Today's Top News Tuesday, March 15, 2016 | By Damian Garde
| | Eli Lilly CEO John Lechleiter |
Eli Lilly ($LLY), at work on a huge Alzheimer's disease study with implications for the whole drug industry, is changing the main goal of its trial to reflect what the company called "emerging scientific evidence." The study, dubbed EXPEDITION3, is testing Lilly's twice-failed solanezumab in about 2,100 patients with early-stage, mild Alzheimer's. The trial was first designed to measure both cognition and physical function as co-primary endpoints, but Lilly has now amended EXPEDITION3 to feature only cognition as its main goal, with function demoted to a secondary endpoint. "Emerging scientific evidence supports the idea that cognitive decline precedes and predicts functional decline in Alzheimer's disease, particularly in earlier stages of the disease," Lilly said in a statement. The change doesn't affect how Lilly is handling the global trial, the company said, and investigators will remain blinded to all study data until the trial wraps up in the fourth quarter of this year. EXPEDITION3 is Lilly's third attempt to prove solanezumab, an antibody therapy, can change the course of Alzheimer's disease. The treatment is part of a class of therapies that target misshapen proteins in the brain called beta amyloids in hopes of slowing the memory-destroying advance of Alzheimer's. Lilly's antibody managed to effectively reduce amyloid buildups in a pair of Phase III trials back in 2012, but the treatment failed to beat placebo at actually improving cognition and function. Later, poring over the data from those studies, Lilly noted that solanezumab did chart a significant cognitive effect on subjects with mild forms of the disease, and the company designed EXPEDITION3 to focus solely on those patients with the goal of finally winning approval for the treatment. - read the statement Related Articles:
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Tuesday, March 15, 2016 | By Damian Garde
Roche ($RHHBY) is promising up to $1 billion in exchange for as many as 5 targeted cancer therapies from Blueprint Medicines ($BPMC), looking to bolster its own work in immuno-oncology. Under the deal, Cambridge, MA's Blueprint gets $45 million in upfront cash, and Roche has laid out another $965 million tied to option fees and milestone payments for all 5 potential collaborations. Roche can buy into each Blueprint therapy once it achieves proof of concept in a Phase I trial, the companies said, and Blueprint has the option to retain commercial rights to as many as two of the 5 programs, receiving royalties on the other three. For Roche, the deal brings in a handful of small-molecule projects focused on immunokinases, enzymes that regulate immune response. Most of today's cancer immunotherapies, including Roche's in-development atezolizumab, are antibody treatments that work by removing the brakes from cancer-fighting T cells. Roche is betting that Blueprint's early-stage work could bring about a complementary force, dovetailing with the effects of so-called checkpoint inhibitors like atezolizumab to create a more powerful weapon against tumors. And Blueprint sees the partnership as a means to open up a new chapter in immuno-oncology, CEO Jeff Albers said. "We believe Blueprint Medicines' proprietary drug discovery platform and expertise in immunokinases, combined with our proven ability to move quickly through drug discovery, is a perfect complement to Roche's expertise with cancer immunotherapy biology and in developing and commercializing innovative therapies," Albers said in a statement. Blueprint, a brainchild of Third Rock Ventures, went public last year on the strength of its R&D platform, which uses genomic profiling to match compounds from a library of kinase inhibitors with the cancers they're best suited to treat, crafting precision therapies. The company inked a similar licensing deal with the rare disease-focused Alexion ($ALXN) in 2015, signing up to reap as much as $265 million. Meanwhile, Blueprint is at work on a stable of proprietary cancer therapies, led by the liver cancer treatment BLU-554 and gastric cancer medicine BLU-285, both in Phase I development. The company is also planning to take BLU-285 into clinical trials for mastocytosis and has a discovery-stage program targeting the RET kinase to treat multiple cancers. - read the statement Related Articles:
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Alexion bets up to $265M on Blueprint's precision medicine platform Monday, March 14, 2016 | By Damian Garde
Celator Pharmaceuticals ($CPXX) saw its shares roughly quintuple in value on Monday afternoon after the biotech posted positive Phase III results for its nano-powered blood cancer therapy, putting the tiny company in line for an FDA filing this year. The Ewing, NJ, company's lead drug, CPX-351, is a 5:1 formulation of the standard cancer treatments cytarabine and daunorubicin, packaged in a nano-scale delivery platform that Celator says can boost efficacy while limiting the risks of administering each drug on its own. Now the company has Phase III data to back up that claim. In a trial on 309 patients with high-risk acute myeloid leukemia, Celator tested CPX-351 against standard cytarabine and daunorubicin therapy, finding that its combo treatment notched a statistically significant effect on overall survival, meeting the study's primary endpoint. Patients in the treatment arm posted a median survival rate of 9.56 months, besting the 5.95 months observed in the other group, Celator said. CPX-351 patients also demonstrated a 31% reduction in risk of death compared with the old drugs, according to the company, and Celator's therapy came through with a statistically significant improvement in response rate. On the safety side, Celator said the rates of serious side effects were comparable across the two study arms, and CPX-351 charted a lower rate of all-cause mortality than cytarabine and daunorubicin after 60 days. Celator said it's holding off on disclosing detailed safety and efficacy data until it can present its findings at the American Society of Clinical Oncology meeting in Chicago this summer. The data are particularly impressive considering that acute myeloid leukemia, or AML, has a dismal survival rate, and fewer than 10% of patients with high-risk forms of the disease live more than 5 years after diagnosis, according to Celator. Furthermore, if CPX-351's top-line results survive future scrutiny, Celator will have done something no small-cap biotech has ever done: successfully execute a Phase III trial and break the Feuerstein-Ratain Rule. The data sent Celator's shares, trading at $1.68 as of Monday's close, up as much as 500% postmarket before they settled at around 3.5 times their earlier value. The company, which reported having just 20 employees in its last annual report, is now planning to file CPX-351 for FDA approval in AML this year. - read the results (PDF) Related Articles:
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Roche ($RHHBY), the world's biggest oncology drugmaker, is set to have its new bladder cancer treatment atezolizumab approved by this fall as the FDA green-lights a speedy review. The Swiss company said in a statement that the U.S. regulator has accepted the company's biologics license application and granted priority review for atezolizumab in certain patients with a late-stage form of the disease. The drug was given breakthrough therapy status by the FDA back in 2014. Specifically, Roche is seeking a license for the treatment of patients with locally advanced or metastatic urothelial carcinoma who had disease progression during or following platinum-based chemotherapy in the metastatic setting, or whose disease worsened within a year of receiving platinum-based chemotherapy before surgery or after surgery. Urothelial carcinoma accounts for 90% of all bladder cancers and can also be found in the renal pelvis, ureter and urethra. There are currently few drug options for treating bladder cancer outside of chemotherapy--although a number of drugs from Novartis ($NVS), Merck ($MRK) and Allergan ($AGN) are also looking to get in on this burgeoning new market. A priority review is usually given by the FDA when a new drug treats a disease with few options, or is particularly effective. It speeds up its marketing review to just 6 months--meaning the FDA will make a decision on approval by Sept. 12. Roche's most recent study for the drug--the Phase II, open-label IMvigor 210 trial--found that after 11.7 months of median follow-up, atezolizumab shrank tumors in 15% of the 310 patients tested whose disease progressed after platinum-based chemotherapy. Atezolizumab also shrank tumors in 26% of 100 patients whose disease had medium and high levels of PD-L1 expression. The drug was originally developed by Roche's Californian biotech arm Genentech. Roche is looking to be the first company to bring an anti-PD-L1 immunotherapy to the market for this particular cancer. It is however playing catch-up in the PD-1 (programmed death-1) and PD-L1 (programmed death ligand-1) market race, as rivals Bristol-Myers Squibb ($BMY) and Merck have already gained approval for their PD-1 medicines Opdivo and Keytruda, which both received the FDA nod for melanoma in 2014. Roche is also battling with AstraZeneca ($AZN) to be the third to market in this new therapy area--although its PD-L1 drug works slightly differently to the approved PD-1 medicines on the market. Atezolizumab directly binds to PD-L1 expressed on tumor cells and tumor-infiltrating immune cells, blocking its interactions with PD-1 and B7.1 receptors. The Swiss major is betting that its mechanism of action will help separate it from the PD-1 field, and also lead to better cure rates in the long term. Merck is however also testing Keytruda in certain bladder cancer patients who exhibit PD-L1 biomarkers--meaning Roche could have an immediate competitor should both drugs gain approval. More than around 76,000 Americans will be diagnosed with bladder cancer in 2016, and about 11% of new diagnoses are made when bladder cancer is in advanced stages. The PD-1 and PD-L1 market is set to be worth around $30 billion by the end of the decade, although Bristol-Myers is expected to take around a $6 billion share of this as Opdivo is set to gain further cancer licenses in the coming years. Atezolizumab is also being studied in a Phase I trial in combination with Amgen's ($AMGN) cancer immunotherapy Imlygic in patients with triple-negative breast cancer and colorectal cancer with liver metastases. It's also posted survival data among patients with non-small cell lung cancer who express high levels of PD-L1, as well as starting new trials into assessing its efficacy in certain kidney cancer patients. Roche will hope for this to become a blockbuster treatment and help shore up sales in the next decade, given that a slew of copycat biosimilars are set to erode sales of its big-selling oncology products Avastin and Rituxan. - read the statement Related Articles:
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| | Spectrum CEO Rajesh Shrotriya |
Spectrum Pharmaceuticals ($SPPI) won FDA approval for its take on a common chemotherapy, reversing an October rejection and clearing the way for the acquisitive company's 6th product launch. The drug, to be sold as Evomela, is a version of the widely used melphalan, reformulated to increase stability, according to Spectrum. The injected treatment is now approved for use in patients with multiple myeloma who are about to receive stem cell transplants or who cannot tolerate oral therapies like Celgene's ($CELG) Revlimid and Pomalyst, the company said. The FDA rejected Spectrum's first attempt approval last year for reasons the company never disclosed, dealing a blow to the biotech's share value from which it is yet to recover. With Evomela finally approved, Spectrum is planning to launch the drug with its existing sales force, believing it can cut in on the roughly $100 million market for intravenous melphalan. Spectrum bought the drug from Ligand Pharmaceuticals ($LGND) in 2013, paying $3 million up front and promising up to $50 million more tied to clinical and regulatory success, plus royalties on sales. - read the statement Related Articles:
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