Here’s the blistering action on the Nasdaq from last week, with four of the top gainers and one of the sorely downtrodden:
- ChemoCentryx Inc. (Nasdaq:CCXI) led advancing issues, skyrocketing 282% over the week to $30.33, following encouraging top-line data from its Phase 3 study, called ADVOCATE. The study is evaluating its orally administered, selective complement 5a receptor inhibitor, avacopan. The pivotal study assessed avacopan in patients with anti-neutrophil cytoplasmic antibody-associated vasculitis (ANCA vasculitis), a rare disease affecting small blood vessels. Data from the study exceeded management and investor expectations. The Mountain View, CA-based company is planning to file regulatory application seeking approval for avacopan for treating ANCA vasculitis in the US and Europe next year. The ADVOCATE study compared efficacy of avacopan in combination with Roche RHHBY/Biogen’s BIIB Rituxan (rituximab) or chemotherapy, cyclophosphamide to current standard of care (SOC) of prednisone, a glucocorticoid or steroid, plus Rituxan or cyclophosphamide. Data showed that patients in the study treated with avacopan achieved both primary endpoints of clinical remission at weeks 26 and 52.
- Elsewhere, Hepion Pharmaceuticals Inc. (Nasdaq:HEPA) soared 95% over the week to $6.99. Edison, NJ-based Hepion announced that an animal study of its lead product CRV431 had managed to produce highly positive results. The medicine in question is meant to stop the development of cirrhosis of the liver in patients who suffer aggressive forms of liver disease. When the liver tries to repair and develop new cells, it can lead to the development of unnaturally large scar tissues. The phenomenon is known as fibrosis. Eventually, it can lead to cirrhosis, and this is what the medicine CRV431 seeks to tackle. The animal study of the product was managed by Physiogenex. During the course of the first nine weeks, the rats were given hepatotoxic compound thioacetamide in order to cause fibrosis in their liver. It was administered in combination with either CRV431 or a control. At the end of the study period, it was discovered that half of the rats in the control group developed cirrhosis of the liver. However, none of the rats who had been administered CRV431 developed the condition.
- Sorrento Therapeutics Inc. (Nasdaq:SRNE) surged 83% to $2.92 after the company turned down an unsolicited, non-binding offer from two biopharma companies to acquire all outstanding stock at $3-$5 per share in cash. The offer rejected by Sorrento represented a roughly 88% to 331% premium to the $1.16 at which the shares closed the previous Friday ahead of the company making the approach public. San Diego-based Sorrento said the offer, which was made Nov. 23, significantly undervalued the company and was not in its best interest, leading the board to reject the proposal. Sorrento has one Phase 1 CD38 CART-T asset being developed for multiple myeloma and three other immuno-oncology assets in pre-clinical development. Its non-opioid pain management portfolio has a commercial product in ZTildo 1.8%, which has been approved for postherpetic neuralgia. ZTildo is in clinical development for additional indications. The company is also evaluating another pain medication, resiniferatoxin, in clinics for several indications.
- And Akebia Therapeutics Inc. (Nasdaq:AKBA) shot up 49% to $6.29. The Cambridge, MA-based biotech didn’t announce any new developments. However, it appears that investors are continuing to grab shares after two key events: regulatory filings the week prior revealed that key insiders were buying more of the stock, and on Nov. 9, the company announced positive results after 52 weeks for its lead candidate vadadustat in two late-stage studies conducted in Japan. Sustained momentum that continues for days and weeks after big news for a small biotech stock sometimes is a sign that institutional investors are scooping up shares. These big investors typically don’t build their positions all at once but instead buy relatively smaller positions over a period of time. There’s no way to know for sure at this point if that’s what is happening with Akebia. However, it wouldn’t be surprising if that’s the case. The company’s positive results from the Japanese studies of vadadustat in treating anemia due to chronic kidney disease (CKD) have heightened expectations for good results from US late-stage studies of the drug.
- But La Jolla Pharmaceutical Co. (Nasdaq:LJPC) plunged 51% to $2.74 after saying it was halting a clinical trial testing LJPC-401, its drug candidate for iron overload in patients with beta thalassemia. The San Diego-based company said an interim analysis showed no benefit compared to the placebo. At the same time the company said CEO George Tidmarsh had left “to pursue other interests,” and that a search for a new CEO was underway. The La Jolla Pharma drug is a synthetic version of human hepcidin, a hormone that regulates iron metabolism. In the wake of the failure it plans to “reassess” the continued development of the investigational drug, including tests in patients with another iron overload disorder. Beta thalassemia is caused by a mutation in the gene that codes for hemoglobin, which carries oxygen throughout the body. Some patients require regular blood transfusions to handle the symptoms, and those transfusions can cause dangerous levels of iron to accumulate in their organs. Patients need another treatment, chelation therapy, to remove the buildup.