- Biogen reported Tuesday that one of its experimental lupus medicines succeeded in a mid-stage clinical study, providing a rare win in lupus drugmaking and support for the company's recent move to make immunology a core area of interest.
- The study was two parts, testing Biogen's BIIB059 in patients who have active cutaneous lupus erythematosus or systemic lupus erythematosus. The CLE part evaluated three doses of the drug, and found that after 16 weeks of treatment, each significantly outperformed placebo in reducing disease activity.
- The SLE part, meanwhile, tested just the highest, 450 mg dose of BIIB059 against placebo. While the drug also hit this portion's primary endpoint by reducing disease activity, the statistical margin by which it did so wasn't as strong as what was seen in CLE patients. Biogen didn't provide much in the way of safety data either, noting simply that the drug's safety and tolerability "supports its continued development."
The topline results from Biogen's LILAC study come as a surprise, given how difficult lupus is to treat. Last year alone saw Ablynx, ImmuPharma and AstraZeneca report clinical failures for their respective lupus drugs — though the latter would go on to succeed in a Phase 3 study that used a different primary endpoint.
In spite of that poor track record, Biogen has pointed to its two experimental lupus drugs as the cornerstone of its new immunology focus. The latest data appear to support the gamble, though Biogen will need to study BIIB059 further to confirm its benefits.
CLE patients who received 50 mg, 150 mg and 450 mg of the drug ended up experiencing decreases in disease severity of 41%, 48% and 43%, respectively, from when they started treatment to 16 weeks. CLE patients on placebo experienced a 14.5% reduction over the same time span.
For the SLE patients, researchers looked at how many tender or swollen joints patients had at baseline and then at week 24. They found the patients receiving BIIB059 had 3.4 fewer than those on placebo.
While the results read positive, RBC Capital Markets analyst Brian Abrahams notes that it's difficult to contextualize them because of the various ways drugmakers measure and evaluate lupus data.
For example, Johnson & Johnson's Phase 2 study of Stelara (ustekinumab) in SLE patients used a test known as SRI-4 for the primary endpoint, whereas the LILAC study used that test as a secondary endpoint and active joint count as the primary outcome measure in SLE.
Still, the first glance at LILAC data suggests BIIB059 is "active and potentially competitive," according to Abrahams.
"[T]hough historical challenges in lupus drug development make it difficult to say whether '059 could represent a future cornerstone of an [inflammation] franchise, we believe this positive step towards diversification from a relatively under-the-radar program could help improve confidence around" Biogen's long-term value prospects, Abrahams wrote in a Dec. 3 note to clients.
While best known for its work in neuroscience, Biogen has been under immense pressure to diversify due to its own series of clinical setbacks as well as the competitive challenges facing its marketed drugs for multiple sclerosis and spinal muscular atrophy. Likely in response to that pressure, the company in June said it would put a greater emphasis on drugs targeting eye and immune system-related diseases.
Should BIIB059 progress through the clinic and to market, it won't find many competitors. The only targeted lupus therapy to have cleared the Food and Drug Administration is GlaxoSmithKline's Benlysta (belimumab), a monoclonal antibody that goes after a protein called B-lymphocyte stimulator.
Benlysta came to market in 2011. Over the first nine months of this year, GSK recorded 443 million British pounds (or roughly $575 million) in sales from the drug.
BIIB059 is also a monoclonal antibody, though it targets a different immune-regulating protein known as BDCA2. In addition to BIIB059, Biogen has another lupus drug in its pipeline called dapirolizumab pegol, an antibody that blocks the CD40L protein.