On August 4, 2016 Raptor Pharmaceutical Corp. (NASDAQ:RPTP), a biopharmaceutical company developing and commercializing transformative treatments for rare diseases, reported its financial results for the second quarter of 2016 and provided an update on recent corporate developments (Press release, Raptor Pharmaceutical, AUG 4, 2016, View Source [SID:1234514301]).

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Summary

Global net product revenue was $32.0 million for the second quarter ended June 30, 2016, a 37.3% increase compared to $23.3 million for the same period in 2015.

Net loss on a GAAP basis was $14.0 million, or $0.16 per share, for the second quarter of 2016 compared to a GAAP net loss of $13.9 million, or $0.17 per share, for the same period in 2015.

Non-GAAP net loss, which excludes non-cash expenditures such as stock compensation, amortization and impairment of IPR&D, and change in the fair value of the contingent consideration liability related to acquisitions, was $11.3 million, or $0.13 per share, for the second quarter of 2016 compared to a non-GAAP net loss of $9.6 million, or $0.12 per share, for the same period in 2015.

Cash and cash equivalents were $124.2 million as of June 30, 2016.

Raptor is raising its 2016 global net revenue guidance to $125 to 135 million from $115 to $125 million and affirming its guidance for non-GAAP operating expenses, excluding non-cash expenditures such as stock compensation, amortization and impairment of IPR&D, and change in the fair value of the contingent consideration liability related to acquisitions, of between $125 and $135 million.

"I’m delighted that we delivered another record quarter for sales, driven primarily by growing patient demand for PROCYSBI ," said Julie Anne Smith, President and CEO of Raptor. "For the first time, PROCYSBI revenue was augmented by sales of QUINSAIR, which is off to a terrific launch in Europe. Based on our outstanding commercial performance, we are pleased to raise our 2016 revenue guidance. We look forward to continued growth from both products and supporting patients living with rare diseases and with limited options."

Second Quarter 2016 Business Highlights

In April, Raptor commenced the first commercial sales of QUINSAIR (levofloxacin inhalation solution) in Germany and Denmark for the management of chronic pulmonary infections due to Pseudomonas aeruginosa in adult patients with cystic fibrosis. To date, approval for reimbursement for QUINSAIR has been achieved in numerous European countries.

In June, Raptor participated in the 39th European Cystic Fibrosis Conference and presented new analyses of data from its Phase 3 clinical study (MPEX-209) comparing QUINSAIR to inhaled tobramycin solution in patients with cystic fibrosis and chronic Pseudomonas aeruginosa infections. The data suggest that subjects with three or more pulmonary exacerbations in the prior year who were randomized to receive QUINSAIR had a significantly lower incidence of pulmonary exacerbations when compared with their peers who were randomized to receive the active comparator, tobramycin inhalation solution (p=0.026).

Second Quarter 2016 Financial Results

Raptor provides non-GAAP financial measures, which it believes can enhance an overall understanding of its financial performance when considered together with GAAP figures. Refer to the section of this press release below entitled "Non-GAAP Financial Information and Other Disclosures" for further discussion on this subject.

Net Product Revenue

Global net revenue for the second quarter of 2016 was $32.0 million, compared to $23.3 million for the second quarter of 2015. The 37.3% revenue growth was driven by further market penetration in the U.S. and in Europe and an increase in named patient sales in other international territories for PROCYSBI, and by the introduction of QUINSAIR during the quarter.

Cost of Sales

Cost of sales was $4.9 million for the second quarter of 2016, compared to $2.6 million for the second quarter of 2015. The increase was due to higher quarterly sales year-over-year, leading to higher direct costs and royalty expenses in 2016.

Research & Development (R&D)

R&D expenses for the second quarter of 2016 were $15.7 million, compared to $11.9 million for the second quarter of 2015. The increased expense for the three month period ended June 30, 2016 was primarily due to increased activities associated with our product portfolio, support for the European QUINSAIR launch and related to preparation for a potential NDA filing for QUINSAIR in a cystic fibrosis indication in the US.

Selling, General and Administrative (SG&A)

SG&A expenses were $20.9 million for the second quarter of 2016 compared to $17.8 million for the second quarter of 2015. The increase in SG&A expenses was primarily a result of increased personnel costs and promotional support for the worldwide commercial operations of PROCYSBI and QUINSAIR in Europe.

Interest Expense

Interest expense for the second quarter of 2016 was $4.3 million, compared to $4.8 million for the second quarter of 2015. The decrease in interest expense was primarily due to a decrease in principal from which Raptor’s interest expense is calculated.

Net Loss

GAAP net loss in the second quarter of 2016 was $14.0 million, or $0.16 per share, compared to a net loss of $13.9 million, or $0.17 per share for the second quarter of 2015.

Non-GAAP net loss, which excludes non-cash expenditures such as stock compensation, amortization and impairment of IPR&D, and change in the fair value of the contingent consideration liability related to acquisitions, for the second quarter of 2016 was $11.3 million, or $0.13 per share, compared to a non-GAAP net loss of $9.6 million, or $0.12 per share, in the second quarter of 2015.

Cash and Cash Equivalents

As of June 30, 2016, Raptor had $124.2 million in cash and cash equivalents compared to $132.0 million in cash and cash equivalents at March 31, 2016.

