Thursday, June 27, 2013

Oncology Clinical Trial News Update Peregrine Pharmaceuticals (NASDAQ: PPHM)


Peregrine Pharmaceuticals (NASDAQ: PPHM) Provides Update on Bavituximab Clinical Program

Company Strengthens Cash Position as It Prepares to Initiate Phase III Trial in Second-Line NSCLC by Year-End While Partnering Discussions Continue; Recent Data Supporting Novel Immunotherapy Mechanism of Action Prompts Review of Ongoing Trials Including Early Review of Phase II Front-Line NSCLC Trial; Company Accelerating Collection of Immune Correlative Data From Ongoing Trials While New Immunotherapy Combination Studies Are Being Planned

Full Press Release Follows.

SOURCE: Peregrine Pharmaceuticals, June 27, 2013 08:30 ET

TUSTIN, CA--(Marketwired - Jun 27, 2013) - Peregrine Pharmaceuticals, Inc. (NASDAQ: PPHM) today provided an update on its bavituximab oncology program including financing activities focused on advancing its lead program into a pivotal Phase III trial and adapting its clinical development plan in accordance with the recent increased understanding of the immune-stimulatory properties of bavituximab. In May, the company reached an agreement with the Food and Drug Administration (FDA) on a Phase III trial design in second-line non-small cell lung cancer (NSCLC) with bavituximab and docetaxel, presented data from its trial supporting this study in early June and is now in the process of initiating the pivotal Phase III trial by year-end. In line with the planning for the Phase III trial and continued development in other indications, the company received over $24 million in net proceeds since its quarter-end January 31, 2013 through an At Market Issuance Sales Agreement and had over $42 million in cash as of June 24, 2013.

Data recently presented at the annual meeting of the American Association for Cancer Research (AACR) yielded definitive insight into bavituximab's immunotherapy mechanism of action. These data prompted Peregrine to undertake a review of its entire ongoing bavituximab clinical program in order to better direct its clinical development and further explore its therapeutic potential. Several of the ongoing investigator-sponsored trials (IST) include immune correlative testing which could corroborate bavituximab's immunotherapy mechanism of action in the clinic. The company is also actively working with its clinical collaborators on how best to design future trials evaluating the potential of combining bavituximab with other immunotherapy agents in addition to the chemotherapy combinations that are currently underway.

To further focus the strategic direction for potential upcoming trials, Peregrine just completed an analysis of overall survival (OS) data from its Phase II clinical trial comparing bavituximab plus carboplatin and paclitaxel versus carboplatin and paclitaxel alone in front-line patients with Stage IIIb and Stage IV NSCLC. This analysis, with less than 60% of survival events, indicated that while the bavituximab containing treatment arm currently demonstrates a median overall survival of over 14 months, there was not a meaningful enough difference in survival between the two arms of the trial that would support the advancement of this combination. Full results from the trial will be presented at a future scientific meeting or through publication.

"These recent data supporting an immunotherapy mechanism of action for bavituximab opens many new development opportunities including new combinations not previously planned and has created a lot of excitement around the potential of bavituximab in combination with other immunotherapeutic agents," stated Joseph Shan, vice president of clinical and regulatory affairs at Peregrine. "While exploring these opportunities, our primary focus remains on the initiation of the Phase III trial in second-line NSCLC by year-end based on promising survival data and we are on track to achieve that goal."

The Phase III clinical trial design will be a randomized, double-blind, placebo-controlled trial evaluating bavituximab plus docetaxel versus docetaxel alone. The trial will enroll approximately 600 patients with metastatic, non-squamous NSCLC who have progressed after standard front-line therapy. The primary endpoint of the trial will be OS.

"The recent agreement with the FDA on the Phase III trial design, along with the successful fundraising efforts have positioned us well for advancing the program into Phase III development, and have strengthened our position to develop the compound including ongoing partnering discussions," stated Steven W. King, president and chief executive officer of Peregrine. "We expect these developments will lead to important milestones during the second-half of the year as we advance the bavituximab program and gain further insight into its potential."

