0001157523-12-006348.txt : 20121211 0001157523-12-006348.hdr.sgml : 20121211 20121211163043 ACCESSION NUMBER: 0001157523-12-006348 CONFORMED SUBMISSION TYPE: 8-K PUBLIC DOCUMENT COUNT: 4 CONFORMED PERIOD OF REPORT: 20121209 ITEM INFORMATION: Other Events ITEM INFORMATION: Financial Statements and Exhibits FILED AS OF DATE: 20121211 DATE AS OF CHANGE: 20121211 FILER: COMPANY DATA: COMPANY CONFORMED NAME: ARIAD PHARMACEUTICALS INC CENTRAL INDEX KEY: 0000884731 STANDARD INDUSTRIAL CLASSIFICATION: BIOLOGICAL PRODUCTS (NO DIAGNOSTIC SUBSTANCES) [2836] IRS NUMBER: 223106987 STATE OF INCORPORATION: DE FISCAL YEAR END: 1231 FILING VALUES: FORM TYPE: 8-K SEC ACT: 1934 Act SEC FILE NUMBER: 000-21696 FILM NUMBER: 121256751 BUSINESS ADDRESS: STREET 1: 26 LANDSDOWNE ST CITY: CAMBRIDGE STATE: MA ZIP: 02139 BUSINESS PHONE: 6174940400 MAIL ADDRESS: STREET 1: 26 LANDSDOWNE CITY: CAMBRIDGE STATE: MA ZIP: 02139 8-K 1 a50504422.htm ARIAD PHARMACEUTICALS, INC. 8-K


UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549

FORM 8-K

CURRENT REPORT
Pursuant to Section 13 or 15(d) of The Securities Exchange Act of 1934

Date of Report (Date of earliest event reported): December 9, 2012

ARIAD Pharmaceuticals, Inc.
(Exact name of registrant as specified in its charter)

Delaware

0-21696

22-3106987

(State or other jurisdiction

of incorporation)

(Commission

File Number)

(I.R.S. Employer

Identification No.)

26 Landsdowne Street, Cambridge, Massachusetts

02139

(Address of principal executive offices)

(Zip Code)


Registrant's telephone number, including area code: (617) 494-0400

Not Applicable
(Former name or former address, if changed since last report)

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):

Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))


ITEM 8.01       Other Events.

On December 9, 2012, ARIAD Pharmaceuticals, Inc. (the “Company”) issued the first of three press releases on data featured at the 54th Annual Meeting of the American Society of Hematology (ASH) which was held in Atlanta, Georgia. The first press release announced twelve-month follow-up data from the Company’s pivotal PACE trial of ponatinib, its investigational BCR-ABL inhibitor, in heavily pretreated patients with resistant or refractory chronic myeloid leukemia (CML) or Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). The data reported showed that 56 percent of chronic-phase CML patients in the trial, including 70 percent of patients with a T315I mutation, achieved a major cytogenetic response (MCyR), the primary end-point for chronic-phase CML patients.  A copy of the press release is attached hereto as Exhibit 99.1 and is incorporated by reference into this Item 8.01 of this Current Report on Form 8-K.

In a press release issued on December 10, 2012, the Company announced updated molecular response data from ARIAD’s Phase 1 and pivotal Phase 2 trials of ponatinib in heavily pretreated patients with resistant or intolerant CML or Ph+ ALL.  The data reported showed that 51 percent of chronic-phase CML patients in the Phase 1 trial achieved a major molecular response (MMR) with a median follow-up of 30 months, and 34 percent of chronic-phase patients achieved MMR in the PACE trial with a median follow-up of 15 months. A copy of the press release is attached hereto as Exhibit 99.2 and is incorporated by reference into this Item 8.01 of this Current Report on Form 8-K.

In the third press release, issued on December 11, 2012, the Company announced twelve-month follow-up data from the pivotal PACE trial of ponatinib in heavily pretreated patients with advanced forms of CML or Ph+ ALL.  The data reported showed that 57 percent of accelerated-phase CML patients in the trial, including 50 percent of patients with the T315I mutation, achieved a major hematologic response (MaHR), the primary end-point for patients with advanced disease in the trial.  A copy of the press release is attached hereto as Exhibit 99.3 and is incorporated by reference into this Item 8.01 of this Current Report on Form 8-K.

ITEM 9.01 Financial Statements and Exhibits.