2016 Full-Year Financial Guidance

Raising 2016 global net revenue guidance to $125 to $135 million from $115 to $125 million.
Refining PROCYSBI revenue growth to at least 30% over 2015 revenues.
Reiterating non-GAAP operating expense guidance, which excludes non-cash expenditures such as stock compensation, amortization and impairment of IPR&D, and change in the fair value of the contingent consideration liability related to acquisitions, of $125 to $135 million. Raptor is unable to reconcile non-GAAP operating expense guidance to GAAP operating expense guidance without unreasonable efforts and believes any such reconciliation would imply a degree of precision that could be confusing to investors. Raptor is unable to predict the probable significance of any of the above-listed adjustments.
Product and Pipeline Updates

PROCYSBI for Nephropathic Cystinosis

In the second quarter, Raptor recorded the first PROCYSBI sales in several additional European countries.
A New Drug Submission (NDS) for PROCYSBI for the treatment of nephropathic cystinosis is currently under review by Health Canada. Recently, Raptor received notice from Health Canada that it is seeking additional information to complete its review. Raptor intends to submit a response to Health Canada in a timely fashion to preserve Priority Review status and expects that Health Canada’s timeline for review of the NDS will re-commence when it accepts Raptor’s response. As a result, Raptor anticipates a delay in Health Canada’s decision on the potential marketing approval of PROCYSBI.
QUINSAIR for Cystic Fibrosis

In April, Raptor launched QUINSAIR in Germany and Denmark for the management of chronic pulmonary infections due to Pseudomonas aeruginosa in adults with cystic fibrosis, and to date, QUINSAIR has become available for reimbursement in several territories in the EU and Raptor is actively expanding into new countries.
Raptor anticipates launching QUINSAIR in Canada for the same indication, for which it has received marketing approval, in the second half of 2016.
In August, the European Patent Office issued EP Pat. No. 1 901 749 with claims covering Raptor’s QUINSAIR formulation. This patent will expire in May 2026, supplementing Raptor’s 10 years of data exclusivity.
MP-376 (Investigational Form of QUINSAIR)

Raptor had a meeting with the FDA in the second quarter concerning MP-376 for the management of chronic pulmonary infections due to Pseudomonas aeruginosa in adult patients with cystic fibrosis in the U.S. The FDA requested additional information pertinent to Raptor’s existing trials prior to the company’s potential submission of an NDA. Raptor submitted a response to the FDA’s request and expects to have additional discussions with the FDA.
The company intends to initiate a clinical study for MP-376 in bronchiectasis (BE) in 2016. Raptor plans to provide additional clarity on the timing of key activities once the study design has been finalized.
RP103 for Huntington’s Disease

With current efforts focused on prioritizing clinical programs that represent Raptor’s best use of current capital, the company continues to explore non-dilutive funding and partnering options for RP103 in Huntington’s disease.
RP103 for Mitochondrial Diseases

The second interim analysis, which occurred after 12 patients completed 24 weeks of treatment, indicated the ongoing trial should continue as planned, which is currently ongoing.
Anticipated Upcoming Milestones

2H 2016 — Potential QUINSAIR launch in Canada
2016 — Advancement towards an NDA submission for MP-376 in cystic fibrosis in the U.S.
2016 — Initiation of a clinical study for MP-376 in BE
1H 2017 — Phase 2 RP103 data in mitochondrial diseases

On Auguest 4, 2016 Ironwood Pharmaceuticals, Inc. (NASDAQ: IRWD), a commercial biotechnology company, reported an update on its second quarter 2016 results and recent business activities (Press release, Ironwood Pharmaceuticals, AUG 4, 2016, View Source [SID:1234514298]).

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"During the second quarter of 2016, Ironwood continued to deliver strong operational performance, resulting in a near-doubling of Ironwood revenue, year-over-year. We are well-positioned to advance our four priority franchises, including the launch of two new products in the next nine months, and we remain on track to become cash flow positive in 2018," said Peter Hecht, chief executive officer of Ironwood. "Our flagship product LINZESS demonstrated solid growth as the branded prescription market leader in adult patients with IBS-C or CIC and is on track to exceed $1 billion in net sales by 2020, with increased commercial margins driven by strong demand."

Second Quarter 2016 and Recent Highlights

Irritable Bowel Syndrome with Constipation (IBS-C) / Chronic Idiopathic Constipation (CIC) Franchise

LINZESS. U.S. net sales, as provided by Ironwood’s U.S. collaboration partner Allergan, were $150.5 million in the second quarter of 2016, a 34% increase compared to the second quarter of 2015. Ironwood and Allergan share equally in brand collaboration profits or losses.
More than 650,000 total LINZESS prescriptions were filled in the second quarter of 2016, a 29% increase compared to the second quarter of 2015, according to IMS Health. Since launch, more than 5 million prescriptions for LINZESS have been filled by more than 1 million unique patients, making LINZESS the branded prescription market leader in IBS-C and CIC.
Net profit for the LINZESS U.S. brand collaboration, including commercial and research and development (R&D) expenses, was $58.3 million in the second quarter of 2016, a 289% increase compared to the second quarter of 2015. LINZESS commercial margin was 52% in the second quarter of 2016, compared to 31% in the second quarter of 2015.
Ironwood and Allergan expect to launch a 72 mcg dose of linaclotide in early 2017 that, if approved, can increase physician prescribing of LINZESS within the large, heterogeneous adult CIC patient population. The companies announced during the second quarter of 2016 that the U.S. Food & Drug Administration (FDA) accepted the supplemental new drug application filing for this dose.
Linaclotide Colonic Release. Ironwood and Allergan completed enrollment in a Phase IIb clinical trial and expect data later this year; if positive, the companies anticipate initiating a Phase III trial in 2017. This second-generation guanylate cyclase-C (GC-C) agonist product candidate has the potential, if approved, to provide greater and faster abdominal pain relief in adult IBS-C patients, expand the IBS-C and CIC markets, and extend patent protection to the mid-2030s.
Uncontrolled Gout Franchise

