#bio2015

Tuesday, June 16, included a day of programs dedicated to state government affairs issues and leaders. The morning opened with the Legislative Best Practices Panel featuring Governors Dennis Daugaard of South Dakota and Jack Markell of Delaware.

It continued with presentations of BIO’s Legislators of the Year Awards. The recipients were Tennessee state Rep. Cameron Sexton, Texas state Rep. John Zerwas, Washington state Sen. Linda Evans Parlette, and New Jersey Assemblywoman Pam Lampitt. Each of these members were chosen for their work regarding state legislation that would provide a pathway for substitution of interchangeable biologic medicines in each of their respective states.

Assemblywoman Pam Lampitt participated in a panel discussion later in the afternoon on the Impact of Biosimilars on States where she discussed her experience shepherding her legislation through the Assembly and into the Senate where it is poised for a vote by the full Senate.

Our very own President of Communications, Topper Ray, served as moderator for the panel. He opened with an overview of the issues surrounding biosimilar substitution as well as an overview of Discovering Cures - a national coalition focused on current issues and trends impacting the biotechnology industry. Bravo Group and BIO collaborated to create Discovering Cures and since its inception in 2013, we have engaged in a national stakeholder education campaign on the substitution of biologic medicines.

Part of that education is understanding the difference between biologic medicines and traditional small molecule medicines. Dr. Javier Coindreau from Pfizer provided that overview during the panel discussion. His presentation included an explanation of the scientific differences of the two types of medicine as well as an explanation of biologics, biosimilars and interchangeable biologics.

Dr. Earl Dye, Genentech, provided an overview of the regulatory process for a biosimilar approval and an update on the FDA guidance for approval. Dr. Dye also reviewed the requirements for interchangeability.

With the approval of the first biosimilar in March 2015, there is anticipation within the healthcare industry that savings from the use of biosimilars will be seen in the market. Angela Lively, ADVI, gave her insights into the likely savings the healthcare industry may see from biosimilars. Although savings are estimated, the healthcare system is not likely to see the steep savings like it has with generic medications. RAND estimates $44.2 billion in national biosimilar savings from 2014-2024. With regards to direct savings within states, Lively stressed that managed care and carve-out status will affect anticipated savings.

Kipp Snider, Amgen, provided a national overview of biosimilars substitution legislation and shared that there is a national coalition working closely on this legislation throughout the country.

Thirteen states passed this legislation with three more bills pending gubernatorial signatures. Bills are before state legislatures in five additional states. Snider also reviewed the five principles adopted by BIO that should be included in every piece of legislation and clarified some misconceptions about the legislation - such as, the measure addresses the practice of pharmacy and has no impact on reimbursements.

By: Lisa LaMotta

10) The convergence of tech and medicine – everything from Twitter to Big Data to wearable devices

9) Merck’s soft serve booth in the exhibit hall

8) Combos in immunotherapy – best deal structures for testing the most combinations on a biotech budget

7) Acorda’s Ron Cohen named Chairman of BIO for 2016

6) How the microbiome can affect a range of diseases, from obesity to irritable bowel syndrome

5) How to get approval and please payers

4) Are asset swaps the future of big pharma dealmaking?

3) Efforts to increase clinical trial data transparency

2) NIH Director Francis Collins can’t choose between his Apple Watch and his FitBit

By: Sue Sutter

In early February, biosimilar developer Pfenex Inc. was on the verge of a transformative deal with Hospira Inc. to bring a proposed biosimilar of Genentech Inc.'s ocular VEGF-inhibitor Lucentis (ranibizumab) to market.

Then it got some news that caused a bit of gastroesophageal reflux: Pfizer Inc. was acquiring Hospira for $17 billion.

"We  were unaware of the Pfizer transaction" during negotiations with Hospira "so certainly that created a little bit of heartburn," Pfenex Chief Business Officer Patrick Lucy said during a June 16 interview with "The Pink Sheet" DAILY at the BIO annual convention in Philadelphia.

However, executives at the San Diego-based firm, which started life as a spinout from Dow Chemical Co., were quickly assuaged.

