On November 3, 2016 Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical company focused on the unmet needs of patients with rare diseases, reported financial results for the quarter ended September 30, 2016 and provided a business update (Press release, Insmed, NOV 3, 2016, View Source [SID1234516280]).

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Business Update

Achieved enrollment objective in global phase 3 study of ARIKAYCE. Today the company announced that it has achieved its patient enrollment objective in the phase 3 study of ARIKAYCE (liposomal amikacin for inhalation). The study, which is known as CONVERT or INS-212, is evaluating ARIKAYCE in treatment refractory nontuberculous mycobacteria (NTM) lung disease caused by Mycobacterium avium complex (MAC). The primary efficacy endpoint is the proportion of subjects who achieve culture conversion at Month 6 in the ARIKAYCE plus multi-drug regimen arm compared to the multi-drug regimen without ARIKAYCE arm.
Published phase 2 study of ARIKAYCE. In October 2016, the American Journal of Respiratory and Critical Care Medicine published the company’s phase 2 study of ARIKAYCE in NTM lung disease. The manuscript, which is entitled "Randomized Trial of Liposomal Amikacin for Inhalation in Nontuberculous Mycobacterial Lung Disease" by Olivier et al. is accessible online at View Source
Presented new analyses of phase 2 study of ARIKAYCE at CHEST. A poster describing the stability and consistency of effect with ARIKAYCE treatment in the phase 2 study was recently presented at the CHEST annual meeting.
Presented new NTM disease burden data at ISPOR European Congress. Three posters describing the burden of NTM lung disease were recently presented at the International Society for Pharmacoeconomic and Outcomes Research (ISPOR) Annual European Congress.
Secured global exclusive rights to novel oral inhibitor of dipeptidyl peptidase I.
In October, the company acquired the global exclusive rights to INS1007 (formerly known as AZD7986) from AstraZeneca. INS1007 is a small molecule, reversible inhibitor of dipeptidyl peptidase I (DPP1), an enzyme responsible for activating neutrophil serine proteases (NSPs) in neutrophils when they are formed in the bone marrow. In chronic inflammatory lung diseases, neutrophils accumulate in the airways and result in excessive active NSPs that cause lung destruction and inflammation. The company expects to begin a phase 2 dose-ranging study of INS1007 in non-cystic fibrosis (non-CF) bronchiectasis in 2017. Non-CF bronchiectasis is a rare, progressive, neutrophil-driven pulmonary disorder with no approved therapies.
Presented phase 1 study of INS1009 at ERS 2016. In September, results from a phase 1 study of INS1009 were presented at the European Respiratory Society International Congress. The study was a randomized, double-blind, placebo-controlled single ascending dose study of INS1009 to determine its safety, tolerability, and pharmacokinetics in healthy volunteers. The pharmacokinetic characteristics supported once- or twice-daily whereas existing inhaled therapies are dosed four to nine times per day. The adverse event profile was consistent with other inhaled prostanoids. INS1009 is the company’s inhaled treprostinil prodrug, which may offer therapeutic potential in rare pulmonary disorders.
Appointed chief commercial officer. The company enhanced its senior leadership team with the appointment of Roger Adsett as chief commercial officer. Prior to joining Insmed, Mr. Adsett was senior vice president, head of the gastrointestinal and internal medicine business unit at Shire plc. In that capacity he oversaw Shire’s global commercial P&L across six specialty and two rare disease brands. Before joining Shire, Mr. Adsett worked at AstraZeneca for 11 years. Mr. Adsett will be charged with overseeing the development and execution of Insmed’s global commercial strategy.
"Recent months have been marked by strong progress across all aspects of our business," said Will Lewis, president and chief executive officer of Insmed. "We are pleased to report that the enrollment phase of the CONVERT study is now complete, which positions us for top-line data next year. There is a significant need for new treatments for patients with refractory NTM lung disease and we look forward to advancing the development of ARIKAYCE. With respect to our earlier-stage pipeline, we are encouraged by the high-level of inbound physician interest in our clinical program for INS1007 in non-CF bronchiectasis. Physicians are eagerly awaiting new treatment options for this debilitating disorder and we believe INS1007 has the potential to achieve disease modification by impeding tissue destruction, inflammation, and mucus hypersecretion through the inhibition of DPP1."

Third Quarter Financial Results

For the third quarter of 2016, Insmed posted a net loss of $37.8 million, or $0.61 per share, compared with a net loss of $31.0 million, or $0.50 per share, for the third quarter of 2015.

Research and development expenses were $23.4 million for the third quarter of 2016, compared with $19.2 million for the third quarter of 2015. The increase was primarily due to the advancement of the company’s global phase 3 CONVERT study of ARIKAYCE in NTM lung disease, as well as an increase in headcount and related expenses. These increases were partially offset by a decrease in manufacturing expenses primarily due to the completion of the build-out of additional production capacity at a contract manufacturer in 2015.

