My Biotech & Medical Watchlist - June 2015

Disclosure - I do not short stocks

Northwest Biotherapeutics - promising vaccines for all solid cancers

Here is the link to my interview on Forbes.com


Below are my responses in an article format -

On the 30^th of May at ASCO, Northwest Biotherapeutics (NWBO) presented new positive results from their Phase1 clinical trial of DCVax-Direct for treating various inoperable solid tumor cancers. DCVax-Direct is NWBO’s second immunotherapy vaccine candidate. 

The Phase 1 trial has so far easily met its primary endpoints of safety and tolerability, the trial is on going. The vaccine continues to show an excellent safety profile without chemo/radiation like side effects. The vaccine also shows promising signs of effectiveness across a broad range of cancers. NWBO are now preparing to move to Phase2 to test its effectiveness. 

The Phase1 study enrolled 40 patients with late stage4 cancers, the patients had 3 inoperable tumors on average and a poor prognosis. The trial covered 13 different cancers included lung, sarcoma, pancreatic, breast and melanoma. 

The purpose of the Phase 1 trial is mainly to test the vaccine’s safety and tolerability. The safety findings are excellent. Patients injected with DCVAX-Direct mainly experience minor side-effects usually mild fevers after their injection. 2 grade3/4 Adverse Events were reported, one for dehydration and one for systemic inflammatory response syndrome. A maximum tolerable vaccine dose was not reached. 

The trial also looked at patient survival and tumor response. The findings are encouraging. The vaccine appears to invoke an immune response at the tumors and in many cases stabilizes the disease across the broad range of cancers tested. 

27 of 39 patients (69%) are still alive at up to 18 months after their first injection. While these are impressive survival figures they are from a very small patient population. The next Phase2 trials will specifically test the vaccine’s effectiveness.

Two different versions of the DCVax-Direct vaccine were tested.  A group of 21 patients were injected with the Method B version of the vaccine. 18 of those patients (86%) are still alive at up to 18 months after their first injection, another impressive survival response.

The other version of the vaccine, Method A, was considered inactive, 9 of 18 (50%) patients are still alive.

Considering these are survival results from very sick stage4 cancer patients from a range of different cancers NWBO’s vaccine shows positive enough results to move onto Phase2 clinical testing.  

NWBO is planning to now run three separate Phase2 trials. Two trials will test separate cancer indications, one for non-small cell Lung Cancer and the other for Sarcoma. The third trial will test various solid cancers like their Phase1 study did. 

NWBO will be making several trial treatment changes for Phase2 to improve their vaccine’s performance. Their next trials will only use one vaccine version (Method B), more tumors will be injected (up to three per patient), and the frequency of injections and the duration of treatment will be increased. These changes should improve patient responses.

NWBO will do a final Phase1 report-out after patients pass 24 months survival.

NWBO’s approach to treating cancer is unique and exciting. They claim their dendritic cell based cancer vaccine is able to educate and direct the human immune system to attack all cancer cell antigens. Cancers are known to each carry thousands of different antigens so it makes sense that attacking all of them is your best approach at defeating the cancer. 

There are other companies also developing promising dendritic cell based vaccines but their treatments are designed to attack a single or several cancer antigens so their approaches are likely to have more limited successes with the fight against cancer. Examples of other companies developing dendritic cell based cancer vaccines include Immunocellular Therapeutics (IMUC), Argos Therapeutics (ARGS), Prima Biomed (PBMD), and the former Dendreon (DNDNQ). 

Since March, NWBO’s share price has moved up from $7 to near $9. I think the share price will continue trending up this year. Without surprises, I think the share price will climb near the $13 level by the end of 2015.

There are several upcoming catalysts to support this positive share price trend. One imminent catalyst is a full enrollment announcement for NWBO’s lead vaccine candidate, DCVax-L at Phase3 for Glioblastoma (GBM). DCVax-L’s first interim efficiency analysis will closely follow. Also an announcement is likely in the coming months on commercial negotiations with German hospitals (HE) for early compassionate use of DCVax-L. Final Phase3 DCVax-L data is expected near summer in 2016. 

The market valuation for NWBO is around $660M, still significantly lower than many small biotech companies developing immunotherapy treatments. I think a Market Cap (MC) around the $2.0B mark would be a fairer value. $2.0B would place NWBO amongst the leaders of its peers (KITE, JUNO). The market does not consider NWBO a leader in immunotherapy. I do, I see them as having an exciting vaccine platform with a superior mechanism of action (attacks all cancer antigens) that can theoretically be used to treat all solid cancers, and their vaccines are also showing superior safety profiles. When the market realizes NWBO’s full potential its MC will quickly reach the levels of its peers.  

Short interest remains high in this stock at 23%. More positive patient data will eventually soften and sway many of these cancer vaccine and NWBO skeptics. 

NWBO’s financial position is improving. Recently, UK’s famous investor Neil Woodford backed NWBO with investments over $65M. NWBO should have enough funds to run operations into late 2015. Their future financing will come easier now that their vaccines are further advanced and less uncertain.