Puma Presents on Phase 3 Neratinib Breast Cancer Trial Results at ASCO

 Puma Biotechnology Inc. (Nasdaq: PBYI), a biopharmaceutical company, announced that results from the Phase III NALA trial of PB272 (neratinib) in patients with HER2-positive metastatic breast cancer who have failed two or more prior lines of HER2-directed treatments (third-line disease) in the setting of metastatic disease, were presented at the American Society of Clinical Oncology (ASCO) 2019 Annual Meeting in Chicago. “Neratinib + capecitabine versus lapatinib + capecitabine in patients with HER2+ metastatic breast cancer previously treated with ≥ 2 HER2-directed regimens: Findings from the multinational, randomized, phase III NALA trial,” was presented as an oral presentation by Adam M. Brufsky, MD, PhD, Professor of Medicine, Co-Director, Comprehensive Breast Cancer Center, Magee Women’s Hospital of the University of Pittsburgh Medical Center. Slides from the presentation are available on the Puma Biotechnology website.

The Phase III NALA trial is a randomized controlled trial of neratinib plus capecitabine versus Tykerb® (lapatinib) plus capecitabine in patients with third-line HER2-positive metastatic breast cancer. The trial enrolled 621 patients who were randomized (1:1) to receive either neratinib plus capecitabine or lapatinib plus capecitabine. The trial was conducted globally at sites in North America, Europe, Asia-Pacific and South America. The co-primary endpoints of the trial are centrally confirmed progression free survival (PFS) and overall survival (OS). An alpha level of 1% was allocated to the PFS and 4% allocated to OS. The study was to be considered positive if either of the co-primary endpoints was positive. Puma reached agreement with the U.S. Food and Drug Administration (FDA) under a Special Protocol Assessment (SPA) for the design of the Phase III clinical trial and the European Medicines Agency (EMA) also provided follow-on scientific advice (SA) consistent with that of the FDA regarding the Company’s Phase III trial design and endpoints used in the trial.

For the primary analysis of centrally confirmed PFS, treatment with neratinib plus capecitabine resulted in a statistically significant improvement in centrally confirmed PFS (hazard ratio=0.76, p=0.0059) compared to treatment with lapatinib plus capecitabine. Because the proportional hazard assumption did not hold, the statistical analysis plan for the NALA trial prespecified that a restricted means survival analysis at 24 months would be performed. In this prespecified analysis the mean PFS for the patients treated with neratinib plus capecitabine was 8.8 months and the mean PFS for the patients treated with lapatinib plus capecitabine was 6.6 months.

For the primary analyses of OS, neratinib plus capecitabine resulted in an improvement in OS that trended positively in favor of the neratinib plus capecitabine arm of the study (hazard ratio = 0.88, p=0.21). The median OS for the patients treated with neratinib plus capecitabine was 21.0 months and the median OS for the patients treated with lapatinib plus capecitabine was 18.7 months. In the prespecified restricted means analysis the mean OS at 48 months for the patients treated with neratinib plus capecitabine was 24.0 months and the mean OS for the patients treated with lapatinib plus capecitabine was 22.2 months.

For the secondary endpoint of time to intervention for symptomatic central nervous system disease (also referred to as brain metastases), the results of the trial showed that treatment with neratinib plus capecitabine led to an improvement over the combination of lapatinib plus capecitabine. The overall cumulative incidence of CNS metastases was 22.8% for the neratinib plus capecitabine arm and 29.2% for the lapatinib plus capecitabine arm (p=0.043, descriptive). For the secondary enpoint of duration of response, neratinib plus capecitabine treatment resulted in a longer duration of response compared to lapatinib and capecitabine treatment, with a median response of 8.54 months compared to a median response of 5.55 months (HR = 0.495, p = 0.0004, descriptive).

https://www.biospace.com/article/releases/puma-biotechnology-presents-results-from-phase-iii-nala-trial-of-neratinib-in-patients-with-her2-positive-metastatic-breast-cancer-at-the-asco-2019-annual-meeting/

Cerner Calls for App Ideas That Improve Consumer Access to Health Records

Cerner, a global health platform and innovation company, challenges innovators and application developers to advance consumers’ access to electronic health records (EHRs) with the 2019 code App Challenge. Participants will join Cerner’s pursuit of the next groundbreaking advancement, with a focus on helping consumers easily access and understand health records.