In March, data from a series of preclinical studies presented at the Annual Meeting of the American Association for Cancer Research (AACR) demonstrated that phosphatidylserine (PS)-targeting antibodies, such as bavituximab, mediate immuno-stimulatory changes in tumors by acting on upstream immune checkpoints and transforming those immune cells that are inhibiting immune recognition (MDSC's) into tumor-fighting (M1) macrophages and activated dendritic cells that lead to the formation of tumor fighting T-cells. Leveraging these findings, Peregrine has initiated studies to further validate this mechanism and evaluate how to best assess this in the four ongoing ISTs of breast, front-line NSCLC, rectal and liver cancers.

"By strengthening our cash position we have given ourselves the needed flexibility to prepare for the upcoming Phase III trial, explore the potential of bavituximab's immune mechanism and to strengthen our position in partnering discussions," stated Paul Lytle, chief financial officer at Peregrine. "Based on our current financial projections, we expect our cash resources to be sufficient to fund our operations for at the least the next twelve months as we continue to explore all of our opportunities."

As of June 24, 2013, the company has $42.0 million in cash and cash equivalents compared to $26.3 million at its quarter-end January 31, 2013 and $35.2 million at its year-end April 30, 2013. Prior to the recent front-line NSCLC data analysis, the company received $24.8 million in net proceeds since January 31, 2013 through the issuance of equity under its At Market Issuance Sales Agreement in exchange for 16.8 million shares of common stock sold at market prices. Peregrine intends to use these proceeds to prepare for the initiation of the pivotal Phase III clinical trial of bavituximab in second-line non-small cell lung cancer and for other general corporate purposes. Peregrine will report financial results for the fourth quarter of the fiscal year 2013 on July 11, 2013 after market.

About Peregrine Pharmaceuticals, Inc.
Peregrine Pharmaceuticals, Inc. is a biopharmaceutical company with a portfolio of innovative monoclonal antibodies in clinical trials focused on the treatment and diagnosis of cancer. The company is pursuing multiple clinical programs in cancer with its lead immunotherapy candidate bavituximab and novel brain cancer agent Cotara®. Peregrine also has in-house cGMP manufacturing capabilities through its wholly-owned subsidiary Avid Bioservices, Inc. (www.avidbio.com), which provides development and biomanufacturing services for both Peregrine and third-party customers. Additional information about Peregrine can be found at www.peregrineinc.com.

Safe Harbor Statement: Statements in this press release which are not purely historical, including statements regarding Peregrine Pharmaceuticals' intentions, hopes, beliefs, expectations, representations, projections, plans or predictions of the future are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. The forward-looking statements involve risks and uncertainties including, but not limited to, the risk that the company may not be able to initiate the Phase III trial within its anticipated timeline, the risk that the results from the Phase III trial may not support a future BLA submission, the risk that the company may not have or raise adequate financial resources to complete the Phase III trial and the risk that the company may not find a suitable partner for the Phase III trial or the PS program. It is important to note that the company's actual results could differ materially from those in any such forward-looking statements. Factors that could cause actual results to differ materially include, but are not limited to, uncertainties associated with completing preclinical and clinical trials for our technologies; the early stage of product development; the significant costs to develop our products as all of our products are currently in development, preclinical studies or clinical trials; obtaining additional financing to support our operations and the development of our products; obtaining regulatory approval for our technologies; anticipated timing of regulatory filings and the potential success in gaining regulatory approval and complying with governmental regulations applicable to our business. Our business could be affected by a number of other factors, including the risk factors listed from time to time in our reports filed with the Securities and Exchange Commission including, but not limited to, our annual report on Form 10-K for the fiscal year ended April 30, 2012 and our quarterly report on Form 10-Q for the quarter ended January 31, 2013. The company cautions investors not to place undue reliance on the forward-looking statements contained in this press release. Peregrine Pharmaceuticals, Inc. disclaims any obligation, and does not undertake to update or revise any forward-looking statements in this press release.


Contact Information

Contact:  

Christopher Keenan or Jay Carlson

Peregrine Pharmaceuticals, Inc.