ZURAMPIC. Ironwood expects to launch FDA-approved ZURAMPIC in October 2016 for use in combination with a xanthine oxidase inhibitor (XOI) for the treatment of hyperuricemia associated with uncontrolled gout. Gout is a form of inflammatory arthritis, and an estimated two million patients in the U.S. suffer from uncontrolled gout in which traditional first-line XOI treatment alone is not sufficient to achieve target serum uric acid (sUA) levels. Many of these patients experience painful flares due to hyperuricemia despite treatment with an XOI. In two Phase III clinical trials, the combination of the XOI allopurinol, which decreases production of uric acid, and ZURAMPIC, which increases excretion of uric acid, demonstrated nearly a two-fold increase in the percentage of patients reaching target serum uric acid levels under 6 mg/dL over allopurinol alone at month six. ZURAMPIC is not recommended for the treatment of asymptomatic hyperuricemia and should not be used as monotherapy. Ironwood closed the transaction with AstraZeneca for the exclusive U.S. rights to all products containing lesinurad during the second quarter of 2016.
Lesinurad-allopurinol fixed-dose combination product. The fixed-dose combination of lesinurad and allopurinol is expected to be submitted for FDA review during the second half of 2016.
Refractory Gastroesophageal Reflux Disease (rGERD) Franchise

IW-3718. Ironwood continues to enroll patients in a dose-ranging Phase IIb clinical trial of IW-3718, a wholly-owned asset for the potential treatment of rGERD. Data are expected in 2017. IW-3718 is a novel, investigational gastric retentive formulation of a bile acid sequestrant designed to work with a proton pump inhibitor (PPI) to reduce the detrimental effects of bile and acid on the esophagus. An estimated 10 million people in the U.S. suffer from rGERD and continue to experience heartburn symptoms despite treatment with PPIs.
Vascular and Fibrotic Franchise

IW-1973. Ironwood generated positive topline data from its Phase Ib multiple ascending dose study of IW-1973. The data were consistent with previous preclinical and Phase Ia findings and support advancement of IW-1973 into Phase II clinical trials, expected to begin later this year. Specifically, in the Phase Ib study, IW-1973 demonstrated characteristics that Ironwood believes could be important pharmacokinetic differentiators in the field of soluble guanylate cyclase (sGC) stimulators, including a narrow peak-to-trough ratio indicative of the potential to maintain a durable and consistent therapeutic effect; a profile that indicates suitability for once-daily dosing; and a volume of distribution consistent with penetration beyond the vasculature and into the tissues.
IW-1701. Ironwood completed enrollment in a Phase Ib multiple ascending dose study, with data expected later this year and a Phase II trial expected to begin before year-end.
Global Collaborations and Partnerships

Linaclotide is currently under review by the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan for potential approval for the treatment of adult patients with IBS-C. Additionally, Ironwood’s partner Astellas Pharma Inc. initiated a Phase III clinical trial of linaclotide in Japan for adults with chronic constipation.
Ironwood continued co-promoting Allergan’s VIBERZI (eluxadoline) for adults suffering from IBS with diarrhea (IBS-D) and Exact Sciences’ Cologuard noninvasive stool DNA screening test for colorectal cancer, in the U.S.
Corporate and Financials

Collaborative Arrangements Revenue
Collaborative arrangements revenue was $54.4 million in the second quarter of 2016, compared to $27.7 million in the second quarter of 2015. Revenue primarily consisted of $48.3 million in revenue associated with Ironwood’s share of the net profits from the sales of LINZESS in the U.S., compared to $24.3 million in the second quarter of 2015.
Operating Expenses
Operating expenses were $69.7 million in the second quarter of 2016, compared to $61.6 million in the second quarter of 2015. Operating expenses in the second quarter of 2016 consisted of $31.7 million in R&D expenses; $36.9 million in selling, general and administrative (SG&A) expenses; and $1.1 million in acquired intangible asset amortization expenses resulting from Ironwood’s U.S. licensing agreement with AstraZeneca for the exclusive rights to all products containing lesinurad.
Other Expense
Interest Expense. Net interest expense was $9.5 million in the second quarter of 2016, in connection with the $175 million debt financing executed in January 2013 and the approximately $336 million convertible debt financing executed in June 2015. Interest expense recorded in the second quarter of 2016 includes $6.2 million in cash expense and $3.6 million in non-cash expense. Both the cash and non-cash components of the 2022 convertible notes are recorded quarterly.
Gain/Loss on Derivatives. Ironwood records a gain/loss on derivatives related to the change in fair value of the convertible note hedges and note hedge warrants issued in connection with the convertible debt financing in June 2015. A gain on derivatives of $3.1 million was recorded in the second quarter of 2016.
Net Loss
GAAP net loss was $21.7 million, or $0.15 per share, in the second quarter of 2016, compared to $48.0 million, or $0.34 per share, in the second quarter of 2015.
Non-GAAP net loss was $23.8 million, or $0.16 per share, in the second quarter of 2016, compared to $47.8 million, or $0.34 per share, in the second quarter of 2015. Non-GAAP net loss excludes the impact of mark-to-market adjustments on the derivatives related to Ironwood’s convertible debt and the amortization of acquired intangible assets related to Ironwood’s U.S. lesinurad license. See Non-GAAP Financial Measures below.
Cash Position
Ironwood ended the second quarter of 2016 with $325 million of cash, cash equivalents and available-for-sale securities, a decrease of $109 million from the end of the first quarter of 2016. This figure includes the $100 million upfront payment to AstraZeneca from cash on hand under the lesinurad licensing agreement. Cash used in operations was $6 million, a 77% decline from the $26 million used during the same period a year ago.
2016 Financial Guidance
With the completion of its U.S. licensing transaction for all products containing lesinurad and support of the anticipated launch of FDA-approved ZURAMPIC in October 2016, Ironwood is updating its guidance for 2016. Ironwood expects:

R&D expenses to fall within a range of $140 million to $150 million, (previously $130 million to $145 million),
SG&A expenses to fall within a range of $170 million to $180 million, (previously $125 million to $140 million), and
Amortization of intangible assets to be $8 million (not applicable prior to the U.S. lesinurad license).
Consistent with its guidance following the announcement of the U.S. lesinurad license, Ironwood continues to expect to use less than $70 million in cash for operations in 2016.

Allergan and Ironwood continue to expect total 2016 marketing and sales expenses for LINZESS to be in the range of $230 million to $260 million, and the companies now expect these expenses to be in the mid to higher end of this range.

Non-GAAP Financial Measures

The company presents non-GAAP net loss and non-GAAP net loss per share to exclude the impact of net gains and losses on the derivatives related to our convertible notes that are required to be marked-to-market, and the amortization of acquired intangible assets. The derivative gains and losses may be highly variable, difficult to predict and of a size that could have a substantial impact on the company’s reported results of operations in any given period. The acquired intangible assets are valued at the time of acquisition and are amortized over their estimated economic useful life, and management believes excluding the amortization of acquired intangible assets provides more consistency with internally developed intangible assets for which research and development costs were previously expensed. The company has presented non-GAAP net loss and non-GAAP net loss per share in prior calendar quarters, and this is the first calendar quarter in which the company has amortization of acquired intangible assets that can be excluded from such non-GAAP financial measures. Management believes this non-GAAP information is useful for investors, taken in conjunction with Ironwood’s GAAP financial statements, because it provides greater transparency and period-over-period comparability with respect to Ironwood’s operating performance. These measures are also used by management to assess the performance of the business. Investors should consider these non-GAAP measures only as a supplement to, not as a substitute for or as superior to, measures of financial performance prepared in accordance with GAAP. In addition, these non-GAAP financial measures are unlikely to be comparable with non-GAAP information provided by other companies. For a reconciliation of these non-GAAP financial measures to the most comparable GAAP measures, please refer to the table at the end of this press release.

On August 4, 2016 Radius Health, Inc. ("Radius" or the "Company") (Nasdaq:RDUS), a science-driven biopharmaceutical company that is committed to developing innovative therapeutics in the areas of osteoporosis, oncology and endocrine diseases, reported its financial results for the second quarter ended June 30, 2016, and provided a business update (Press release, Radius, AUG 4, 2016, View Source [SID:1234514295]). As of June 30, 2016, Radius had $400.9 million in cash, cash equivalents and marketable securities.

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"We are pleased to be working with the U.S. Food and Drug Administration and European Medicines Agency as they review our regulatory submissions for abaloparatide-SC for the treatment of postmenopausal osteoporosis. In anticipation of our first potential launch, we are building our commercial organization in the U.S. and continuing our productive partnering discussions," said Robert Ward, President and Chief Executive Officer of Radius. "At the same time, we continue to make steady progress in the development of an optimized transdermal patch line extension for abaloparatide as well as the advancement of our two oncology programs, both of which we believe can add substantial value to Radius."

Pipeline Updates

Abaloparatide-SC

In May 2016, Radius’ new drug application ("NDA") in the United States for abaloparatide-SC for the treatment of postmenopausal women with osteoporosis was accepted for filing by the FDA and was granted a Prescription Drug User Fee Act (PDUFA) date of March 30, 2017. Radius’ marketing authorisation application ("MAA") to the European Medicines Agency ("EMA"), was validated in December 2015 and is currently undergoing regulatory review. We anticipate a CHMP scientific opinion in late 2016 or 2017.

Abaloparatide-TD

Radius is developing abaloparatide-transdermal, or abaloparatide-TD, based on 3M’s patented Microstructured Transdermal System technology for potential use as a short wear-time transdermal patch. Radius commenced a human replicative clinical evaluation of the optimized abaloparatide-TD patch in December 2015 with the goal of achieving comparability to abaloparatide-SC. An abstract submitted to the American Society for Bone Mineral Research 2016 Annual Meeting (ASBMR) was accepted as an oral late-breaking presentation to be made on September 19, 2016 in Atlanta, Georgia.

