On February 23, 2017 Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical company focused on the unmet needs of patients with rare diseases, reported financial results for the fourth quarter and year ended December 31, 2016 and provided a business update (Press release, Insmed, FEB 23, 2017, View Source [SID1234517842]).

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Business Update

Achieved enrollment objective in global phase 3 study of ARIKAYCE. In November 2016, the company announced that it has achieved its patient enrollment objective in the phase 3 study of ARIKAYCE (liposomal amikacin for inhalation). The study, which is known as CONVERT or INS-212, is evaluating ARIKAYCE in treatment refractory nontuberculous mycobacteria (NTM) lung disease caused by Mycobacterium avium complex (MAC). The primary efficacy endpoint is the proportion of subjects who achieve culture conversion at Month 6 in the ARIKAYCE plus multi-drug regimen arm compared to the multi-drug regimen without ARIKAYCE arm. The Company expects to report the top-line data in the second half of 2017.
Granted U.S. Patent for ARIKAYCE, extending patent protection by more than five years into 2034. U.S. Patent 9,566,234 was issued to Insmed on February 14, 2017. The claims of the patent relate in part to systems and methods for treating pulmonary infections, including NTM infections, comprised of an aqueous dispersion of liposomal complexed aminoglycoside, which can be amikacin sulfate, with a nebulizer.
Advancing INS1007 toward Phase 2 trial. In October, the company exclusively licensed global rights to INS1007 (previously AZD7986) from AstraZeneca. INS1007 is a small molecule, oral reversible inhibitor of dipeptidyl peptidase I (DPP1), an enzyme responsible for activating neutrophil serine proteases (NSPs) in neutrophils when they are formed in the bone marrow. In chronic inflammatory lung diseases, neutrophils accumulate in the airways and result in excessive active NSPs that cause lung destruction and inflammation. The company expects to begin a phase 2 dose-ranging study of INS1007 in non-cystic fibrosis (non-CF) bronchiectasis in 2017. Non-CF bronchiectasis is a rare, progressive, neutrophil-driven pulmonary disorder with no approved therapies. Insmed is also evaluating the potential of INS1007 in other indications and expects to announce plans for phase 2 studies in additional disease states by the end of 2017.
“2017 represents a pivotal year for Insmed, with the potential to set the company on a new growth trajectory supported by a portfolio of novel products that address rare diseases,” said Will Lewis, president and chief executive officer of Insmed. “We anticipate announcing top-line results from our ongoing phase 3 CONVERT study of ARIKAYCE for the treatment of NTM lung disease which, if positive, may provide the basis for accelerated approval in the U.S. and potentially other parts of the world. We are also focused on advancing our pre-regulatory activities to support a timely filing and continuing to build NTM lung disease awareness among physicians. We plan to manage our clinical, pre-commercial and operational priorities while maintaining our commitment to efficient use of capital.”

Fourth Quarter Financial Results

For the fourth quarter of 2016, Insmed posted a net loss of $68.4 million, or $1.10 per share, compared with a net loss of $31.2 million, or $0.51 per share, for the fourth quarter of 2015. The fourth quarter 2016 results include $30.0 million of expense related to an upfront payment to AstraZeneca for INS1007.

Research and development expenses were $54.9 million for the fourth quarter of 2016, compared with $19.6 million for the fourth quarter of 2015. The increase was primarily due to the $30.0 million upfront payment related to INS1007, as well as the advancement of the company’s global phase 3 CONVERT study of ARIKAYCE in NTM lung disease and an increase in headcount and related expenses.

General and administrative expenses for the fourth quarter of 2016 were $12.2 million, compared with $12.9 million for the fourth quarter of 2015. The expenses in the fourth quarter of 2016 were relatively flat compared to the prior year and included $3.7 million of expenses related to pre-commercial activities for ARIKAYCE.

Balance Sheet Highlights and Cash Guidance

As of December 31, 2016, Insmed had cash and cash equivalents of approximately $163 million. The company’s cash operating expenses for the second half of 2016 were $64 million, not including the $30 million upfront payment related to INS1007 and excluding depreciation and stock-based compensation expense. Insmed ended the fourth quarter of 2016 with $55 million in debt.

