On March 6, 2017 Kura Oncology, Inc. (NASDAQ:KURA), a clinical stage biopharmaceutical company focused on the development of precision medicines for oncology, reported updated results from the ongoing, Phase 2 trial of tipifarnib, a selective inhibitor of farnesyl transferase, and additional preclinical data in HRAS mutant squamous cell carcinomas of the head and neck (SCCHN) (Press release, Kura Oncology, MAR 6, 2017, View Source [SID1234518010]). The data were presented today at the 15th International Congress on Targeted Anticancer Therapies (TAT 2017) in Paris, France.

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This update on the patients in the first stage of the second cohort of the Phase 2 clinical trial in HRAS mutant tumors reported that, as of February 28, 2017, the two patients with HRAS mutant SCCHN with objective responses remain on study and are currently at treatment cycle 19 and cycle 12. Among the five patients with HRAS mutant salivary gland cancer, although no objective responses were observed, three patients experienced disease stabilization and were on study for 9, 10 and 14 cycles. In addition, tipifarnib was generally well tolerated with adverse events consistent with its known safety profile.

"The durability of the responses we have observed with tipifarnib is compelling considering the setting of relapsed and/or refractory SCCHN," said Alan L. Ho, M.D., Ph.D., a medical oncologist at Memorial Sloan Kettering Cancer Center and a lead investigator on the study. "These data reinforce the relevance of testing for HRAS mutations in these patients."

"We are very encouraged by the durable responses we have observed in patients with relapsed and/or refractory HRAS mutant SCCHN who are treated with tipifarnib," said Troy Wilson, Ph.D., J.D., President and CEO of Kura Oncology. "The two patients with partial responses have now been on treatment with tipifarnib for more than 16 months and 10 months, respectively. Recruitment of patients with SCCHN has become more challenging in the United States with the approval of immune therapy agents. To facilitate enrollment in the second stage of our trial we are adding additional clinical sites in Western Europe and Asia, and are working with third party laboratories to facilitate HRAS screening. We anticipate that additional results from this ongoing Phase 2 trial of tipifarnib in HRAS mutant SCCHN will be available during the second half of 2017."

Dr. Ho’s slide presentation will be available at View Source

On March 6, 2017 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) reported that Phase 1 clinical data for IPI-549, an orally administered immuno-oncology development candidate that selectively inhibits phosphoinositide-3-kinase gamma (PI3K-gamma), were presented during a plenary session at the 15th International Congress on Targeted Anticancer Therapies (TAT 2017) taking place in Paris, France, March 6 – 8 (Press release, Infinity Pharmaceuticals, MAR 6, 2017, View Source [SID1234518009]). A Phase 1 clinical study is ongoing to explore the safety and activity of IPI-549 both as a monotherapy and in combination with Opdivo (nivolumab), a PD-1 immune checkpoint inhibitor, in patients with advanced solid tumors. IPI-549 is believed to be the only selective PI3K-gamma inhibitor in clinical development.

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"The TAT congress has become a leading conference for Phase 1 clinical studies in oncology, so it is an honor to be invited to participate in a plenary session to discuss our progress with IPI-549 at this year’s congress," commented Claudio Dansky Ullmann, M.D., senior vice president, clinical development at Infinity.

Today’s presentation at TAT 2017 included updated Phase 1 data from 12 patients enrolled in the monotherapy dose-escalation phase of the ongoing study. The presentation, "A Phase 1/1b, First-in-Human Study of IPI-549, a PI3K-Gamma Inhibitor and Myeloid Targeting Agent, as Monotherapy and in Combination with Nivolumab in Patients with Advanced Solid Tumors," was given by Anthony Tolcher, M.D., FRCP(C), clinical director at South Texas Accelerated Research Therapeutics, an investigator for the Phase 1 study and the recipient of the TAT 2016 Honorary Award Invited Lecture.

"Based on the experience of patients in my clinic, I am encouraged about the potential of IPI-549, and the data to date suggest that the safety, pharmacokinetics and pharmacodynamics of monotherapy treatment appear favorable," stated Dr. Tolcher. "IPI-549 holds the promise of offering a unique approach for enhancing the body’s anti-tumor immune response, and I look forward to continuing to participate in this Phase 1 clinical study."

