On May 05, 2016 Threshold Pharmaceuticals, Inc. (Nasdaq:THLD), a clinical-stage biopharmaceutical company developing novel therapies for cancer, reported financial results for the first quarter ended March 31, 2016 and provided an update on the Company’s corporate and clinical development activities (Press release, Threshold Pharmaceuticals, MAY 5, 2016, View Source [SID:1234512061]).

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"We remain focused on establishing a potential regulatory path forward for evofosfamide as well as a possible strategic partnering initiative, and we are making progress on both of these fronts," said Barry Selick, Ph.D., Chief Executive Officer of Threshold. "I am also pleased with the appointment of Stew Kroll as Chief Operating Officer who most capably leads the strategy, design and execution of our clinical development programs."

Recent Highlights
Evofosfamide – The Company’s lead product candidate is an investigational hypoxia-activated prodrug that is designed to be activated under tumor hypoxic conditions, a hallmark of many cancers. Additional analysis of the MASTRO Phase 3 data combined with previous Phase 2 experience strongly suggests that evofosfamide plus gemcitabine is an active regimen in patients with pancreatic cancer, most notably in the Japanese patients.

Conducted additional analyses of evofosfamide data in pancreatic cancer; the Company intends to discuss potential registration pathways with health regulatory authorities; and
Continued ongoing clinical development collaborations investigating evofosfamide in patients with pancreatic neuroendocrine tumors (pNET), recurrent glioblastoma (GBM) and hepatocellular carcinoma (HCC).
Tarloxotinib – Beyond the Company’s evofosfamide program, the clinical development team is focused on tarloxotinib, a hypoxia-activated epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor (TKI), which is designed to selectively release an irreversible EGFR-TKI in hypoxic tumors.

Continued to enroll patients in two proof-of-concept Phase 2 clinical trials in patients with advanced non-small cell lung cancer (NSCLC) as well as metastatic head and neck squamous cell carcinoma; the Company plans to share preliminary results from both trials in mid-2016.
First Quarter 2016 Financial Results

Cash, cash equivalents and marketable securities totaled $38.0 million at March 31, 2016 compared to $48.7 million at December 31, 2015; the net decrease was a result of operating cash requirements for the quarter ended March 31, 2016, including the payment of $2.3 million of accrued severance benefits related to the previously announced workforce reduction in December of 2015. With the previously announced decision to cease joint development of evofosfamide under the Company’s former collaboration with Merck KGaA and the workforce reduction, the Company expects its quarterly operating cash requirements to decrease for the remainder of fiscal year 2016 compared to the first quarter ended March 31, 2016.

No revenue was recognized in the first quarter ended March 31, 2016 compared to $3.7 million for the same period of 2015. Revenue for the quarter ended March 31, 2015 related to the amortization of the aggregate of $110 million in upfront and milestone payments received from the Company’s former collaboration with Merck KGaA, Darmstadt, Germany. The revenue from the upfront payment and milestone payments received under the agreement were previously being amortized over the relevant performance period, rather than being immediately recognized when the upfront payment and milestones were earned or received. As a result of Merck KGaA, Darmstadt, Germany’s and the Company’s decision to cease further joint development of evofosfamide in December 2015, the Company immediately recognized all of the remaining deferred revenue into revenue during the quarter ending December 31, 2015. Also as a result of the termination of the agreement, the Company is no longer eligible to receive any further milestone payments from Merck KGaA, Darmstadt, Germany.

Research and development expenses were $6.0 million for the first quarter ended March 31, 2016, compared to $10.7 million for the same period in 2015. The decrease in research and development expenses, net of reimbursement for Merck KGaA, Darmstadt, Germany’s 70 percent share of total eligible collaboration expenses for evofosfamide, was due primarily to a $3.1 million decrease in employee related expenses, including a $0.5 million decrease in non-cash stock-based compensation expense and a $1.6 million decrease in clinical development expenses and consulting expenses. The Company expects research and development expenses to continue to decline in 2016 as result of the decision to cease further joint development of evofosfamide under the Company’s former collaboration with Merck KGaA and the workforce reduction.

General and administrative expenses were $2.2 million for the first quarter of 2016 compared to $2.6 million for the same period in 2015. The decrease in general and administrative expenses was due primarily to a $0.2 million decrease in consulting expenses and a $0.2 million decrease in employee related expenses.

Non-cash stock-based compensation expense included in total operating expenses was $0.8 million for the first quarter of 2016 compared to $1.4 million for the same period in 2015. The decrease in stock-based compensation expense was due to the amortization of a smaller number of options with lower fair values.

Net loss for the first quarter of 2016 was $7.9 million compared to $11.2 million for the same period in 2015. Included in the net loss for the first quarter of 2016 was an operating loss of $8.3 million and non-cash income of $0.4 million compared to an operating loss of $9.6 million and non-cash expense of $1.5 million for the first quarter of 2015. The non-cash income or expense is related to changes in the fair value of the Company’s outstanding and exercised warrants that was classified as other income (expense).
About Evofosfamide
Evofosfamide (previously known as TH-302) is an investigational hypoxia-activated prodrug of a bis-alkylating agent that is preferentially activated under severe hypoxic tumor conditions, a feature of many solid tumors. Areas of low oxygen levels (hypoxia) in solid tumors are due to insufficient blood vessel supply. Similarly, the bone marrow of patients with hematological malignancies has also been shown, in some cases, to be severely hypoxic. On December 6, 2015, the Company announced the outcomes of two Phase 3 studies (MAESTRO and TH-CR-406/SARC021) of evofosfamide stating that neither study met its primary endpoint.

