On October 27, 2016 Cempra, Inc. (Nasdaq:CEMP), a clinical-stage pharmaceutical company focused on developing antibiotics to meet critical medical needs in the treatment of bacterial infectious diseases, reported financial results for the quarter ended September 30, 2016 and provided an update on recent corporate developments (Press release, Cempra, OCT 27, 2016, View Source [SID1234516066]). The company will host a webcast and conference call today at 5:00 p.m. EDT.

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Third Quarter 2016 and Recent Corporate Highlights

On October 25, Cempra completed enrollment in its ongoing Phase 3 study of fusidic acid as an oral treatment for acute bacterial skin and skin structure infections. The company expects to have topline data to report in the first quarter of 2017.

On October 4, Cempra presented an analysis of patient data at the Academy of Managed Care Pharmacy meeting. The presentation included three abstracts based upon analyzing treatment failures with existing antibiotics from more than 400,000 records of patients diagnosed with community-acquired bacterial pneumonia (CABP) in a large U.S. insurance claims database. This analysis showed that more than one out of five adult CABP patients failed initial antibiotic monotherapy, with the failure rate exceeding one out of four for an elderly population with certain comorbidities.

On September 29, Cempra announced interim results showing anti-NASH effects in the first six nonalcoholic steatohepatitis (NASH) patients dosed with solithromycin in a Phase 2 study. After 90 days of solithromycin treatment, all six NASH patients had a reduction in their nonalcoholic fatty liver disease activity score (NAS), as well as alanine aminotransferase (ALT) reduction. Based on the safety profile and activity seen in the first six patients, Cempra plans to continue the Phase 2 study to obtain data from up to 15 NASH patients. Enrollment is expected to complete in the first quarter of 2017.
On August 30, Cempra announced that the U.S. Food and Drug Administration (FDA) scheduled a meeting of the Antimicrobial Drugs Advisory Committee on November 4, 2016 in Silver Spring, Maryland, to discuss the safety and efficacy of solithromycin in the treatment of CABP. The FDA is currently reviewing New Drug Applications (NDAs) for IV and oral solithromycin for CABP, with PDUFA dates in late December.

On August 25, Cempra announced the publication in Clinical Infectious Diseases of its pivotal Phase 3 study, SOLITAIRE-IV, comparing the efficacy and safety of intravenous-to-oral solithromycin to intravenous-to-oral moxifloxacin for the treatment of CABP. Both of Cempra’s pivotal Phase 3 studies have been published in leading infectious disease journals.

On August 23, Cempra announced that the European Medicines Agency (EMA) had validated the company’s marketing authorization application (MAA) seeking approval of oral capsule and intravenous formulations of solithromycin for the treatment of CABP. The EMA’s Committee for Medicinal Products for Human Use (CHMP) has begun its assessment of solithromycin through the centralized review procedure. If the CHMP review results in a positive opinion, the next step would be for the European Commission to grant marketing clearance for solithromycin, which would apply to all EU member states.

On August 11, Cempra appointed David Zaccardelli, Pharm.D. to serve on the company’s board of directors.

On July 5, Cempra announced that the FDA had accepted for filing Cempra’s two NDAs for intravenous and oral capsule formulations of solithromycin in CABP. Having been granted qualified infectious diseases product (QIDP) designation in 2013, solithromycin’s NDAs have qualified for an eight month priority review, with 2016 PDUFA dates of December 27 for the oral formulation and December 28 for intravenous.
"Cempra continued an exceptional 2016 with further progress in the third quarter, including FDA acceptance of our NDAs for intravenous and oral capsule formulations of solithromycin, the submission of our solithromycin MAA in Europe, publication of our IV to oral Phase 3 solithromycin study in a prestigious journal, and the announcement of exciting interim results from our Phase 2 NASH study," said Prabhavathi Fernandes, Ph.D., president and chief executive officer of Cempra.

"The company is preparing for the potential approval and 2017 launch of solithromycin and we look forward to discussing the safety and efficacy profile of solithromycin, along with the urgent and growing unmet medical need for a new macrolide antibiotic, at our advisory committee meeting next week," Fernandes added.