(800) 987-8256

info@peregrineinc.com

Thursday, June 20, 2013

Google's Insights, Recruitment & Hiring


In the ongoing battle for talent, no company musters the same resources quite like Google. Google recently shared its insights on recruitment and hiring. One of the key insights: the importance of effectively evaluating candidates for desired behavioral traits, such as solving problems where “there isn’t an obvious answer.” Forget about asking the candidate to calculate how many golf balls will fit into a 747 airplane. “The interesting thing about the behavioral interview is that when you ask somebody to speak to their own experience, and you drill into that, you get two kinds of information. One is you get to see how they actually interacted in a real-world situation, and the valuable ‘meta’ information you get about the candidate is a sense of what they consider to be difficult.”
Full article appears below.

SOURCE: On GPAs and Brainteasers: New Insights From Google On Recruiting and Hiring (Date: June 20 2013)
Author: Adam Bryant

We found that brainteasers are a complete waste of time. How many golf balls can you fit into an airplane? How many gas stations in Manhattan? A complete waste of time. They don’t predict anything. They serve primarily to make the interviewer feel smart.”
That was just one of the many fascinating revelations that Laszlo Bock, Google’s senior vice president for people operations, shared with me in an interview that was part of the New York Times’ special section on Big Data published Thursday.

Bock’s insights are particularly valuable because Google focuses its data-centric approach internally, not just on the outside world. It collects and analyzes a tremendous amount of information from employees (people generally participate anonymously or confidentially), and often tackles big questions such as, “What are the qualities of an effective manager?” That was question at the core of its Project Oxygen, which I wrote about for the Times in 2011.
I asked Bock in our recent conversation about other revelations about leadership and management that had emerged from its research.
The full interview is definitely worth your time, but here are some of the highlights:
The ability to hire well is random. “Years ago, we did a study to determine whether anyone at Google is particularly good at hiring,” Bock said. “We looked at tens of thousands of interviews, and everyone who had done the interviews and what they scored the candidate, and how that person ultimately performed in their job. We found zero relationship. It’s a complete random mess, except for one guy who was highly predictive because he only interviewed people for a very specialized area, where he happened to be the world’s leading expert.”
Forget brain-teasers. Focus on behavioral questions in interviews, rather than hypotheticals. Bock said it’s better to use questions like, “Give me an example of a time when you solved an analytically difficult problem.” He added: “The interesting thing about the behavioral interview is that when you ask somebody to speak to their own experience, and you drill into that, you get two kinds of information. One is you get to see how they actually interacted in a real-world situation, and the valuable ‘meta’ information you get about the candidate is a sense of what they consider to be difficult.”
Consistency matters for leaders. “It’s important that people know you are consistent and fair in how you think about making decisions and that there’s an element of predictability. If a leader is consistent, people on their teams experience tremendous freedom, because then they know that within certain parameters, they can do whatever they want. If your manager is all over the place, you’re never going to know what you can do, and you’re going to experience it as very restrictive.
GPAs don’t predict anything about who is going to be a successful employee. “One of the things we’ve seen from all our data crunching is that G.P.A.’s are worthless as a criteria for hiring, and test scores are worthless — no correlation at all except for brand-new college grads, where there’s a slight correlation,” Bock said. “Google famously used to ask everyone for a transcript and G.P.A.’s and test scores, but we don’t anymore, unless you’re just a few years out of school. We found that they don’t predict anything. What’s interesting is the proportion of people without any college education at Google has increased over time as well. So we have teams where you have 14 percent of the team made up of people who’ve never gone to college.”
That was a pretty remarkable insight, and I asked Bock to elaborate.
“After two or three years, your ability to perform at Google is completely unrelated to how you performed when you were in school, because the skills you required in college are very different,” he said. “You’re also fundamentally a different person. You learn and grow, you think about things differently. Another reason is that I think academic environments are artificial environments. People who succeed there are sort of finely trained, they’re conditioned to succeed in that environment. One of my own frustrations when I was in college and grad school is that you knew the professor was looking for a specific answer. You could figure that out, but it’s much more interesting to solve problems where there isn’t an obvious answer. You want people who like figuring out stuff where there is no obvious answer.”
Please share your thoughts on these insights below, and as I’ll be writing frequently on LinkedIn, please hit my FOLLOW button to see future posts.
ABOUT THE AUTHOR:
Adam Bryant has interviewed more than 200 leaders for his "Corner Office" feature that runs every Friday and Sunday in The New York Times. He is the author of the New York Times bestseller, "The Corner Office: Indispensable and Unexpected Lessons from CEOs on How to Lead and Succeed." His second book,“Quick and Nimble: Creating a Corporate Culture of Innovation," will be published in January.