Additionally, IPI-549 was discussed today during the TAT 2017 Honorary Award Invited Lecture, "Immunologic Checkpoint Blockade: Exploring Combinations and Mechanisms," which was given by Jedd Wolchok, M.D., Ph.D., Chief of the Melanoma and Immunotherapeutics Service at Memorial Sloan Kettering Cancer Center (MSK), as well as Associate Director of the Ludwig Center for Cancer Immunotherapy and Director of the Parker Institute for Cancer Immunotherapy, both at MSK. Dr. Wolchok serves as the lead investigator for the Phase 1 clinical study of IPI-549. He is also an author on the recently published Nature paper which described that the presence of suppressive myeloid cells is critical in tumor resistance to checkpoint inhibitors and targeting PI3K-gamma in these cells with IPI-549 is able to help overcome this resistance.1

"This is an exciting time in the field of immuno-oncology. New immunotherapies, such as checkpoint inhibitors, underscore the progress we have made in the treatment of various cancers. Even so, we are always exploring new avenues of investigation that we hope will benefit even more patients," stated Dr. Wolchok. "IPI-549 is one of the newer and potentially promising immunotherapies in clinical development. Our research suggests IPI-549 could offer a unique way to both enhance the activity of checkpoint inhibition in sensitive tumors, as well as to overcome tumor resistance to checkpoint inhibition."

IPI-549 Poster Presentation at Upcoming AACR (Free AACR Whitepaper) Annual Meeting 2017
Last week, Infinity announced that updated Phase 1 data for IPI-549 will be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2017 taking place in Washington, D.C. April 1 – 5. Details of the presentation are as follows:
Poster presentation time: Tuesday, April 4, 2017, 8:00 a.m. – 12:00 p.m. ET
Title: IPI-549-01 – A Phase 1/1b, First-in-Human Study of IPI-549, a PI3K-Gamma Inhibitor, as Monotherapy and in Combination with Nivolumab in Patients with Advanced Solid Tumors
Abstract number: CT089
Lead author: Jedd Wolchok, M.D., Ph.D., Chief of the Melanoma and Immunotherapeutics Service at Memorial Sloan Kettering Cancer Center (MSK), as well as Associate Director of the Ludwig Center for Cancer Immunotherapy and Director of the Parker Institute for Cancer Immunotherapy, both at MSK
Location: Convention Center, Halls A-C, Poster Section 33

Due to AACR (Free AACR Whitepaper) embargo policies, the data presented today at TAT 2017 will be made available during the AACR (Free AACR Whitepaper) Annual Meeting 2017.

About the IPI-549 and the Ongoing Phase 1 Study
IPI-549 is an investigational, orally administered immuno-oncology development candidate that selectively inhibits PI3K-gamma. In preclinical studies, IPI-549 increases antitumor immunity by targeting tumor-associated myeloid cells and overcomes immune checkpoint blockade resistance in preclinical tumor models.1,2 As such, IPI-549 may have the potential to treat a broad range of solid tumors and represents a potentially complementary approach to restoring anti-tumor immunity in combination with other immunotherapies such as checkpoint inhibitors.

A Phase 1 study of IPI-549 in patients with advanced solid tumors is ongoing to explore the activity, safety, tolerability, pharmacokinetics and pharmacodynamics of IPI-549 as a monotherapy and in combination with Opdivo in patients with advanced solid tumors.3 The study includes monotherapy and combination dose-escalation phases, in addition to a monotherapy expansion cohort and combination expansion cohorts. Overall, the study is expected to enroll approximately 175 patients.

The expansion cohorts evaluating IPI-549 plus Opdivo will include patients with non-small cell lung cancer (NSCLC), melanoma and squamous cell carcinoma of the head and neck (SCCHN). There is a great need for additional treatment options for the growing number of patients living with these types of cancers, which account for more than 17 percent of all new cancer cases in the U.S.4,5 Additionally, patients enrolled in the combination expansion cohorts represent a difficult-to-treat population, as they must have demonstrated initial resistance or subsequently develop resistance to a PD-1 or PD-L1 therapy immediately prior to enrolling in the study.