(d)   The following exhibit is filed with this report:

Exhibit

Number

Description

 
99.1 Press Release dated December 9, 2012.
 
99.2 Press Release dated December 10, 2012.
 
99.3 Press Release dated December 11, 2012.

The press releases contain hypertext links to information on our website.  The information on our website is not incorporated by reference into this Current Report on Form 8-K and does not constitute a part of this Form 8-K.

2

SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

ARIAD Pharmaceuticals, Inc.

 

 

 

By:  

/s/ Edward M. Fitzgerald

 

Edward M. Fitzgerald

Executive Vice President, Chief Financial Officer

 

Date:

December 11, 2012

3

EX-99.1 2 a50504422ex99_1.htm EXHIBIT 99.1

Exhibit 99.1

ARIAD Announces 12-Month Data from Pivotal PACE Trial of Ponatinib in Heavily Pretreated Chronic-Phase CML Patients

56 percent major cytogenetic response and 34 percent major molecular response reported

ATLANTA, & CAMBRIDGE, Mass.--(BUSINESS WIRE)--December 9, 2012--ARIAD Pharmaceuticals, Inc. (NASDAQ: ARIA) today announced twelve-month follow-up data from the pivotal PACE trial of ponatinib, its investigational BCR-ABL inhibitor, in heavily pretreated patients with resistant or refractory chronic myeloid leukemia (CML) or Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). The study now shows that 56 percent of chronic-phase CML patients in the trial, including 70 percent of patients with a T315I mutation, achieved a major cytogenetic response (MCyR), the primary end-point for chronic-phase CML patients.

The data are being featured today at 4:30 p.m. (ET) in an oral presentation at the 54th Annual Meeting of the American Society of Hematology (ASH) being held in Atlanta, Georgia. ARIAD filed for regulatory approval of ponatinib in the third quarter of 2012 in the U.S. and in the E.U. based on clinical data from the pivotal PACE trial.

“The 12-month results from the global PACE trial of ponatinib reinforce its impressive anti-leukemic activity in heavily pretreated CML patients, regardless of their mutation status or disease stage,” stated Jorge Cortes, M.D., professor and deputy chair, Department of Leukemia, The University of Texas M.D. Anderson Cancer Center, Houston, TX.

“Ponatinib demonstrated early responses in chronic-phase patients with thirty-four percent of these patients achieving a major molecular response and fifteen percent of those patients achieving a complete molecular response,” he added. “Of particular importance, responses to ponatinib appear to be durable, with 91 percent of chronic-phase CML patients projected to remain in major cytogenetic response at one year.”


  • Trial Design
    • Efficacy data were reported at ASH on 444 treated patients in six pre-specified cohorts at 45 mg of ponatinib administered orally once daily, including 267 patients with chronic-phase CML. Findings were based on a minimum follow-up of 12 months in patients remaining on study.
    • Ninety-three percent of the patients in the trial had received at least two tyrosine kinase inhibitors prior to enrollment. Fifty-eight percent of the patients had received three or more tyrosine kinase inhibitors prior to enrollment.
    • Chronic-phase patients had bone marrow assessments approximately every three months for determination of cytogenetic response.
  • Chronic-phase CML patients evaluable for cytogenetic response (N=267)
    • Based on assessment of all evaluable chronic-phase patients in the trial, 56 percent (149 of 267) of patients achieved a MCyR, with 46 percent achieving a complete cytogenetic response (CCyR). The median follow up of the chronic-phase CML patients is 15.3 months.
    • Of the 64 evaluable chronic-phase CML patients with the T315I mutation, 70 percent (45 of 64) of these patients achieved a MCyR, with 66 percent achieving a CCyR. The MCyR rate in evaluable chronic-phase patients without the T315I mutation was 51 percent (104 of 203).
    • Thirty-four percent (91 of 267) of chronic-phase CML patients achieved a major molecular response (MMR).
    • Fifteen percent (39 of 267) of chronic-phase CML patients achieved a 4.5-log reduction of BCR-ABL transcripts (MR4.5).
  • Responses in chronic-phase patients who had received only one prior TKI (N=19)
    • There were 19 chronic-phase patients treated with ponatinib in the PACE trial who had previously received only one tyrosine kinase inhibitor (TKI). Thirteen of these patients had previously been treated with imatinib only, and six had previously received either dasatinib or nilotinib. Of these 19 patients, 84 percent (16 of 19) achieved a MCyR.
  • Safety profile (N=449)
    • The most common non-hematologic treatment-emergent adverse events in the PACE trial included rash (in 38% of patients), abdominal pain (38%), headache (35%), dry skin (35%), and constipation (34%), with the majority of these being grades 1 or 2 in severity.
    • The most common hematologic treatment-emergent adverse events were thrombocytopenia (42%), neutropenia (24%), and anemia (20%), which were primarily grades 3 or 4 in severity.
    • Pancreatitis and pneumonia were the most common non-hematologic treatment-emergent serious adverse events (5% each), followed by abdominal pain (4%), myocardial infarction (3%), congestive heart failure (3%), atrial fibrillation (3%), and pyrexia (3%). The most common hematologic serious adverse events were anemia, febrile neutropenia, and thrombocytopenia (3% each).

