On March 14, 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 fourth quarter and full year ended December 31, 2017 and provided a corporate update (Press release, BioSpecifics Technologies, MAR 14, 2018, View Source [SID1234524755]).

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"2018 is off to a solid start with the Phase 3 cellulite trials for CCH now initiated and positive interim data recently presented for our ongoing Phase 1 uterine fibroids trial, which is advancing toward a top-line data readout this year. In 2017, we continued to be pleased with our royalty revenues from XIAFLEX net sales and look forward to the continued growth of this product and the ongoing profitability of our business," said Thomas L. Wegman, President of BioSpecifics. "In 2018, we will also continue to work with our partner Endo to ensure that XIAFLEX and CCH are able to fulfill their commercial and clinical potential."

Fourth Quarter and Full Year 2017 Financial Results

BioSpecifics reported net income of $2.6 million for the fourth quarter ended December 31, 2017, or $0.37 per basic share and $0.36 per share on a fully diluted basis, compared to net income of $2.9 million, or $0.41 per basic share and $0.40 per share on a fully diluted basis, for the same period in 2016. For the full year ended December 31, 2017, the Company reported a net income of $11.3 million, or $1.58 per basic share and $1.55 per share on a fully diluted basis, compared to net income of $11.4 million, or $1.61 per basic share and $1.56 per share on a fully diluted basis for the same period in 2016.

Total revenue for the fourth quarter ended December 31, 2017 was $6.7 million, compared to $6.6 million for the same period in 2016. For the full year ended December 31, 2017, total revenue was $27.4 million, compared to $26.3 million for the same period in 2016. The increase in total revenues for the quarterly and year-end periods was primarily due to royalty revenues related to the net sales of XIAFLEX.

Royalty and mark-up on cost of goods sold (COGS) revenues recognized under BioSpecifics’ agreement with Endo for the fourth quarter ended December 31, 2017 were $6.7 million compared to $6.6 million for the same period in 2016, an increase of $0.1 million or 2 percent. Total royalty and mark-up on COGS revenues for the year ended December 31, 2017 increased to $27.4 million as compared to $25.4 million in the same period in 2016. This increase in royalties and the mark-up on COGS in each quarterly and year end periods was primarily due to royalty revenues related to the net sales of XIAFLEX.

Licensing revenue consists of licensing fees, sublicensing fees and milestones. BioSpecifics recognized licensing revenue for the fourth quarter ended December 31, 2017 and 2016 of approximately $4,000 in each period. Licensing fees recognized for the years ended December 31, 2017 and 2016 were $18,000 and $0.8 million, respectively. In the 2017 period, licensing fees recognized were related to the cash payments received under the agreement with Endo in prior years and amortized over the expected development period. In the 2016 period, licensing fees of $0.8 million were recognized related to the exercise of an opt-in right by Endo for the human lipoma indication.

Milestone revenue recognized for the years ended December 31, 2017 and 2016 was zero and $28,500, respectively. The $28,500 milestone revenue recognized in the 2016 period related to the approval of XIAFLEX in Australia for the treatment of Peyronie’s Disease by Actelion.

Research and development (R&D) expenses for the fourth quarter ended December 31, 2017 and 2016 were $0.3 million in each period. For the year ended December 31, 2017, R&D expenses were $1.2 million, compared to $1.3 million in the same period in 2016.

General and administrative expenses for the fourth quarter ended December 31, 2017 were $1.6 million, compared to $2.0 million for the same period in 2016. For the year ended, December 31, 2017, general and administrative expenses were $8.5 million, compared to $7.9 million in the same period in 2016.

Provision for income taxes for the fourth quarter ended December 31, 2017 were $2.4 million, compared to $1.5 million for the same period in 2016. For the year ended December 31, 2017, provision for income taxes were $7.0 million as compared to $6.0 million in the same period of 2016. The increase in 2017 as compared to 2016 was primarily due to the Company’s initial analysis of the Tax Cuts and Job Act. The Company has made reasonable estimates of its 2017 impact and due to the federal corporate rate reduction, a re-measurement of deferred tax assets and liabilities resulted in the recording of a charge of approximately $1.1 million.

As of December 31, 2017, BioSpecifics had cash and cash equivalents and investments of $65.1 million, compared to $52.8 million as of December 31, 2016.