The company is investing in the following activities in 2017: (i) clinical development of ARIKAYCE, (ii) regulatory and pre-commercial initiatives for ARIKAYCE, and (iii) preclinical and clinical activities for its earlier-stage development candidate INS1007. As a result of these activities, Insmed expects its cash-based operating expenses to be in the range of $67 million to $77 million for the first half of 2017. This range primarily reflects spending for the CONVERT study, the follow on 312 study for those NTM patients that do not convert, and continued regulatory and commercial development of ARIKAYCE. The estimates also include expenses related to INS1007, including inventory purchases as well as preclinical and clinical start-up activities.

On February 23, 2017 Heron Therapeutics, Inc. (NASDAQ: HRTX) (the Company or Heron), a commercial-stage biotechnology company focused on developing novel best-in-class treatment solutions to address some of the biggest unmet patient needs, reported fourth quarter and full year 2016 financial results and highlighted recent corporate progress (Press release, Heron Therapeutics, FEB 23, 2017, View Source [SID1234517840]).

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Recent Corporate Progress

Pain Franchise
Advanced HTX-011 through Four Positive Phase 2 Studies. HTX-011 is Heron’s investigational, long-acting formulation of the local anesthetic bupivacaine in a fixed-dose combination with the anti-inflammatory meloxicam for the prevention of post-operative pain. In four Phase 2 studies, HTX-011 produced statistically significant reductions in both pain intensity and the need for opioids compared to saline placebo following procedures with incisions ranging from very large (abdominoplasty) to very small (hernia repair and bunionectomy surgeries). Statistically significant reductions in pain and opioid use were also demonstrated when compared against bupivacaine solution, the current standard-of-care. Heron anticipates initiating Phase 3 studies in 2017 and filing a New Drug Application (NDA) in 2018.

Established Synergy of Heron’s Novel Formulation of Bupivacaine and Meloxicam. HTX-011 demonstrated a statistically significant benefit over each individual component of the product alone, providing evidence of the synergistic activity of bupivacaine and meloxicam in the HTX-011 formulation.

CINV Franchise
Launched First Commercial Product, SUSTOL. SUSTOL (granisetron) extended-release injection is indicated in combination with other antiemetics in adults for the prevention of acute and delayed nausea and vomiting associated with initial and repeat courses of moderately emetogenic chemotherapy (MEC) or anthracycline and cyclophosphamide (AC) combination chemotherapy regimens. Heron launched SUSTOL in October 2016, and we anticipate net sales of SUSTOL in 2017 to range from $15 to $25 million.

Submitted NDA for CINVANTITM (HTX-019). CINVANTI is the first surfactant-free, intravenous formulation of aprepitant for the prevention of chemotherapy-induced nausea and vomiting (CINV). If approved by the FDA, CINVANTI will strengthen Heron’s CINV portfolio by adding a second, complementary therapeutic agent. In January 2017, Heron submitted an NDA with the FDA for CINVANTI and expects to receive approval in the fourth quarter of 2017.

"2016 was an exciting and transformational year for Heron, highlighted by the release of our best-in-class Phase 2 data for HTX-011 and our FDA approval and commercial launch of SUSTOL," said Barry D. Quart, Pharm.D., Chief Executive Officer of Heron. "2017 is off to a promising start as well, highlighted by our submission of an NDA for CINVANTI, our second, complementary therapeutic agent in the CINV category, and the release of strong Phase 2 results of HTX-011 in abdominoplasty."

Financial Results
As of December 31, 2016, we had cash, cash equivalents and short-term investments of $51.1 million. In January 2017, we completed an underwritten public offering of 14.1 million shares of our common stock for net proceeds of $163.7 million. As of December 31, 2016, our pro-forma cash, cash equivalents and short-term investments, adjusting for the January 2017 public offering, was $214.8 million. This compares to $131.2 million in cash, cash equivalents and short-term investments as of December 31, 2015.

For the quarter and year ended December 31, 2016, we recognized net product sales of $1.3 million related to sales of SUSTOL. Due to the recent launch, we have recognized revenue only when our specialty distributors have resold SUSTOL to healthcare providers, the end users. We have deferred an additional $1.1 million in SUSTOL sales to the specialty distributors.

Our net loss for the quarter and year ended December 31, 2016 was $48.0 million and $173.1 million, or $1.22 per share and $4.56 per share, respectively, compared to a net loss of $31.2 million and $97.6 million, or $0.87 per share and $2.95 per share, respectively, for the same periods in 2015. Net loss for the quarter and year ended December 31, 2016, included non-cash, stock-based compensation expense of $7.3 million and $26.0 million, respectively, compared to $5.8 million and $14.4 million for the same periods in 2015.