General and administrative expenses for the third quarter of 2016 were $13.7 million, compared with $11.0 million for the third quarter of 2015. The increase was primarily related to pre-commercial activities, namely the buildout of the company’s infrastructure and NTM disease awareness activities.

Balance Sheet Highlights and Cash Guidance

As of September 30, 2016, Insmed had cash and cash equivalents of $201 million. Excluding depreciation and stock-based compensation expense, the company’s cash operating expenses for the nine months ended September 30, 2016 were $91 million. Insmed ended the third quarter of 2016 with $35 million in debt and $183 million of working capital.

On September 30, 2016, Insmed closed a $55 million debt agreement with Hercules Capital, Inc. The transaction refinanced the company’s existing debt of $25 million and added a total of $30 million of new debt, $20 million of which was funded in early October in connection with the upfront payment for the global exclusive rights to INS1007.

The company is investing in the following activities in 2016: (i) clinical development of ARIKAYCE, (ii) regulatory and pre-commercial initiatives for ARIKAYCE, and (iii) preclinical and clinical activities for its earlier-stage pipeline. Insmed continues to expect its cash-based operating expenses for the second half of 2016 to be in the range of $62 to $72 million.

On November 3, 2016 Oncolytics Biotech Inc. (TSX: ONC) (OTCQX: ONCYF) (FRA: ONY) ("Oncolytics" or the "Company") reported its financial results and operational highlights for the third quarter ended September 30, 2016 (Filing, Q3, Oncolytics Biotech, 2016, NOV 3, 2016, View Source [SID1234516308]).

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"During the quarter we reported additional statistically significant, randomized clinical data that further strengthened the linkage between genetic status and survival outcomes in patients with adenocarcinoma of the lung," said Dr. Matt Coffey, Interim President and CEO of Oncolytics Biotech. "In parallel with reporting data from sponsored Phase 2 randomized studies, we continued to push ahead with preparations for a registration study."

Selected Highlights

Since July 1, 2016, selected highlights announced by the Company include:

Clinical Program

· Additional data from a randomized, sponsored Phase II clinical study of REOLYSIN in non-small cell lung cancer (IND 211), which showed that:
· progression free survival ("PFS") was statistically significantly better for female patients in the test arm (n=20) than for those in the control arm (n=16) in patients with adenocarcinoma. Median PFS was 5.39 months compared with 3.02 months, respectively (p=0.0201);
· evolving overall survival ("OS") demonstrated a strong trend towards survival benefit for female patients in the test arm with adenocarcinoma (n=20, six of whom remained alive at the time of the analysis) over those in the control arm (n=16, three of whom remained alive at the time of the analysis). Median OS was 10.68 months compared with 7.59 months, respectively (p=0.145) (Figure B). By contrast, no PFS or OS benefit was noted for the male patients with adenocarcinoma; and
· patients with adenocarcinoma treated with REOLYSIN with one or more target biomarkers (EGFR, Hras, Kras, Nras, Braf and/or p53 mutations) had a greater PFS (p=0.039) and OS (p=0.031) than patients treated with REOLYSIN without any of these biomarkers. The presence of these biomarkers may account, at least in part, for the difference between the survival outcomes for male and female patients; target biomarkers were present in a higher proportion of the female patients in the study than the male patients (66.7% versus 43.6%). As a result, pre-screening for target biomarkers in patients with adenocarcinoma of the lung is warranted.
Financial
At September 30, 2016, the Company reported $17.7 million in cash, cash equivalents and short-term investments. At November 2, 2016, the Company had approximately $16.7 million in cash, cash equivalents and short-term investments.

ONCOLYTICS BIOTECH INC.
INTERIM CONSOLIDATED STATEMENTS OF FINANCIAL POSITION
(unaudited)

September 30,
2016
$ December 31,
2015
$
Assets
Current assets
Cash and cash equivalents 15,612,729 24,016,275
Short-term investments 2,088,800 2,060,977
Accounts receivable 44,991 340,059
Prepaid expenses 356,741 506,669
Total current assets 18,103,261 26,923,980
Non-current assets
Property and equipment 333,926 459,818
Total non-current assets 333,926 459,818

Total assets 18,437,187 27,383,798

Liabilities And Shareholders’ Equity
Current Liabilities
Accounts payable and accrued liabilities 2,809,008 2,709,492
Total current liabilities 2,809,008 2,709,492

Shareholders’ equity
Share capital
Authorized: unlimited
Issued:
September 30, 2016 – 120,873,222
December 31, 2015 – 118,151,622 262,218,228 261,324,692
Contributed surplus 26,536,601 26,277,966
Accumulated other comprehensive income 492,637 760,978
Accumulated deficit (273,619,287) (263,689,330)
Total shareholders’ equity 15,628,179 24,674,306
Total liabilities and equity 18,437,187 27,383,798

ONCOLYTICS BIOTECH INC.
INTERIM CONSOLIDATED STATEMENTS OF LOSS AND COMPREHENSIVE LOSS
(unaudited)