Hospira Chief Scientific Officer Sumant Ramachandra reached out to Pfenex executives "literally minutes after the press release hit the wire" to assure them the biosimilar deal would happen, Lucy said.

Pfizer's familiarity with Pfenex and its unique protein production platform also may have helped calm some nerves at the smaller company.

"We are not an unknown quantity to Pfizer," Lucy said. "Pfenex has had a lot of interactions with Pfizer in the past on this platform. The team at Pfizer that focuses on microbial expressed proteins has a long history of working with Pfenex and Pfenex technology. And certainly Pfizer's been supportive of biosimilars [with] significant efforts there. So I think there's a nice match in terms of history and commitment to biosimilars."

Five days after the Pfizer/Hospira deal was announced, Hospira and Pfenex announced their own development and commercialization partnership for PF582, Pfenex's proposed biosimilar to Lucentis.

Under the terms of the deal, Pfenex received an up-front payment of $51 million and is eligible for development and sales-based milestones of up to an additional $291 million, as well as a tiered double-digit royalty on net product sales.

The compound is currently in Phase I/II testing but is expected to enter Phase III in 2016. Pfenex and Hospira will share the Phase III equivalence trial costs, and Hospira will be responsible for manufacturing and commercializing the product worldwide.

But, if all goes as planned, Pfenex will soon be working with a partner that shares the first two letters of its name.

by John Hodgson 

What key message about biotechnology is coming out of Philadelphia? Well, hopefully it's nothing to do with what was bothering the Twittersphere yesterday. 

Scrip looked at a day's worth of #BIO2015 Tweets from the first day of the BIO convention expecting to find some interesting discussion on drug payment, Obamacare, PD1/PDL-1 inhibitors or even some abstruse self-promotion from thought-provoking biotech companies. We thought that assessing the interest in a subject from the number of retweets or "favoriting" would be a good way of assessing what the BIO2015 Twitterati found fascinating.  

For the sake of the BIO convention, we hope that is not so.  

At #11 with 10 re-tweets and six favorites, and setting a very low bar for the rest of the list, it's @eezzed with the surprising news that the BIO meeting started yesterday: "And so #BIO2015 starts today! Meetings have already begun."

At #10 with 10 re-tweets and seven favorites, and managing quite comfortably to limbo under @eezzed's bar, it's @BIOConvention (the organizers) with a host-serving huzzah for Philadelphia: "Today at 3:30: learn how #LifeSciences are growing in #Philly and how you can be a part of it!  

At #9 with 10 re-tweets and seven favorites, it's clean and shaved @WKuketz telling us that he and his team are in the shower before the meeting: "@consultcollab Our Life Science team is getting ready for the conference."

At #8 with 10 re-tweets and 10 favorites, it's @RaleighWake with a punt for Raleigh, NC as a place to do business. "Forbes ranked Raleigh as #1 Best Place for Business and Careers. Biz Make the smart move here! http://bit.ly/1IzVs96 @raleigh4u"

At #7 with 12 re-tweets and two favorites, it's a plug from @meetPHL for delegates not to bother to go to the meeting at all (and who could blame them, given this list): "Welcome @BIOConvention attendees! Follow @discoverPHL for free time ideas & start tours @PHLVisitorCntr"

In joint seventh place, with 11 re-tweets and five favorites, it's yummy consumable distractions from @PHLVisitorCntr: "@PatsSteaks @GenosSteaks What a delicious way to prep for #BIO2015!"

At #6 with 13 re-tweets and three favorites, it's actually a Tweet about doing something about disease from @JNJInnovation: "Imagine if we could stop T1 #diabetes before it starts. We've teamed w/ @WUSTLmed researchers to explore http://bit.ly/1f4KZJg."

Also at sixth place, with 11 re-tweets and seven favorites, it's @AvalonVentures with a plug for a session on business development: "Jay and @GSK's Damien McDevitt will talk collaboration and new company details on today's #buildtobuy panel: http://bit.ly/1MWrGgk.

At #5 with 14 re-tweets and 17 Favorites, it's @PHLCommerce with yet another Philly fillip, this time a plug for the entire set of local biotech exhibitors: "More than 70 companies are showcasing in the #PAPavilion. It is clear that Pennsylvania's life sciences industry is thriving!" Yay!!  