About Ponatinib

Internally discovered at ARIAD, ponatinib is an investigational BCR-ABL inhibitor that also selectively inhibits certain other tyrosine kinases in preclinical studies, including FLT3, RET, KIT, and the members of the FGFR, PDGFR and VEGFR families of kinases.

The primary target for ponatinib is BCR-ABL, an abnormal tyrosine kinase that is expressed in chronic myeloid leukemia (CML) and Philadelphia-chromosome positive acute lymphoblastic leukemia (Ph+ ALL). Ponatinib was designed using ARIAD’s computational and structure-based drug design platform to inhibit the activity of BCR-ABL with high potency and broad specificity. Ponatinib targets not only native BCR-ABL but also its isoforms that carry mutations that confer resistance to treatment with existing tyrosine kinase inhibitors, including the T315I mutation for which no effective therapy currently exists.

About CML and Ph+ ALL

CML is a cancer of the white blood cells that is diagnosed in approximately 5,000 patients each year in the United States. CML is a type of leukemia characterized by the increased and unregulated growth of predominantly myeloid cells in the bone marrow and the accumulation of these cells in the blood. The genetic hallmark of CML is the Philadelphia chromosome, an abnormality resulting in a fusion of the BCR and ABL genes. This is known as Philadelphia chromosome-positive CML, or Ph+ CML.

Treatment of CML usually includes a targeted therapy, a tyrosine kinase inhibitor (TKI) (e.g., imatinib, dasatinib or nilotinib), followed by chemotherapy if the disease progresses. Ph+ ALL is a subtype of acute lymphoblastic leukemia that carries the Ph+ chromosome that produces the fused BCR-ABL gene. It is known to have a more aggressive course than CML and is often treated with a combination of chemotherapy and TKIs. Because both of these diseases express the BCR-ABL protein, this would render them potentially susceptible to treatment with ponatinib.


About ARIAD

ARIAD Pharmaceuticals, Inc. is an emerging global oncology company focused on the discovery, development and commercialization of medicines to transform the lives of cancer patients. ARIAD’s approach to structure-based drug design has led to several internally discovered, molecularly targeted product candidates for drug-resistant and difficult-to-treat cancers, including certain forms of chronic myeloid leukemia and non-small cell lung cancer. For additional information, visit http://www.ariad.com.

This press release contains “forward-looking statements” including, but not limited to, statements relating to the updated clinical data for ponatinib, the positive treatment effects of ponatinib over time and the timing of regulatory filings for marketing approvals. Forward-looking statements are based on management's expectations and are subject to certain factors, risks and uncertainties that may cause actual results, outcome of events, timing and performance to differ materially from those expressed or implied by such statements. These risks and uncertainties include, but are not limited to, preclinical data and early-stage clinical data that may not be replicated in later-stage clinical studies, the costs associated with our research, development, manufacturing and other activities, the conduct, timing and results of pre-clinical and clinical studies of our product candidates, the adequacy of our capital resources and the availability of additional funding, and other factors detailed in the Company's public filings with the U.S. Securities and Exchange Commission. The information contained in this press release is believed to be current as of the date of original issue. The Company does not intend to update any of the forward-looking statements after the date of this document to conform these statements to actual results or to changes in the Company's expectations, except as required by law.