Our net cash used for operating activities for the quarter and year ended December 31, 2016 was $38.5 million and $134.1 million, respectively, compared to net cash used for operating activities of $23.2 million and $78.5 million, respectively, for the same periods in 2015.

The increases in net loss and net cash used for operating activities in the 2016 periods as compared to the 2015 periods were primarily due to costs incurred in preparation for the commercial launch of SUSTOL, as well as clinical and manufacturing costs related to the development of CINVANTI and HTX-011.

On February 23, 2017 Five Prime Therapeutics, Inc. (Nasdaq:FPRX), a clinical-stage biotechnology company focused on discovering and developing innovative immuno-oncology protein therapeutics, reported a corporate update and reported financial results for the fourth quarter and full year ending December 31, 2016 (Press release, Five Prime Therapeutics, FEB 23, 2017, View Source [SID1234517836]).

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"2016 was a year of tremendous progress in Five Prime’s clinical and preclinical pipeline," said Lewis T. "Rusty" Williams, M.D., Ph.D., president and chief executive officer of Five Prime. "We advanced our three clinical-stage programs and look forward to announcing data from each of these programs this year. With a concerted focus on building out our pipeline, we also unveiled three preclinical programs that we advanced into IND-enabling activities. We are on track to meet our goal of filing at least one IND application for a new molecule each year for the foreseeable future, beginning this year."

2016 Business Highlights and Recent Developments

Clinical Pipeline:

Cabiralizumab (FPA008): an investigational antibody that inhibits CSF1R and has been shown to block the activation and survival of monocytes and macrophages. In the setting of advanced cancer, tumor-associated macrophages can inhibit the immune system’s ability to eradicate the disease. In pigmented villonodular synovitis (PVNS), a CSF-1-driven tumor, the bulk of the tumor mass in joints is formed by the macrophages themselves. Five Prime and Bristol-Myers Squibb (BMS) have an exclusive worldwide collaboration agreement for the development and commercialization of cabiralizumab for these and potentially additional indications.
– Initiated Phase 1b portion of cabiralizumab/OPDIVO trial.
In October 2016, Five Prime initiated the Phase 1b portion of the clinical trial evaluating the immunotherapy combination of cabiralizumab with the PD- 1 immune checkpoint inhibitor OPDIVO (nivolumab) in multiple tumor types. Five Prime and BMS are evaluating the safety, tolerability and preliminary efficacy of the combination in advanced solid tumors, including non-small cell lung cancer, squamous cell carcinoma of the head and neck, pancreatic cancer, glioblastoma, renal cell carcinoma and ovarian cancer. Five Prime expects to complete enrollment in the current Phase 1b trial cohorts in the second half of 2017.

– Advanced the Phase 2 trial of cabiralizumab in patients with tenosynovial giant cell tumor (TGCT), also known as pigmented villonodular synovitis (PVNS).
Five Prime expects to complete enrollment of the Phase 2 trial of cabiralizumab in PVNS in the first half of 2017. Five Prime is evaluating clinical measures, including response rate, pain and range of motion in approximately 30 PVNS patients.

– Five Prime plans to seek regulatory guidance on a pivotal trial in diffuse PVNS.

– Five Prime plans to disclose clinical data from the cabiralizumab PVNS trial at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2017 Annual Meeting and from the cabiralizumab immuno-oncology trial in the second half of 2017.

FPA144: an isoform-selective antibody in development as a targeted immuno-therapy for tumors that overexpress FGFR2b, a splice variant of a receptor for some members of the fibroblast growth factor (FGF) family. FPA144 has been engineered for enhanced antibody-dependent cell-mediated cytotoxicity (ADCC) to increase direct tumor cell killing by recruiting natural killer (NK) cells. Five Prime retains global development and commercialization rights to FPA144.
– Opened new gastric cancer cohorts and added a bladder cancer cohort in Phase 1 monotherapy trial of FPA144. Enrollment continues in the expansion portion of the trial, evaluating the safety, PK and efficacy of biweekly 15 mg/kg infusions of FPA144 in patients with gastric cancer whose tumors highly overexpress FGFR2b. During the third quarter of 2016, Five Prime added cohorts to evaluate FPA144 in patients with bladder cancer whose tumors overexpress FGFR2b and in patients with gastric cancer whose tumors express moderate or low levels of FGFR2b. Five Prime reported initial single-agent efficacy and safety data at the ASCO (Free ASCO Whitepaper) 2016 Annual Meeting and at the ASCO (Free ASCO Whitepaper) 2016 Gastrointestinal Cancers Symposium.