Three
Month
Period
Ending
September
30, 2016
$ Three
Month
Period
Ending
September
30, 2015
$ Nine Month
Period
Ending
September
30, 2016
$ Nine Month
Period
Ending
September 30,
2015
$
Expenses
Research and development 2,141,737 1,704,784 6,358,822 6,601,877
Operating 1,222,447 1,176,023 3,708,317 3,780,812
Operating loss (3,364,184) (2,880,807) (10,067,139) (10,382,689)
Interest 31,691 52,756 136,849 153,313
Loss before income taxes (3,332,493) (2,828,051) (9,930,290) (10,229,376)
Income tax expense 19 4,074 333 3,303
Net loss (3,332,474) (2,823,977) (9,929,957) (10,226,073)
Other comprehensive income items that may be
reclassified to net loss

Translation adjustment 32,545 192,586 (268,341) 377,060

Net comprehensive loss (3,299,929) (2,631,391) (10,198,298) (9,849,013)
Basic and diluted loss per common share (0.03) (0.02) (0.08) (0.09)
Weighted average number of shares (basic and
diluted) 120,552,638 117,963,979 119,455,440 110,757,811

ONCOLYTICS BIOTECH INC.
INTERIM CONSOLIDATED STATEMENTS OF CHANGES IN EQUITY
(unaudited)


Share Capital
$
Contributed
Surplus
$
Accumulated
Other
Comprehensive
Income
$
Accumulated
Deficit
$
Total
$
As at December 31, 2014 237,657,056 25,848,429 280,043 (249,966,335) 13,819,193
Net loss and other comprehensive income — — 377,060 (10,226,073) (9,849,013)
Issued, pursuant to Share Purchase Agreement 4,305,396 — — — 4,305,396
Issued, pursuant to "At the Market" Agreement 19,267,267 — — — 19,267,267
Share based compensation — 181,436 — — 181,436
As at September 30, 2015 261,229,719 26,029,865 657,103 (260,192,408) 27,724,279

Share Capital
$ Contributed
Surplus
$
Accumulated
Other
Comprehensive
Income
$
Accumulated
Deficit
$ Total
$
As at December 31, 2015 261,324,692 26,277,966 760,978 (263,689,330) 24,674,306
Net loss and other comprehensive loss — — (268,341) (9,929,957) (10,198,298)
Issued, pursuant to "At the Market" Agreement 852,536 — — — 852,536
Issued, pursuant to incentive share award plan 41,000 (41,000) — — —
Share based compensation — 299,635 — — 299,635
As at September 30, 2016 262,218,228 26,536,601 492,637 (273,619,287) 15,628,179

ONCOLYTICS BIOTECH INC.
INTERIM CONSOLIDATED STATEMENTS OF CASH FLOWS
(unaudited)

Three
Month
Period
Ending
September
30, 2016
$ Three
Month
Period
Ending
September
30, 2015
$ Nine Month
Period
Ending
September
30, 2016
$ Nine Month
Period
Ending
September
30, 2015
$

Operating Activities
Net loss for the period (3,332,474) (2,823,977) (9,929,957) (10,226,073)
Amortization – property and equipment 44,014 44,761 134,631 134,743
Share based compensation 98,369 10,791 299,635 181,436
Unrealized foreign exchange gain (49,400) (182,131) (152,019) (485,653)
Net change in non-cash working capital 216,611 92,792 978,847 (327,690)
Cash used in operating activities (3,022,880) (2,857,764) (8,668,863) (10,723,237)
Investing Activities
Acquisition of property and equipment (4,851) (17,695) (10,553) (47,292)
Purchase of short-term investments — — (27,823) (29,292)
Cash used in investing activities (4,851) (17,695) (38,376) (76,584)
Financing Activities
Proceeds from Share Purchase Agreement — — — 4,305,396
Proceeds from "At the Market" equity distribution agreement 242,706 213,742 852,536 19,267,267
Cash provided by financing activities 242,706 213,742 852,536 23,572,663
(Decrease) increase in cash (2,785,025) (2,661,717) (7,854,703) 12,772,842
Cash and cash equivalents, beginning of period 18,320,981 30,018,217 24,016,275 14,152,825
Impact of foreign exchange on cash and cash equivalents 76,773 605,962 (548,843) 1,036,795
Cash and cash equivalents, end of period 15,612,729 27,962,462 15,612,729 27,962,462

On November 3, 2016 Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) reported financial results for the three and nine months ended September 30, 2016, and provided an operating forecast and program updates (Press release, Ligand, NOV 3, 2016, View Source [SID1234516346]). Ligand management will host a conference call today beginning at 4:30 p.m. Eastern time to discuss this announcement and answer questions.