About Tarloxotinib Bromide
Tarloxotinib bromide (the proposed International Nonproprietary Name, previously known as TH-4000), or "tarloxotinib", is a prodrug designed to selectively release a covalent (irreversible) EGFR tyrosine kinase inhibitor under severe hypoxia, a feature of many solid tumors. Accordingly, tarloxotinib has the potential to effectively shut down aberrant EGFR signaling in a tumor-selective manner, thus potentially avoiding or reducing the systemic side effects associated with currently available EGFR tyrosine kinase inhibitors. Tarloxotinib is currently being evaluated in two Phase 2 proof-of-concept trials: one for the treatment of patients with mutant EGFR-positive, T790M-negative advanced non-small cell lung cancer progressing on an EGFR tyrosine kinase inhibitor, and the other for patients with recurrent or metastatic squamous cell carcinomas of the head and neck or skin. Threshold licensed exclusive worldwide rights to tarloxotinib from the University of Auckland, New Zealand, in September 2014.

On May 5, 2016 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported financial results for the first quarter of 2016 and provided a business update (Press release, Regeneron, MAY 5, 2016, View Source [SID:1234512060]).

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Financial Highlights

($ in millions, except per share data)

Three Months Ended
March 31,

2016

2015

% Change
EYLEA U.S. net product sales

$
781

$
541

44%
Total revenues

$
1,201

$
870

38%
Non-GAAP net income(2)

$
293

$
336

(13%)
Non-GAAP net income per share – diluted(2)

$
2.57

$
2.88

(11%)
GAAP net income

$
166

$
76

118%
GAAP net income per share – diluted

$
1.45

$
0.66

120%

"The year is off to a very productive start at Regeneron. This quarter, we saw continued strong sales growth with EYLEA, made additional launch progress with Praluent, prepared for the potential launch of sarilumab, and reported important new data across our pipeline," said Leonard S. Schleifer, M.D., Ph.D., President and Chief Executive Officer of Regeneron. "Our innovative new therapy dupilumab showed positive results across two Phase 3 trials in moderate-to-severe atopic dermatitis, a debilitating disease with very limited treatment options, and we look forward to submitting a Biologics License Application to the U.S. FDA in the third quarter."

Business Highlights

Marketed Product Update

EYLEA (aflibercept) Injection for Intravitreal Injection

In the first quarter of 2016, net sales of EYLEA in the United States increased 44% to $781 million from $541 million in the first quarter of 2015. Overall distributor inventory levels remained within the Company’s one- to two-week targeted range.
Bayer commercializes EYLEA outside the United States. In the first quarter of 2016, net sales of EYLEA outside of the United States(1) were $419 million, compared to $292 million in the first quarter of 2015. In the first quarter of 2016, Regeneron recognized $146 million from its share of net profit from EYLEA sales outside the United States, compared to $89 million in the first quarter of 2015.
A Phase 3 study of EYLEA for the treatment of non-proliferative diabetic retinopathy in patients without diabetic macular edema (DME) was initiated in the first quarter of 2016.
Praluent (alirocumab) Injection for the Treatment of High Low-Density Lipoprotein (LDL) Cholesterol

In the first quarter of 2016, net sales of Praluent were $13 million. Product sales for Praluent are recorded by Sanofi, and the Company shares in any profits or losses from the commercialization of Praluent. Praluent was launched in the United States in the third quarter of 2015 and in certain countries in the European Union commencing in the fourth quarter of 2015.
In March 2016, the Company and Sanofi reported data from the Phase 3 ODYSSEY ESCAPE study in patients with heterozygous familial hypercholesterolemia (HeFH) who were undergoing LDL apheresis therapy. The trial achieved its primary endpoint, demonstrating that patients who added Praluent to their existing treatment regimen significantly reduced the frequency of their apheresis therapy by 75%, compared to placebo.
In the first quarter of 2016, the Data Monitoring Committee (DMC) of the ODYSSEY OUTCOMES study for Praluent completed the first interim analysis. In accordance with the protocol, the DMC performed a futility assessment. The DMC recommended the study continue with no changes. Regeneron remains blinded to the actual results of this analysis. The ongoing ODYSSEY OUTCOMES trial is assessing the potential of Praluent to demonstrate cardiovascular benefit.
Pipeline Progress

Regeneron has thirteen product candidates in clinical development. These consist of EYLEA and twelve fully human monoclonal antibodies generated using the Company’s VelocImmune technology, including four in collaboration with Sanofi. In addition to EYLEA and Praluent, highlights from the antibody pipeline include:

Sarilumab, the Company’s antibody targeting IL-6R for rheumatoid arthritis, is currently being studied in the global Phase 3 SARIL-RA program.

In March 2016, the Company and Sanofi reported results from the 24-week Phase 3 SARIL-RA-MONARCH study in adult patients with active rheumatoid arthritis who were inadequate responders to, intolerant of, or inappropriate candidates for methotrexate (MTX) therapy. The study met its primary endpoint, demonstrating that sarilumab monotherapy was superior to adalimumab monotherapy (marketed by AbbVie Inc. as HUMIRA).
In December 2015, the U.S. Food and Drug Administration (FDA) accepted for review a Biologics License Application (BLA) for sarilumab, with a target action date of October 30, 2016.
Dupilumab, the Company’s antibody that blocks signaling of IL-4 and IL-13, is currently being studied in atopic dermatitis, asthma, nasal polyps, and eosinophilic esophagitis.