– Five Prime plans to seek regulatory guidance on a registrational path for FPA144 in combination with chemotherapy as an early-line gastric cancer therapy.

– Five Prime plans to disclose updated clinical data from the FPA144 program at the ASCO (Free ASCO Whitepaper) 2017 Annual Meeting.

FP-1039: a protein drug designed to block FGF signaling. As a ligand trap, FP-1039 binds to and neutralizes a subset of FGF ligands (such as FGF2), preventing these growth-promoting and angiogenic proteins from reaching FGFR1 on the surface of tumor cells.
– Five Prime plans to make decisions on potential future development of FP-1039 in mesothelioma after data on objective response rate, disease control rate and progression-free survival are mature. Five Prime regained full rights to FP-1039 from GlaxoSmithKline (GSK) in September 2016. GSK is completing the ongoing Phase 1b trial combining FP-1039 with 1st-line pemetrexed and cisplatin in untreated, unresectable mesothelioma. GSK concluded trial recruitment with 25 patients enrolled at the 15 mg/kg dose in June 2016, and continues to dose and follow patients.

– Five Prime plans to disclose clinical data from this program at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2017 Congress.

Preclinical Research and Development:

Five Prime unveiled three preclinical development candidates in IND-enabling studies at its R&D Day in New York City in December 2016.
– FPA150 (anti-B7-H4)

— An antibody designed for two mechanisms of action: to block an inhibitory T cell checkpoint pathway and to enhance killing of B7-H4-expressing tumors by ADCC.
— Investigational New Drug (IND) application planned for the fourth quarter of 2017.

– FPA154 (GITR agonist antibody)

— A tetravalent agonist antibody designed for greater GITR activation versus conventional antibodies. Conventional GITR agonist antibodies have two GITR binding sites while FPA154 has four.
— IND application planned for the fourth quarter of 2017.

– FPT155 (CD80)

— A natural, multi-targeting immune modulator that stimulates T cell responses through three critical pathways: CTLA4 blockade, CD28 agonism (without superagonism) and PD-L1 blockade that removes a potent inhibitory checkpoint.
— IND planned in 2018.

Progress in pre-clinical and research programs is on track for the company to achieve the goal of filing at least one IND application for a new molecule each year for the foreseeable future, beginning this year.

Completed multiple immuno-oncology research screens to identify new targets and drug candidates. Five Prime’s research team completed functional screens on CD8 T cells and regulatory T cells, as well as a comprehensive screen of all extracellular binding interactions in the "immunome," a defined subset of 700 extracellular proteins enriched for potential immune cell modulators. Five Prime conducted the screens to identify new immuno-oncology targets, which the company is prioritizing for further development as targets for new drug candidates or drug candidates themselves, either as monotherapies or as part of rational combination regimens.

Five Prime will feature three preclinical research poster presentations during the 2017 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, April 1 – 5, 2017, in Washington, D.C.
Summary of Financial Results and Guidance:

Cash Position. Cash, cash equivalents and marketable securities totaled $421.7 million on December 31, 2016 compared to $517.5 million on December 31, 2015. The decrease in year-end cash in 2016 was primarily attributable to net cash used in operations to advance the company’s clinical and preclinical pipeline.
Revenue. Collaboration and license revenue for the fourth quarter of 2016 was $8.3 million compared to $363.3 million for the fourth quarter of 2015. During the fourth quarter of 2015, the company recognized the entire upfront payment of $350 million as revenue under the cabiralizumab license and collaboration agreement with BMS. Collaboration and license revenue for the full year 2016 was $30.7 million compared to $379.8 million for the full year 2015.
R&D Expenses. Research and development expenses for the fourth quarter of 2016 increased by $8.2 million, or 39%, to $29.1 million from $21.0 million in the fourth quarter of 2015. Full year 2016 research and development expenses increased by $23.9 million, or 34%, to $94.1 million in 2016 from $70.2 million in 2015. These increases were primarily related to advancing the FPA144 program in a phase 1 clinical trial, advancing the cabiralizumab program in immuno-oncology and PVNS, and advancing our internal immuno-oncology preclinical and research activities.
G&A Expenses. General and administrative expenses for the fourth quarter of 2016 increased by $1.9 million, or 22%, to $10.5 million from $8.6 million in the fourth quarter of 2015. Full year 2016 general and administrative expenses were $35.8 million, an increase of $13.2 million, or 58%, from $22.6 million in 2015. This increase was primarily due to increases in personnel related expenses, including stock-based compensation.
Net Income (Loss). Net loss for the fourth quarter of 2016 was $20.1 million, or $0.73 per basic share and diluted share, compared to net income of $296.1 million, or $11.37 per basic share and $10.63 per diluted share, for the fourth quarter of 2015. Full year 2016 net loss was $65.7 million, or $2.44 per basic share and diluted share, compared to net income of $249.6 million, or $9.73 per basic share and $9.23 per diluted share. These decreases in net income were primarily related to recognizing the 2015 upfront payment of $350 million as revenue under the cabiralizumab license and collaboration agreement with BMS.
Cash Guidance. Five Prime expects full-year 2017 net cash used in operating activities to be less than $120 million. The company estimates ending 2017 with approximately $300 million in cash, cash equivalents and marketable securities.