XIAFLEX Commercial Highlights

On February 27, 2018, Endo reported commercial highlights for XIAFLEX for the fourth quarter and full year of 2017 (Endo’s fourth quarter 2017 financials are reported in BioSpecifics’ first quarter 2018 financials). For the fourth quarter of 2017, net revenues were $61.3 million compared to $55.5 million for the fourth quarter of 2016, a 10 percent increase, primarily driven by a full year of direct-to-consumer initiatives intended to increase patient awareness. Full year 2017 net revenues were $213.4 million compared to $189.7 million for the full year of 2016, a 12 percent increase.

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.

BioSpecifics presented interim data from the ongoing Phase 1 study of CCH for the treatment of uterine fibroids in a poster on March 9, 2018 at the Society for Reproductive Investigation’s 65th Annual Scientific Meeting in San Diego, CA. The data show the safety and effectiveness of the injection method in five patients. Three patients were injected with CCH and underwent a hysterectomy 24-96 hours after the injection. Two patients subsequently were injected with a higher dose of collagenase, and underwent a hysterectomy 63 days after the injection. The collagenase-treated tissue samples showed not only the reduction of collagen content but also the disruption of the tissue pattern, while in control tissues the collagen remained abundant and compact. The digestion of collagen did not extend beyond the capsule of any fibroid. No adverse event occurred in these patients.
In February 2018, Endo announced the initiation of two pivotal Phase 3 RELEASE clinical trials of CCH for the treatment of cellulite. These multicenter, randomized, double-blind, placebo-controlled studies will evaluate the safety and efficacy of CCH in reducing the appearance of cellulite. These two U.S. studies are expected to enroll 840 women, 420 per trial, aged 18 years or older with moderate-to-severe buttock cellulite. Each subject will receive up to three treatments in which CCH, 0.84 mg per treatment area, or placebo will be administered. The two treatment areas include the left and right buttock. Each treatment visit will occur approximately 21 days apart. A total of 12 injections will be administered into cellulite dimples per treatment area per visit. Cellulite severity will be assessed at the beginning and conclusion of treatment. The assessments will be conducted by each patient and clinician using two validated photonumeric cellulite severity scales developed by Endo and third-party psychometric experts. The primary endpoint of the studies is a composite responder analysis demonstrating at least a 2-level composite improvement on the photonumeric cellulite severity scale, independently reported by both the patient and clinician. Key secondary endpoints include the percentage of subjects that experience at least a 1-level or 2-level improvement in patient reported assessment, percentage of subjects with a 1-level composite improvement, percentage of satisfied subjects, change from baseline in a cellulite impact scale and the percentage of subjects with at least a 1-level or 2-level improvement in the global aesthetic improvement scale (GAIS).
Top-line results for the Phase 3 trials of CCH for cellulite 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 in 2018. 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’s primary endpoint 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.
Endo continues its commercial review of additional licensed indications, including adhesive capsulitis (frozen shoulder) and plantar fibromatosis.

On March 14, 2018 Bristol-Myers Squibb Company (NYSE:BMY) reported that it will announce results for the first quarter of 2018 on Thursday, April 26, 2018. During a conference call at 10:30 a.m. EDT on April 26, company executives will review financial information and will address inquiries from investors and analysts (Press release, Bristol-Myers Squibb, MAR 14, 2018, View Source [SID1234524756]).

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Investors and the general public are invited to listen to a live webcast of the call at View Source or by dialing in the U.S. toll free 323-794-2093 or international 866-548-4713, confirmation code: 4713257. Materials related to the call will be available at the same website prior to the conference call. A replay of the call will be available beginning at 1:30 p.m. EDT on April 26 through 1:30 p.m. EDT on, May 10, 2018. The replay will also be available through View Source or by dialing in the U.S. toll free 888-203-1112 or international 719-457-0820, confirmation code: 4713257.

On March 14, 2018 Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE:CFBI), a biotechnology company advancing a pipeline of proprietary small-molecule drugs that address cancer, liver disease and inflammatory diseases, reported that Dr. Pnina Fishman, Can-Fite’s Chief Executive Officer, will present at the 2nd Annual H.C. Wainwright NASH Investor Conference on March 19 at 5:20 p.m. ET in New York City (Press release, Can-Fite BioPharma, MAR 14, 2018, View Source [SID1234524757]). The presentation will focus on its drug candidate Namodenoson (CF102), A3 adenosine receptor (A3AR), currently in a Phase II trial for the treatment of NAFLD, and non-alcoholic steatohepatitis (NASH).

