Amicus Therapeutics Inc. (NASDAQ:FOLD) rose $0.93 (20%) to $5.50 on Wednesday after reporting that Amigal migalastat (AT1001) met the co-primary endpoints of comparability to enzyme replacement therapy (ERT) on two measures of kidney function in the Phase III ATTRACT (Study 012) trial to treat Fabry's disease. The measures were mean annualized estimated glomerular filtration and mean annualized measured glomerular filtration rates evaluated over the 18-month treatment period. According to Amicus, comparability was defined as either a 50% or greater overlap in the confidence intervals between the treatment groups; or mean annualized changes in the rates for patients receiving Amigal that is within a prespecified level compared with patients receiving ERT. By early next year, Amicus said it will submit an MAA to EMA for Amigal based primarily on data from Study 012. Next quarter, the company plans to meet with FDA to discuss a U.S. regulatory path for the small molecule that enhances alpha galactosidase A activity.
The Study 012 data came from 56 patients who had received standard ERT for 12 months prior to enrolling in the double-blind trial and who had genetic mutations amenable to Amigal. According to Amicus, about 30-50% of the worldwide Fabry's population has amenable mutations, which are defined as mutations leading to a 3% absolute increase in wild-type alpha galactosidase A activity and a 20% relative increase when exposed to Amigal in a cell-based in vitro assay. Amicus estimates that Fabry's affects 5,000-10,000 people worldwide.
Standard ERT in Study 012 comprised Fabrazyme agalsidase beta from Sanofi (Euronext:SAN; NYSE:SNY) and Replagal agalsidase alfa from Shire plc (LSE:SHP; NASDAQ:SHPG), which AbbVie Inc. (NYSE:ABBV) is acquiring.
Hospira Inc. (NYSE:HSP) filed suit against FDA in the U.S. District Court for the District of Maryland alleging FDA's decision to approve generic versions of Hospira's Precedex dexmedetomidine so long as the labels for the generics do not indicate use in an intensive care unit (ICU) setting is "wrong and unlawful." According to its U.S. label, Precedex is approved to sedate initially intubated and mechanically ventilated patients in an ICU setting; and to sedate non-intubated patients prior to and/or during surgeries and other procedures.
Hospira is arguing that FDA's "proposed carved-out indications" for the generics' labels overlap with the use code for Precedex. According to Hospira's complaint, the use code states "ICU sedation, including sedation of non-intubated patients prior to and/or during surgical and other procedures." Use codes are descriptions of the methods of use protected by patents, which FDA uses to evaluate whether an ANDA infringes patents for a branded drug. A method of use patent covering Precedex in non-intubated patients in an ICU setting expires in October 2019.
The district court granted a stay until Sept. 2, temporarily preventing the U.S. launch of two dexmedetomidine generics that FDA approved this week, when FDA simultaneously said it had the authority to approve Precedex generics.
According to Hospira, Precedex accounted for about 17% of the $2.2 billion in 2013 net sales of its U.S. specialty injectable pharmaceutical products. The company was up $0.99 to $54.91 on Wednesday.
The U.K.'s NICE issued a final appraisal determination (FAD) recommending Revlimid lenalidomide from Celgene Corp. (NASDAQ:CELG) to treat patients with transfusion-dependent anemia with low- or intermediate-1-risk myelodysplastic syndromes (MDS) with a 5q chromosomal deletion when other therapeutics are insufficient or inadequate -- one of its approved EU indications. The recommendation is contingent on a patient access scheme (PAS) in which Celgene will provide Revlimid free of charge to all patients who receive more than 26 monthly cycles.
The FAD reverses draft guidance issued in May, in which NICE recommended against the use of Revlimid for the indication and concluded that because the proportion of patients surviving more than 26 cycles in clinical practice was uncertain, so were the potential cost-savings from the PAS. In the FAD, NICE reiterated that the incremental cost-effectiveness ratio (ICER) was uncertain because of the PAS, but accepted that a commitment from Celgene to publish data on the proportion of people on treatment beyond 26 cycles would "provide reassurance." The cost of a 28-day cycle of treatment with 10 mg of Revlimid (excluding VAT) is £3,780 ($6,325).
NICE already backs Revlimid in combination with dexamethasone to treat relapsed or refractory multiple myeloma (MM) in patients who have received two or more prior therapies. The thalidomide analog is approved in the EU for the MDS indication and in combination with dexamethasone to treat relapsed or refractory MM in patients who have received one or more prior therapies.
Celgene was up $0.64 to $92 on Wednesday.
Bristol-Myers Squibb Co. (NYSE:BMY) and Celgene Corp. (NASDAQ:CELG) partnered to conduct a Phase I trial to evaluate Celgene's Abraxane nab-paclitaxel in combination with BMS's cancer immunotherapy Opdivo nivolumab. Celgene will conduct the trial, which is slated to start next quarter and will evaluate the drugs in patients with HER2-negative metastatic breast cancer, non-small cell lung cancer (NSCLC) and pancreatic cancer. BMS and Celgene will jointly share trial costs.
Opdivo is approved for melanoma in Japan, where Ono Pharmaceutical Co. Ltd. (Tokyo:4528) retains rights. BMS is submitting a rolling BLA for Opdivo for third-line treatment of squamous NSCLC. This quarter, it plans to submit a BLA to FDA for the human IgG4 mAb against programmed cell death 1 (PDCD1; PD-1; CD279) for melanoma.