CONTACT:
ARIAD Pharmaceuticals, Inc.
For Investors
Kendra Adams, 617-503-7028
Kendra.adams@ariad.com
or
For Media
Liza Heapes, 617-621-2315
Liza.heapes@ariad.com

EX-99.2 3 a50504422ex99_2.htm EXHIBIT 99.2

Exhibit 99.2

ARIAD Announces Long-Term Molecular Response Data on Ponatinib

51 percent of chronic-phase CML patients in Phase 1 trial and 34 percent in Phase 2 PACE trial achieve major molecular response

ATLANTA & CAMBRIDGE, Mass.--(BUSINESS WIRE)--December 10, 2012--ARIAD Pharmaceuticals, Inc. (NASDAQ: ARIA) today announced updated molecular response data from its Phase 1 and pivotal Phase 2 trials of ponatinib, its investigational BCR-ABL inhibitor, in heavily pretreated patients with resistant or intolerant chronic myeloid leukemia (CML) or Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). The studies now show that 51 percent of chronic-phase CML patients in the Phase 1 trial achieved a major molecular response (MMR) with a median follow-up of 30 months, and 34 percent of chronic-phase patients achieved MMR in the PACE trial with a median follow-up of 15 months.

These data are being featured today at 6:00 p.m. in two poster presentations at the 54th Annual Meeting of the American Society of Hematology (ASH) being held in Atlanta, Georgia.

Molecular response is a measurement of blood levels of the transcript product of the BCR-ABL oncogene. MMR is defined as a value less than or equal to 0.1% on the accepted International Scale. All patient samples were evaluated for molecular response at a single central laboratory (Molecular MD) using a standardized assay. MMR is a secondary efficacy endpoint for chronic-phase CML patients in ARIAD’s Phase 1 and pivotal Phase 2 PACE trials of ponatinib.

Phase 1 Trial MMR Rates in Chronic-Phase CML Patients

The ongoing Phase 1 dose-escalation study of ponatinib enrolled 81 patients with resistant or refractory hematologic cancers, including 43 patients with chronic-phase CML. Sixty-one percent of the chronic-phase CML patients in this study had failed at least three prior tyrosine kinase inhibitors (TKI).


  • With a median follow-up of 30 months, 51 percent (22 of 43) of patients with chronic-phase CML enrolled in the study achieved MMR, including 75 percent (9 of 12) who had the T315I mutation, which is the most common mutation among resistant patients.
  • The median time to MMR was 5.6 months, and the median duration of MMR in chronic-phase CML has not yet been reached. At the time of analysis, 21 of 22 patients who achieved MMR remained in the study and continued to receive ponatinib.
  • Molecular response rates increased over time with nine percent (4 of 43) of chronic-phase CML patients achieving MMR by 3 months and 51 percent (22 of 43) achieving MMR overall. Patients continued to achieve MMRs after 12 months of follow-up.
  • Thirty-three percent (14 of 43) of chronic-phase CML patients achieved MR4 (4-log reduction in BCR-ABL transcripts).
  • The most common non-hematologic treatment-related adverse events in all patients in this trial included rash (42%), arthralgia (20%), increased lipase (20%), fatigue (19%) and dry skin (19%), with the majority of these being grades 1 or 2 in severity. The most common hematologic treatment-related adverse events included thrombocytopenia (34%), neutropenia (14%) and anemia (12%), with thrombocytopenia and neutropenia being primarily grades 3 or 4 in severity.

PACE Trial MMR Rates in Chronic-Phase CML Patients

The ongoing pivotal Phase 2 PACE trial enrolled 449 patients with chronic myeloid leukemia (CML) or Philadelphia-positive acute lymphoblastic leukemia (Ph+ ALL), who are resistant or intolerant to dasatinib or nilotinib or who have the T315I mutation.

  • With a median follow up of 15 months, 34 percent (91 of 267) of chronic-phase CML patients achieved MMR; the reported prior MMR rate to their most recent TKI was three percent.
  • Fifteen percent (39 of 267) of patients achieved a 4.5-log reduction of BCR-ABL transcripts (MR4.5).
  • Fifty-three percent (10 of 19) of chronic-phase patients who failed only one prior approved TKI achieved MMR with ponatinib.
  • The median time to MMR among responders was 6 months. MMR was durable with 81 percent of patients estimated to remain in MMR at 12 months (by Kaplan-Meier analysis). Median duration of MMR among chronic-phase CML patients has not yet been reached.