IPI-549 is an investigational compound and its safety and efficacy has not been evaluated by the U.S. Food and Drug Administration or any other health authority.

On March 6, 2017 Cellectis S.A. (Alternext: ALCLS – Nasdaq: CLLS), a biopharmaceutical company focused on developing immunotherapies based on gene edited CAR T-cells (UCART), reported its results for the three-month period ended December 31, 2016 and for the year ended December 31, 2016 (Press release, Cellectis, MAR 6, 2017, View Source [SID1234518008]).

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– FDA approval to conduct Phase I clinical trials for UCART123 in AML & BPDCN patients
– UCART191 Phase I clinical trials ongoing in ALL patients; partial data presented at the NIH’s RAC’s meeting in December 2016
– Constitution of Cellectis’ Clinical Advisory Board
– Strong cash position of $291 million2 (€276 million) as of December 31, 2016
– Revenues and other income of $56 million3 (€51 million) in 2016
– Excluding non-cash stock based compensation expense, Adjusted net loss4 of $9 million2 (€8 million) for full year 2016

1 Cellectis granted Servier an exclusive license to UCART19 product candidate, and Pfizer has been exclusively licensed by Servier for the development and commercialization of UCART19 in U.S.

2 Translated only for convenience into U.S. dollars at an exchange rate of €1.00=$1.0541, the daily reference rate reported by the European Central Bank ("ECB") as of December 31, 2016

3 Translated only for convenience into U.S. dollars at an exchange rate of €1.00=$1.1066, the arithmetic average of the ECB’s monthly average reference rates for the twelve months comprising full year 2016

4 See the section related to the reconciliation of GAAP to non-GAAP net income. GAAP Net Loss attributable to shareholders amounts to $67 million (€61 million) for the year ended December, 312016.

________________________________________

Earnings Call Details

Cellectis will host an earnings call on March 7, 2017 at 8:00am Eastern Time to discuss its financial results and provide a general business update.

Dial-In Numbers:

Live PARTICIPANT Dial-In (Toll-Free US & Canada): 877-407-3104

Live PARTICIPANT Dial-In (International): +1 201-493-6792

Replay Information:

Conference ID #: 13625168

Replay Dial-In (Toll Free US & Canada): 877-660-6853

Replay Dial-In (International): +1 201-612-7415

Expiration Date: 3/21/17

Webcast URL (Archived for 12 months): http://cellectis.equisolvewebcast.com/q4-2016

________________________________________

RECENT CORPORATE HIGHLIGHTS

Cellectis – Therapeutics

UCART123 – Cellectis’ most advanced, wholly owned TALEN gene-edited product candidate

– Pre-clinical data presented at the 2016 American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting by Dr. Monica Guzman, MD, Weill Cornell, showed long-lasting molecular remission in mice, using UCART123, compared to Cytarabine alone.

– Successful National Institutes of Health’s (NIH) Recombinant DNA Advisory Committee’s (RAC) meeting with unanimous approval of Phase I clinical trials for UCART123 in acute myeloid leukemia (AML) and blastic plasmacytoid dendritic cell neoplasm (BPDCN).

– Investigational New Drug (IND) approval received from the U.S. Food and Drug Administration (FDA) to conduct Phase I clinical trials in patients with AML and BPDCN.

– First clinical trial approval by the FDA for an allogeneic, "off-the-shelf" gene-edited CAR T-cell product candidate.

– AML clinical program to be led, at Weill Cornell, by Gail J. Roboz, MD, Director of the Clinical and Translational Leukemia Programs and Professor of Medicine.

– BPDCN clinical program to be led, at MD Anderson Cancer Center, by Naveen Pemmaraju, MD, Assistant Professor, and Hagop Kantarjian, MD, Professor and Department Chair, Department of Leukemia, Division of Cancer Medicine.