Monday, June 17, 2013

Navigating the Health Care Mobile Device Revolution


Health Care Mobile Device Revolution

An FDA letter in May admonished an Indian software developer that it needed FDA clearance for its urinalysis app. This action sent a warning to other mobile health firms that many medical marketing apps could be interpreted as medical devices. 

The FDA's action alone does not establish nor spell out which kinds of apps could be regulated; and the FDA's final guidance on mobile health apps, due this fall, will not settle some important issues in this area, which include:
·      Differentiation between disease-related and wellness-related apps. For instance, weight loss/diet app that includes a calorie counter and a pedometer, for example, doesn't need to be regulated if it's promoted for general wellness and fitness. However, if that same app is marketed it to people with a specific disease, such as diabetes, as a diabetes management tool, it can be interpreted as a medical device.
·      Accessories (which will be defined/dealt with in separate guidance) used with a medical device to enable it to operate--is defined as an accessory to the device and must be regulated. An example includes software that connects a glucose meter to a cellphone ‘could be’ defined as an accessory.

For a detailed overview of mobile medical apps & the regulatory landscape, see article below.

SOURCE: The Speed of Innovation: Mobile Radiology Apps are Evolving at the Speed of Imagination – But Can Regulators Keep Up?
American College of Radiology, author: Chris Hobson (Feb 27, 2013)

Full article and cites appear below.

Apple’s iPhone kicked off the mobile device revolution just five years ago, and now there are well over a million apps in existence. Tens of thousands of these apps are designed to either improve or maintain user health, doing everything from measuring blood pressure to monitoring a patient’s adherence to certain medical treatments. A fraction of these medical apps are designed for radiologists, transforming a smartphone or tablet into, for instance, a portable workstation on which to perform occasional primary diagnoses. 

The number of these mobile medical apps is growing rapidly. According to Research2Guidance, a research firm specializing in mobile technology, 500 million smartphone users will be running health-related apps by the year 2015.1 Before a subset of these apps can be marketed to the public, however, software developers must make their existence known to the federal government. But with so many new tools being developed so fast, some have begun questioning whether the government can regulate them quickly and efficiently enough to avoid stifling innovation. 



Regulations for the 21st Century 
Since health-related apps are being created at a breakneck pace, some software vendors worry that government oversight could slow the development process. The U.S. Federal Drug Administration (FDA) regulates medical devices, which include a subset of health-related apps known as “mobile medical apps.” Important benchmarks of the agency’s efficiency in regulating these devices and apps had steadily worsened over the past decade so, in 2011, the FDA laid out a plan of action that has improved the review process.2 Later that year, the agency put forth a draft guidance, to be finalized in 2013, that designated a small subset of mobile medical apps that the agency will oversee. In addition, the guidance outlined into which pre-established medical device categories mobile medical apps should be placed, further clarifying the process for developers. These categories are named Class I, Class II, and Class III. 

When evaluating the market readiness for mobile medical apps, the FDA applies a risk-based approach to classification: Class I represents apps that pose the least risk to a patient, while Class III designates the apps that carry the greatest risk. Apps that fall into Class I or Class II, which are deemed substantially equivalent in safety and effectiveness to an already legally marketed app, use what is called a 510(k) pathway, or a premarket notification, to gain FDA marketing clearance. In other words, these mobile medical apps do not represent a total departure from other apps already in existence that have proven safe and effective for users. Software developers who submit information about their apps for an FDA premarket clearance through the 510(k) pathway are usually notified within 90 days that their product meets the requirements, after which time they may proceed to market their product with the FDA’s clearance. 

If, on the other hand, the vendor is developing a Class III app, then they must apply for premarket approval (PMA). Class III apps are comprised of “those that support or sustain human life, are of substantial importance in preventing impairment of human health, or which present a potential, unreasonable risk of illness or injury.”3 PMA represents a more rigorous review process, and developers must demonstrate safety and effectiveness of their product to the FDA in order to receive approval to market it. This designation applies even if a similar app is already being legally marketed. 