  • The most common non-hematologic treatment-emergent adverse events in all patients in the PACE trial included rash (38%), abdominal pain (38%), headache (35%), dry skin (35%), and constipation (34%), with the majority of these being grades 1 or 2 in severity.
  • The most common hematologic treatment-emergent adverse events were thrombocytopenia (42%), neutropenia (24%), and anemia (20%), which were primarily grades 3 or 4 in severity.
  • Pancreatitis and pneumonia were the most common non-hematologic treatment-emergent serious adverse events (5% each), followed by abdominal pain (4%), myocardial infarction (3%), congestive heart failure (3%), atrial fibrillation (3%), and pyrexia (3%). The most common hematologic serious adverse events were anemia, febrile neutropenia, and thrombocytopenia (3% each).

About Ponatinib

Internally discovered at ARIAD, ponatinib is an investigational BCR-ABL inhibitor that also selectively inhibits certain other tyrosine kinases in preclinical studies, including FLT3, RET, KIT, and the members of the FGFR, PDGFR and VEGFR families of kinases.

The primary target for ponatinib is BCR-ABL, an abnormal tyrosine kinase that is expressed in chronic myeloid leukemia (CML) and Philadelphia-chromosome positive acute lymphoblastic leukemia (Ph+ ALL). Ponatinib was designed using ARIAD’s computational and structure-based drug design platform to inhibit the activity of BCR-ABL with high potency and broad specificity. Ponatinib targets not only native BCR-ABL but also its isoforms that carry mutations that confer resistance to treatment with existing tyrosine kinase inhibitors, including the T315I mutation for which no effective therapy currently exists.

About CML and Ph+ ALL

CML is a cancer of the white blood cells that is diagnosed in approximately 5,000 patients each year in the United States. CML is a type of leukemia characterized by the increased and unregulated growth of predominantly myeloid cells in the bone marrow and the accumulation of these cells in the blood. The genetic hallmark of CML is the Philadelphia chromosome, an abnormality resulting in a fusion of the BCR and ABL genes. This is known as Philadelphia chromosome-positive CML, or Ph+ CML.

Treatment of CML usually includes a targeted therapy, a tyrosine kinase inhibitor (TKI) (e.g., imatinib, dasatinib or nilotinib), followed by chemotherapy if the disease progresses. Ph+ ALL is a subtype of acute lymphoblastic leukemia that carries the Ph+ chromosome that produces the fused BCR-ABL gene. It is known to have a more aggressive course than CML and is often treated with a combination of chemotherapy and TKIs. Because both of these diseases express the BCR-ABL protein, this would render them potentially susceptible to treatment with ponatinib.


About ARIAD

ARIAD Pharmaceuticals, Inc. is an emerging global oncology company focused on the discovery, development and commercialization of medicines to transform the lives of cancer patients. ARIAD’s approach to structure-based drug design has led to several internally discovered, molecularly targeted product candidates for drug-resistant and difficult-to-treat cancers, including certain forms of chronic myeloid leukemia and non-small cell lung cancer. For additional information, visit http://www.ariad.com.

This press release contains “forward-looking statements” including, but not limited to, statements relating to the updated clinical data for ponatinib, the positive treatment effects of ponatinib over time and the timing of regulatory filings for marketing approvals. Forward-looking statements are based on management's expectations and are subject to certain factors, risks and uncertainties that may cause actual results, outcome of events, timing and performance to differ materially from those expressed or implied by such statements. These risks and uncertainties include, but are not limited to, preclinical data and early-stage clinical data that may not be replicated in later-stage clinical studies, the costs associated with our research, development, manufacturing and other activities, the conduct, timing and results of pre-clinical and clinical studies of our product candidates, the adequacy of our capital resources and the availability of additional funding, and other factors detailed in the Company's public filings with the U.S. Securities and Exchange Commission. The information contained in this press release is believed to be current as of the date of original issue. The Company does not intend to update any of the forward-looking statements after the date of this document to conform these statements to actual results or to changes in the Company's expectations, except as required by law.