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"Our solid third quarter financial performance is highlighted by all-time high royalty revenues. In addition, we had a very active quarter with new licensings further increasing our portfolio of partnered assets," said John Higgins, Chief Executive Officer of Ligand. "Novartis continues to make excellent progress with growing sales, expanding indications and entering new geographies with Promacta. Amgen is focused on growing Kyprolis, and Spectrum’s product Evomela is showing good momentum in sales growth following its launch earlier this year. Also, recent positive clinical and regulatory milestones were achieved with a few of our more important portfolio programs, notably for programs or technologies licensed to Retrophin, Lundbeck and Melinta. We also initiated a Phase 2 clinical trial with LGD-6972 for the treatment of type 2 diabetes mellitus."

Ligand is in the process of evaluating certain deferred tax assets (DTA) recorded in the third quarter of 2015. Ligand is reviewing the amount of net operating loss carryforwards recorded as a result of capitalized R&D expenses associated with two acquisitions accounted for in 2010. As a result, the amount of DTA Ligand recorded in connection with the release of its valuation allowance could be reduced by approximately 10% of the $217 million DTA booked in the third quarter of 2015. The impact of the reduction would reduce the one-time DTA gain and would reduce GAAP net income for that period by the same amount. The balance sheet for the third quarter of 2015 and every subsequent period would reflect the reduction in DTA. The 2015 GAAP net income and earnings per share, which would be impacted by the reduction in DTA, are not available at this time, but will be reported in Ligand’s Form 10-Q for the third quarter of 2016, which will include Ligand’s conclusion regarding the reduction in DTA. The GAAP and adjusted net income and EPS figures for the three and nine month periods ended September 30, 2016 would be unaffected by the changes.

Third Quarter 2016 Financial Results

Total revenues for the third quarter of 2016 were $21.6 million, compared with $17.7 million for the same period in 2015. Royalty revenues were $15.7 million, compared with $9.8 million for the same period in 2015 primarily due to higher royalties from Promacta and Kyprolis. Material sales were $4.2 million, compared with $6.0 million for the same period in 2015 due to timing of Captisol purchases for use in clinical trials and commercial products. License and milestone revenues were $1.7 million, compared with $1.9 million for the same period in 2015.

Cost of goods sold was $1.0 million for the third quarter of 2016, compared with $1.3 million for the same period in 2015 due to the timing and mix of Captisol sales. Amortization of intangibles was $2.7 million, compared with $0.6 million for the same period in 2015 due primarily to additional amortization of intangibles related to the acquisition of OMT. Research and development expense was $6.3 million, compared with $1.9 million for the same period of 2015 as a result of the addition of OMT-related expenses, timing of spending on internal development programs and non-cash stock-based compensation expense. General and administrative expense was $6.3 million, compared with $5.0 million for the same period in 2015 due to costs associated with OMT and non-cash stock-based compensation expense.

GAAP net income for the third quarter of 2016 was $0.6 million, or $0.03 per share. Adjusted net income for the third quarter of 2016 was $13.5 million, or $0.62 per diluted share, compared with adjusted net income for the same period in 2015 of $12.0 million, or $0.57 per diluted share. See "Adjusted Financial Measures" and the accompanying table below for the adjusted calculations and reconciliation to comparable GAAP financial measures.

As of September 30, 2016, Ligand had cash, cash equivalents and short-term investments of $124.1 million.

Year-to-Date Financial Results

Total revenues for the nine months ended September 30, 2016 were $70.8 million, compared with $50.7 million for the same period in 2015. Royalty revenues were $39.8 million, compared with $26.6 million for the same period in 2015 primarily due to higher royalties from Promacta and Kyprolis. Material sales were $13.5 million, compared with $20.5 million for the same period in 2015 due to timing of Captisol purchases for use in clinical trials and commercial products. License and milestone revenues were $17.5 million, compared with $3.6 million for the same period in 2015 due to the addition of OmniAb revenue as of January 2016 and the timing of milestones and upfront license fees.

Cost of goods sold was $2.7 million for the nine months ended September 30, 2016, compared with $4.9 million for the same period in 2015 due to the timing and mix of Captisol sales. Amortization of intangibles was $7.9 million, compared with $1.8 million for the same period in 2015 due primarily to additional amortization of intangibles related to the acquisition of OMT. Research and development expense was $14.8 million, compared with $8.7 million for the same period of 2015 as a result of timing of spending on internal development programs and non-cash stock-based compensation expense. General and administrative expense was $20.0 million, compared with $18.2 million for the same period in 2015 due to costs associated with OMT and non-cash stock-based compensation expense.

GAAP net income for the nine months ended September 30, 2016 was $1.5 million, or $0.07 per diluted share. Adjusted net income for the nine months ended September 30, 2016 was $45.3 million, or $2.09 per diluted share, compared with adjusted net income for the same period in 2015 of $57.3 million, or $2.75 per diluted share.

Revised Financial Forecast

The Company is revising expectations for full-year 2016 total revenues to be between $110 million and $114 million, and adjusted earnings per diluted share to be between $3.37 and $3.44. This compares to previous Company expectations for full-year 2016 total revenues to be between $115 million and $119 million, and adjusted earnings per diluted share to be between $3.41 and $3.46. The revised outlook for total revenues reflects expected lower sales for Captisol due to timing of certain regulatory and clinical events. The Company expects total revenues for the fourth quarter of 2016 to be between $39 million and $43 million, including approximately $19 million in royalty revenues. Fourth quarter adjusted earnings per diluted share is expected to be between $1.29 and $1.36. For 2017, we are maintaining our previously disclosed guidance of greater than $160 million of revenue and greater than $5.03 of adjusted earnings per diluted share.