– Successful cGMP manufacturing runs of UCART123 at large scale, to provide doses for initiating planned Phase I clinical trials in AML and BPDCN patients.

UCART19, exclusively licensed to Servier

– Phase I clinical trials in pediatric and adult ALL patients are ongoing at University College London (UCL) and Kings College London (KCL), UK, sponsored by Servier. Additional sites in other European countries are planned to be opened subject to approval of concerned regulatory bodies.

– Partial data presented on first 7 patients treated with UCART19 at NIH’s Recombinant DNA Advisory Committee (RAC) meeting in December 2016.

– Pfizer, in collaboration with Servier, plans to open sites in the U.S. for the ongoing Phase I clinical trials in adult ALL patients, as presented at the RAC meeting in December 2016.

Scientific Publications

– Publication of a study in Scientific Reports, a Nature Publishing Group journal, describing a novel approach to a CAR design with an integrated environmental signal utilizing oxygen concentration to manipulate the CAR T-cell response.

Clinical Advisory Board

– Formation of a Clinical Advisory Board (CAB) comprising leading experts in the hematologic malignancies / stem cell transplant, immunotherapy and hematology-oncology clinical research fields to serve as a strategic resource to Cellectis in connection with the clinical development of UCART123.

Calyxt – Cellectis’ plant science subsidiary

– Publication of a study in BMC Plant Biology describing the use of genome editing technology to modulate soybean oil composition for increased shelf-life, higher frying stability and improved nutritional characteristics.

– Calyxt completed an expansion of its high-oleic/no trans-fat soybean variety (CAL1501) in the U.S. with a production of 1,200 tons of soybeans.

Financial Results
Cellectis’ consolidated financial statements have been prepared in accordance with International Financial Reporting Standards, or IFRS, as issued by the International Accounting Standards Board ("IASB"). The audit procedures have been carried out by the independent auditors and their audit report relating to the certification of the financials is in the process of being issued. The audited report for Cellectis’ consolidated financial statements will be included in the Company’s annual report.

Fourth quarter 2016 Financial Results

Cash: As of December 31, 2016 Cellectis had €276.2 million in total cash, cash equivalents and current financial assets compared to €264.0 million as of September 30, 2016. This increase of €12.2 million notably reflects (i) the receipt of R&D tax credits of €9.2 million, (ii) proceeds of €7.0 million related to the supply agreement with Servier, (iii) the unrealized positive translation effect of exchange rate fluctuations on our U.S. dollar cash, cash equivalents and current financial assets of €12.1 million, partially offset by (iv) other net cash flows used by operating activities of €15.0 million and (v) fixed assets expenditures of €1.1 million.

Revenues and Other Income: During the quarters ended December 31, 2015 and 2016, we recorded €29.2 million and €12.1 million, respectively, in revenues and other income. This decrease is mainly due to the decrease of €20.0 million in collaboration revenues, notably due to the €18.8 million revenue recorded in 2015 in relation to early exercise by Servier of its option to acquire the exclusive worldwide rights to further develop and commercialize UCART19, partially offset by increases of €1.0 million in research tax credit and €1.3 million in subsidies.

Total Operating Expenses: Total operating expenses for the fourth quarter of 2016 were €30.9 million, compared to €28.0 million for the fourth quarter of 2015. The non-cash stock-based compensation expenses included in these amounts were €13.1 million and €12.6 million, respectively.

R&D Expenses: For the quarters ended December 31, 2015 and 2016, research and development expenses increased by €2.6 million from €16.0 million in 2015 to €18.7 million in 2016. Personnel expenses increased by €0.5 million from €11.2 million in 2015 to €11.6 million in 2016, due to a €1.8 million increase in social charges on stock options, partially offset by a €1.1 million decrease in non-cash stock based compensation expense and a €0.2 million decrease in wages and salaries. Purchases, external expenses and other expenses increased by €2.2 million from €4.3 million in 2015 to €6.5 million in 2016.