In April 2016, the Company and Sanofi reported that the Phase 3 LIBERTY AD SOLO 1 and SOLO 2 trials evaluating dupilumab in adult patients with inadequately controlled moderate-to-severe atopic dermatitis met their primary endpoints.
A Phase 2 study of dupilumab in pediatric patients (6-17 years of age) with moderate-to-severe atopic dermatitis is fully enrolled and ongoing.
A Phase 3 pivotal study of dupilumab in patients with uncontrolled persistent asthma continues to enroll patients.
A Phase 2 study of dupilumab in eosinophilic esophagitis is ongoing.
Fasinumab, the Company’s antibody targeting Nerve Growth Factor (NGF), is currently being studied in patients with pain due to osteoarthritis and lower back pain.

The Company recently reported results from a Phase 2/3 study evaluating fasinumab in patients with moderate-to-severe osteoarthritis pain of the hip or knee who have a history of inadequate pain relief or intolerance to current analgesic therapies. The study met its primary endpoint at 16 weeks.
In the first quarter of 2016, the Company initiated a Phase 3 long-term safety and efficacy study of fasinumab in patients with pain due to osteoarthritis of the knee or hip, and this trial is currently enrolling patients.
In the first quarter of 2016, the Company also initiated a Phase 2b/3 study of fasinumab in chronic lower back pain.
REGN2810, an antibody to programmed cell death protein 1 (PD-1), entered a potentially pivotal clinical study for the treatment of advanced cutaneous squamous cell carcinoma in the second quarter of 2016.

Nesvacumab/aflibercept, a combination product comprised of an antibody to angiopoietin-2 (Ang2) co-formulated with aflibercept for intravitreal injection for use in ophthalmology, entered Phase 2 clinical development for the treatment of neovascular age-related macular degeneration (wet AMD) and DME in the first quarter of 2016.

Evinacumab, an antibody to Angptl-3, was granted orphan-drug designation by the FDA in the first quarter of 2016. Clinical studies are ongoing for the treatment of homozygous familial hypercholesterolemia and severe forms of hyperlipidemia.

Select Upcoming 2016 Milestones

Clinical Programs

Milestones
REGN2176-3 (PDGFR-beta
Antibody co-formulated with
aflibercept)
•
Report results from Phase 2 study

Praluent
•
DMC interim analysis of ODYSSEY OUTCOMES trial
•
Ongoing launch in additional countries

Sarilumab (IL-6R Antibody)
•
FDA target action date of October 30, 2016
•
File for regulatory approvals outside the United States

Dupilumab (IL-4R Antibody)
•
Report primary endpoint results from Phase 3
CHRONOS study in atopic dermatitis
•
Complete rolling BLA submission for atopic dermatitis in
the United States
•
Initiate Phase 3 study in pediatric patients in atopic
dermatitis

REGN2810 (PD-1 Antibody)
•
Report data from Phase 1 study in patients with cancer
Human Genetics Initiative

In the first quarter of 2016, the New England Journal of Medicine published a Regeneron Genetics Center paper showing that inactivating mutations of the angiopoeitin-like 4 (ANGPTL4) gene are associated with a significantly reduced risk of coronary artery disease in humans. ANGPTL4 and ANGPTL3 are thought to be related inhibitors of lipoprotein lipase (LPL).
Business Development Update

In March 2016, the Company and Bayer entered into a collaboration agreement to jointly develop a combination therapy of the Ang2 antibody nesvacumab and aflibercept for the treatment of serious eye diseases.
In April 2016, the Company and Intellia Therapeutics, Inc. entered into a license and collaboration agreement to advance CRISPR/Cas gene-editing technology for in vivo therapeutic development. In addition to the discovery, development and commercialization of new therapies, the companies will focus on technology development of the CRISPR/Cas platform.
First Quarter 2016 Financial Results

Product Revenues: Net product sales were $784 million in the first quarter of 2016, compared to $545 million in the first quarter of 2015. EYLEA net product sales in the United States were $781 million in the first quarter of 2016, compared to $541 million in the first quarter of 2015.

Total Revenues: Total revenues, which include product revenues described above, increased by 38% to $1.201 billion in the first quarter of 2016, compared to $870 million in the first quarter of 2015. Total revenues also include collaboration revenues of $399 million in the first quarter of 2016, compared to $297 million in the first quarter of 2015. Collaboration revenues in the first quarter of 2016 increased primarily due to higher reimbursement of the Company’s research and development expenses under its antibody collaboration with Sanofi, an increase in the Company’s net profit from commercialization of EYLEA outside the United States, and reimbursement of the Company’s research and development expenses and amortization of up-front payments received in connection with the Company’s July 2015 immuno-oncology collaboration with Sanofi.

Refer to Table 4 for a summary of collaboration revenue.