The adjusted net income per diluted share guidance excludes non-cash stock-based compensation expense, non-cash debt-related costs, amortization related to acquisitions, changes in contingent liabilities, non-cash net losses of Viking Therapeutics equity, mark-to-market adjustment for amounts owed to licensors, fair value adjustments to Viking Therapeutics convertible note receivable and warrants, non-cash tax benefit (expense), unissued shares relating to the Senior Convertible Note and adjustments for discontinued operations, net of non-cash tax expense.

Third Quarter 2016 and Recent Business Highlights

Portfolio Program Progress

Promacta/Revolade

Novartis announced Q3 2016 net sales of Promacta (eltrombopag) of $168 million, a $51 million or 44% increase over Q3 2015. Novartis also announced that Promacta is now approved in more than 100 countries.
Kyprolis (carfilzomib), an Amgen Product Utilizing Captisol

On September 27, 2016, Amgen announced top-line results of the Phase 3 CLARION trial, which evaluated an investigational regimen of Kyprolis (carfilzomib), melphalan and prednisone (KMP) versus Velcade (bortezomib), melphalan and prednisone (VMP) for 54 weeks in patients with newly diagnosed multiple myeloma who were ineligible for hematopoietic stem-cell transplant. The trial did not meet the primary endpoint of superiority in progression-free survival (PFS). A Phase 3 study evaluating Kyprolis in combination with lenalidomide plus dexamethasone (KRd) versus Velcade in combination with lenalidomide plus dexamethasone (VRd) in newly diagnosed multiple myeloma patients called ENDURANCE, an investigator-sponsored study, is underway independently by the ECOG-ACRIN Cancer Research Group.
In July 2016, Amgen announced that the European Commission approved an extended indication for Kyprolis (carfilzomib), to be used in combination with dexamethasone alone, for adult patients with multiple myeloma who have received at least one prior therapy. Also, Ono Pharmaceuticals, holder of Kyprolis (carfilzomib) marketing rights in Japan, announced approval in Japan for treatment of patients with relapsed or refractory multiple myeloma.
Additional Pipeline and Partner Developments

Retrophin announced positive top-line results from the Phase 2 DUET study of sparsentan for the treatment of focal segmental glomerulosclerosis. The study achieved statistical significance in the primary efficacy endpoint for the overall sparsentan treatment group, demonstrating a greater than two-fold reduction of proteinuria compared to irbesartan after the eight-week, double-blind treatment period. Additional data from the Phase 2 DUET study of sparsentan for the treatment of focal segmental glomerulosclerosis will be presented at the late-breaking High-Impact Clinical Trials oral session at the American Society of Nephrology (ASN) Kidney Week 2016.
Lundbeck announced FDA approval of Carnexiv (carbamazepine) injection as a short-term replacement therapy for oral carbamazepine formulations in adults with certain seizure types when oral administration is temporarily not feasible. Ligand earned a $1.25 million milestone payment upon approval and is entitled to receive a royalty of 2.75% on net sales of Carnexiv.
Melinta Therapeutics announced that it has submitted NDAs to the FDA for approval of IV and oral Baxdela (delafloxacin) for the treatment of patients with acute bacterial skin and skin structure infections (ABSSSI). With the submission, Ligand earned a $1.5 million milestone payment. If approved, Ligand is entitled to receive a 2.5% royalty on net sales of the IV formulation of Baxdela and an additional $1.5 million approval milestone payment. Baxdela was the subject of several poster presentations at IDWeek 2016, held October 26-30 at the New Orleans Ernest N. Morial Convention Center.
The FDA granted orphan designation to Merck’s Noxafil for treatment of invasive aspergillosis.
Viking Therapeutics announced the first patient has been dosed in the company’s Phase 2 clinical trial of VK2809 in patients with primary hypercholesterolemia and non-alcoholic fatty liver disease.
Viking Therapeutics announced positive top-line results from a proof-of-concept study of VK0214 in a mouse model of X-linked adrenoleukodystrophy (X-ALD), showing VK0214 rapidly reduced plasma very long chain fatty acid levels by more than 25% in treated animals compared with vehicle controls (p<0.01). Detailed study results were presented at the 86th Annual Meeting of the American Thyroid Association.
Aldeyra Therapeutics announced plans for ADX-102 (formerly NS2) for the first-ever vehicle-controlled Phase 3 clinical trial in noninfectious anterior uveitis, as well as a Phase 3 clinical trial in Sjögren-Larsson Syndrome. Aldeyra also announced the expected advancement of ADX-102 to a Phase 2b clinical trial in allergic conjunctivitis and the addition of a clinical program in dry eye syndrome.
Eli Lilly presented data on Prexasertib (LY2606368) demonstrating activity in patients with BRCA wild type sporadic high-grade serous ovarian cancer at the European Society for Medical Oncology 2016 Congress.
Merrimack Pharmaceuticals announced the FDA granted seribantumab (MM-121) Fast Track designation for development in patients with heregulin-positive, locally advanced or metastatic non-small cell lung cancer whose disease has progressed following immunotherapy.
Lubris BioPharma announced positive results of a clinical trial that showed recombinant human lubricin demonstrated significant improvement in both signs and symptoms of dry eye disease compared to sodium hyaluronate (HA). Results were published in the September issue of The Ocular Surface.
Opthea announced that the Phase 1 dose-escalation study of OPT-302 met its primary objective demonstrating safety and tolerability as monotherapy and in combination with the current wet AMD standard of care Lucentis. Opthea is recruiting patients for its Phase 2a dose-expansion trial and expects data by the end of 2016.
New Licensing Deals

