May
02
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After initially being rebuffed at the FDA Pfizer ($PFE) and Protalix have snagged an FDA OK for their new Gaucher disease drug Elelyso (taliglucerase), an orphan enzyme replacement therapy which will appear on the market at a discount over Genzyme's mainstay and carrying a pointed promise to avoid supply interruptions.
Israel's Protalix heralded the approval as an endorsement of its plant cell-based manufacturing system. Elelyso is made from carrot cells rather than the more complex mammalian cell-based system that Genzyme--now owned by Sanofi ($SNY)--uses. And with its first approval complete and a new revenue stream to rely on, Protalix's chances of inking more favorable deals on its pipeline drugs just improved considerably.
Pfizer bought global rights to the drug--outside of Israel--with a $60 million upfront and the promise of $55 million more in regulatory milestones. Protalix, which saw its share price ($PLX) spike 23% last night, gains a $25 million milestone on the approval. And now the two companies will split the revenue 60/40.
Only about 6,000 people in the U.S. have Gaucher's disease. But that's been a big enough market for Genzyme, which has turned Cerezyme into a big winner over the years. Pfizer, which needs all the new products it can get to replace the Lipitor franchise, says it will price the new therapy at a 25% discount to Cerezyme. In another jab at its rival, Pfizer also promises to carry a 24-month supply of the drug to avoid the manufacturing snafu that created lingering supply problems for Genzyme.
Protalix was handed a CRL on Elelyso a year ago, but regulators limited their demands for more information to a few technical issues, without requiring more data from clinical studies.
- read the press release
- here's the Globes story
- get the report from Bloomberg
Related Articles:
Protalix shares dive on Gaucher's drug delay at FDA
Genzyme's shortage opens door to rival Protalix
Protalix showcases PhIII data for rare-disease drug in Pfizer pact
FDA hands Protalix a CRL for Gaucher's disease drug
Apr
25
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Riding positive data on a late-phase multiple sclerosis drug, Genzyme execs talked up the company’s willingness to go head-to-head against Rebif, EMD Serono and Pfizer’s blockbuster beta interferon.
Genzyme execs didn’t go so far as to reiterate former CEO Henri Termeer’s 2010 prediction of $3 billion plus in potential sales for Lemtrada (alemtuzumab), but they did tout clinical results suggesting a reversal in MS-related disability for some patients.
New phase III data from Genzyme’s CARE-MS II was unveiled yesterday at the American Academy of Neurology’s annual meeting, and while Biogen Idec’s BG-12 (dimethyl fumarate) – a twice or thrice daily oral drug – has gotten more and better attention over the last year, Michael Panzara, therapeutic area head, multiple sclerosis, immune diseases and neurology at Sanofi-owned Genzyme, says consider the trial design. Without speaking directly to other clinical programs in the MS space, Panzara tells PharmExec that Genzyme’s decision to use an active comparator, rather than a placebo, raises the bar. “When you have a therapy as efficacious as [Lemtrada] with a risk profile that’s manageable – but it does have a risk profile – you want to set a standard of going against what is viewed as the most effective platform therapy, which is Rebif,” says Panzara.
This strategy, while riskier than testing against placebo, pays dividends if the results suggest superiority, according to Panzara. “I think [active comparator data] will be viewed favorably by physicians, who practice medicine and have to make these treatment decisions every day,” says Panzara. “And I would hope regulatory authorities would take notice as well, because they’re always looking to better understand risk and benefit versus therapies that are already available, especially in Europe.” Bill Sibold, senior vice president, head of multiple sclerosis at Genzyme, says the comparator data will aid in reimbursement discussions with payers, so the risk of knockout in a head-to-head battle is warranted. “I think most companies hesitate to do [active comparator] trials, so I think it says a lot about Genzyme and the approach that we’ve taken, specifically with this program.”
But is Rebif still the lead contender in MS? Raghuram Selvaraju*, head of healthcare equity research at Aegis Capital, says going head-to-head against Rebif in MS is akin to playing a game of chicken and waiting a bit longer before turning away. “Rebif is no longer the standard as far as efficacy is concerned…that honor now belongs to [Novartis'] Gilenya,” says Selvaraju. Gilenya was approved in late 2010, which illustrates a key difficulty with comparator trials; when Lemtrada went into the clinic, Gilenya wasn’t yet on the market. Concerns regarding Gilenya in patients with a history of cardiovascular issues have recently reemerged, however, which could add a few pounds to the scale for Lemtrada.
If approved – Genzyme is on track to file for US and EU approval of Lemtrada for relapsing MS this quarter, according to Panzara and Sibold – the company has worldwide rights and the lead role in development and commercialization, although Bayer HealthCare retains an option to co-promote the drug, and would receive contingent payments based on sales, according to a company statement. Sibold says Genzyme will be hiring sales reps closer to launch. “You’ll see a lot of growth” with respect to marketing and promotional capabilities in MS, says Sibold. Sales force headcount and the specifics of a co-promote with Bayer are details for a later date, he said.