Research and Development (R&D) Expenses: GAAP R&D expenses were $470 million in the first quarter of 2016, compared to $343 million in the first quarter of 2015. The higher R&D expenses in the first quarter of 2016 were principally due to higher development costs primarily related to dupilumab and fasinumab, and higher headcount to support the Company’s increased R&D activities, partly offset by lower development costs primarily related to Praluent. In addition, in the first quarter of 2016, R&D-related non-cash share-based compensation expense was $78 million, compared to $60 million in the first quarter of 2015.

Selling, General, and Administrative (SG&A) Expenses: GAAP SG&A expenses were $290 million in the first quarter of 2016, compared to $159 million in the first quarter of 2015. The increase was primarily due to higher headcount, and higher commercialization expenses related to EYLEA and Praluent. In addition, in the first quarter of 2016, SG&A-related non-cash share-based compensation expense was $60 million, compared to $42 million in the first quarter of 2015.

Cost of Goods Sold (COGS): GAAP COGS was $79 million in the first quarter of 2016, compared to $43 million in the first quarter of 2015. COGS primarily consists of royalties as well as costs in connection with producing U.S. EYLEA commercial supplies, and various start-up costs in connection with the Company’s Limerick, Ireland commercial manufacturing facility. COGS increased principally due to the increase in U.S. EYLEA net product sales, as well as an increase in Limerick start-up costs.

Income Tax Expense: In the first quarter of 2016, GAAP income tax expense was $164 million and the effective tax rate was 49.8%, compared to $201 million and 72.5% in the first quarter of 2015. The effective tax rate for the first quarter of 2016 was negatively impacted, compared to the U.S. federal statutory rate, by losses incurred in foreign jurisdictions with rates lower than the federal statutory rate and the non-tax deductible Branded Prescription Drug Fee, partly offset by the federal tax credit for increased research activities and the domestic manufacturing deduction. The effective tax rate for the first quarter of 2015 was negatively impacted primarily by losses incurred in foreign jurisdictions with rates lower than the federal statutory rate, the non-tax deductible Branded Prescription Drug Fee, and expiration, at the end of 2014, of the federal tax credit for increased research activities.

The non-GAAP income tax adjustment in the first quarter of 2016 is primarily related to the cash taxes the Company expects to be paid or payable in 2016 in connection with the immuno-oncology up-front payment that the Company received in 2015, partly offset by the excess tax benefit associated with stock option exercises. The non-GAAP income tax adjustment in the first quarter of 2015 was primarily related to the Company’s tax credit carry-forwards available for tax purposes and excess tax benefits in connection with stock option exercises.

Non-GAAP and GAAP Net Income: The Company reported non-GAAP net income of $293 million, or $2.81 per basic share and $2.57 per diluted share, in the first quarter of 2016, compared to non-GAAP net income of $336 million, or $3.28 per basic share and $2.88 per diluted share, in the first quarter of 2015.

The Company reported GAAP net income of $166 million, or $1.59 per basic share and $1.45 per diluted share, in the first quarter of 2016, compared to GAAP net income of $76 million, or $0.74 per basic share and $0.66 per diluted share, in the first quarter of 2015.

A reconciliation of the Company’s GAAP to non-GAAP results is included in Table 3 of this press release.

On May 05, 2016 Raptor Pharmaceutical Corp. (NASDAQ:RPTP) ("Raptor" or the "Company"), a biopharmaceutical company developing and commercializing transformative treatments for rare diseases, reported its financial results for the first quarter of 2016 and provided an update on recent corporate developments (Press release, Raptor Pharmaceutical, MAY 5, 2016, View Source [SID:1234512059]).

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Summary

Global net product revenue for PROCYSBI was $27.5 million for the first quarter ended March 31, 2016, a 34% increase compared to $20.5 million for the same period in 2015.

Net loss, including In-Process R&D (IPR&D) impairment and changes in the fair value of contingent consideration liability, on a GAAP basis was $41.6 million, or $0.49 per share, for the first quarter of 2016 compared to a GAAP net loss of $19.7 million, or $0.28 per share, for the same period in 2015.

Non-GAAP net loss, which excludes non-cash expenditures such as stock compensation, impairment of intangible assets and adjustment to the fair value of contingent consideration liability, was $14.1 million, or $0.17 per share compared to a net loss of $16.8 million, or $0.24 per share in the first quarter of 2015.

Cash and cash equivalents were $132.0 million as of March 31, 2016.

"Raptor achieved several significant milestones during the quarter, underscoring the progress we are making in building a premiere rare disease company," said Julie Anne Smith, President and CEO of Raptor. "PROCYSBI continues to deliver strong growth as a result of significant market penetration and continued high levels of patient compliance and we now look forward to revenues from the commercialization of QUINSAIRTM. We are focused on prioritizing key clinical programs to drive growth and being measured with our investments in selected development to extend our cash runway. As a result we are adjusting our non-GAAP cash operating expense guidance favorably for 2016. We look forward to continuing momentum as we work to bring QUINSAIR to patients in Europe and Canada, to make progress towards an NDA filing for MP-376 in cystic fibrosis (CF) and to initiate a Phase 2 MP-376 study in non-cystic fibrosis bronchiectasis (BE)."

First Quarter 2016 Business Highlights

Four new patents related to PROCYSBI were listed in the Orange Book during the quarter, bringing the total number of Orange Book-listed patents to five. In addition, PROCYSBI was granted additional U.S. orphan exclusivity in the two-to-six year old nephropathic cystinosis population until August 2022.

