33.1 million compared to the same quarter of fiscal 2017. The increase was primarily due to the ASLAN Pharmaceuticals upfront license fee.
· Cost of partnered programs increased $10.3 million compared to the third quarter of fiscal 2017. The increase was primarily due to higher costs incurred for the BEACON CRC trial, as well as more resources engaged on collaborations.
· Research and development expense for proprietary programs increased $7.6 million, compared to the third quarter of fiscal 2017. The increase was driven by higher activity on the Novartis transitioned studies and pre-commercial manufacturing costs for encorafenib and binimetinib.
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Net loss for the third quarter of fiscal 2018 was $22.9 million, or ($0.11) per share, compared to $35.3 million, or ($0.21) per share, for the same quarter in fiscal 2017. The decrease in net loss was primarily due to increased revenue, which was partially offset by increased research and development expense.

CONFERENCE CALL INFORMATION
Array will hold a conference call on Wednesday, May 9, 2018 at 9:00 a.m. Eastern Time to discuss these results and provide an update on the progress of its key clinical development programs. Ron Squarer, Chief Executive Officer, will lead the call.

Date: Wednesday, May 9, 2018
Time: 9:00 a.m. Eastern Time
Toll-Free: (844) 464-3927
Toll: (765) 507-2598
Pass Code: 6465079

Webcast, including Replay and Conference Call Slides:
View Source

On May 9, 2018 Cellectar Biosciences (Nasdaq: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported that the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development has granted Orphan Drug Designation to CLR 131, the company’s lead Phospholipid Drug Conjugate (PDC) product candidate, for the treatment of rhabdomyosarcoma, a rare pediatric cancer (Press release, Cellectar Biosciences, MAY 9, 2018, View Source [SID1234526363]).

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"Rhabdomyosarcoma is the most common type of tissue sarcoma in children. While initial response to treatment is generally favorable, there is an important need for new treatments, especially in children who experience relapse." said John Friend, M.D., chief medical officer of Cellectar. "Cellectar is committed to working closely with the FDA to fully evaluate the potential for targeted delivery of CLR 131 to address this currently unmet medical need."

Orphan drug designation provides seven-year market exclusivity, increased engagement and assistance from the FDA, tax credits for certain research, research grants and a waiver of the New Drug Application user fee. Rhabdomyosarcoma is recognized by the FDA as an orphan disease, usually defined as a condition that affects fewer than 200,000 people nationwide.

About Rhabdomyosarcoma

Rhabdomyosarcoma (RMS), a malignant tumor of mesenchymal origin, is the most common soft tissue sarcoma in children, accounting for approximately 40% of childhood soft tissue sarcomas in the United States. The incidence is about 4.5 cases per 1 million per year in children younger than 15 years and more than 50% are younger than 10 years at diagnosis. Approximately 340 new cases are diagnosed each year in North America and the prognosis is favorable with a 64% 5-year survival in children aged birth to 19 years [Ward 2014]. At least one-third of all patients will experience disease progression or relapse, and 95% of all failures occur within 3 years. The median progression free survival following the first recurrence or progression is approximately nine months.

About CLR 131

CLR 131 is Cellectar’s investigational radioiodinated PDC therapy that exploits the tumor-targeting properties of the company’s proprietary phospholipid ether (PLE) and PLE analogs to selectively deliver radiation to malignant tumor cells, thus minimizing radiation exposure to normal tissues. CLR 131, is in a Phase 2 clinical study in relapsed or refractory (R/R) MM and a range of B-cell malignancies and a Phase 1 clinical study in patients with (R/R) MM exploring fractionated dosing. In 2018 the company plans to initiate a Phase 1 study with CLR 131 in pediatric solid tumors and lymphoma, and a second Phase 1 study in combination with external beam radiation for head and neck cancer.