Cerner’s 40-year history of innovation and technological developments has helped reduce the complexities of health care and drive breakthrough advancements, directly benefiting providers and consumers. By creating a single patient record with a complete picture of a person’s health and financial information, providers can engage people in meaningful and informed discussions to improve health and well-being. Following health care’s digitization era, the focus is on shaping the future with a person-centered mission that requires joint efforts among developers, technology companies and provider organizations. Cerner’s platforms, technology and culture of openness emphasize the importance of collaboration to advance health care.

With the 2019 code App Challenge, Cerner encourages innovators to build apps on its industry-leading platforms that further transform health care. Proposed apps must focus on consumers’ ability to access, understand and use health information in the EHR.

“We challenge innovators and organizations across the industry to join us in advancing interoperability, secure data exchange and consumer access to health records. People should be able to access and understand their own health information regardless of facility or EHR, which is why collaboration through open platforms and shared data is critical to our mission,” said John Gresham, senior vice president, Health Networks, Cerner. “Putting the person at the center of care by improving consumer access to medical information encourages active participation in health care. Embedding consumer-centric apps in the EHR can drive patient engagement, increase awareness of health care costs and improve overall health outcomes.”

Innovation and collaboration are fostered through openness and transparency, exemplified by the launch of the Cerner Open Developer Experience (code) in 2016. The code program provides third-party developers with tools and resources to realize ideas and create software apps that can be embedded across all EHRs, not only Cerner’s platform. The Cerner code App Gallery now has more than 30 apps accessed by nearly 53,000 unique and active monthly users. Cerner’s rigorous validation process helps ensure providers and consumers access high-quality and secure apps that bring differentiated benefits to the delivery of care and the patient experience. Cerner’s open platforms and technology provide new ways for organizations to adjust to health care’s rapidly evolving demands.

Submissions for the 2019 code App Challenge can be at any stage of development, from an idea to a working demo. Developers can create apps independently, as a team, or in collaboration with hospitals or health systems. The winning individual or team will receive a one-year membership to the code program, with Cerner engineers helping guide the app through the validation process. If the app completes validation, it will be added to the code App Gallery. The deadline for submissions is Aug. 16, 2019.

For more information, including official rules and the submission form, click the following link.

https://www.marketscreener.com/CERNER-CORPORATION-8744/news/Cerner-Calls-for-App-Ideas-That-Improve-Consumer-Access-to-Health-Records-28703796/

Atara: Positive Phase 1 for Mesothelioma CAR T Immunotherapy

Memorial Sloan Kettering Cancer Center collaborators presented results demonstrating that a regionally delivered mesothelin-targeted, autologous CAR T was well tolerated and showed encouraging anti-tumor activity in combination with pembrolizumab, a PD-1 checkpoint inhibitor

 In a subset of 16 malignant pleural mesothelioma patients with minimum follow-up time of 3 months who also received pembrolizumab and lymphodepleting chemotherapy, 12-month overall survival was 80% and best overall response rate was 63%, including 3 investigator-assessed complete responses

Clinical findings presented at the American Society of Clinical Oncology (ASCO) Annual Meeting 2019

Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leading off-the-shelf, allogeneic T-cell immunotherapy company developing novel treatments for patients with cancer, autoimmune and viral diseases, today announced that the Company’s collaborators at Memorial Sloan Kettering Cancer Center (MSK), Prasad S. Adusumilli, M.D. and Michel Sadelain, M.D., Ph.D., presented an update on encouraging results from an ongoing MSK investigator-sponsored Phase 1 clinical study (NCT02414269) of a mesothelin-targeted CAR T immunotherapy for patients with mesothelin-associated malignant pleural solid tumors, primarily mesothelioma, who progressed following platinum-containing chemotherapy. Mesothelin-targeted, autologous CAR T cells delivered regionally were well-tolerated and showed encouraging anti-tumor activity in combination with pembrolizumab, a PD-1 checkpoint inhibitor. The findings were presented today at the American Society of Clinical Oncology (ASCO) Annual Meeting 2019 in Chicago.

https://www.marketscreener.com/ATARA-BIOTHERAPEUTICS-INC-16917577/news/Atara-Biotherapeutics-Announces-Collaborator-Presentation-Updating-Positive-Phase-1-Clinical-Results-28704213/

Genentech Phase 3: Xofluza Effective at Preventing Influenza Infection

– Compared with placebo, XOFLUZA treatment significantly reduced the likelihood of people developing influenza (flu) after exposure to an infected household member

– Data from the Phase III BLOCKSTONE study will be submitted to health authorities globally

* * *

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the Phase III BLOCKSTONE study, conducted by Shionogi & Co., Ltd., met its primary endpoint showing that people exposed to a household member with influenza (flu) and treated preventatively with XOFLUZA(TM) (baloxavir marboxil) were significantly less likely to develop the disease compared to those treated with placebo (1.9% versus 13.6%, p<0.0001). Furthermore, XOFLUZA was well tolerated with no safety signals identified. Full results from the BLOCKSTONE study will be presented at an upcoming medical meeting.