In March 2016, MP-376 received Qualified Infectious Disease Product (QIDP) designation for three distinct indications: the treatment of chronic pulmonary infections due to Pseudomonas aeruginosa, in patients with CF and in patients with BE, and in patients with nontuberculous mycobacteria infections (NTM). QIDP designation confers five years exclusivity under the Hatch-Waxman Act, which, in the case of CF, would be added to the seven years of orphan exclusivity for a total potential of 12 years of exclusivity, if approved for the indication.

In March 2016, Health Canada accepted the new drug submission for PROCYSBI in nephropathic cystinosis with Priority Review, which provides for a shortened review period of 180 days, compared to a standard review of 300 days. If approved, the shorter review time is expected to enable Raptor to bring PROCYSBI to market faster in Canada.

First 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

PROCYSBI global net revenue for the first quarter of 2016 was $27.5 million, compared to $20.5 million for the first quarter of 2015. The 34% revenue growth was driven by further market penetration in the U.S. and in Europe and named patient sales in other international territories.

Cost of Sales

Cost of sales was $4.3 million for the first quarter of 2016, compared to $3.7 million for the first quarter of 2015. The increase was due to higher quarterly sales year-over-year, which yielded higher direct costs and royalty expenses, and lower inventory reserves in 2016.

Research & Development (R&D)

R&D expenses for the first quarter of 2016 were $14.0 million compared to $16.6 million for the first quarter of 2015. The change was primarily due to a decrease in clinical trial expenses.

Selling, General and Administrative (SG&A)

SG&A expenses were $20.4 million for the first quarter of 2016 compared to $14.8 million for the first quarter of 2015. The increase in SG&A expenses was primarily a result of increased staffing and promotional support for commercial operations of PROCYSBI worldwide and administrative expenses.

In-Process R&D (IPR&D) and Contingent Consideration Liability Related to QUINSAIR Acquisition

The Company has revised its clinical development plans for MP-376. As a result, for the quarterly period ended March 31, 2016, based on a fair value assessment, Raptor recorded a non-cash impairment charge to IPR&D of $39.6 million and a partially offsetting contingent consideration credit adjustment of $14.7 million related to R&D stage projects from its October 2015 acquisition of QUINSAIR.

Interest Expense

Interest expense for the first quarter of 2016 was $5.0 million, compared to $4.5 million for the first quarter of 2015. The increase in interest expense was due to an increase in royalty fees based on net sales for the period.

Net Loss

GAAP net loss in the first quarter of 2016 was $41.6 million, or $0.49 per share, compared to a net loss of $19.7 million, or $0.28 per share, in the first quarter of 2015.

Non-GAAP net loss, which excludes the non-cash expenses of stock compensation expenses, impairment of intangible assets and adjustment to the fair value of the contingent consideration liability, for the first quarter of 2016 was $14.1 million, or $0.17 per share, compared to a net loss of $16.8 million, or $0.24 per share, in the first quarter of 2015.

Cash, Cash Equivalents

As of March 31, 2016, Raptor had $132.0 million in cash and cash equivalents compared to $157.4 million in cash and cash equivalents at the end of 2015.

2016 Full-Year Financial Guidance

Reiterating 2016 global net revenue guidance of $115 to $125 million.
Reiterating PROCYSBI revenue growth of 25% to 30% over 2015 revenues.
Lowering non-GAAP operating expense guidance, which excludes non-cash expenditures such as stock compensation, amortization and impairment of IPR&D and adjustment to the fair value of the contingent consideration liability, to $125 to $135 million from $135 to $145 million.
Product and Pipeline Updates

PROCYSBI for Nephropathic Cystinosis

PROCYSBI is currently commercially available to patients in the U.S., where it is the market leader in nephropathic cystinosis. PROCYSBI is also commercially available in Europe and is made available in additional markets through named patient sales.
In March 2016, Health Canada accepted for review the Company’s New Drug Submission (NDS) for PROCYSBI for the treatment of nephropathic cystinosis, with Priority Review status.
QUINSAIRTM

In April 2016, the Company launched QUINSAIR in Germany and Denmark for the management of chronic pulmonary infections due to Pseudomonas aeruginosa in adults with CF. Raptor anticipates launching the product in Canada in the second half of 2016.
MP-376 (Investigational Form of QUINSAIRTM)

In March 2016, MP-376 received QIDP designation for three distinct indications: the treatment of chronic pulmonary infections due to Pseudomonas aeruginosa, in patients with CF and in patients with BE, and in patients with NTM. Granting of the QIDP designation by the FDA highlights the potential of MP-376 to address a number of serious and life-threatening infections and provides Raptor with significant incentives for the development of MP-376, including Priority Review by the FDA, eligibility for Fast Track designation and a five-year extension of marketing exclusivity under the Hatch-Waxman Act. QIDP also paves the way for the FDA to apply greater regulatory flexibility in the case of high unmet medical need for serious life-threatening infections. Raptor holds orphan drug designation in the U.S. for MP-376 for the treatment of CF, which, when added to the five years of exclusivity associated with QIDP designation, would confer 12 years of regulatory exclusivity upon FDA approval.
Raptor expects to provide an update on regulatory discussions with the FDA concerning MP-376 in CF.
The Company intends to initiate a Phase 2 study for MP-376 in BE this year. The Company will provide additional clarity on the timing of key activities once the study design has been finalized.
RP103 for Huntington’s Disease (HD)