SG&A Expenses: During the quarters ended December 31, 2015 and 2016, we recorded €8.1 million and €11.4 million, respectively, of selling, general and administrative expenses. The increase of €3.3 million primarily reflects the increase in personnel expenses from €5.6 million to €8.9 million, attributable to the increase of €1.5 million in non-cash stock-based compensation expense, €1.4 million in social charges on stock options, and €0.4 million in wages and salaries. No material variance has been identified on purchases, external expenses and other expenses.

Financial Gain (Loss): The financial gain was €7.0 million for the fourth quarter of 2015 compared with a financial gain of €6.4 million for the fourth quarter of 2016. The change in financial result was primarily attributable to the increase of €1.3 million in fair value adjustment expense on our foreign exchange derivatives and current financial assets, partially offset by the gain of €0.8 million due to the effect of exchange rate fluctuations on our U.S. dollar cash and cash equivalent accounts.

Net Income (Loss) Attributable to Shareholders of Cellectis: During the quarters ended December 31, 2015 and 2016, we recorded a net gain of €8.2 million (or €0.23 per share on both a basic and a diluted basis) and net loss of €12.5 million (or €0.35 per share on both a basic and a diluted basis), respectively. Adjusted income attributable to shareholders of Cellectis for the fourth quarter of 2016 was €0.6 million (€0.02 per share on both a basic and a diluted basis) compared to adjusted income attributable to shareholders of Cellectis of €20.9 million (€0.59 per share on both a basic and a diluted basis), for the fourth quarter of 2015. Adjusted income (loss) attributable to shareholders of Cellectis for the fourth quarter of 2016 and 2015 excludes non-cash stock-based compensation expense of €13.1 million and €12.6 million, respectively. Please see "Note Regarding Use of Non-GAAP Financial Measures" for reconciliation of GAAP net income (loss) attributable to shareholders of Cellectis to Adjusted income (loss) attributable to shareholders of Cellectis.

Full year 2016 Financial Results

Cash: As of December 31, 2016 Cellectis had €276.2 million in total cash, cash equivalents and current financial assets compared to € 314.2 million as of December 31, 2015. This decrease of €38.0 million was primarily driven by (i) €29.6 million of cash used in operating activities, related to our research and development and manufacturing efforts, including the advancement of UCART123, for which an IND was filed in the United States in early 2017, partially offset by payments received from Servier and Pfizer pursuant to our collaboration agreements and R&D tax credit, and (ii) €12.5 million of cash used in investment activities, primarily through Calyxt’s land acquisition and greenhouse construction in an aggregate amount of €9.5 million. The decrease was also partially offset by the positive unrealized translation effect of exchange rate fluctuations on our U.S. dollar cash, cash equivalents and current financial assets of €4.4 million.

Cellectis expects that its cash, cash equivalents and Current financial assets of €276.2 million as of December 31, 2016 will be sufficient to fund its current operations to 2019.

Revenues and Other Income: During the year ended December 31, 2015 and 2016, we recorded €56.4 million and €51.0 million, respectively, in revenues and other income. This decrease is mainly due (i) to the decrease of €10.4 million in collaboration revenues notably due to revenue recorded in 2015 in relation to early exercise by Servier of its option to acquire the exclusive worldwide rights to further develop and commercialize UCART19 (€18.8 million) partially offset by the revenue from an agreement to provide Servier with raw materials and additional batches of UCART19 products and the achievement of two milestones in 2016 (totaling €11.9 million), (ii) increases of €4.0 million in research tax credit, €0.5 million in licenses fees and €0.4 million in research subsidies.

Total Operating Expenses: Total operating expenses for the year ended December 31, 2016 were €111.8 million, compared to €84.3 million for the year ended December 31, 2015. The non-cash stock-based compensation expenses included in these amounts were €53.0 million and €30.1 million, respectively.

