On May 10, 2017 Stemline Therapeutics, Inc. (Nasdaq: STML), a clinical-stage biopharmaceutical company developing novel therapeutics for oncology indications of unmet medical need, reported financial results for the quarter ended March 31, 2017 (Press release, Stemline Therapeutics, MAY 10, 2017, View Source [SID1234519066]). The company also reviewed clinical and regulatory events from the past quarter, and outlined key upcoming milestones:

SL-401 In Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

· During the quarter, we announced completion of enrollment in the Stage 3 cohort of the Phase 2 trial. Stage 3 enrolled 13 patients, and statistical analysis will be conducted on evaluable first-line BPDCN patients.

· Depending on the data from the trial, we plan to use the results generated, along with other relevant data, to support the potential filing of a Biologics License Application (BLA) for approval in BPDCN. A possible BLA filing could begin in 4Q17 or 1Q18.

· To ensure ongoing patient access to SL-401, we are enrolling both first-line and relapsed/refractory BPDCN patients under the current protocol in a Stage 4 cohort.

· We plan to provide an update on Stage 1 and 2 patients mid-year at an upcoming hematology-focused medical conference and on Stage 3 patients in the second half of the year.

Additional Clinical Trials

· Clinical trials evaluating SL-401 are ongoing in additional indications including certain myeloproliferative neoplasms (MPN), acute myeloid leukemia (AML) in complete remission with minimal residual disease, and relapsed/refractory multiple myeloma, and we expect to provide updates on these studies later this year and into next year.

· SL-801 is being evaluated in a Phase 1 dose escalation trial of advanced solid tumor patients, and we recently opened the sixth dosing cohort. SL-701 has completed dosing in a Phase 2 trial in second-line glioblastoma. Updates from both of these studies are expected later this year and into next year.

First Quarter 2017 Financial Results Review

Stemline ended the first quarter of 2017 with $105.8 million in cash, cash equivalents and investments, as compared to $67.6 million as of December 31, 2016, which reflects a cash increase of $38.2 million for the quarter. The $38.2 million increase in cash represents the $48.2 million in net cash proceeds received from the company’s follow-on public offering during January 2017 offset by a $10.0 million cash burn for operating activities during the first quarter 2017. The company ended the first quarter of 2017 with 25.1 million shares outstanding.

For the first quarter of 2017, Stemline had a net loss of $14.6 million, or $0.67 per share, compared with a net loss of $9.0 million, or $0.51 per share, for the same period in 2016.

Research and development expenses were $9.6 million for the first quarter of 2017, which reflects an increase of $3.1 million, or 47%, compared with $6.5 million for the first quarter of 2016. The higher costs are primarily driven by an increase of $2.3 million in manufacturing development expenses to support our upcoming potential BLA filing for SL-401. The manufacturing development costs include process characterization and process development work relating to the manufacture of drug substance and drug product for SL-401. Additionally, we incurred an increase in costs due to higher compensation expense as a result of increased headcount. Partially offsetting these higher expenses was a decrease in clinical trial costs for SL-701 resulting from the study attaining full patient enrollment during 2016.

General and administrative expenses were $5.4 million for the first quarter of 2017, which reflects an increase of $2.5 million, or 87%, compared with $2.9 million for the first quarter of 2016. The increase in expense was attributable to higher legal and audit fees of $1.9 million as a result of the class action lawsuits filed against us arising from our January 2017 follow-on public offering. Additionally, the higher costs were driven by $0.5 million in commercial-related pre-launch expenses in support of preparing for a potential product launch of SL-401 in BPDCN if marketing approval from the FDA is received.

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On May 10, 2017 SciClone Pharmaceuticals, Inc. (NASDAQ: SCLN) reported financial results for the quarter ended March 31, 2017 (Press release, SciClone Pharmaceuticals, MAY 10, 2017, View Source [SID1234519065]).

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Revenues: In the first quarter 2017, SciClone reported revenues of $42.9 million, compared to $36.5 million for the same period in 2016.
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GAAP Diluted EPS: In the first quarter 2017, SciClone reported GAAP diluted earnings per share of $0.28, compared to $0.15 for the same period in 2016.
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Non-GAAP Diluted EPS: In the first quarter 2017, SciClone reported non-GAAP diluted earnings per share of $0.31, compared to $0.19 for the same period in 2016.