At #4 with 14 re-tweets and 14 favorites, it's naughty child and    persistent Tweeter @ChristianeTrue (79 Tweets sent on Monday 15 June)    reacting to Bill Clinton's call for "disruptors" in biotech: "I    was a disruptor in grade school too. All it got me was bad grades in conduct."

At #3 with 15 re-tweets and five favorites, it's a retweet from @livecellassays informing the world that a venture capitalist wants to invest in drug discovery stuff: "Domain Associates at #BIO2015 ready to make connections with others to reduce the risk & cost of drug discovery."

At #2 with 24 re-tweets and 15 favorites, it's @alexmccann with appreciation for the comestibles from the USA's northern neighbor: "#NovaScotia enjoying #BiotechCanada reception. https://lnkd.in/ezW5PB7."

Finally at #1, with 34 re-tweets and 22 favorites, it's @NeilPBardhan showing off his knowledge of Eastern US socio-geography: "40% of the American population lives within a day's train ride or drive from    Philadelphia."

You are a very dull lot. Please find something more interesting to tell those of us not at BIO (#notatBIO2015) about tomorrow.  

by Sue Sutter  

FDA's "breakthrough therapy" program should be broadened in a way that helps increase market competition and drive down costs for first-in-class medicines, some stakeholders suggested at the Biotechnology Industry Organization's annual convention in Philadelphia June 16.

During a panel discussion entitled "Paying for the 21st Century Cure," Dan Durham, acting CEO of America's Health Insurance Plans, and American Enterprise Institute Resident Fellow Dr Scott Gottlieb made the argument for moving beyond the current product-based approach to granting breakthrough status and, instead, extending the expedited pathway's benefits to subsequent treatments that target the same indication.

"When you have that breakthrough therapy that gets an expedited approval, you have several other products in the pipeline," Mr Durham said. Allowing these subsequent products to enjoy the same advantages as those on the expedited regulatory pathway benefits the consumer by bringing more competitors into the marketplace more quickly, he said.

Mr Durham also suggested a safety benefit that would come with more competition sooner for breakthrough therapies.

"If there is a problem with the first drug to market, you then have a second and third that can go to those patients as well," he said.

Looking to 'Cures' bill to make the competition argument

The breakthrough program was created through the FDA Safety and Innovation Act to expedite the development and review of drugs that treat a serious or life-threatening disease or condition and for which preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints.

The program has proven enormously popular with the biopharma industry, far more so than was ever anticipated. Through the end of May, FDA had received 308 requests for designation, 90 of which were granted.

The health insurance industry has been far less enthusiastic about breakthrough, pointing not only to the treatments' high costs but also to the more limited clinical dataset upon which they are approved.

AHIP's focus is clearly on the cost-savings that result from greater competition.

Mr Durham pointed to what he called the "poster child" for high-priced breakthrough treatments: Gilead Sciences hepatitis C drug Sovaldi (sofosbuvir), which entered the market with a price tag of $1,000 a day.

The market entry a year later of another breakthrough hep C treatment, AbbVie Viekira Pak (paritaprevir/ritonavir/ombitasvir/dasabuvir), gave health plans leverage to negotiate price in the category, Durham noted.

Mr Durham said AHIP has advocated for the extension of breakthrough to later-in-class drugs in the context of the 21st Century Cures legislation.

Such a provision has not been included in the measure now making its way through the House. However, similar legislation is under development in the Senate, and Mr Durham said AHIP officials are continuing to talk to lawmakers about the importance of competition in the marketplace.

Helping to ensure later-in-class agents reach market

The idea drew strong support from Dr Gottlieb, a former FDA official, who said that taking an indication-based approach to awarding the designation would help ensure that later-in-class products actually reach market.

"What we've seen I think over the last number of years, probably since [Medicare] Part D, is the drug makers really getting away from being second, third, fourth in class," Dr Gottlieb said. "Even in some of these higher reimbursed classes, if a drug maker thinks that they're going to be third to market, sometimes they will close down the program … and that's not a good thing."