R&D Expenses: For the year ended December 31, 2015 and 2016, research and development expenses increased by €18.5 million from €52.4 million in 2015 to €70.9 million in 2016. Personnel expenses increased by €8.8 million from €35.5 million in 2015 to €44.3 million in 2016, notably due to a €1.6 million increase in wages and salaries, and a €11.5 million increase in non-cash stock based compensation expense, partially offset by a €4.3 million decrease in social charges on stock options and free share grants. Purchases and external expenses increased by €9.8 million from €15.2 million in 2015 to €25.0 million in 2016, due to increased expenses related to UCART123 and other product candidates’ development, including payments to third parties and costs related to preparation of UCART123 clinical trials, purchases of biological materials and expenses associated with the use of laboratories and other facilities.

SG&A Expenses: During the year ended December 31, 2015 and 2016, we recorded €27.2 million and €39.2 million, respectively, of selling, general and administrative expenses. The increase of €12.0 million primarily reflects (i) an increase of €10.7 million in personnel expenses from €19.6 million to €30.3 million attributable to a €0.9 million increase in wages and salaries, an increase of €11.4 million of non-cash stock-based compensation expense, partially offset by a decrease of €1.6 million of social charges on stock options and free share grants, (ii) an increase of €1.9 million in purchases and external expenses and (iii) a decrease of €0.6 in other expenses due to lower business taxes and lower provisions.

Financial Gain (Loss): The financial gain was €7.6 million for the year ended December 31, 2015 and the financial gain was null for the year ended December 31, 2016. The decrease in financial income and expenses between 2016 and 2015 was mainly attributable to the decrease of €5.9 million in net foreign exchange gain, €1.6 million foreign exchange derivatives fair value expense, and €0.9 million current financial assets fair value expenses, partially offset by an increase of €0.5 million in interest income.

Net Income (Loss) Attributable to Shareholders of Cellectis: During the year ended December 31, 2015 and 2016, we recorded a net loss of €20.5 million (or € 0.60 per share on both a basic and a diluted basis) and a net loss of €60.8 million (or €1.72 per share on both a basic and diluted basis), respectively. Adjusted loss attributable to shareholders of Cellectis for the year ended December 31, 2016 was €7.8 million (€0.22 per share on both a basic and a diluted basis) compared to Adjusted income attributable to shareholders of Cellectis of € 9.6 million (€0.28 per share on both a basic and a diluted basis), for the year ended December 31, 2015. Adjusted income (loss) attributable to shareholders of Cellectis for the year ended December 31, 2016 and 2015 excludes non-cash stock-based compensation expense of €53.0 million and €30.1 million, respectively. Please see "Note Regarding Use of Non-GAAP Financial Measures" for a reconciliation of GAAP net income (loss) attributable to shareholders of Cellectis to Adjusted income (loss) attributable to shareholders of Cellectis.

On March 7, 2017 Actelion Ltd (SIX: ATLN) reported that the European Commission has granted marketing authorization for the use of Ledaga (chlormethine gel) 160 micrograms/g for the treatment of mycosis fungoides-type cutaneous T-cell lymphoma (MF-CTCL) (Press release, Actelion, MAR 6, 2017, View Source [SID1234518007]).

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MF-CTCL is a rare, potentially life-threatening immune system cancer that is chronic and usually progresses slowly. The course of disease in individual patients is unpredictable. In about 34% of cases, a progression of the disease is observed, and in the most advanced stages, MF-CTCL cells can metastasize to other body tissues, including the liver, spleen and lungs.

Ledaga is indicated for the topical treatment of mycosis fungoides-type cutaneous T-cell lymphoma (MF type CTCL) in adult patients.

The market authorization for Ledaga is based on the results of the pivotal 201 study, the largest randomized controlled study ever conducted in early stage MF-CTCL, involving 260 patients. In this study, within the efficacy evaluable (EE) population, 77% of patients who were treated for at least 6 months with chlormethine gel achieved a clinical response in the Composite Assessment of Index Lesion Severity (CAILS) score, while 59% of those treated with the compounded control had a clinical response. A response was defined as at least a 50% improvement in the baseline CAILS score. Complete response was achieved in 19% of patients treated with chlormethine gel in the EE population versus 15% of patients treated with the compounded control. Reductions in mean CAILS scores were seen as early as four weeks into the study, with further reductions observed with continuing therapy.