RAD1901

In December 2014, Radius commenced a Phase 1 multicenter, open-label, two-part, dose-escalation study of RAD1901 in postmenopausal women with estrogen receptor positive (ER+) and HER2-negative advanced breast cancer in the United States. The Phase 1 study is designed to evaluate escalating doses of RAD1901 in Part A. The Part B expansion cohorts allow for an evaluation of additional safety, tolerability and preliminary efficacy. In addition, in December 2015, Radius commenced a Phase 1 FES-PET study in patients with ER+, HER2-negative advanced breast cancer in the European Union, which includes the use of FES-PET imaging to assess estrogen receptor occupancy in tumor lesions following RAD1901 treatment.

As of the end of July, the Phase I Part B expansion cohort for RAD1901 that initiated in March 2016 at 400 mg daily in ER+, HER2-negative advanced breast cancer has enrolled 19 out of 20 patients. We continue to enroll patients in the European Phase I FES-PET trial — the first three-patient dosing cohort is enrolled. We are pleased with the progress across these trials and expect to provide an update on the RAD1901 program at an upcoming scientific meeting.

RAD140

We have reported that RAD140 in preclinical xenograft models of breast cancer has demonstrated potent tumor growth inhibition when administered alone or in combinations with CDK4/6 inhibitors. It is estimated that 77% of breast cancers show expression of the androgen receptor. Our preclinical data suggest that RAD140 activity at the androgen receptor stimulates up-regulation of a tumor suppression pathway. We expect to provide an update on the RAD140 program at an upcoming scientific meeting.

Radius Expects the Following Upcoming Milestones

Abaloparatide-SC
Receive opinion from the Committee for Medicinal Products for Human Use regarding the EMA’s review of the abaloparatide-SC MAA in late 2016 or 2017
FDA PDUFA date of March 30, 2017
Enter into a collaboration for the potential commercialization of abaloparatide-SC outside the U.S. prior to commercial launch
Three abstracts accepted for poster presentations at ASBMR from the Phase 3 ACTIVE clinical trial
Abaloparatide-TD
Oral late-breaker presentation of the results of the human replicative PK pilot study of an optimized abaloparatide transdermal patch at ASBMR, on September 19, 2016 in Atlanta, Georgia
RAD1901
Expect to provide an update on the RAD1901 program at an upcoming scientific meeting.
RAD140
Expect to provide an update on the RAD140 program at an upcoming scientific meeting.
Radius Expects To Make Presentations at the Following Upcoming Conferences

Radius President and CEO, Robert E. Ward will make a presentation and company management will host one-on-ones at the Canaccord Genuity Growth Conference, August 10, 2016 at the InterContinental Hotel in Boston, MA.
Radius President and CEO, Robert E. Ward, will make a presentation and company management will host one-on-ones at the Rodman & Renshaw 18th Annual Global Investment Conference at the Lotte New York Palace Hotel in New York on September 12.
Four abstracts for abaloparatide were accepted for presentation at ASBMR September 16-19, 2016 in Atlanta, Georgia. Three data presentations are from the Phase 3 ACTIVE trial for abaloparatide-SC, and the fourth is an oral presentation in the Late-Breaking Abstracts session from the pilot PK human replicative study of an optimized transdermal patch titled: "Clinical Development of an Optimized Abaloparatide Transdermal Patch" on September 19, 2016 at 11:36 AM — 11:48 AM.
Recent Corporate Highlights

In July 2016, Radius entered into a pre-clinical collaboration with Takeda Pharmaceutical Company Limited to evaluate the combination of investigational drug RAD1901 with investigational drug TAK-228, an oral mTORC 1/2 inhibitor in Phase 2b development for the treatment of breast, endometrial and renal cancer, with the goal of potentially exploring such combination in a clinical study.

In June, Radius announced the opening of its Wayne, Pennsylvania office, which will serve as the Company’s Commercial and Medical hub and house marketing, sales, human resources, IT, pharmacovigilance, health economics, outcomes research, medical education, clinical affairs and medical affairs staff.

In May, Radius announced that its New Drug Application (NDA) for abaloparatide—SC had been accepted for filing by the U.S. Food and Drug Administration (FDA). The acceptance of the NDA reflects the FDA’s determination that the application is sufficiently complete to permit a substantive review. A PDUFA date of March 30, 2017 has been assigned.
Abaloparatide-SC as a treatment for postmenopausal women with osteoporosis is an investigational product and its safety and efficacy have not been established.

Second Quarter 2016 Financial Results

For the three months ended June 30, 2016, Radius reported a net loss of $43.4 million, or $1.01 per share, as compared to a net loss of $23.0 million, or $0.61 per share for the three months ended June 30, 2015. The increase in net loss for the three months ended June 30, 2016 as compared to the three months ended June 30, 2015 was primarily due to an increase in research and development and general and administrative expenses, partially offset by a decrease in interest expense and an increase in interest income.

Research and development expenses for the three months ended June 30, 2016 were $26.9 million, compared to $16.3 million for the same period in 2015. This increase was primarily driven by higher professional contract services costs associated with the development of RAD1901 to support a Phase 1 study in metastatic breast cancer that commenced in late 2014 and a Phase 2b study in postmenopausal vasomotor symptoms that commenced in December 2015. This increase was also a result of an increase in compensation expense, including stock-based compensation, due to an increase in headcount from June 30, 2015 to June 30, 2016.