Upcoming Clinical Development Milestones

Solithromycin

Solithromycin pediatric
Patient enrollment for Phase 1b trial continues.
Phase 2/3 pivotal trial with solithromycin for bacterial infections in pediatric patients has been initiated. Global sites have been added.
Phase 3 trial for solithromycin in urogenital gonorrhea is ongoing.
Enrollment of the Phase 2 NASH trial is ongoing.
FDA Antimicrobial Drugs Advisory Committee to discuss solithromycin November 4, 2016.
Fusidic acid

Data from Phase 3 study in ABSSSI expected in first quarter of 2017.
An exploratory trial for Taksta in patients with refractory bone or joint infections is ongoing.
Financial Results for the Three Months Ended September 30, 2016

For the quarter ended September 30, 2016, Cempra reported a net loss of $32.3 million, or $0.62 per share, compared to a net loss of $27.6 million, or $0.63 per share for the third quarter in 2015. Research and development expense in the third quarter of 2016 was $21.1million, a decrease of 10.2% compared to $23.5 million in the third quarter of 2015. The lower R&D expense was primarily due to a decrease of clinical trial expenses for solithromycin and reduced fusidic acid clinical trial supply purchases. General and administrative expense was $15.0 million compared to $5.8 million in the third quarter of 2015, driven primarily by commercial readiness activities and increased headcount as the company continues to plan for commercialization of solithromycin in 2017.

As of September 30, 2016, Cempra had cash and equivalents of $248.9 million and 52.4 million shares outstanding. In the third quarter, the company utilized its ATM financing facility with the sale of approximately 1.6 million shares resulting in net proceeds of approximately $29.2 million.

On October 27, 2016 BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) reported financial results for the third quarter ended September 30, 2016 (Press release, BioMarin, OCT 27, 2016, View Source [SID1234516056]). GAAP net loss was $43 million, or $(0.26) per basic and diluted share, for the third quarter of 2016, compared to GAAP net loss of $91 million, or $(0.57) and $(0.60) per basic and diluted share, respectively, for the third quarter of 2015. Non-GAAP income was $3 million for the quarter ended September 30, 2016, compared to non-GAAP loss of $41 million for the third quarter of 2015.

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The change in GAAP net loss and non-GAAP income and loss compared to the prior year quarter was primarily due to increased gross margins from Naglazyme, Kuvan and Vimizim net product revenues, partially offset by increased selling, general and administrative expenses for Vimizim and Kuvan.

Total BioMarin Revenues were $280 million for the third quarter of 2016, an increase of 34% compared to the same period in 2015. Vimizim net product revenues increased to $81 million, a 25% year over year increase. Patients on therapy for Vimizim increased 46% year over year. The decrease in Vimizim net product revenues quarter to quarter was attributable to forward buying in Latin America and the Middle East in the second quarter of 2016. Naglazyme net product revenues increased to $78 million, a 44%, year over year increase, due to the timing of central government orders from Latin America in the current quarter. Naglazyme patients on therapy continue to show consistent growth with an increase of 10% year over year. Kuvan net product revenues increased to $91 million, a 42% year over year increase, including $71 million contributed from revenue in North America due to a 15% increase in patients on therapy, and $20 million contributed from net product revenues in the newly acquired ex-North American territories.

As of September 30, 2016, BioMarin had cash, cash equivalents and investments totaling $1.4 billion, which includes $713 million of net proceeds from the August 12, 2016 public offering, as compared to $1.0 billion on December 31, 2015.

Commenting on the quarter, Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, said, "In the third quarter of 2016 we shared proof-of-concept data from our BMN 270 gene therapy program for the treatment of Hemophilia A, currently the only factor VIII product in clinical development in this indication. In addition, our regulatory filings for approval of Brineura, for the treatment of Batten disease, were accepted and validated in both the U.S. and EU. With the Prescription Drug User Fee Act (PDUFA) goal date for an FDA approval decision of April 27, 2017, we hope to have an approved treatment option for this devastating childhood disease in the near future."

Mr. Bienaimé continued, "In addition, just last week we announced the results of the 30µg/kg dose cohort from our Phase 2 study with vosoritide in achondroplasia, which demonstrated similar efficacy as the lower dose of 15µg/kg. Based on these results, we intend to initiate a one-year, randomized, placebo-controlled Phase 3 study in children with achondroplasia ages 5-14 at the 15µg/kg dose with a subsequent open-label extension by year-end. If the data from both the vosoritide and BMN 270 gene therapy programs continue to mature as we hope, we believe that each of these product candidates has the potential to ultimately drive a billion dollars in annual revenue, if approved and successfully commercialized."