Ligand announced worldwide license agreements with Gilead Sciences, F-Star Biotechnology Limited and TeneoBio to use certain or all of the OmniAb platform technologies to discover fully human antibodies. Ligand is eligible to receive annual access payments, sublicensing fees, milestone payments and royalties on future net sales of any antibodies discovered under these licenses.
Ligand announced licensing rights to four programs to Seelos Therapeutics including aplindore for the treatment of various CNS disorders, a CRTH2 antagonist for the treatment of respiratory disorders, a Captisol-enabled acetaminophen program for pain and fever management and an H3 receptor antagonist program for the treatment of narcolepsy. Ligand is entitled to receive milestones and net sales royalties ranging from 4% to 10% for the various programs licensed.
Ligand announced a license agreement for its LTP technology with Nucorion Pharmaceuticals, a venture-funded biotechnology company focused on developing anti-cancer and anti-viral agents initially directed to China, of which Ligand is a minority shareholder. Three initial programs fall under the license: NUC-202, a targeted anticancer analog for the treatment of hepatocellular carcinoma; NUC-404, a targeted nucleotide analog for the treatment of hepatitis B; and NUC-101, a targeted nucleotide analog for the treatment of hepatitis C. Ligand is eligible to receive milestones in addition to royalties ranging from 5% to 9% on future net sales of any approved program.
Internal Glucagon Receptor Antagonist (GRA) Program

Ligand announced initiation of a Phase 2 clinical trial with LGD-6972 for the treatment of type 2 diabetes mellitus (T2DM). The randomized, double-blind, placebo-controlled study will evaluate the safety and efficacy of LGD-6972, as an adjunct to diet and exercise, in subjects with T2DM whose blood glucose levels are inadequately controlled with metformin. Results from two Phase 1 clinical trials with LGD-6972 were published online in the August 2016 issue of the journal Diabetes, Obesity and Metabolism.
Adjusted Financial Measures

The adjusted financial measures discussed above and in the tables below for the three and nine months ended September 30, 2016 and 2015 exclude non-cash stock-based compensation expense, non-cash debt-related costs, amortization related to acquisitions, changes in contingent liabilities, non-cash net losses of Viking Therapeutics equity, mark-to-market adjustment for amounts owed to licensors, fair value adjustments to Viking Therapeutics convertible note receivable and warrants, non-cash tax benefit (expense), unissued shares relating to the Senior Convertible Note and adjustments for discontinued operations, net of non-cash tax expense.

Management has presented net income and net income per share on an adjusted basis. Ligand believes the presentation of adjusted financial measures provides useful supplementary information to investors and reflects amounts that are more closely aligned with the cash profits for the period. Ligand uses these adjusted financial measures in connection with its own budgeting and financial planning. These adjusted financial measures are in addition to, and not a substitute for, or superior to, measures of financial performance prepared in conformity with GAAP.

On November 2, 2016 MacroGenics, Inc. (NASDAQ:MGNX), a clinical-stage biopharmaceutical company focused on discovering and developing innovative monoclonal antibody-based therapeutics for the treatment of cancer, as well as autoimmune disorders and infectious diseases, reported a corporate progress update and reported financial results for the quarter ended September 30, 2016 (Press release, MacroGenics, NOV 2, 2016, View Source [SID1234516198]).

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"MacroGenics continues to make progress across its broad pipeline of clinical compounds, including margetuximab, our Fc-optimized anti-HER2 monoclonal antibody, our two clinical programs targeting B7-H3 as well as several bispecific product candidates based on our DART platform," said Scott Koenig, M.D., Ph.D., President and CEO of MacroGenics. "During the third quarter, an IND for MGA012 cleared the FDA and we remain on track to submit two DART molecule INDs in 2017, which would result in a total of eight DART molecules in clinical development. We look forward to providing an update on multiple clinical and preclinical programs at our R&D Day on December 13."

