Modus Therapeutics has begun a Phase I study with subcutaneously administered sevuparin. Its CEO says this new formulation of its lead asset has the potential to be self-administered by sickle cell disease patients at home for the treatment of vaso-occlusive crises.

South Korean biotech Alteogen is progressing the development of its Eylea biosimilar both at home and abroad, with the hope of becoming a global first mover with its version in wet AMD, backed by formulation and manufacturing patents.

A surprisingly strong improvement in vision has accompanied the use of proprietary stem cells in the first three retinitis pigmentosa patients in a US Phase II study, intriguing the UK biotech, ReNeuron, and maybe attracting development partners too.

Once suspended due to safety issues, bardoxolone now has completed a Phase II study that showed kidney function benefit in four rare types of CKD. A pivotal study is underway, with another planned and more possible.

In its latest TANGO trial for chronic lower back pain, Eli Lilly and Pfizer’s tanezumab has shown efficacy at 10 mg, but its 5 mg dose missed the statistically significant mark. Vitally, analysts are encouraged by safety data.

The drug demonstrates improvements in overall survival and progression-free survival, but not enough for success in KEYNOTE-240.

Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.

A controversial cell therapy for spinal injuries receives coverage under Japan’s national health scheme, amid signs that the pricing of regenerative therapies may come under more scrutiny, while the first CAR-T treatment in the country takes a step closer to market.

Novo Nodisk's CSO says FDA approval of extended half-life Factor VIII Esperoct treatment for people with hemophilia A will give more choice to those patients and "effectively completes" the Danish group's hitherto R&D efforts based on clotting factor deficiencies. 

Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.

With four new entrants readying to be prescribed in the next four years, the market for diabetic nephropathy is looking more buoyant than ever. However, competition from GLP-1 agonists and eventually from generics could put a dampener on proceedings.

New potentially effective therapy for drug-resistant epilepsy, seen as the most pressing unmet need in this market, is on the horizon as FDA begins approval review of SK’s cenobamate, also marking a first for the Korean pharma industry.

Roche is working towards implementing PMS studies for Avastin (bevacizumab) and ensuring that a suspension of marketing authorization for the product in first line ovarian cancer is lifted in India.

Patients with heart failure on Entresto say they have fewer symptoms and greater quality of life, according to a registry study and Novartis is hoping that this and other real world data will propel the drug towards its peak sales target of $5bn.

Europe is expected to follow where the US led in expanding the label for Amgen's PCSK9 inhibitor Repatha to include a prevention indication for heart attacks and strokes. But growth will be hard-won.

Valeant is touting Siliq to lead new product growth, but the drug's significant safety concerns in a competitive psoriasis market likely will offset efficacy advantages it could offer.

With few remaining unmet needs in hepatitis C, Gilead, Merck and AbbVie are focusing on tough-to-treat niches with their next-gen combination regimens, especially treatment-experienced patients who failed on prior direct-acting antiviral therapy. Merck also is studying injectable drug users in an effort to eradicate HCV entirely.