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Recent Positive Safety Data on Namodenoson

Can-Fite’s current Phase II study is being conducted in three Israeli sites, including Hadassah Medical Center, Jerusalem, and the Rabin Medical Center, Petach Tikva. Patients who suffer from NAFLD/NASH with evidence of active inflammation are treated twice daily with 12.5 or 25 mg of oral Namodenoson vs. placebo. The primary end point of the Phase II study is the anti-inflammatory effect of the drug, as determined by ALT blood levels, and the secondary end points include percentage of liver fat, as measured by MRI-PDFF (proton density fat fraction). The Company anticipates the completion of patient enrollment toward the end of 2018 and data release in the first half of 2019.

Recent safety data showed that Namodenoson has a favorable safety profile and lack of hepatotoxicity in patients. Preclinical data demonstrate robust anti-inflammatory, anti-fibrogenic and anti-steatotic effects, supporting its development for the NAFLD/NASH indication.

On March 14, 2018 CBT Pharmaceuticals (CBT), a U.S. and China-based innovative biopharmaceutical company committed to becoming a leader in the discovery and development of oncology combination therapies, reported that the company reported that it will present at two upcoming investor conferences (Press release, CBT Pharmaceuticals, MAR 14, 2018, View Source [SID1234524758]):

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March 23, 2018 – Future Leaders in the Biotech Industry at 2:45 – 3:00 p.m. EST in New York, N.Y.
March 28, 2018 – China Healthcare Investment Conference (CHIC) at 3:15 – 5:15 p.m. CST in Shanghai, P.R. China

On March 14, 2018 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR technology, reported financial results and operational progress for the fourth quarter and full year of 2017 (Press release, Intellia Therapeutics, MAR 14, 2018, View Source [SID1234524759]).

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Using our proprietary lipid nanoparticle (LNP) delivery system, Intellia has achieved near-complete knockout editing of the transthyretin amyloidosis target gene (TTR) in rodents and most recently shared data on the progress of non-human primate (NHP) studies. In the fourth quarter, our NHP studies continued, yielding higher levels of editing as the Company improved the LNP formulation and dosing regimens, including repeated doses. Ongoing efforts in the NHP studies focus on consistently achieving a therapeutically relevant level of editing for the TTR gene as determined by transthyretin level reductions and we continue to observe and, as in prior animal studies, gather long-term durability data.

In parallel with the continuous progress on ATTR, during the fourth quarter, the Company expanded its research in the liver in two ways. First, in collaboration with partner Regeneron Pharmaceuticals, Inc. (Regeneron), we successfully inserted a functional gene into a specific site in the mouse genome, achieving therapeutically relevant levels of gene expression and demonstrating proficiency in executing complex editing procedures. These results provide a proof-of-principle for a large class of genetic defects that cannot be addressed solely by gene knockout. Second, knockout editing in livers of mice was successful for a second therapeutic target, the SERPINA1 gene that gives rise to liver complications in certain alpha-1 antitrypsin deficiency patients. The Company plans to present these data at upcoming scientific conferences and extend our evaluation in additional in vivo studies.

Through the ongoing collaboration with Novartis Institutes for BioMedical Research, Inc. (Novartis), the Company made further progress on complex ex vivo editing. Data presented at the 2017 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition showed that an Intellia-qualified gRNA targeting the erythroid specific enhancer region of the BCL11A gene in human CD34+ cells could edit greater than 80 percent of these healthy bone marrow-derived cells, which led to the majority of the cells expressing fetal hemoglobin. These edited hematopoietic stem cells maintained engraftment over a 16-week period and demonstrated multi-lineage reconstitution, retaining their ability to complete normal hematopoiesis (blood cell formation) after the gene edit.

"2017 marked significant progress for Intellia with the CRISPR/Cas9 technology and our lead ATTR program. We demonstrated editing with our proprietary lipid nanoparticle delivery system across multiple animal species, including NHPs, and advanced this program towards clinical studies. After raising an additional $141 million, we are advancing the Company’s emerging pipeline of indications in the liver, including transthyretin amyloidosis and alpha-1 antitrypsin deficiency, expanding our in vivo editing work beyond the liver into CNS, and accelerating our ex vivo cellular therapy programs," said President and Chief Executive Officer, John Leonard, M.D., Intellia Therapeutics. "As we begin 2018, our team remains focused on the patient and making genome editing-based therapies a reality. We are well-positioned to deliver against our goals and mission."