oN February 23, 2017 Ipsen (Euronext: IPN; ADR: IPSEY), a global specialty-driven pharmaceutical group, reported financial results for the full year 2016.

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Commenting on the 2016 full year performance, David Meek, Chief Executive Officer of Ipsen, said: "The strong operating performance in 2016 serves as a solid foundation for the company in this new era of accelerated momentum and transformation. Sales grew by nearly 12% year-on-year, a record high for Ipsen, and core operating margin improved despite additional investments for the Cabometyx launch in Europe."

David Meek added: "2016 was a very productive year for Ipsen with the Cabometyx approval and launch for second line renal cell carcinoma in Europe, the launch of new indications for Dysport in the U.S., a new corporate governance structure implemented, and most recently, the acquisition of Onivyde, which reinforces our specialty oncology strategy. The focus for 2017 will be on building upon the strong momentum of the current business and the successful launch of Cabometyx, which combined with the expected addition of Onivyde and the new Primary Care products, will significantly contribute to the growth and profitability of the company in the coming years."



New definition of Core Financial Measures

Ipsen has updated its definition of Core financial measures (Core Operating income, Core consolidated net profit, Core EPS) to exclude the amortization of intangible assets (excluding software) and the gain or loss on disposal of fixed assets.

Core financial measures are the key performance indicators for understanding and measuring the performance of the Group. Ipsen believes that the updated financial indicators reflect with better clarity the Group’s underlying business trends and enable more meaningful comparisons year on year, as they exclude non-core items which may vary significantly.

These performance indicators do not replace IFRS indicators, and should not be relied upon as such.

Reconciliations between IFRS 2015/2016 results and the newly defined Core financial measures are presented in Appendix 4 and in the "Reconciliation from Core consolidated net profit to IFRS consolidated net profit" table on page 12.



Review of the full year 2016 results

Note: Unless stated otherwise, all variations in sales are stated excluding foreign exchange impacts.

In 2016, Group sales reached €1,584.6 million, up 11.8% year-on-year.

Specialty Care sales reached €1,273.0 million, up 16.1%, driven by the strong growth of Somatuline in North America, as well as a solid performance throughout Europe.

Dysport good sales performance in aesthetics in the U.S. through Galderma, and in Russia and the Middle East was offset by importation issues in Brazil that occurred in the second half of the year due to a temporary cancellation of the certificate of Good Manufacturing Practices (cGMP). Decapeptyl sales reflect good volume growth in Europe and China offset by price pressure in the region. The Group booked during the fourth quarter the first sales of Cabometyx in Europe, mainly in Germany, Austria and France following the product approval by EMA in September.

Primary Care sales reached €311.6 million, down 2.7%, impacted by lower sales in Russia for Tanakan and other Primary Care products, while Smecta sales were slightly up driven by the implementation of the new OTx6 commercial model.

Core Operating Income totaled €363.9 million, up 11.1%. Core operating margin reached 23.0%, up 0.3 points compared to 2015, mainly driven by strong business performance, partially offset by investments for the Cabometyx launch and the adverse impact of foreign currencies.

Core consolidated net profit was €263.6 million, up 12.8% over the period, compared to €233.8 million in 2015.

Core earnings per share – fully diluted (see Appendix 4) grew by 13.0% year-on-year to reach €3.18 for 2016, compared to €2.82 in 2015.

Free cash flow generated in 2016 reached €228.8 million, up by €52.5 million, driven by the strong operating performance and a good management of working capital and capital expenditures.

Closing net cash reached €68.6 million at the end of the period, compared to €186.9 million in 2015, notably after payments to Exelixis for the original cabozantinib license and subsequent extension to Canada, as well as regulatory and commercial milestones, for a total of €257.3 million in 2016.