Based on prioritization of clinical programs and the best use of current capital, the Company will be exploring non-dilutive funding and partnering options.
RP103 for Mitochondrial Diseases

The second interim analysis, which occurred after 12 patients completed 24 weeks of treatment, indicated the trial should continue as planned. The treatment was generally safe and well tolerated and no unexpected safety signals were observed in this patient population.
Anticipated Upcoming Milestones

4Q 2016 — Potential PROCYSBI approval in Canada
2H 2016 — Potential PROCYSBI and QUINSAIR launches in Canada
2016 — Advancements toward an NDA submission for MP-376 in CF in the U.S.
2016 — Initiation of a Phase 2 study for MP-376 in BE
Conference Call and Webcast Information

Raptor will conduct a conference call and live audio webcast at 4:30 p.m. ET (1:30 p.m. PT) today. The live call may be accessed by dialing (877) 710-6201 for domestic callers or (616) 548-5611 for international callers and using the conference ID number 1390038. A live webcast of the conference call will be available online from the investor relations section of the Raptor website at www.raptorpharma.com. After the call, a webcast replay will be available on the Raptor website for 90 days while a telephone replay will be available for five days. This can be accessed by dialing (855) 859-2056 for domestic callers, or (404) 537-3406 for international callers, and using the conference ID number 1390038.

Non-GAAP Financial Information and Other Disclosures

Raptor uses non-GAAP financial measures, such as non-GAAP net loss, non-GAAP net loss per share and non-GAAP operating expense, to assess and analyze its operational results and trends, provide operating expense guidance, and to make financial and operational decisions. Raptor believes these non-GAAP financial measures are also useful to investors because they provide greater transparency regarding Raptor’s operating performance and exclude 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. These non-GAAP financial measures should not be considered an alternative to measurements required by GAAP, such as net loss, net loss per share and total operating expense, and should not be considered measures of Raptor’s liquidity. Non-GAAP financial measures should not be considered as a substitute for measures of financial performance calculated in accordance with GAAP and should only be used to supplement an understanding of Raptor’s operating results as reported under GAAP. In addition, these non-GAAP financial measures are unlikely to be comparable with non-GAAP information provided by other companies. Reconciliations between non-GAAP financial measures and GAAP financial measures are included in the table accompanying this press release after the unaudited consolidated financial statements. Raptor has not provided a GAAP reconciliation for its forward-looking non-GAAP financial measures, such as 2016 operating expense, because such measures are not readily determinable and not available without unreasonable efforts. Raptor cannot reliably predict without unreasonable efforts the timing or likelihood of outcomes that determine future impairments or changes in the fair value of contingent consideration liability nor the timing or amount of the factors that substantially contribute to the projection of stock compensation expense. Both are excluded from the forward-looking non-GAAP financial measures. The Company does not believe that such a reconciliation would be meaningful to stockholders.

On May 5, 2016 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and reported financial results for the first quarter ending March 31, 2016 (Press release, PTC Therapeutics, MAY 5, 2016, View Source [SID:1234512058]).

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"We are working hard to make Translarna available to all Duchene muscular dystrophy patients globally who may benefit," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "To that end, we are in dialogue with the FDA about the Refuse to File letter we received earlier this year. In Europe, we are also continuing discussions with the CHMP surrounding our regulatory submissions for DMD and CF. We are seeing growth in Translarna global sales and continue to expand access for DMD patients outside of the U.S. We were pleased with the positive recommendation from NICE, and anticipate access for patients in England once the market access agreement with NHS England is finalized. We are also excited that the second SMA compound in our collaboration is advancing in the clinic."

Key First Quarter and other Corporate Highlights:

Actively pursuing regulatory approvals for Translarna in DMD globally. PTC has engaged in dialogue with the U.S. Food and Drug Administration (FDA) to discuss the matters described in the Agency’s Refuse to File letter regarding Translarna for nonsense mutation Duchenne muscular dystrophy (nmDMD) and to seek a potential path forward to bring Translarna to patients in the U.S. Given the sensitivity around these discussions as well as their iterative nature, PTC will provide an update once it has clarified the regulatory strategy for Translarna in the U.S.
In the first quarter of 2016, PTC submitted the ACT DMD Phase 3 results to the European Medicines Agency (EMA) in support of the marketing authorization for Translarna for the treatment of nmDMD in ambulatory patients aged five and over, and separately, submitted a request for renewal of its current conditional marketing authorization. PTC is in the process of responding to requests for supplementary information from the Committee for Medicinal Products for Human Use (CHMP) to assist in determining whether the results of ACT DMD support a positive benefit-risk assessment for Translarna. PTC anticipates that the CHMP will issue its recommendation in mid-2016.

In Canada, PTC plans to submit a New Drug Submission for Translarna to Health Canada incorporating the results of the company’s Phase 3 ACT DMD study in the second half of 2016.

PTC recently initiated a Phase 1 pharmacokinetics study to assess the effects of Translarna in Japanese healthy volunteers. This study is the first step in pursuing regulatory approval for Translarna for nmDMD in Japan, one of the world’s largest pharmaceutical markets.