Net Product Revenues (in millions of U.S. dollars, unaudited)

Total BioMarin Revenues

Three Months Ended September 30, Nine Months Ended September 30,
2016 2015 $ Change % Change 2016 2015 $ Change % Change

Vimizim (1) $ 81 $ 65 $ 16 25 % $ 260 $ 170 $ 90 53 %
Naglazyme (1) 78 54 24 44 % 222 243 (21 ) (9 )%
Kuvan (2) 91 64 27 42 % 258 175 83 47 %
Aldurazyme 24 21 3 14 % 59 59 — —
Firdapse 4 4 — — 13 11 2 18 %
Net product revenues $ 278 $ 208 $ 70 34 % $ 812 $ 658 $ 154 23 %

Collaborative, royalty, license and other revenues $ 2 $ 1 $ 1 $ 5 $ 4 $ 1
Total BioMarin Revenues $ 280 $ 209 $ 71 34 % $ 817 $ 662 $ 155 23 %

(1) Vimizim and Naglazyme net product revenues experience quarterly fluctuations primarily due to the timing of government ordering patterns in certain countries. The Company does not believe these fluctuations reflect a change in underlying demand.
(2) North America contributed $71 million in the third quarter with an additional $20 million coming from the newly acquired ex-North American territories.

Details of Net Product Revenues Attributable to Aldurazyme

Three Months Ended September 30, Nine Months Ended September 30,
2016 2015 $ Change % Change 2016 2015 $ Change % Change
Aldurazyme revenue reported by Genzyme $ 59 $ 54 $ 5 9 % $ 169 $ 164 $ 5 3 %

Three Months Ended September 30, Nine Months Ended September 30,
2016 2015 $ Change 2016 2015 $ Change
Royalties earned from Genzyme $ 27 $ 23 $ 4 $ 71 $ 69 $ 2
Net product transfer revenues (3) $ (3 ) $ (2 ) $ (1 ) $ (12 ) $ (10 ) $ (2 )
Total Aldurazyme net product revenues $ 24 $ 21 $ 3 $ 59 $ 59 $ —

(3) To the extent units shipped to third party customers by Genzyme exceed BioMarin inventory transfers to Genzyme, BioMarin will record a decrease in net product revenues from the royalty payable to BioMarin for the amount of previously recognized product transfer revenue. If BioMarin inventory transfers exceed units shipped to third party customers by Genzyme, BioMarin will record incremental net product transfer revenues for the period. Positive net product transfer revenues result in the period if BioMarin transferred more units to Genzyme than Genzyme sold to third-party customers.

Updated 2016 Financial Guidance

Revenue Guidance ($ in millions)

Item
Provided August 4, 2016 Updated October 27, 2016
Total BioMarin Revenues $1,100 to $1,150 Unchanged
Vimizim Net Product Revenues $340 to $360 Unchanged
Naglazyme Net Product Revenues $290 to $320 Unchanged
Kuvan Net Product Revenues $340 to $360 Unchanged
Select Income Statement Guidance ($ in millions, except percentages)

Item
Provided August 4, 2016 Updated October 27, 2016
Cost of Sales (% of Total BioMarin Revenues) 18.0% to 19.0% Unchanged
Selling, General and Admin. Expense $470 to $490 $460 to $480
Research and Development Expense $670 to $690 $650 to $670
GAAP Net Loss $(620) to $(650) $(600) to $(630)
non-GAAP Loss $(30) to $(50) $(10) to $(30)
Recent Key Program Updates

BMN 270 gene therapy product for hemophilia A: On October 13, 2016, the Company announced that the Medicines and Healthcare products Regulatory Agency (MHRA) in the United Kingdom approved continued enrollment into the open-label Phase 1/2 study of BMN 270. BioMarin had previously announced that after enrolling the first nine patients in the study, that dosing of patients had been suspended due to observed increases in ALT levels that exceeded a pre- specified threshold set by the Company.

The agency also approved the Company’s proposed amendments to the study, which included eliminating the requirement for prophylactic corticosteroids and increasing potential additional enrollment from up to three additional patients to up to six additional patients. Based on protocol amendments agreed to with the MHRA three patients will be enrolled at a dose of 4 x 1013 vg/kg, and an additional three may be enrolled at this dose or the previously tested high dose of 6 x 1013 vg/kg. In the up to six additional patients, the threshold for starting therapeutic corticosteroids has been increased. BioMarin intends to provide an update on the ongoing Phase 1/2 study in December 2016. Safety and efficacy data from these patients will inform the Phase 2b study planned to begin in the second half of 2017.