” This positive Phase III study adds to robust existing clinical data for XOFLUZA, and is the first to show that a single treatment with XOFLUZA reduced the likelihood that people living with an infected household member would develop the flu,” said Sandra Horning, M.D., chief medical officer and head of Global Product Development. “Preventing otherwise healthy people from developing the flu virus will reduce the overall societal burden of disease, and we look forward to sharing these data with health authorities around the world. ”

XOFLUZA is the first and only one-dose oral medicine approved to treat the flu, and the first new flu medicine approved by the U.S. Food and Drug Administration (FDA) with a novel proposed mechanism of action in nearly 20 years. XOFLUZA is also the only flu treatment with a new mechanism of action shown to be efficacious in both otherwise healthy people with the flu (in the CAPSTONE-1 study) and people at high risk of flu complications (in the CAPSTONE-2 study).

XOFLUZA is currently approved in Japan for the treatment of influenza types A and B in children, adolescents and adults, and in the U.S. for the treatment of acute, uncomplicated influenza in people 12 years of age and older. In addition, the FDA recently accepted a supplemental New Drug Application (sNDA) for XOFLUZA as a one-dose oral treatment for people at high risk of complications from the flu, which includes adults 65 years of age or older, or those who have conditions such as asthma, chronic lung disease, morbid obesity or heart disease – for these people the flu can be particularly serious or deadly. The FDA is expected to decide on whether to approve this additional indication by November 4, 2019.

https://www.marketscreener.com/ROCHE-HOLDING-68997/news/Genentech-Phase-III-Study-Showed-Xofluza-Is-Effective-at-Preventing-Influenza-Infection-28703327/

Biomarin gets China OK for rare disease med

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that Vimizim® (elosulfase alfa) has been approved by the National Medical Products Administration (NMPA) for the treatment of patients with mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome. Vimizim is the first treatment in China approved for this condition.

In May 2018, the Chinese government issued the country’s first national list of rare diseases, which included MPS.  The list also includes phenylketonuria (PKU), Tetrahydrobiopterin Deficiency, and hemophilia—diseases that BioMarin either has an approved therapy for or is developing one.  The list gives priority to rare diseases with a relatively high prevalence, that pose a heavy burden and that are highly treatable.  Also, in August 2018, the Chinese Drug Evaluation Center posted a list of 48 drugs already approved in the U.S., EU or Japanthat could be eligible for Priority Review in China, which included Vimizim.

“Vimizim is the first, and currently only, disease-specific treatment option for this very rare, progressively degenerative, autosomal-recessive lysosomal storage disorder,” said Luo Xiaoping, Vice Chairman of the Pediatrics society of the Chinese Medical Association and head of Endocrine Genetic Metabolism Group of Chinese Medical Association.

“Morquio A syndrome is an ultra-rare and difficult condition to treat. Vimizim is the only specific treatment available and offers improved endurance to these patients,” said Gu Xuefan, Deputy Director of Shanghai Institute of Pediatrics, Director of Pediatric Endocrinology and Inherited Metabolic Diseases Research Office of Xinhua Hospital, Director of Pediatric Genetic Diseases Diagnosis and Treatment Center, and Director of Pediatric Endocrinology and Inherited Metabolism Group of Shanghai Medical Association.

https://www.marketscreener.com/BIOMARIN-PHARMACEUTICAL-I-8587/news/BioMarin-Pharmaceutical-Announces-Approval-of-Vimizim-elosulfase-alfa-in-China-for-Treatment-of-28703454/

Pfizer reinstated at Overweight by Morgan Stanley

Target $48

https://www.benzinga.com/stock/PFE/ratings

Neon Therapeutics started at Buy by Mizuho

Target $21

https://www.benzinga.com/stock/NTGN/ratings