Pipeline Update

Margetuximab. Recent highlights related to the Company’s Fc-optimized monoclonal antibody that targets the human epidermal growth factor receptor 2, or HER2, include:

SOPHIA Study: MacroGenics’ Phase 3 pivotal study in patients with HER2-positive metastatic breast cancer is ongoing, as the Company continues to initiate sites and enroll patients. This study is evaluating the efficacy of margetuximab plus chemotherapy compared to trastuzumab plus chemotherapy in approximately 530 relapsed/refractory patients. Approximately 90% of the anticipated study sites have been activated as of September 30, 2016.
Phase 1b/2 Gastric Cancer Study: MacroGenics continues to recruit and dose patients in a clinical trial of margetuximab in combination with pembrolizumab, an anti-PD-1 therapy, in patients with advanced HER2-positive gastric cancer. Treatment options for these patients are limited and the combination regimen being explored avoids chemotherapy while exploiting the potential for enhancing the antitumor immune response. This trial is being conducted in collaboration with Merck and is currently recruiting patients in North America. We anticipate the start of this study in Asia by year-end.
B7-H3 Franchise. MacroGenics is developing a portfolio of therapeutics that target B7-H3, a member of the B7 family of molecules involved in immune regulation. The Company is advancing multiple programs that target B7-H3 through complementary mechanisms of action and take advantage of this antigen’s broad expression across multiple solid tumor types. Current ongoing clinical-stage development programs include:

Enoblituzumab: The Company continues to recruit patients in three ongoing studies of enoblituzumab, an Fc-optimized monoclonal antibody that targets B7-H3. These studies include one monotherapy study and two combination studies with each of ipilimumab and pembrolizumab. As previously reported, the monotherapy study was expanded to include additional prostate and bladder cancer cohorts. An additional monotherapy study is planned for children with neuroblastoma and other tumors, and we also anticipate an investigator-sponsored monotherapy study in neo-adjuvant prostate cancer.
MGD009: This DART molecule targeting B7-H3 and CD3 is being evaluated in a Phase 1 study across multiple solid tumor types.
DART Product Candidates. There are currently six DART molecules in Phase 1 clinical development, including MGD006 (CD123 x CD3, also known as S80880), MGD007 (gpA33 x CD3), MGD009 (B7-H3 x CD3), MGD010 (CD32B x CD79B), MGD011 (CD19 x CD3, also known as JNJ-64052781 or duvortuxizumab) and PF-06671008 (P-cadherin x CD3).

During the third quarter and as previously reported, MacroGenics and Takeda Pharmaceutical Company Limited announced the conclusion of their License and Option Agreement for MGD010, a bispecific molecule targeting CD32B and CD79B. MacroGenics regained the worldwide rights to MGD010, for which the Company plans to continue to advance development based on the encouraging study results reported to date.

An Investigational New Drug (IND) application for MGA012, a monoclonal antibody, recently cleared the FDA and the Company plans to submit INDs for two DART molecules in 2017. These two DART molecules are:

MGD013: MacroGenics is developing MGD013 to simultaneously block two immune checkpoint molecules, PD-1 and LAG-3.
MGD014: MGD014 is a DART molecule that is being developed to eliminate latent HIV infection.
Beyond MGD013 and MGD014, MacroGenics continues to generate and evaluate multiple other candidates that target a range of immune regulatory and other molecules using its proprietary platforms.

Corporate Update

R&D Day: MacroGenics plans to host an R&D Day in New York on Tuesday, December 13, 2016. At this meeting, the Company plans to both provide an update on its clinical pipeline as well as preview its next set of development candidates.
Universal Shelf Registration Statement Filing: MacroGenics has filed a shelf registration statement with the SEC and believes that this filing provides the Company with the flexibility to access the equity or debt capital markets most efficiently should the need arise. However, the Company has no immediate plan to undertake an offering.
Third Quarter 2016 Financial Results

Cash Position: Cash, cash equivalents and marketable securities as of September 30, 2016, were $314.1 million, compared to $339.0 million as of December 31, 2015.
Revenue: Total revenue, consisting primarily of revenue from collaborative agreements, was $3.3 million for the quarter ended September 30, 2016, compared to $14.7 million for the quarter ended September 30, 2015. This decrease was primarily due to recognition of a one-time milestone received from Janssen Biotech, Inc. in 2015.
R&D Expenses: Research and development expenses were $30.3 million for the quarter ended September 30, 2016, compared to $24.1 million for the quarter ended September 30, 2015. This increase was due primarily to increased activity in the Company’s preclinical immune checkpoint programs, including MGD013, MGD014 (funded by NIAID/NIH) and the initiation of two Phase 1 clinical trials combining enoblituzumab with other compounds.
G&A Expenses: General and administrative expenses were $7.2 million for the quarter ended September 30, 2016, compared to $6.0 million for the quarter ended September 30, 2015. This increase was primarily due to increased staff, recruiting costs and stock-based compensation expense.
Net Income/Loss: Net loss was $33.9 million for the quarter ended September 30, 2016, compared to net loss of $15.4 million for the quarter ended September 30, 2015.
Shares Outstanding: Shares outstanding as of September 30, 2016 were 34,813,334.