Vosoritide for achondroplasia: On October 19, 2016, the Company provided an update on its Phase 2 study of vosoritide, an analog of C-type Natriuretic Peptide (CNP), in children with achondroplasia, the most common form of dwarfism, at the American Society of Human Genetics 2016 Meeting. Results from eight children in cohort 4, who completed six months of daily dosing at 30 µg/kg/daily experienced a 46% or 2.1 cm/year increase in mean annualized growth velocity from baseline (p-value = 0.03). These data are comparable to those observed at the lower dose of 15 µg/kg/day in cohort 3. Results from 10 children in cohort 3, who completed six months of daily dosing at 15 µg/kg/day experienced a 50% or 2.0 cm/year increase in mean annualized growth velocity from baseline (p-value = 0.01). Based on these data, the Company intends to initiate a one-year, randomized, placebo-controlled Phase 3 study in children with achondroplasia ages 5-14 with a subsequent open-label extension by year-end with the 15 µg/kg/day dose. Children in this study will have completed a minimum six-month natural history study to determine their respective baseline growth velocity prior to entering the Phase 3 study.

Brineura for CLN2, late-infantile form of Batten disease: During the quarter, the Company announced that the U.S. Food and Drug Administration (FDA) had accepted for review the submission of a Biologics License Application (BLA) for Brineura, an investigational therapy to treat children with CLN2 disease, a form of Batten disease. The Prescription Drug User Fee Act (PDUFA) goal date for a decision is April 27, 2017. The FDA granted Brineura Priority Review status, which is designated for drugs that offer major advances in treatment or provide a treatment where no adequate therapy exists. Brineura was previously granted Orphan Drug Designation and Breakthrough Therapy Designation by the FDA. BioMarin also received validation of the Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for Brineura. Assuming a positive opinion from the CHMP and standard assessment timing, a decision from the European Commission is anticipated by the third quarter of 2017. The EMA previously granted Brineura Orphan Drug Designation.

Pegvaliase for phenylketonuria (PKU): Pivotal results for the Phase 3 PRISM-2 study (formerly referred to as 165-302) that pegvaliase met the primary endpoint of change in blood phe compared with placebo (p<0.0001) were announced in the first quarter of 2016. The pegvaliase treated group maintained mean blood phe levels at 527.2 umol/L compared to their Randomized Discontinuation Trial (RDT) baseline of 503.9 umol/L, whereas the placebo treated group mean blood phe levels increased to 1385.7 umol/L compared to their RDT baseline of 536.0 umol/L. The treatment effect demonstrated in this study represents an approximately 62% improvement in blood phe compared to placebo. Based on the supportive data results, the Company plans to submit a BLA to the FDA in the first quarter of 2017.

On October 28, 2016 Astellas Pharma Inc. (TSE: 4503, President and CEO: Yoshihiko Hatanaka, "Astellas") and Ganymed Pharmaceuticals AG (CEO; Özlem Türeci, "Ganymed"), a biopharmaceutical company located in Mainz, Germany which focuses on the development of antibodies against cancer, reported that Astellas and Ganymed’s shareholders have entered into an agreement for Astellas to acquire Ganymed (Press release, Astellas, OCT 27, 2016, View Source [SID1234516055]). The transaction would enable Astellas to continue to build upon its leading oncology franchise as a platform for sustainable growth.

Under the agreement, Astellas will pay EUR 422 million to acquire 100% of the equity in Ganymed. In addition, Ganymed’s shareholders will become eligible to receive up to EUR 860 million in further contingent payments based on progress in the development of IMAB362, Ganymed’s most advanced clinical program. Upon completion of the transaction, Ganymed would become a wholly owned subsidiary of Astellas. The closing of the transaction is subject to customary regulatory approvals, and is expected to be finalized in the next several weeks.

"Oncology is one of our focus therapeutic areas and key drivers for our growth. The acquisition of Ganymed will enable Astellas to further expand our oncology presence by adding a late-stage antibody asset with the potential to establish a new pillar following XTANDI," commented Yoshihiko Hatanaka, President and CEO, Astellas. "We aim to deliver a potential new therapeutic option to cancer patients who currently have limited treatment options available to them."