In the 201 study, the most frequent adverse reactions reported with chlormethine gel were skin related: dermatitis (54.7%; e.g., skin irritation, erythema, rash, urticaria, skin-burning sensation, pain of the skin), pruritus (20.3%), skin infections (11.7%), skin ulceration and blistering (6.3%), and skin hyperpigmentation (5.5%). No evidence of systemic absorption of chlormethine was observed with the treatment.

Actelion is working diligently to launch Ledaga in the EU as rapidly as possible. Actelion has agreed to a list of post-approval measures with the CHMP (Committee for Medical Products for Human Use, the scientific committee of the European Medicines Agency). Subject to fulfilling the agreed commitments and achieving market access in various countries, a potential first European launch of Ledaga is not expected before January 2018.

ABOUT LEDAGA (CHLORMETHINE)

Ledaga (chlormethine) is an alkylating drug indicated for the treatment of mycosis fungoides-type cutaneous T-Cell lymphoma (MF-CTCL) formulated as a topical, once-daily, colorless gel.

Chlormethine gel, under the brand name Valchlor (mechlorethamine) is commercially available in the US (since 2013) and in Israel through special import authorization procedure (since 2016). In France, patients benefit from the drug under a temporary authorization for use ("ATU") program initiated during the second half of 2014.

ABOUT MF-CTCL

Mycosis fungoides-type cutaneous T-Cell lymphoma (MF-CTCL) is a rare, but serious and life-threatening, immune system cancer that appears in the skin. MF-CTCL is the most common form of cutaneous T-cell lymphoma.

MF-CTLC typically appears in patients over 50 years of age (median age is 54), and is more common in men. It presents first as dry skin and a red rash, with or without itching. As a result, MF-CTLC is often mistaken for eczema or psoriasis, delaying diagnosis. MF-CTLC goes on to form scaly plaques on the skin, which can cover small or large areas of the skin. Large bumps or tumor nodules may also develop, and lymph nodes may be involved.

While MF-CTCL is a chronic and usually slowly progressing disease, the course of disease in individual patients is unpredictable, with some patient progressing into advanced stages. In about 34% of cases, a progression of the disease is observed, and in the most advanced stages, MF-CTCL cells can metastasize to other body tissues, including the liver, spleen and lungs.

Current research suggests that patients who are diagnosed in early stages of MF-CTCL have a normal life expectancy, however, the average time to diagnosis ranges from two to seven years. An important therapeutic objective in treating MF-CTLC is prevention of disease progression. Failure to maintain MF-CTLC in its early stages results in a drastically reduced median survival.

ABOUT STUDY 201

Study 201 was a multicenter, randomized, observer-blinded, active-controlled, 12-month study of Stage I and IIA MF-type CTCL patients, conducted in 13 centers in the US to evaluate the efficacy and safety of chlormethine gel compared with chlormethine HCl 0.02% compounded in Aquaphor ointment. In total, 260 patients were randomized 1:1 to topical treatment with chlormethine gel or chlormethine HCl 0.02% compounded in Aquaphor ointment once daily for up to 12 months.

In the study, within the efficacy evaluable (EE) population, 77% of patients treated with chlormethine gel had a clinical response at 12 months, in the Composite Assessment of Index Lesion Severity (CAILS*) score, while 59% of those treated with the compounded control achieved a confirmed response. (*A response was defined as at least a 50% improvement in the baseline CAILS score).

Complete response was achieved in 19% of patients treated with chlormethine gel versus 15% of patients treated with the compounded control in the EE population. Reductions in mean lesion severity (CAILS) were seen as early as four weeks, with further reductions observed with continuing therapy.

The most frequent adverse reactions reported with chlormethine gel were skin related: dermatitis (54.7%; e.g., skin irritation, erythema, rash, urticaria, skin-burning sensation, pain of the skin), pruritus (20.3%), skin infections (11.7%), skin ulceration and blistering (6.3%), and skin hyperpigmentation (5.5%). No clinical evidence of systemic absorption of chlormethine was observed with the treatment.