IFRS Operating Income totaled €304.7 million, up 24.8% from €244.0 million in 2015, impacted by lower impairment charge, with an Operating margin at 19.2%, up 2.3 points compared to 2015.

IFRS Consolidated net profit was €226.6 million, up 18.8% over the period, compared to €190.7 million in 2015 and fully diluted EPS at €2.73 in 2016, was up 18.7% from €2.30 in 2015.



Comparison of 2016 performance with financial objectives

The Group exceeded the raised guidance provided on 26 October 2016 for Specialty Care sales and Core operating margin and came in at the favorable end of revised guidance for Primary Care sales.

The table below shows the comparison between the financial objectives provided on 26 October 2016 and 2016 actuals, both including the amortization of intangible assets.

Picture 2

Below is a reconciliation of the Core Operating Income from the previous definition to the new reported definition:

Picture 3



Dividend for the 2016 financial year proposed for the approval of Ipsen’s shareholders

The Ipsen S.A. Board of Directors, which met on 22 February 2017, has decided to propose at the annual shareholders’ meeting on 7 June 2017 the payment of a dividend of €0.85 per share, stable year-on-year.



2017 Financial objectives

The Group has set the following financial targets for 2017 assuming a successful closing of the Onivyde transaction with Merrimack by the end of the first quarter 2017, and of the Consumer Healthcare transaction with Sanofi in the second quarter of 2017:

Specialty Care sales growth year-on-year greater than +18.0%;
Primary Care sales growth year-on-year greater than +4.0%;
Core operating margin (excluding amortization of intangible assets) greater than 24% of net sales.
Sales objectives are set at constant currency.

On February 23, 2017 CBT Pharmaceuticals, Inc. (CBT), a life sciences company focused on developing innovative oncology therapeutics, reported preclinical data demonstrating the efficacy of its Programmed Death-1 (PD-1) antibody, CBT-501 (genolimzumab, GB-226), in stimulating various immune cells, generating anti-tumor immunity, and suppressing tumor growth and delaying tumor progression in a preclinical model of colon cancer (Press release, CBT Pharmaceuticals, FEB 23, 2017, View Source [SID1234517818]). The data were presented in a poster at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) – Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Clinical Immuno-Oncology Symposium being held from February 23 – 25, 2017 in Orlando, Florida. The symposium is focused on the latest clinical and translational research in immuno-oncology and the implications for clinical care.

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"The strong in-vivo preclinical data suggest that CBT-501 may have clinical benefit in a variety of tumor types," said Sanjeev Redkar, Ph.D., Chief Executive Officer and President at CBT Pharmaceuticals. "CBT-501’s novel epitope, with overlapping yet distinct regions compared to incumbent PD-1 inhibitors, may offer a point of differentiation in the clinical setting."

Presentation Highlights:

CBT-501 efficiently inhibited binding of PD-L1/L2 to PD-1 through competitive action.
CBT-501 enhanced human T-cell activation, as shown by increased release of IL-2 and INF-gamma.
In a humanized preclinical model expressing human PD-1 and implanted with a colon adenocarcinoma (MC38) cell line, CBT-501 significantly inhibited tumor growth in a dose-dependent manner that was comparable or improved over nivolumab.
"These studies support our commitment to advancing the clinical development of genolimzumab (CBT-501) as an immuno-oncology therapy for many types of cancer. Based on these findings, a Phase 1 dose escalation and dose and disease expansion study will be initiated in the first half of 2017," said Gavin Choy, Pharm.D., Chief Operating Officer at CBT Pharmaceuticals.

Genolimzumab Injection (CBT-501)

CBT-501 is a novel humanized IgG4 monoclonal antibody targeting the Programmed Death-1 (PD-1) membrane receptor on T lymphocytes and other cells of the immune system. CBT-501 has a comparable efficacy profile in in vitro and in vivo studies to marketed anti-PD-1 antibodies and has a superior safety profile with very low undesirable antibody-dependent cell-mediated cytotoxicity (ADCC) and complement-dependent cytotoxicity (CDC) activity. The antibody (GB226) has been developed by Genor BioPharma Co. Ltd., a Walvax Company, who owns development and commercialization rights in China. CBT Pharmaceuticals, Inc. retains rest of the world (ROW) rights. An investigational new drug application has been approved by the China Food and Drug Administration (CFDA), and a phase 1 trial will be initiated in China by Genor.