CONTACT:
ARIAD Pharmaceuticals, Inc.
For Investors
Kendra Adams, 617-503-7028
Kendra.adams@ariad.com
or
For Media
Liza Heapes, 617-621-2315
Liza.heapes@ariad.com

EX-99.3 4 a50504422ex99_3.htm EXHIBIT 99.3

Exhibit 99.3

ARIAD Announces 12-Month Data from Pivotal PACE Trial of Ponatinib in Heavily Pretreated Patients with Advanced-Phase CML and Ph+ ALL

57 percent major hematologic response and median response duration of 12 months in accelerated-phase CML patients reported

Median overall survival not yet reached in accelerated-phase CML patients

ATLANTA & CAMBRIDGE, Mass.--(BUSINESS WIRE)--December 11, 2012--ARIAD Pharmaceuticals, Inc. (NASDAQ: ARIA) today announced twelve-month follow-up data from the pivotal PACE trial of ponatinib, its investigational BCR-ABL inhibitor, in heavily pretreated patients with advanced forms of chronic myeloid leukemia (CML) or Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). The study now shows that 57 percent of accelerated-phase CML patients in the trial, including 50 percent of patients with the T315I mutation, achieved a major hematologic response (MaHR), the primary end-point for patients with advanced disease in the trial.

The data are being featured today at 8:00 a.m. (ET) in an oral presentation at the 54th Annual Meeting of the American Society of Hematology (ASH) being held in Atlanta, Georgia. ARIAD filed for regulatory approval of ponatinib in the third quarter of 2012 in the U.S. and in the E.U. based on clinical data from the pivotal PACE trial.

“Patients with advanced forms of Philadelphia chromosome-positive leukemia and those who have failed currently available therapy have limited treatment options available to them,” said Hagop M. Kantarjian, M.D., chairman and professor, Department of Leukemia, University of Texas M.D. Anderson Cancer Center. “The overall prognosis is poor for patients with advanced disease.”

“The pivotal PACE trial data show that ponatinib has robust activity in heavily pretreated patients with accelerated phase CML, more than doubling their reported best prior responses to available TKI therapy,” he added. “What is equally striking is that the median time to achieve a response to ponatinib among accelerated phase patients was only three weeks and that the median duration of major hematologic response in these patients is one year.”


  • Trial Design
    • Efficacy data were reported at ASH on 444 treated patients in six pre-specified cohorts at 45 mg of ponatinib administered orally once daily, including 177 treated patients with advanced disease (i.e., accelerated and blast phase CML and Ph+ ALL).
    • Sixty percent of accelerated phase CML patients and 53 percent of blast-phase CML and Ph+ ALL patients in the trial had received three or more tyrosine kinase inhibitors (TKI) prior to enrollment.
    • Advanced disease patients had a blood test approximately every month for determination of hematologic response and a bone-marrow assessment approximately every two months for determination of cytogenetic response.
  • Advanced CML and Ph+ ALL patients evaluable for response (N=177)
    • Fifty-seven percent (47 of 83) of accelerated-phase patients achieved a MaHR, including 50 percent (9 of 18) of accelerated-phase patients with the T315I mutation. At study entry, the reported best prior response of MaHR or better to their most recent TKI among accelerated-phase patients was 21 percent.
    • Thirty-four percent (32 of 94) of blast-phase CML or Ph+ ALL patients achieved a MaHR, including 33 percent (15 of 46) of blast-phase CML or Ph+ ALL patients with the T315I mutation. At study entry, the reported best prior response of MaHR or better to their most recent treatment with a TKI among blast-phase CML or Ph+ ALL patients was 24 percent.
    • Thirty-nine percent (32 of 83) of accelerated-phase CML patients and 31 percent (29 of 94) of blast-phase CML or Ph+ ALL patients achieved a MCyR. Furthermore, 24 percent (20 of 83) of patients with accelerated-phase CML and 24 percent (23 of 94) of patients with blast-phase CML or Ph+ ALL achieved a complete cytogenetic response.
  • Median duration, progression-free survival and overall survival
    • In accelerated-phase CML patients, the median time to achieve a MaHR was 21 days, and the median duration of this response was 12 months. In blast-phase CML or Ph+ ALL patients, the median time to achieve a MaHR was 26 days, and the median duration of this response was 5 months.
    • Progression-free survival (PFS) in accelerated-phase CML patients was estimated to be 55 percent at 12 months (median, 18 months). Progression-free survival in blast-phase CML or Ph+ ALL patients was estimated to be 15 percent at 12 months (median, 3 months).
    • Overall survival at 12 months in accelerated-phase CML patients was estimated to be 84 percent (median not yet reached). Overall survival at 12 months in blast-phase CML or Ph+ ALL patients was estimated to be 33 percent, with a median overall survival of 7 months.
  • Safety profile (N=449)
    • The most common non-hematologic treatment-emergent adverse events across all patients in the PACE trial included rash (in 38% of patients), abdominal pain (38%), headache (35%), dry skin (35%), and constipation (34%), with the majority of these being grades 1 or 2 in severity.
    • The most common hematologic treatment-emergent adverse events were thrombocytopenia (42%), neutropenia (24%), and anemia (20%), which were primarily grades 3 or 4 in severity.
    • Pancreatitis and pneumonia were the most common non-hematologic treatment-emergent serious adverse events (5% each), followed by abdominal pain (4%), myocardial infarction (3%), congestive heart failure (3%), atrial fibrillation (3%), and pyrexia (3%). The most common hematologic serious adverse events were anemia, febrile neutropenia, and thrombocytopenia (3% each).