FDA Office of New Drugs Director Dr John Jenkins has also lamented the decline in so-called "me too" drugs in primary care settings.

"It would be a very simple change," Dr Gottlieb said. "Rather than giving breakthrough designation on the basis of a specific molecule, you'd grant the designation on the basis of a therapeutic indication, just like we do with orphan drugs."

He noted that already some products that are second or third in class for a condition with high unmet need are designated as breakthroughs or receiving benefits under FDA's other expedited pathways.

Any effort to expand the program's breadth seems likely to meet with strong objections from FDA as well as some innovator companies and patient advocacy groups, who can be expected to argue that the program's benefits will be diluted if they are opened up to a much broader group of drugs than currently qualify under the existing designation process.

FDA, which has cited the resource demands of having to respond to so many designation requests that are ultimately denied, likely would make the case that such an expansion demands new and dedicated funding.

Dr Gottlieb said he believed the additional resources that FDA would need to implement a breakthrough expansion would likely be small relative to the projected savings resulting from earlier competition and lower prices.

Panel moderator Dan Mendelson, Avalere Health's CEO and founder, said he supported the idea but suggested such an expansion could undercut existing incentives to expedite development of novel, innovative therapies.

"The idea that if you were able to do that, you were able to bring in more competition, it strikes as a kind of a free market-oriented approach," he said.

"I think what's on the flip side of that is if you want to set up a system that really incents getting that first drug to market, are you starting to take away the incentive for that innovator, the Gilead, to kind of push things through really rapidly, and get that drug out there more rapidly," Mr Mendelson said.

by Donna Young

Too often, biopharmaceutical firms put their investment and attention mostly on research and development and gaining regulatory approval and fail to provide equal support to the commercial market access side of their businesses – which can ultimately result in a successfully approved drug being a flop in the marketplace, three expert consultants said during a 16 June session at the annual Biotechnology Industry Organization (BIO) International Conference in Philadelphia.

Indeed, it's not all about just getting a drug on the market, they said. To be successful, firms must ensure those products are paid for by insurers and government reimbursement agencies – a fact some companies have learned too late and to their detriment.

Companies make mistakes by designing their programs for regulatory approval but not market access success, said Dr Laurie Smaldone, president and chief scientific officer at PharmApprove, a regulatory and scientific communications consultancy firm, which helps drug makers prepare for regulatory and payer negotiation meetings.

Drug manufacturers must not only engage with regulators, like the FDA and the European Medicines Agency, but also with health technology assessment (HTA) bodies, such as National Institute for Health and Care Excellence and Agenzia Italiana del Farmaco in Europe, US government payers, like Medicare and Medicaid, along with private insurers, she said.

Dr Smaldone noted, however, those interactions with government reimbursement bodies currently occur more in Europe than in the US.

But, Dr Steffen Thirstrup, director of the NDA Regulatory Advisory Board at the NDA Group, a regulatory and HTA consultancy, told Scrip....

by Sue Sutter

Clinical trial data transparency initiatives could make it difficult for drug developers to secure patents for new products and, in turn, investment for their compounds, an industry attorney said on 15 June.

During a panel discussion at the Biotechnology Industry Organization's annual convention in Philadelphia, Covington and Burling partner Richard Kingham said global transparency initiatives that require publication of trial results while a compound is still early in development could close off a drug developer's ability to patent an innovation.

"The moment the information is disclosed it becomes part of the prior art, and therefore you can't get a patent," Kingham said.

While Kingham asserted that transparency initiatives could undercut intellectual property, deterring investment and drug development in the process, European Medicines Agency Senior Medical Officer Hans-Georg Eichler had a decidedly more optimistic view of the investor impact of public and private data transparency initiatives sweeping across the globe. Greater data-sharing will increase efficiency in drug development and reduce the high rate of failure in the clinical stages, which is something that investors should welcome, Eichler said.

EMA led, and industry followed

The panel discussion at BIO brought together representatives from industry and the European Union regulator to discuss recent developments in global initiatives to increase clinical data transparency.

EMA has taken a driving role in these initiatives, and its interest in the subject has led industry to develop its own voluntary measures...