Dr. Özlem Türeci, CEO and co-founder of Ganymed said, "I am impressed by the competence, commitment and vision of Astellas. Recognizing their global resources, I am also confident that Astellas will be able to turn innovative science and promising clinical results into near-term value for patients". And Helmut Jeggle, Ganymed’s Supervisory Board member and General Manager of the family office of the Strüngmann brothers (Athos Beteiligungsverwaltung GmbH) added, "We are pleased to hand over the Ganymed portfolio with its important novel treatment approaches for unmet needs in solid tumors to a strong and dedicated player in healthcare".

Ganymed is a privately-held biopharmaceutical company founded in 2001 and focuses on the development of a new class of cancer drugs. Ganymed has several oncology pipeline assets in pre-clinical and clinical stages including IMAB362. Recent results of a Phase 2b study (FAST) of IMAB362 in gastroesophageal cancer patients positive for Claudin18.2 showed that IMAB362 extended the median progression-free survival (7.9 months vs. 4.8 months, HR 0.47, p=0.0001) and the median overall survival (13.2 months vs. 8.4 months, HR 0.51, p=0.0001) when added to standard chemotherapy. In the subgroup of patients with the highest levels of Claudin18.2, IMAB362 resulted in near-doubling of overall survival (16.7 months vs. 9.0 months, HR 0.45, p<0.0005). The most frequent adverse effects observed during the study were vomiting, nausea and neutropenia.

Astellas is still reviewing the impact of this transaction on its financial forecasts for the fiscal year ending March 31, 2017.

Acquisition Summary
Acquiring company: Astellas Pharma Inc.
Major shareholders of Ganymed: ATS Beteiligungsverwaltung GmbH, MIG Fonds, FCP Gany GmbH and Future Capital AG
Payment: Cash on hand
Amount:
EUR 422 million upon the acquisition of 100% equity in Ganymed
Up to EUR 860 million in further contingent payments based on progress in the development of IMAB362, Ganymed’s most advanced clinical program
Expected timing of closing: Next several weeks, subject to customary regulatory approvals
Finance Advisor to Astellas: None
Legal Advisor to Astellas: Latham & Watkins LLP
Finance Advisor to Ganymed’ shareholders: klugeconcepts, JP Morgan
Legal Advisor to Ganymed`shareholders: Freshfields Bruckhaus Deringer, honert + partner

Overview of Acquired Company
Corporate Name: Ganymed Pharmaceuticals AG
Location: Mainz, Germany
Representative: Özlem Türeci, CEO
Founded year: 2001
Capital Stock: EUR 1,416,308 (as of end of May 2016)
Number of employees: 85
Relationship with Astellas: There is no relationship between Astellas and Ganymed required to be disclosed

###
About IMAB362
IMAB362 is a new investigational antibody drug that is specific for the tight junction protein Claudin18.2. This unique target is restricted to stomach cells only and is absent from all other healthy tissues. Claudin 18.2 is expressed in various high medical need cancers including in about 80% of gastrointestinal adenocarcinomas, 60% pancreatic tumors as well as in biliary, ovarian and lung cancer. Its mechanism of action includes activation of antibody-dependent cell-mediated cytotoxicity (ADCC), complement-dependent cytotoxicity (CDC) and – in combination with chemotherapy – T-cell infiltration and modulation of the tumor microenvironment. IMAB362 has received orphan drug designation in the US and Europe for gastric and pancreatic cancer.

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On October 27, 2016 Myriad Genetics, Inc. (NASDAQ:MYGN), a leader in molecular diagnostics and personalized medicine, reported it will present two new studies at the American Society of Dermatopathology (ASDP) annual meeting being held Oct. 27-30, 2016 in Chicago, Ill (Press release, Myriad Genetics, OCT 27, 2016, View Source [SID1234516051]). The research being presented validates the accuracy of Myriad myPath Melanoma in differentiating benign skin nevi from malignant melanoma.

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"We are presenting landmark data from the largest outcomes-based study ever performed with a melanoma diagnostic," said Loren Clarke, M.D., medical director, Dermatology, Myriad Genetic Laboratories. "The myPath Melanoma test performed very well and identified patients with melanoma versus benign skin lesions with greater than 95 percent diagnostic accuracy, which is exceptional in molecular diagnostics for cancer, particularly given the extreme heterogeneity of melanoma."

"Pigmented or suspect skin lesions are difficult to diagnose in approximately 15 percent of cases," said Sancy Leachman, M.D., Ph.D., chair of the Department of Dermatology in the Oregon Health & Science University School of Medicine and director of the Melanoma Research Program at the Knight Cancer Institute. "A highly accurate biomarker like the myPath Melanoma test should help dermatologists augment their diagnosis of melanoma, improve patient care and lower healthcare costs."