Translarna revenue of $18.9M in first quarter, which represents 49% growth over prior quarter. PTC has established a strong global commercial footprint launching the first approved therapy in nmDMD. By the end of 2016, PTC anticipates expanding commercial access to Translarna in over 35 countries across Europe, the Middle East, Latin America and Asia Pacific.
Translarna received recommendation in draft guidance from NICE. In the UK, the National Institute for Health and Care Excellence (NICE) has recommended Translarna for ambulatory patients aged five years and older with nmDMD in connection with a Managed Access Agreement (MAA) with NHS England. The provision of patient access is subject to the completion of the MAA with NHS England and finalization of the NICE draft guidance. NICE recently extended the timeframe for finalization of its guidance.
German patients accessing Translarna through alternate distribution channel. In Germany, PTC has delisted Translarna from the pharmacy ordering system. Since delisting in April, initial orders for German patients have already been successfully fulfilled via a foreign importation pathway. This pathway allows patients with high unmet medical needs to receive reimbursed access to treatments not directly available in Germany.
ACT CF Phase 3 clinical trial on track for completion by year-end 2016 with top-line results expected early 2017. During the third quarter of 2015, PTC submitted a variation to its marketing authorization requesting EMA approval of Translarna for the treatment of nonsense mutation cystic fibrosis (nmCF) based on the company’s previous Phase 3 study. The company anticipates the CHMP will issue its opinion regarding this submission in mid-2016. PTC’s confirmatory Phase 3 ACT CF trial is currently ongoing, and there is substantial risk that the results from this trial will be required for approval.
SMA program progressing with proof of concept achieved in Phase 1 study of RG7916 and clinical study in SMA patients planned for second half of 2016. Clinical development of the spinal muscular atrophy (SMA) program, a collaboration with Roche and the SMA Foundation, continued with the completion of a Phase 1 study of RG7916 in healthy volunteers. Preliminary results indicate that RG7916 was well tolerated and treatment resulted in increases of full length SMN2 mRNA. A clinical study of RG7916 in SMA patients is expected to begin in the second half of 2016.
First Quarter Financial Highlights:

Translarna net product sales were $18.9 million for the first quarter of 2016, representing 49% growth over $12.7 million reported in the fourth quarter of 2015; and significant growth versus $5.1 million in the first quarter of 2015. Translarna net product sales were positively impacted by a significant order from Brazil, where purchasing is often fulfilled in six-month orders, partially offset by a lower sales price for existing stock in Germany as a result of a mandatory discount imposed by the German Federal Association of the Statutory Health Insurances (GKV-SV) prior to delisting. 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 first quarter of 2016 were $18.9 million compared to $7.5 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.
Non-GAAP R&D expenses were $27.1 million for the first quarter of 2016, excluding $4.3 million in non-cash, stock-based compensation expense, compared to $23.3 million for the same period in 2015, excluding $4.6 million in non-cash, stock-based compensation expense. GAAP R&D expenses were $31.4 million for the first quarter of 2016 compared to $27.9 million for the same period in 2015. The increase in R&D expense for the first quarter of 2016 as compared to the prior year period was primarily due to additional costs associated with our ongoing clinical trials.
Non-GAAP SG&A expenses were $21.3 million for the first quarter of 2016, excluding $4.6 million in non-cash, stock-based compensation expense, compared to $12.5 million for the same period in 2015, excluding $5.1 million in non-cash, stock-based compensation expense. GAAP SG&A expenses were $25.9 million for the first quarter of 2016 compared to $17.6 million for the same period in 2015. The increase in SG&A expense for the first 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 first quarter of 2016 was $2.0 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 first quarter of 2016 was $41.2 million compared to a net loss of $37.9 million for the same period in 2015.
During the quarter, PTC announced a workforce reduction of approximately 18% of its employees and contractors, which will result in a one-time charge of approximately $2.6 million. PTC incurred $1.9 million of this charge in the first quarter.
Cash, cash equivalents, and marketable securities totaled approximately $299 million at March 31, 2016 compared to approximately $339 million at December 31, 2015.
Shares issued and outstanding as of March 31, 2016 were 34.3 million, which includes 0.4 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 of approximately $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.

PTC Therapeutics, Inc.
Consolidated Statements of Operations
(In thousands, except per share data)


Three Months Ended

March 31,

2016

2015

Revenues:

Net product revenue
$18,878

$5,069

Collaboration and grant revenue
17

2,413

Total revenues
18,895

7,482

Operating expenses:

Research and development (1)
31,399

27,938

Selling, general and administrative (1)
25,938

17,615

Total operating expenses
57,337

45,553

Loss from operations
(38,442)

(38,071)

Interest (expense)/income, net
(1,956)

524

Other expense, net
(721)

(368)

Loss before income tax expense
(41,119)

(37,915)

Income tax expense
(114)

—

Net loss
($41,233)

($37,915)

Weighted-average shares outstanding (in shares):

Basic and diluted
33,919,169

33,067,752

Net loss per share – basic and diluted (in dollars per share)
($1.22)

($1.15)

(1) Non-cash share-based compensation expense

included in operating expenses are as follows:

Research and development
$4,328

$4,667

Selling, general and administrative
4,587

5,081

Total share-based compensation expense
$8,915

$9,748

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

March 31,

December 31,

2016

2015
Cash, cash equivalents and marketable securities
$298,712

$338,925
Total assets
$335,232

$365,281

Total debt
$93,366

$91,848
Total deferred revenue
396

139
Total liabilities
$139,231

$139,280

Total stockholders’ equity (33,919,684 and 33,916,559 common shares

issued and outstanding at March 31, 2016 and December 31, 2015, respectively)
196,001