Revenues in the first quarter of 2017 were $42.9 million, a $6.4 million or 18% increase, compared to $36.5 million for the same period in 2016. ZADAXIN revenues were $39.5 million in the first quarter of 2017, a $5.9 million or 17% increase, compared to $33.6 million for the same period in 2016. Of the $39.5 million in ZADAXIN revenues, $4.2 million was attributed to revenues from sales generated in the fourth quarter of 2016 but recognized in the first quarter of 2017. This $4.2 million of revenue is a result of fourth quarter sales that were above the reference tender price under a provision in the agreement with the Company’s China distributor to share, in part, in the burden of price reductions. Promotion services revenues were $1.3 million for the first quarter of 2017, a $0.1 million or 6% increase, compared to $1.2 million for the same period in 2016.

On a GAAP basis, SciClone reported net income in the first quarter of 2017 of $14.6 million, or $0.28 and $0.28 per share on a basic and diluted basis, respectively, compared to net income of approximately $7.9 million, or $0.16 and $0.15 per share on a basic and diluted basis, respectively, for the same period in 2016.

SciClone’s non-GAAP net income in the first quarter of 2017 was $16.6 million, or $0.32 and $0.31 per share on a basic and diluted basis, respectively, compared with non-GAAP net income of $9.7 million, or $0.20 and $0.19 per share on a basic and diluted basis, respectively, for the same period of the prior year.Both GAAP and non-GAAP net income were favorably impacted by the additional revenue recognition for ZADAXIN fourth quarter sales and a Chinese government subsidy in the first quarter of 2017.

Friedhelm Blobel, PhD, SciClone’s Chief Executive Officer commented: "We delivered a strong first quarter performance, in line with our expectations, and reflecting the continued demand for, and growth potential of, ZADAXIN and our core business. ZADAXIN’s competitive position remains strong, with continued volume growth despite generic competition. ZADAXIN’s double digit volume growth rate continued this quarter, underscoring its strength as the leading branded thymalfasin, with a 17% volume share and more than a 40% value share. We do face continued pricing pressure, with tenders in two provinces recently announced at prices lower than the reference price with our distributor. We cannot determine at this time with certainty when these prices will take effect, or when they will impact prices in other provinces, but we are likely to experience some effect of those lower prices at some point during the next few quarters. We may also see some pressure on unit volumes in some areas as a result of reduced national-level reimbursement for thymalfasins."

"We have demonstrated our ability to manage the various challenges of the China market effectively to date, and we are continuing to focus on strategies to expand the market for ZADAXIN, manage the impact of potential reimbursement changes and provincial pricing pressures and actively seek to participate in the provincial reimbursement negotiations for thymalfasins to maximize future reimbursement for ZADAXIN. We further expect that pricing pressures on revenue in 2017 will be offset, at least in part, through continued sharing of the burden with our China distributor and potentially through volume increases. We are confident that ZADAXIN has significant growth potential as a differentiated, high quality, Western-manufactured brand."

"We were pleased to see continued strong growth in our oncology portfolio in the first quarter. The market introduction of DC Bead to treat liver cancer is continuing to progress, although slower than anticipated. We are continuing implementation of our academic-focused marketing effort to build the market for DC Bead as an alternative to conventional TACE procedures using gels. Our development portfolio continue to advance, led by Angiomax (bivalirudin) for which we expect to file the NDA in the coming months."

"As we near the mid-year mark for 2017, we have confidence that our core strategies to grow our commercial business, advance and expand our development portfolio through creative partnering, maintain high levels of fiscal responsibility, and strong compliance are on track. We believe we are well positioned to continue to build our reputation and standing as a key player in the evolving China pharma market."

For the first quarter of 2017, sales and marketing (S&M) expenses were $12.8 million, compared with $12.4 million for the same period in 2016. The increase in S&M expenses for first quarter of 2017, compared to the same period in 2016, related to increases in salaries and benefits, mainly from annual increases, and to increased sales commissions based on increased ZADAXIN sales.

For the first quarter of 2017, research and development (R&D) expenses were $2.5 million, compared with $1.5 million of R&D expenses for the same period of 2016. R&D expenses were higher for the first quarter of 2017, compared to the first quarter of 2016, related to R&D activities in China that relate to development expenses of product candidates in-licensed from certain business partners.