General and administrative expenses for the three months ended June 30, 2016 were $17.2 million, compared to $6.0 million for the same period in 2015. This increase was primarily attributable to an increase in professional support costs and legal fees, including the costs associated with increasing headcount and preparing for the potential commercialization of abaloparatide-SC, subject to a favorable regulatory review.
This increase was also driven by an increase in compensation expense due to an increase in headcount from June 30, 2015 to June 30, 2016.

As of June 30, 2016, Radius had $400.9 million in cash, cash equivalents and marketable securities. Based upon Radius’ cash, cash equivalents and marketable securities balance, Radius believes that, prior to the consideration of revenue from the potential future sales of any of its investigational products that may receive regulatory approval or proceeds from collaboration activities, it has sufficient capital to fund its development plans, U.S. commercial scale-up and other operational activities into 2018.

On August 4, 2016 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and reported financial results for the second quarter ending June 30, 2016 (Press release, PTC Therapeutics, AUG 4, 2016, View Source [SID:1234514293]).

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"Making Translarna available to all Duchenne muscular dystrophy patients globally who may benefit continues to be our top priority," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "Global sales are on a strong growth trajectory as we continue to expand access outside of the U.S. We are excited patients in England can now access reimbursed Translarna and have received our first commercial order in England following NICE’s final guidance recommending Translarna. On the regulatory front, we are optimistic that our interactions with the EMA will support the renewal of Translarna’s marketing authorization coupled with an obligation to conduct an agreed upon clinical trial, and we look forward to our next steps in the appeal process of the FDA’s Refuse to File letter."

Key Second Quarter and Other Corporate Highlights:

Translarna revenue of $15.4M in second quarter represents 150% year-over-year growth. PTC continues to grow its global commercial footprint and expand access to Translarna. In addition to Europe, PTC is now providing Translarna to patients on a commercial basis in the Middle East and Latin America.
NICE issues final guidance recommending Translarna for patients in England. The National Institute for Health and Care Excellence (NICE) issued final guidance recommending Translarna for the treatment of ambulatory patients aged five years and older with nmDMD in England in connection with a Managed Access Agreement (MAA) with National Health Services (NHS) England. As part of the MAA, NHS England is waiving the typical three-month implementation period under local regulation, making Translarna immediately available to patients in England.
EMA review of European marketing authorization for Translarna continues. Over the last several months, PTC has been engaged in constructive discussions with the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) regarding the renewal of Translarna’s marketing authorization. The company has been informed that the renewal assessment procedure cannot be completed by mid-year 2016. The CHMP has agreed to the proposal by PTC to submit a draft clinical trial protocol for further discussion, which includes seeking scientific advice from the EMA. The company is optimistic that these interactions will support the renewal of the marketing authorization of Translarna coupled with an obligation to conduct an agreed upon clinical trial.
PTC has submitted an appeal to the FDA via the formal dispute resolution process. PTC has appealed the Refuse to File (RTF) letter issued on February 22, 2016 with respect to the company’s New Drug Application (NDA) for Translarna for the treatment of nmDMD. This process elevates the discussion to the next level of FDA management and exists to encourage open, prompt discussion of scientific and procedural disputes that arise during the drug development process between FDA and companies.
ACT CF Phase 3 clinical trial remains on track while EMA review of cystic fibrosis filing for Translarna is ongoing. PTC’s confirmatory Phase 3 ACT CF trial is currently ongoing with the results from this trial expected in early 2017. The EMA is currently reviewing the company’s variation submission to Translarna’s marketing authorization, which requests EMA approval for the treatment of nonsense mutation cystic fibrosis (nmCF). Based on recent interactions with the CHMP, PTC no longer anticipates that the CHMP will issue its opinion regarding this submission in 2016 and believes that ACT CF results may be required prior to any approval in this indication.
Phase 2 Clinical Trial of Translarna for nmDMD in pediatric patients initiated. PTC has initiated a Phase 2 clinical trial of Translarna for the treatment of nmDMD in patients between the ages of two and five years of age. This open-label, multiple-dose study will evaluate the safety and pharmacokinetics of Translarna in pediatric patients and will support the potential expansion of Translarna’s label to younger patients.
Second Quarter Financial Highlights:

Translarna net product sales were $15.4 million for the second quarter of 2016, representing a 150% increase versus $6.2 million in the second quarter of 2015. The increase in sales is a result from the continued global commercial expansion and access to Translarna. Translarna net product sales decreased sequentially from $18.9 million reported in the first quarter of 2016. In the first quarter, Translarna net product sales were positively impacted by a significant order from Brazil, where purchasing is often fulfilled in national bulk orders. The effect of larger but less frequent orders from Brazil may result in fluctuations in quarterly sales reporting during the course of the year.
Total revenues for the second quarter of 2016 were $15.6 million compared to $6.8 million in the same period of 2015. The change in total revenue was a result of the expanded commercial launch of Translarna, partially offset by lower grant revenue.
GAAP R&D expenses were relatively flat at $28.8 million for the second quarter of 2016 compared to $28.2 million for the same period in 2015. Non-GAAP R&D expenses were $24.7 million for the second quarter of 2016, excluding $4.1 million in non-cash, stock-based compensation expense, compared to $24.2 million for the same period in 2015, excluding $4.0 million in non-cash, stock-based compensation expense.
GAAP SG&A expenses were $23.4 million for the second quarter of 2016 compared to $17.2 million for the same period in 2015. Non-GAAP SG&A expenses were $18.7 million for the second quarter of 2016, excluding $4.6 million in non-cash, stock-based compensation expense, compared to $12.8 million for the same period in 2015, excluding $4.4 million in non-cash, stock-based compensation expense. The increase in SG&A expense for the second quarter 2016 as compared to the prior year period primarily resulted from additional costs associated with commercial activities in support of Translarna across Europe and other regions.
Net interest expense for the second quarter of 2016 was $2.1 million compared to net interest income of $0.5 million in the same period in 2015. The increase in interest expense is primarily a result of the $150 million convertible debt offering completed during the third quarter 2015. The debt was recorded on PTC’s balance sheet at a discount, which will be amortized over the life of the bond.
Net loss for the second quarter of 2016 was $38.9 million compared to a net loss of $38.4 million for the same period in 2015.
During the first quarter of 2016, PTC announced a workforce reduction of approximately 18% of its employees and contractors, which resulted in a one-time charge of approximately $2.5 million. PTC incurred $0.6 million of this charge in the second quarter.
Cash, cash equivalents, and marketable securities totaled approximately $273 million at June 30, 2016 compared to approximately $339 million at December 31, 2015.
Shares issued and outstanding as of June 30, 2016 were 34.3 million, which includes 0.2 million shares of unvested restricted stock.
2016 Guidance:

Total ex-U.S. Translarna nmDMD revenues for 2016 are anticipated to be between $65 and $85 million. This guidance assumes current exchange rates and the continued commercial expansion for Translarna in nmDMD outside of the U.S.
Operating expenses for the full year 2016 are anticipated to be between $185 million and $195 million, excluding expected non-cash stock-based compensation expense of approximately $40 million, for total operating expenses of approximately $225 million to $235 million. These expenses will be primarily in support of our ongoing clinical trials for Translarna in nmDMD and nmCF, commercial launch activities for Translarna outside of the US, and the continued research and clinical development of other product pipeline candidates.
PTC expects to end 2016 with cash and cash equivalents over $200 million.
Non-GAAP Financial Measures

In this press release, PTC’s financial results and financial guidance are provided in accordance with accounting principles generally accepted in the United States (GAAP) and using certain non-GAAP financial measures. In particular, non-GAAP financial results exclude stock-based compensation expense. These results are provided as a complement to results reported in GAAP, because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management’s opinion, these non-GAAP measures are useful to investors and other users of our financial statements by providing greater transparency into the operating performance at PTC and the company’s future outlook.

PTC Therapeutics, Inc.

Consolidated Statements of Operations

(In thousands, except per share data)

Three Months Ended

Six Months Ended

June 30,

June 30,

2016

2015

2016

2015

Revenues:

Net product revenue
$15,437

$6,161

$34,314

$11,230

Collaboration and grant revenue
196

613

214

3,026

Total revenues
15,633

6,774

34,528

14,256

Operating expenses:

Research and development (1)
28,827

28,190

60,226

56,128

Selling, general and administrative (2)
23,366

17,210

49,304

34,825

Total operating expenses
52,193

45,400

109,530

90,953

Loss from operations
(36,560)

(38,626)

(75,002)

(76,697)

Interest (expense) income, net
(2,060)

498

(4,016)

1,022

Other (expense) income, net
(387)

(88)

(1,107)

(456)

Loss before income tax expense
(39,007)

(38,216)

(80,125)

(76,131)

Income tax benefit (expense)
93

(145)

(22)

(145)

Net loss
($38,914)

($38,361)

($80,147)

($76,276)

Weighted-average shares outstanding (in shares):

Basic and diluted
34,000,333

33,600,653

33,959,751

33,335,674

Net loss per share – basic and diluted (in dollars per share)
($1.14)

($1.14)

($2.36)

($2.29)

(1) Research and development reconciliation

GAAP research and development
$28,827

$28,190

$60,226

$56,128

Less stock-based compensation expense
4,087

3,957

8,415

8,624

Non-GAAP research and development
$24,740

$24,233

$51,811

$47,504

(2) Selling, general and administrative reconciliation

GAAP selling, general and administrative
$23,366

$17,210

$49,304

$34,825

Less stock-based compensation expense
4,649

4,371

9,236

9,452

Non-GAAP selling, general and administrative
$18,717

$12,839

$40,068

$25,373

PTC Therapeutics, Inc.
Summary Consolidated Balance Sheet
(In thousands, except share amounts)

June 30,

December 31,

2016

2015
Cash, cash equivalents and marketable securities
$272,893

$338,925
Total assets
$305,563

$365,281

Total debt
$94,936

$91,848
Total deferred revenue
726

139
Total liabilities
$139,939

$139,280

Total stockholders’ equity (34,083,319 and 33,916,559 common shares

issued and outstanding at June 30, 2016 and December 31, 2015, respectively)
165,624

226,001
Total liabilities and stockholders’ equity
$305,563

$365,281
Upcoming Events:

PTC will participate in the following upcoming conference:

2016 Wedbush PacGrow Healthcare Conference, August 17th at 1:20 p.m. in New York, NY
The presentation will be webcast live on the Events and Presentations page under the investor relations section of PTC’s website at www.ptcbio.com and will be archived for two weeks following the presentation. PTC’s current investor presentation is available at the same website location.