Below are the featured presentations at ASDP (#ASDP2016).

Poster Presentation
Title: Diagnostic Distinction of Malignant Melanoma and Benign Nevi by a Gene Expression Signature and Correlation to Clinical Outcome​.
Presenter: Jennifer Ko.
Date: Friday, Oct. 28, 2016: 4:15 — 5:00 p.m. and Saturday, Oct. 29, 2016 10:00 — 10:45 a.m. CT.

In this study, research collaborators from the Cleveland Clinic, Stanford University and Nottingham University assessed the clinical accuracy (sensitivity and specificity) of the myPath Melanoma test against clinical outcomes in 182 patients with skin lesions (99 melanomas and 83 nevi) with more than 5 years of follow up. The results show that the myPath Melanoma test accurately differentiated benign lesions from melanoma with a sensitivity of 93.8 percent and a specificity of 96.2 percent when compared to known clinical outcomes. The diagnostic accuracy of the myPath Melanoma test was high even in a subset of difficult-to-diagnose cases and, in combination with two previous validation studies, the findings support its use as an adjunct method for the early and accurate diagnosis of melanoma.

Podium Presentation
Title: Gene Expression Signature as an Ancillary Method in the Diagnosis of Desmoplastic Melanoma​.
Presenter: Loren Clarke.
Date: Sunday, Oct. 30, 2016: 8:20 — 8:30 a.m. CT.

The objective of this study was to assess the accuracy of the myPath Melanoma test in the differentiation of desmoplastic melanoma (DM) from benign skin lesions. These lesions represent approximately one percent of melanomas, but are known to be very difficult to diagnose. The analysis included samples from 20 patients with DM and 27 from patients with benign moles (nevi). The results showed that the myPath Melanoma test was positive in 15 of the 20 known melanomas, negative in four and indeterminate in one. The myPath score was negative in 24 of the benign nevi and indeterminate in three. Based on these findings, the myPath Melanoma test demonstrated approximately 80 percent diagnostic accuracy in this very difficult-to-diagnose subtype.

For more information about the meeting, please visit the ASDP website at View Source Follow Myriad on Twitter via @MyriadGenetics to stay informed about news and updates from the Company.

About Melanoma
Melanoma is one of the fastest growing cancers in the United States and can strike people of all ages, races and skin types. With a one-in-50 lifetime risk of developing melanoma, nearly 76,000 Americans are expected to be diagnosed with Stage I-IV melanoma and another 68,000 will be diagnosed with melanoma in situ — totaling approximately 144,000 total diagnoses. Early and accurate diagnosis of melanoma is critical for long-term survival. For more information visit: www.mypathmelanoma.com/ and www.myriadpro.com/melanoma.

About Myriad myPath Melanoma
Myriad myPath Melanoma is a clinically validated test to be used as an adjunct to histopathology when the distinction between a benign nevus and a malignant melanoma cannot be made confidently by histopathology alone. The test measures the expression of 23 genes and accurately distinguishes melanoma from benign nevi.

On October 27, 2016 following the recent update on clinicaltrials.gov, AstraZeneca reported that the US FDA has placed a partial clinical hold on the enrolment of new patients with head and neck squamous cell carcinoma (HNSCC) in clinical trials of durvalumab as monotherapy and in combination with tremelimumab or other potential medicines (Press release, AstraZeneca, OCT 27, 2016, View Source [SID1234516048]). All trials are continuing with existing patients.

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The partial clinical hold on new patient enrolment relates only to head and neck cancer. Trials for durvalumab in different cancer types, as monotherapy or in combination with tremelimumab or other potential medicines, are progressing as planned, with pivotal data in lung cancer anticipated in the first half of 2017.

The FDA’s decision follows voluntary action by AstraZeneca to pause enrolment of new HNSCC patients while a detailed analysis is conducted of adverse events related to bleeding that were observed as part of routine safety monitoring of the Phase III KESTREL and EAGLE trials. Bleeding is a known complication in treatments of head and neck cancers primarily due to the nature of the underlying disease, the proximity of tumours to major blood vessels and use of prior cancer therapies, which may involve surgery and radiation.

AstraZeneca has submitted its analysis of the observed bleeding events to the FDA for review and is working closely with the Agency, providing the required information to resume new patient enrolment as soon as possible.