For the first quarter of 2017, general and administrative (G&A) expenses were $7.2 million, compared with $7.4 million for the same period in 2016. G&A expenses were lower for the first quarter of 2017, compared to the first quarter of 2016, due to a foreign currency gain of $615 thousand on re-measuring operational monetary assets partially offset by an increase in salaries and benefits mainly from annual increases.

For the first quarter of 2017, other income, net was $1.0 million, compared with $0.1 million for the same period in 2016. Other income, net was higher principally for the first quarter of 2017, compared to the first quarter of 2016, as a result of a $1.0 million government subsidy related to the Company’s China operations which had no future performance obligations and was recognized upon receipt as other income.

For the first quarter of 2017, income tax provision was approximately $1.0 million, compared with $1.9 million for the same period in 2016. The $1.9 million income tax provision for the first quarter of 2016 included $1.2 million in additional tax expense representing the correction of an error related to a previously unrecognized liability for an uncertain tax provision in China.

As of March 31, 2017, cash and cash equivalents totaled $141.3 million, compared to $134.4 million as of December 31, 2016.

SciClone has presented non-GAAP information above as the Company believes this non-GAAP information is useful for investors, taken in conjunction with SciClone’s GAAP financial statements, because management uses such information internally for its operating, budgeting and financial planning purposes. Non-GAAP information is not prepared under a comprehensive set of accounting rules and should only be used to supplement an understanding of SciClone’s operating results as reported under GAAP. The non-GAAP calculations and reconciliation are provided in the accompanying table titled "Reconciliation of GAAP to Non-GAAP Net Income."

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On May 10, 2017 Onxeo S.A. (Euronext Paris, NASDAQ Copenhagen: ONXEO), a clinical-stage biotechnology company specializing in the development of innovative drugs for the treatment of orphan diseases, in particular in oncology, reported that it has received a Notice of Allowance from the U.S. Patent and Trademark Office for a patent application covering the specific route of administration for Livatag, which is currently in a phase III clinical trial (ReLive) for the second-line treatment of hepatocellular carcinoma (primary liver cancer) (Press release, Onxeo, MAY 10, 2017, View Source [SID1234519054]).

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Livatag (doxorubicin Transdrug) is based on an innovative technology allowing the formulation of doxorubicin (a chemotherapeutic agent) within nanoparticles composed of polyalkylcyanoacrylate, cyclodextrin, and poloxamer. This nanoparticle formulation provides new and promising properties, including overcoming the mechanisms of chemoresistance developed by tumor cells that affect the efficacy of chemotherapy agents.

"The United States represents a significant target market for Livatag. This new U.S. patent significantly strengthens our Livatag intellectual property portfolio, and enhances the value of this late-stage product candidate. We look forward to the availability of data from our ReLive trial in mid-2017," said Judith Greciet, CEO of Onxeo.

The new patent provides protection of the associated claims in the U.S. until 2032, and is in addition to the previously issued patents for the same patent family in other major territories, such as Europe and Japan. An additional patent family has also been filed based on a specific composition of Livatag nanoparticles that, if granted, would extend the patent protection of Livatag to 2036.

On May 10, 2017 Progenra, Inc. reported that it has received the Official Notice of Allowance (dated March 30, 2017) of its patent application entitled "Methods of treating cancer through the inhibition of USP7 and immune system modulation (Press release, Progenra, MAY 10, 2017, View Source [SID1234519053])." The patent, based on work described in a recent publication, is related to a new immune oncology therapy based on the inhibition of the ubiquitin-deconjugating enzyme USP7 by a small molecule. USP7 is a master positive regulator of cancer as it both directly supports the growth and survival of cancer cells and prevents the patient’s immune cells from recognizing and eradicating the tumor. Progenra is developing small molecule inhibitors of USP7 for clinical trial and, according to its President, Dr. Tauseef Butt, hopes to initiate Phase I in early 2018. He stated that "we continue to obtain data showing that our USP7 inhibitors have the potential to eliminate cancer by both direct cytotoxic and indirect immunological mechanisms. This class of drug could become a powerful alternative to the biological immune checkpoint inhibitors currently on the market (such as Opdivo and Keytruda), as well as a component of combination therapy with these agents." In animal efficacy models, Progenra compounds have demonstrated anti-cancer activity that was superior to that of Yervoy, Opdivo, or Keytruda. These results point to a potentially radical development in cancer treatment — a small molecule single agent that works as well as or better than combination protocols.