The Company achieved several key milestones during 2017, including:

Produced interim, top-line NHP data demonstrating, for the first time, liver genome editing using CRISPR/Cas9 delivered through a LNP system;
Confirmed re-dosing in NHPs produced increased levels of editing, where the treatment/drug and regimen was well tolerated as assessed by clinical signs and chemistry;
Presented 12-month data from a long-term mouse study, demonstrating robust and durable in vivo genome editing following a single, systemic intravenous delivery of Intellia’s proprietary, non-viral, LNP delivery system;
Evaluated in vivo delivery by LNPs to a second organ, with successful genome editing by CRISPR/Cas9 in the central nervous system (cerebellum and striatum) in mice;
In collaboration with Regeneron, produced positive insertion editing data in mice, demonstrating capability to perform complex genetic editing;
In collaboration with Novartis, generated positive data in sickle cell anemia in transplant mouse models; and
Continued to enhance and defend the Company’s CRISPR/Cas9 foundational and therapeutic intellectual property position, which included filing multiple patent applications covering our inventions and the issuance in Europe, the United Kingdom, Australia and China of foundational CRISPR/Cas9 patents for which we have in-licensed rights.

Upcoming Goals

The Company has set forth the following for 2018 pipeline progression:

Mid-year: begin IND-enabling activities for lead ATTR development candidate
Mid-year: present additional editing data following in vivo delivery by LNPs to organs beyond the liver
Late 2018: advance lead development candidate for second indication
During 2018: present additional insertion/repair editing data; and
During 2018: present preclinical data in support of our first proprietary ex vivo programs on immuno-oncology and autoimmune/inflammation indications.

Fourth Quarter and Full Year 2017 Financial Results

Collaboration Revenue

Collaboration revenue was $6.7 million for the fourth quarter of 2017, compared to $5.6 million for the fourth quarter of the prior year. The increase in collaboration revenue in 2017 was primarily driven by amounts recognized under Intellia’s collaboration agreement with Regeneron which was entered in April 2016.

Since inception through December 31, 2017, the Company has received $106.1 million in funding from the collaborations with Novartis and Regeneron, excluding amounts received for equity investments, and had an accounts receivable balance of $10.5 million at December 31, 2017. Excluding the $2.6 million of the upfront payment received from Novartis, which was allocated to the purchase of the Company’s equity securities, Intellia has recognized $48.6 million in collaboration revenue under these agreements from inception through December 31, 2017, and had a remaining deferred revenue balance of $65.3 million at December 31, 2017.

Operating Expenses

Research and Development expenses increased by $9.8 million to $21.2 million during the fourth quarter of 2017, compared to $11.3 million during the same period of 2016. This increase was driven primarily by the advancement of Intellia’s research programs, research personnel growth to support these programs, as well as the expansion of the development organization, and includes laboratory supplies, research materials and certain equipment. Additionally, salary and related headcount-based expenses increased, as the Company grew to 143 research and development personnel as of December 31, 2017, from 74 research and development employees as of December 31, 2016.

General and administrative expenses increased by $5.1 million to $10.2 million during the fourth quarter of this year, compared to $5.1 million in the fourth quarter of 2016. This increase was driven primarily by increased salary and related headcount-based expenses as the Company grew to 41 general and administrative employees as of December 31, 2017, from 29 general and administrative employees as of December 31, 2016, to support Intellia’s larger research and development organization, public company compliance and administrative obligations. The Company also incurred increased corporate insurance, legal, and other professional expenses related to our expanding operations since becoming a public company in May 2016.

The Company’s net loss was $24.0 million for the fourth quarter of 2017, compared to $10.6 million for the fourth quarter of 2016.

Balance Sheet

Cash and cash equivalents at December 31, 2017, were $340.7 million, compared to $273.1 million at December 31, 2016. The base period cash and cash equivalents were primarily attributable to $115.5 million in proceeds from the Company’s initial public offering and $55.0 million in concurrent private placements in May 2016, in addition to a $75.0 million upfront payment from Regeneron in April 2016 and a follow-on public offering of $141.0 million in November 2017.

Financial Guidance

The Company’s primary uses of capital will continue to be research and development programs, laboratory and related supplies, compensation costs for current and future employees, consulting, legal and other regulatory expenses, patent prosecution filing and maintenance costs for Intellia’s licensed intellectual property, and general overhead costs.

As of December 31, 2017, the Company had an accumulated deficit of $121.1 million. The Company expects losses to increase as it continues to incur significant research and development expenses related to the advancement of Intellia’s therapeutic programs and ongoing operations. Based on Intellia’s research and development plans and expectations related to the progress with the Company’s programs, the Company expects that the cash and cash equivalents as of December 31, 2017, as well as technology access and research funding from Novartis and Regeneron, will enable Intellia to fund operating expenses and capital expenditures through mid-2020, excluding any potential milestone payments or extension fees that could be earned and distributed under the collaboration agreements with Novartis and Regeneron or any strategic use of capital not currently in the base case planning assumptions.