Innovation Under Threat? 
According to the FDA, all of the radiology mobile apps it has approved have fallen into the Class II category, which, explains David S. Hirschorn, MD, director of radiology informatics at Staten Island University Hospital, has caused little disruption in innovation. This is largely because, to his mind, the 510(k) process is so short. “Of the software vendors I’ve talked to, no one has said that the FDA is dragging its heels during the review process,” he notes, saying that the organization strives not to over-regulate medical apps. Indeed, the agency treads lightly when it comes to medical software. “With the exception of mammography,” he asserts, “states regulate the practice of medicine, not the FDA.”

But while the FDA generally cannot regulate the practice of medicine, medical app manufacturers do come under its purview. The agency distinguishes whether or not it has oversight authority over a product based on a product’s intended use, which largely depends on how a vendor markets it. Hirschorn illustrates the fine line the FDA walks with an example: “If a magnifying glass is advertised as something that can help you see small things, it’s not a medical device. If, however, it’s advertised as helping you to see micro-calcifications in a mammogram, then it’s a medical device,” and the agency has right of approval over it. 


Bradley Merrill Thompson, general counsel of the mHealth regulatory coalition in Washington, D.C.,agrees. While he would like to see the premarket notification process for mobile medical apps cut down, he doesn’t believe that the FDA is overly suppressing innovation. “The pace at which the agency moves is dependent upon its available resources,” says Thompson. “It’s a balancing act between speed and resources — it can only hire so many staff members without getting more resources either from Congress or from user fees. The FDA has to go through a deliberate product-approval process but also try to move as fast as possible.

Time Is of the Essence 
Others, however, are not convinced that the FDA’s approval process paves the way for innovation. Joel C. White, executive director of Health IT Now, in Washington, D.C., states that the life cycle of many mobile apps is shorter than the time it takes to go through the FDA approval process. “Some apps are updated weekly, and in some cases daily,” he says. “Turnaround time will be crucial to ensuring the latest, best information is available for providers to treat patients.” He recommends a more hands-off approach to regulation: “At a minimum, software that poses the least risk should fall outside the FDA review process. Only when a product interacts with a variable like human physiology should the FDA be involved.” 

But there are steps developers can take to reduce turnaround times, argues FDA policy advisor Bakul Patel. “One point I can emphasize to help reduce frustration is the need to talk to us early in the development of mobile medical apps,” he notes. “The FDA can discuss data needed to show safety and effectiveness with developers so they can plan those requirements into the development of the app. This can help developers to find the quickest path to market, help them use resources more efficiently, and shorten review times.” 

Although finding the quickest route to market may be helpful for developers, however, it is the end product — and the end users — that matter most. In Hirschorn’s experience, radiologists tend not to suspect the safety of the software they use, often assuming it is safe unless told otherwise. “They focus more on the clinical task and less on the software itself,” he explains. “As a consequence, though, they are very unnerved if they learn that what they were shown by a computing device was not what they thought it was. This implicit trust can lead to a strong distrust if the radiologist feels she has been betrayed by the technology,” he says, emphasizing the need for government oversight to ensure that products work as they should. 

The rapidly changing landscape of mobile medical app development is a cause of both optimism and unease. By working with developers and vendors, the FDA is making an effort to alleviate this concern. While innovation will only increase the speed at which new technologies are born, spurring unimaginable new treatment options, the public’s safety must also be taken into account. Radiologists would do well to ensure that the apps they use are tested thoroughly so that their trust in the technology remains strong.

ENDNOTES 
1. Mikalajunaite, E. “500m people will be using healthcare mobile applications in 2015.” Available at http://bit.ly/MobileAppStudy. Accessed Dec. 8, 2012. 
2. U.S. Food and Drug Administration. “Improvements in Device Review: Results of CDRH’s Plan of Action for Premarket Review of Devices.” Available at http://bit.ly/FDAPlanOfAction. Accessed Dec. 9, 2012. 
3. U.S. Food and Drug Administration. “Premarket Approval (PMA).” Available at http://bit.ly/FDAPremarketApproval. Accessed Dec. 8, 2012. 


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