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On May 10, 2017 Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported financial results for the first quarter ended March 31, 2017 (Press release, Puma Biotechnology, MAY 10, 2017, View Source [SID1234519047]).

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Unless otherwise stated, all comparisons are for the first quarter 2017 compared to the first quarter 2016.

Based on accounting principles generally accepted in the United States (GAAP), Puma reported a net loss applicable to common stock of $72.9 million, or $1.97 per share, for the first quarter of 2017, compared to a net loss applicable to common stock of $71.0 million, or $2.19 per share, for the first quarter of 2016.

Non-GAAP adjusted net loss was $43.1 million, or $1.16 per share, for the first quarter of 2017, compared to non-GAAP adjusted net loss of $41.5 million, or $1.28 per share, for the first quarter of 2016. Non-GAAP adjusted net loss excludes stock-based compensation expense, which represents a significant portion of overall expense and has no impact on the cash position of the Company. For a reconciliation of GAAP net loss to non-GAAP adjusted net loss and GAAP net loss per share to non-GAAP adjusted net loss per share, please see the financial tables at the end of this news release.

Net cash used in operating activities for the first quarter of 2017 was $36.0 million. At March 31, 2017, Puma had cash and cash equivalents of $105.1 million and marketable securities of $88.9 million, compared to cash and cash equivalents of $194.5 million and marketable securities of $35.0 million at December 31, 2016.

"We made significant progress with our lead investigational drug, neratinib, during the first quarter of 2017," said Alan H. Auerbach, Chairman, Chief Executive Officer and President of Puma. "We look forward to continuing to work with the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) as they review our New Drug Application (NDA) and Marketing Authorization Application (MAA) filings, respectively, and we look forward to presenting the data on neratinib at the upcoming FDA Oncologic Drugs Advisory Committee on May 24th.

"Data on neratinib was also presented at the 2017 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April which included data on the use of antidiarrheal prophylaxis to reduce the diarrhea with neratinib in the extended adjuvant treatment of patients with early stage HER2-overexpressed/amplified breast cancer who have received prior adjuvant trastuzumab-based therapy (CONTROL trial). There was also clinical data presented on neratinib in the treatment of patients who have solid tumors with activating HER2 or HER3 mutations (SUMMIT trial). Additional data was also presented on the combination of T-DM1 and neratinib in patients with HER2 positive metastatic breast cancer (MBC) that has previously been treated with pertuzumab and trastuzumab. We look forward to continuing to achieve our objectives and believe that Puma is very well-positioned to build value for our shareholders."

Mr. Auerbach added, "During 2017, we anticipate the following key milestones with neratinib: (i) reporting data from the Phase III trial in third-line HER2-positive MBC patients in the second quarter of 2017; (ii) reporting data in the second quarter of 2017 from the TBCRC-022 Phase II trial of neratinib plus capecitabine in HER2-positive MBC patients with brain metastases; (iii) reporting final 5-year disease free survival (DFS) data during the second quarter of 2017 from the ExteNET Phase III trial of neratinib as an extended adjuvant treatment in HER2-positive early stage breast cancer; and (iv) announcing regulatory decisions in the United States and European Union on neratinib for the extended adjuvant treatment of patients with HER2-positive early stage breast cancer in the third quarter of 2017."

Operating Expenses

Operating expenses were $73.2 million for the first quarter of 2017, compared to $71.2 million for the first quarter of 2016.

General and Administrative Expenses:

General and administrative expenses were $18.4 million for the first quarter of 2017, compared to $11.0 million for the first quarter of 2016. The approximately $7.4 million increase resulted primarily from increases of approximately $1.4 million for stock-based compensation, $3.9 million for professional fees, $1.3 million for payroll and related costs, and $0.5 million for facility and equipment costs. These increases reflect overall corporate growth.

Research and Development Expenses:

Research and development (R&D) expenses were $54.8 million for the first quarter of 2017, compared to $60.2 million for the first quarter of 2016. The approximately $5.4 million decrease resulted primarily from decreases of approximately $1.1 million for stock-based compensation and $5.0 million for clinical trial expenses, partially offset by an increase of $0.6 million for consultants and contractors. For our existing clinical trials, we expect R&D expenses to decrease in subsequent quarters as clinical trials wind down.