Sibold added that Genzyme has the modest goal of “becoming leaders in MS,” and that “starts with the portfolio.” In addition to Aubagio (teriflunomide), a phase III once-daily oral treatment with “efficacy similar to the platform therapies,” according to Sibold, Genzyme is “looking within our internal pipeline, and looking externally for ways to continually evolve our MS franchise…our plan is to be here for a long time, so we need the supporting portfolio to do so,” says Sibold.
Lemtrada is administered as an infusion over five consecutive days, and then again one year later, for three days. Autoimmune side effects, particularly hypothyroidism and hyperthyroidism, occurred at a rate as high as 30% of patients during phase II, but were down at 15.9% for the CARE-MS II study. A rare autoimmune disorder called immune thrombocytopenic purpura, or ITP, occurred in less than one percent of patients. Only 5% of the Rebif comparator arm had autoimmune thyroid side effect. As a result, analysts have been cool on Lemtrada, but Panzara insists that most of the occurrences were mild to moderate, and they’re well managed. “We’ve gotten very good at detecting and managing [thyroid-related autoimmune side effects], and it doesn’t cause people to leave the trial early, or refuse additional doses; it doesn’t impact that,” says Panzara. “It’s easy to call attention to [Lemtrada’s] side effects, without calling attention to the risks of MS itself,” says Panzara. Of the benefit side of the coin, the CARE-MS II study used the Expanded Disability Status Scale (EDSS) to show that 29% of patients with an EDSS score of two or more – two is characterized as “minimal disability” – had a preexisting disability that was reversed, says Panzara. “Those patients had a reduction in their disability scores that was sustained, so that is a reversal in what they had before,” he says.
Lemtrada’s active ingredient – alemtuzumab – is already on the market in a subcutaneous formulation known as Campath, indicated for the treatment of certain cancers. In 2010, Genzyme execs publicly considered discontinuing Campath and giving it to cancer patients for free, to prevent off-label MS usage, but Sibold wouldn’t elaborate on the company’s current strategy with respect to Campath, beyond expressing a “commitment to ensuring that patients who need Campath receive it.” The pricing issue is problematic, says Selvaraju. “Currently, a dose of Campath that is over 10 times higher than the dose being used in MS patients is sold for roughly $30,00 per year. Just as Genentech/Roche found with their Avastin/Lucentis problem, Genzyme/Sanofi are going to run into trouble if they ask reimbursement agencies to swallow a higher price than roughly $3,000 per patient per year for alemtuzumab in MS,” he says. Taking Campath off the market and providing it for free to cancer patients while putting a premium price on the MS version “is probably not going to fly with reimbursement agencies either here or in Europe,” says Selvaraju.
Lemtrada has patent protection in the US until 2017, and most of Europe until 2014, the company said.
*Raghuram Selvaraju does not currently have ratings or price targets on any of the companies mentioned in this article, nor does he own securities or any derivatives thereof pertaining to these companies. Aegis Capital has not conducted any investment banking business for any of these companies, either.
Apr
10
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Carl Icahn is turning up the heat on Amylin Pharmaceuticals' board of directors. He filed suit against Amylin ($AMLN), claiming that because the company never told investors about Bristol-Myers Squibb's ($BMY) $22 a share offer to buy the company, the company shouldn't be allowed to enforce a rule requiring advance notice of new candidates for the board--opening the door to a bloody proxy fight.
The lawsuit, filed in Delaware, claims that Amylin "rejected a request to reopen the time for stockholders to provide notice" of new nominations to the board. Amylin's deadline for nominations to the board was set for Jan. 25, which came after the BMS offer but long before the takeover attempt was revealed.
Amylin swiftly rejected the claim, saying it was "without merit." Icahn owns 9% of Amylin's shares.
Icahn's strategy is familiar to anyone who has watched him push for the acquisition of ImClone or Genzyme, or new management at Biogen Idec ($BIIB). Once the offer was reported, he charged that Amylin's management was "dysfunctional." A proxy fight could expand his representation on the board and lead to a buyout, offering him another windfall to add to the fortune he's already made in the industry. And investors, who quickly bid up Amylin's stock past BMS' offer, are clearly hopeful of seeing the price go higher.
"Amylin is at a crossroads--it can be sold, now, at a significant premium, or it can continue to attempt to market its products, which may be lucrative if successful, but which entails substantial risk," Icahn's suit claims. "Stockholders of Amylin should be given the choice."
- here's the story from The Wall Street Journal
- get the story from Bloomberg
- read the report from Reuters
Related Articles:
Icahn goes in for the kill at Amylin
Icahn pushes Amylin board to open up for sale
Bristol stays in hunt for Amylin
Is an Amylin takeover inevitable after the BMS rejection?