226,001
Total liabilities and stockholders’ equity
$335,232

$365,281

Upcoming Events:

PTC will participate in the following upcoming conference:

Bank of America 2016 Healthcare Conference, May 12 at 9:20 a.m. in Las Vegas, NV
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 May 05, 2016 pSivida Corp. (NASDAQ:PSDV) (ASX:PVA), a leader in the development of sustained release drug delivery products for treating eye diseases, reported a Company update and announced financial results for its third fiscal quarter ended March 31, 2016 (Press release, pSivida, MAY 5, 2016, View Source [SID:1234512057]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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pSivida continued to advance its lead product candidate, Medidur. The European Commission granted Medidur orphan medicinal product designation for the treatment of posterior uveitis. Orphan designation provides up to 10 years of market exclusivity in Europe upon marketing approval as well as other regulatory and financial incentives. In accordance with published EU regulatory guidance, pSivida plans to file for EU marketing approval based upon the strength of the results from Medidur’s first Phase 3 trial. The trial met its primary efficacy endpoint with high statistical significance with positive safety results.
"We are very pleased with the regulatory position of Medidur in the EU and are planning to file for regulatory approval under the centralized marketing authorization procedure around the end of 2016," said Dr. Paul Ashton, Ph.D., president and Chief Executive Officer of pSivida.

pSivida recently met with the U.S. Food and Drug Administration (FDA) to confirm the data required to support the U.S. New Drug Application (NDA) for Medidur. As a result of the meeting, pSivida continues to plan for an NDA submission based on the results of two Phase 3 trials together with data incorporated from the ILUVIEN Phase 3 trials and data from a short inserter utilization study. The second Phase 3 trial is approximately 60% enrolled. With favorable results, pSivida plans to file an NDA for Medidur around mid-2017.

With the addition of the net proceeds from a $17.8 million underwritten public offering of common stock during the quarter, the Company’s cash position at the end of the quarter was $33.3 million. "With this solid cash position, we should have adequate funding through our planned Medidur NDA filing and into the fourth quarter of calendar 2017," said Dr. Ashton.

Development of our Durasert product candidate for severe knee osteoarthritis (OA) being developed in partnership with Hospital for Special Surgery is proceeding on schedule. "The stability work requested by the FDA for the Investigational New Drug Application for the severe knee OA product candidate has been completed as planned, and we understand the principal investigator will submit it to the FDA shortly," added Dr. Ashton.

pSivida also continued its work on potential new product candidates. Research continued in its evaluation of off-patent or soon-to-be off-patent anti-cancer drugs that inhibit VEGF and PDGF to treat wet and dry age-related macular degeneration and of Tethadur to deliver antibodies.

Results for the Third Quarter and Nine Months Ended March 31, 2016. At March 31, 2016, cash, cash equivalents and marketable securities totaled $33.3 million compared to $21.1 million at the end of the prior quarter. In January 2016, pSivida enhanced its cash position with approximately $16.5 million of net proceeds from the consummation of a $17.8 million underwritten public offering of 4,440,000 shares of common stock. Net operating cash usage in the fiscal 2016 third quarter totaled $4.3 million, a $1.1 million increase over the prior quarter. The increase primarily reflected expected increases in CRO payments for Medidur clinical development. pSivida expects net cash usage to increase in its fiscal fourth quarter and to continue to vary from quarter to quarter, primarily as a result of the amount and timing of payments for Medidur clinical development.

Revenues for the quarter ended March 31, 2016 totaled $324,000 compared to $328,000 for the prior year quarter.

Research and development expense decreased by $265,000, or 8%, to $3.1 million for the three months ended March 31, 2016 compared to $3.3 million for the three months ended March 31, 2015. This was primarily attributable to lower CRO costs, partially offset by higher personnel costs, for the Medidur clinical development program.

General and administrative expense increased by $305,000, or 15%, to $2.3 million for the quarter ended March 31, 2016 compared to $2.0 million for the prior year quarter. The increase was primarily attributable to higher professional fees and personnel costs, including stock-based compensation.

Net loss for the quarter ended March 31, 2016 was $5.0 million, or $0.15 per share, compared to a net loss of $5.0 million, or $0.17 per share, for the prior year quarter.

Revenues for the nine months ended March 31, 2016 totaled $1.3 million compared to $26.2 million for the nine months ended March 31, 2015. The decrease reflected the $25.0 million milestone for FDA approval of ILUVIEN earned in the fiscal 2015 first quarter.

Research and development expense increased by $1.4 million, or 16%, to $10.3 million for the nine months ended March 31, 2016 compared to $8.9 million for the nine months ended March 31, 2015. The increase was primarily attributable to higher costs related to Medidur clinical development.

General and administrative expense increased by $712,000, or 13%, to $6.4 million for the first nine months of fiscal year 2016 compared to $5.6 million for the prior year period. The increase was primarily the result of higher professional fees, stock-based compensation and personnel-related costs.

Income tax benefit was $117,000 for the nine months ended March 31, 2016 compared to income tax expense of $144,000 for the nine months ended March 31, 2015. Both periods included refundable foreign research and development tax credits, which amounts for the nine months ended March 31, 2015 were more than offset by federal alternative minimum tax expense of $263,000 attributable to receipt of the $25.0 million ILUVIEN FDA approval milestone.