On August 4, 2016 Progenics Pharmaceuticals, Inc. (Nasdaq:PGNX) reported financial and business results for the second quarter 2016 (Press release, Progenics Pharmaceuticals, AUG 4, 2016, View Source [SID:1234514291]).

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"The clear highlight in recent weeks was the approval of RELISTOR tablets, which triggered a $50 million milestone payment from our partner Valeant, strengthening our balance sheet as we prepare for a potential AZEDRA launch and continue to advance development of our prostate cancer pipeline," said Mark Baker, Chief Executive Officer of Progenics. "Our cash, together with the potential to earn significant additional royalties and sales milestones from the RELISTOR franchise, puts us in a strong position to achieve multiple value-creating milestones. In particular, our ultra-orphan radiotherapeutic candidate AZEDRA represents a meaningful near-term commercialization opportunity for Progenics, and we are on track to announce registrational topline data later this year or in early 2017. We are also advancing development of our portfolio of prostate cancer imaging agents and therapeutics, which has the potential to significantly improve how we find, fight, and follow all stages of prostate cancer."

Key Business Highlights

RELISTOR, treatment for opioid-induced constipation (partnered with Valeant Pharmaceuticals International, Inc.)

On July 19, the Company Announced that the FDA has approved RELISTOR tablets for the treatment of opioid induced constipation in adults with chronic non-cancer pain. The approval triggered a $50 million milestone payment on July 25 from Progenics’ partner, Valeant, as well as subsequent royalties and up to $200 million in sales milestones.

RELISTOR SC Net Sales for the Second Quarter 2016 Total $15.9 Million. The second quarter 2016 sales, as reported to the Company by Valeant, translated to $2.4 million in royalty revenue for the second quarter of 2016.
AZEDRA, Ultra-orphan radiotherapeutic candidate

AZEDRA Topline Results Expected Between December 2016 and March 2017. In late 2016 or early 2017, Progenics expects to report topline results from its ongoing registrational trial of AZEDRA. If the AZEDRA trial meets the endpoints of the SPA, the Company expects to submit an NDA to the FDA during the first half of 2017.
PSMA-Targeted Prostate Cancer Pipeline

Granted an Exclusive License to Bayer for the Development and Commercialization of Therapeutic Antibodies Combining Progenics’ PSMA Antibody Technology with Bayer’s Targeted Thorium Conjugate Technology. Progenics recognized revenue of $5 million in the second quarter of 2016, constituting the $4 million upfront payment and the first pre-clinical development milestone of $1 million. Under terms of the agreement, the Company is entitled to up to an additional $48 million in potential clinical and regulatory development milestones, single digit royalties on net sales, and potential sales milestone payments up to an aggregate of $130 million.

Enrollment in Pivotal Phase 3 Study of 1404 is Ongoing. The study will enroll up to 450 patients with newly-diagnosed or low-grade prostate cancer who are candidates for active surveillance. The Company’s plans for an interim analysis during the fourth quarter of 2016 to assess futility and evaluate the need for a sample size re-estimation remain unchanged.

Presented Data from its PSMA-Targeted Prostate Cancer Imaging Programs at the Society of Nuclear Medicine and Molecular Imaging 2016 Annual Meeting in San Diego. The data highlighted the potential of the Company’s SPEC/CT imaging agent 1404 and PET/CT imaging agent PyL to detect prostate cancer.

On-Track to Initiate Phase 2/3 Trial of PyL by Year-End. The study is designed to assess the diagnostic accuracy of PyL PET/CT imaging in patients with high risk and/or metastatic prostate cancer.

Company Remains On-Track to Initiate a Phase 1 Trial of 1095 in the Fourth Quarter of 2016. The Phase 1 Study of 1095, a PSMA-Targeted Therapeutic for Metastatic Prostate Cancer, will be conducted at Memorial Sloan Kettering Cancer Center.
Second Quarter 2016 Financial Results

Net loss attributable to Progenics for the quarter was $5.6 million or $0.08 per diluted share, compared to a net loss of $11.7 million or $0.17 per diluted share in the 2015 period. Progenics ended the quarter with cash and cash equivalents of $60.1 million, a decrease of $5.5 million in the quarter.

Second quarter revenue totaled $8.5 million, up from $1.9 million in 2015, reflecting RELISTOR royalty income of $2.4 million compared to $1.8 million in the 2015 period, based on net sales reported to Progenics by Valeant. The increase was primarily attributable to upfront and milestone revenue of $5 million under the Bayer license agreement.

Second quarter and year-to-date research and development expenses increased by $1.3 million and $3.9 million, respectively, compared to the prior year periods, resulting from higher clinical trial and contract manufacturing expenses for AZEDRA, 1404 and PyL, partially offset by lower expenses for PSMA ADC. Second quarter general and administrative expenses decreased by $0.5 million from the prior year period, primarily attributable to lower legal fees as the prior year included costs related to litigation with a former employee. Year-to-date general and administrative expenses increased by $1.6 million compared to prior year period, primarily resulting from higher depreciation expense as a result of a reduction in the remaining useful lives of our leasehold improvements at our Tarrytown, NY location, and higher compensation and consulting expenses. The Company also recorded a non-cash charge of $0.6 million in the second quarter related to an increase in the fair value estimate of the contingent consideration liability.