About Ponatinib

Internally discovered at ARIAD, ponatinib is an investigational BCR-ABL inhibitor that also selectively inhibits certain other tyrosine kinases in preclinical studies, including FLT3, RET, KIT, and the members of the FGFR, PDGFR and VEGFR families of kinases.

The primary target for ponatinib is BCR-ABL, an abnormal tyrosine kinase that is expressed in chronic myeloid leukemia (CML) and Philadelphia-chromosome positive acute lymphoblastic leukemia (Ph+ ALL). Ponatinib was designed using ARIAD’s computational and structure-based drug design platform to inhibit the activity of BCR-ABL with high potency and broad specificity. Ponatinib targets not only native BCR-ABL but also its isoforms that carry mutations that confer resistance to treatment with existing tyrosine kinase inhibitors, including the T315I mutation for which no effective therapy currently exists.

About CML and Ph+ ALL

CML is a cancer of the white blood cells that is diagnosed in approximately 5,000 patients each year in the United States. CML is a type of leukemia characterized by the increased and unregulated growth of predominantly myeloid cells in the bone marrow and the accumulation of these cells in the blood. The genetic hallmark of CML is the Philadelphia chromosome, an abnormality resulting in a fusion of the BCR and ABL genes. This is known as Philadelphia chromosome-positive CML, or Ph+ CML.

Treatment of CML usually includes a targeted therapy, a tyrosine kinase inhibitor (TKI) (e.g., imatinib, dasatinib or nilotinib), followed by chemotherapy if the disease progresses. Ph+ ALL is a subtype of acute lymphoblastic leukemia that carries the Ph+ chromosome that produces the fused BCR-ABL gene. It is known to have a more aggressive course than CML and is often treated with a combination of chemotherapy and TKIs. Because both of these diseases express the BCR-ABL protein, this would render them potentially susceptible to treatment with ponatinib.


About ARIAD

ARIAD Pharmaceuticals, Inc. is an emerging global oncology company focused on the discovery, development and commercialization of medicines to transform the lives of cancer patients. ARIAD’s approach to structure-based drug design has led to several internally discovered, molecularly targeted product candidates for drug-resistant and difficult-to-treat cancers, including certain forms of chronic myeloid leukemia and non-small cell lung cancer. For additional information, visit http://www.ariad.com.

This press release contains “forward-looking statements” including, but not limited to, statements relating to the updated clinical data for ponatinib, the positive treatment effects of ponatinib over time and the timing of regulatory filings for marketing approvals. Forward-looking statements are based on management's expectations and are subject to certain factors, risks and uncertainties that may cause actual results, outcome of events, timing and performance to differ materially from those expressed or implied by such statements. These risks and uncertainties include, but are not limited to, preclinical data and early-stage clinical data that may not be replicated in later-stage clinical studies, the costs associated with our research, development, manufacturing and other activities, the conduct, timing and results of pre-clinical and clinical studies of our product candidates, the adequacy of our capital resources and the availability of additional funding, and other factors detailed in the Company's public filings with the U.S. Securities and Exchange Commission. The information contained in this press release is believed to be current as of the date of original issue. The Company does not intend to update any of the forward-looking statements after the date of this document to conform these statements to actual results or to changes in the Company's expectations, except as required by law.

CONTACT:
ARIAD Pharmaceuticals, Inc.
For Investors
Kendra Adams, 617-503-7028
Kendra.adams@ariad.com
or
For Media
Liza Heapes, 617-621-2315
Liza.heapes@ariad.com