On November 2, 2016 Genmad reported its interim Report for First Nine Months Ended September 30, 2016 (Press release, Genmab, NOV 2, 2016, View Source [SID1234516222]).
Highlights
Net Sales of DARZALEX (daratumumab) by Janssen for the first nine months of 2016 were USD 372 million, resulting in royalty income of USD 45 million (DKK 298 million)
Announced U.S. and European regulatory submissions for daratumumab in relapsed or refractory multiple myeloma, triggering USD 25 million in milestone payments
Daratumumab received second Breakthrough Therapy Designation from U.S. Food and Drug Administration (FDA)
Announced FDA approval of Arzerra (ofatumumab) in combination with fludarabine and cyclophosphamide for relapsed chronic lymphocytic leukemia (CLL)
Entered commercial license agreement with Gilead Sciences for DuoBody Technology
2016 financial guidance improved
"Throughout the third quarter we continued to see excellent progress in our DARZALEX program with Janssen. Regulatory applications to expand the label for daratumumab to include relapsed or refractory multiple myeloma were submitted in the U.S. and Europe, triggering USD 25 million in milestone payments. Daratumumab also received its second Breakthrough Therapy Designation from the FDA. We continued to see progress with Arzerra too, with another CLL indication approved in the U.S., and we made progress with our DuoBody technology, with a new commercial agreement with Gilead Sciences," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.
Financial Performance First Nine Months of 2016
Revenue was DKK 889 million in the first nine months of 2016 compared to DKK 558 million in the first nine months of 2015. The increase of DKK 331 million, or 59%, was mainly driven by higher royalty and milestone revenue under our daratumumab collaboration with Janssen.
Operating expenses were DKK 544 million in the first nine months of 2016 compared to DKK 380 million in the first nine months of 2015. The increase of DKK 164 million, or 43%, was due to the additional investment in our pipeline of products, including the advancement of tisotumab vedotin, HuMax-AXL-ADC, HexaBody-DR5/DR5, DuoBody-CD3xCD20, and our other pre-clinical programs.
Operating income was DKK 345 million in the first nine months of 2016 compared to DKK 355 million in the first nine months of 2015. The decrease of DKK 10 million, or 3%, was driven by the one-time reversal of the ofatumumab funding liability of DKK 176 million in 2015, combined with increased operating expenses in 2016, which were partly offset by higher revenue in 2016.
On September 30, 2016, Genmab had a cash position of DKK 3,942 million compared to DKK 3,493 million at December 31, 2015. This represented a net increase of DKK 449 million, which was driven primarily by income from operations and the proceeds from the exercise of warrants of DKK 184 million, partially offset by the purchase of treasury shares for DKK 118 million.
Business Progress Third Quarter
Daratumumab
August: Regulatory submission in Europe for daratumumab (DARZALEX) in patients with multiple myeloma who have received at least one prior therapy. In addition, a regulatory application was submitted in the U.S. for the use of daratumumab in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of patients with multiple myeloma who received at least one prior therapy. The submissions triggered milestone payments of USD 10 million, and USD 15 million, respectively, to Genmab.
July: The FDA granted Breakthrough Therapy Designation for DARZALEX injection in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone for the treatment of patients with multiple myeloma who have received at least one prior therapy.
Ofatumumab
August: The FDA approved ofatumumab (Arzerra) in combination with fludarabine and cyclophosphamide (FC) for the treatment of patients with relapsed CLL.
DuoBody
August: Entered an agreement to grant Gilead Sciences, Inc. an exclusive license and an option on a second exclusive license, to use the DuoBody technology platform to create and develop bispecific antibody candidates for a therapeutic program targeting HIV. Under the terms of the agreement, Genmab received an upfront payment of USD 5 million from Gilead Sciences.
Subsequent Event
October: The FDA granted Priority Review for the use of daratumumab in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of patients with multiple myeloma who have received at least one prior therapy. The FDA assigned a Prescription Drug User Fee Act (PDUFA) target date of February 17, 2017 to take a decision on daratumumab in this indication. In addition, the FDA granted a Standard Review period for the use of daratumumab in combination with pomalidomide and dexamethasone for the treatment of patients with relapsed or refractory multiple myeloma who have received at least two prior therapies, including a proteasome inhibitor (PI) and an immunomodulatory agent. The PDUFA date for the combination of daratumumab with pomalidomide/dexamethasone is June 17, 2017.
Outlook
Genmab is improving its 2016 financial guidance published on August 9, 2016 due to increased royalty and milestone income related to the sales of DARZALEX resulting in increased operating income and cash position.
MDKK Revised Guidance Previous Guidance
Revenue 1,200 — 1,250 975 — 1,025
Operating expenses (800) — (850) (800) — (850)
Operating income 375 — 425 150 — 200
Cash position at end of year* 3,650 — 3,750 3,550 — 3,650
*Cash, cash equivalents, and marketable securities

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