Abraxane, an albumin stabilized nanoparticle formulation of paclitaxel, is approved in the U.S., EU and elsewhere for indications including second-line treatment of metastatic breast cancer, first-line treatment of NSCLC and first-line treatment of metastatic pancreatic cancer in combination with gemcitabine. Celgene was up $0.64 to $92 on Wednesday.
Pfizer Inc. (NYSE:PFE) and the Crohn's & Colitis Foundation of America will fund up to four academic projects focused on developing small molecules to treat Crohn's disease and ulcerative colitis. A committee comprising members from the not-for-profit and from the pharma's Centers for Therapeutic Innovation (CTI) initiative will select the projects. The organizations will split costs equally, but aren't disclosing how much total funding they will provide for the projects. Pfizer established a similarly structured partnership with the Alliance for Lupus Research in 2012.
Pfizer launched the CTI initiative in 2010 to partner with academic medical centers. According to CTI's website, Pfizer typically takes joint ownership of IP rights in CTI-backed projects and provides milestone payments and royalties for successful programs (see SciBX: Science-Business eXchange, Dec. 2, 2010).
Also this month, Pfizer partnered with 23andMe Inc. (Mountain View, Calif.) to explore the genetic factors associated with the onset, progression, severity and response to inflammatory bowel disease (IBD) (see BioCentury Extra, Aug. 13).
Novartis AG (NYSE:NVS; SIX:NOVN) granted the Global Alliance for TB Drug Development (TB Alliance) exclusive, worldwide rights to the entire portfolio of preclinical tuberculosis (TB) compounds discovered at the Novartis Institutes for Tropical Diseases (NITD). The portfolio includes a group of indolcarboxamide candidates including NITD304, an inhibitor of mycobacterium tuberculosis transmembrane transport protein 3 (MmpL3) that has shown activity against drug-sensitive and multi-drug resistant TB. Financial terms were not disclosed.
AstraZeneca plc (LSE:AZN; NYSE:AZN) and Mitsubishi Tanabe Pharma Corp. (Tokyo:4508) partnered to validate and progress novel targets and small molecules for diabetic nephropathy into the clinic under a three-year research deal. The companies have identified undisclosed targets of interest from their research portfolios, though they may add additional targets to the deal. The research will be performed at AZ's Cardiovascular and Metabolic Disease Innovative Medicines unit in Sweden and at Mitsubishi's facilities in Japan.
FDA will allow Kronus Market Development Inc. (Star, Idaho) to market Kronus' ZnT8Ab ELISA diagnostic, which differentiates Type I diabetes from Type II or gestational diabetes. The blood-based assay detects the autoantibody against solute carrier family 30 zinc-transporter member 8 (SLC30A8; ZNT8), which is produced in many people with Type I diabetes but not in patients with other forms of the disease.
The Shanghai and Hong Kong stock exchanges are conducting final tests and rehearsals ahead of the initial launch of a trading link between the two exchanges. The pilot program -- dubbed Shanghai-Hong Kong Stock Connect -- will establish mutual stock market access between mainland China and Hong Kong. In April, when plans for the program were first announced, the two exchanges said it would take about six months to complete preparations, which would put a formal launch date around October.
The program represents another major push in China's ongoing efforts to open its capital markets to overseas investors and increase its market-oriented reforms. Foreign exchange controls have restricted overseas investments in the country, and it was only until the Qualified Foreign Institutional Investor (QFII) Scheme was introduced in 2002 that foreign investors were granted direct access to China's capital market. Under QFII, only institutional investors that meet certain criteria can qualify for a QFII license. Shanghai-Hong Kong Stock Connect would expand access to the Shanghai stock exchange to all Hong Kong and overseas investors.
One of the goals is to develop Hong Kong as an offshore RMB business center by allowing mainland investors to participate in the Hong Kong stock market using RMB. It will also expand investment channels for offshore RMB funds and facilitate a flow of RMB funds between the two markets. However, it is unlikely that the exchange will support IPOs.
Molecular diagnostics play GeneReach Biotechnology Corp. (GreTai:4171) raised NT$166 million ($5.5 million) last week through the sale of 4 million shares at about NT$41.09 in an IPO on the GreTai Securities Market. GeneReach markets veterinary diagnostics, including its PetNAD assay system. The company plans to expand into human health. GeneReach was off NT$4.45 (11%) to NT$36.50 on Wednesday.
NovaBay Pharmaceuticals Inc. (NYSE-M:NBY) was off $0.44 (35%) to $0.81 on Wednesday after discontinuing development of an ophthalmic formulation of auriclosene (NVC-422) to treat adenoviral conjunctivitis. The decision came after the product missed the primary and secondary endpoints vs. vehicle in a 450-patient Phase IIb trial in the indication. NovaBay said it will focus on developing an irrigation solution of auriclosene that is slated to enter a second Phase II trial this half for blockage and encrustation in indwelling long-term urinary catheters.
NovaBay said it also will focus on the ongoing U.S. launch of a lid and lash cleanser, as well as supporting partner China Pioneer Pharma Holdings Ltd. (HKSE:1345) with the Chinese launch of wound cleanser NeutroPhase, pending the product's approval in the country.
Galderma S.A. (Lausanne, Switzerland) has exclusive, worldwide rights to auriclosene for dermatological indications. Last year, the partners said a topical gel formulation of the synthetic analog of N-dichlorotaurine missed the primary endpoint in a Phase IIb trial to treat impetigo (see BioCentury Extra, Nov. 6, 2013).