IMO it's because no one really knows who Pulmatrix is as a company. Most stock owners are not the general public. Likewise, people don't understand the value of an inhaled anti-fungal however, they need look no further than Acorda (ACOR) Therapeutics who is being very successful thru their inhaled version of an old (generic) Parkinson's drug. I would buy PULM and not look at it again for 18-24months...if their clinical trial goes well, you're a hero...if not, well you just have to get out.
My understanding is that Concert was able to procure the IP for a deuterated version of Kalydeco years ago it is unclear if Concert can do this again for Orkambi or other CF related compounds. I think Vertex has improved their IP protection from deuterium-modified versions of their compounds. Worth mentioning is that CEO @ Concert Pharma was instrumental in the success of Kalydeco for Vertex years ago.
Our DD has been on Orkambi for 75 days, the only initial side affects were some chest tightening and a few days of increased mucus production. This is the longest she's gone without antibiotics in 3 years.
For those interested, here's a list of publicly traded companies with a significant focus on CF, “small cap”
ANTH, Anthera - About to begin phase III trials for a Pancreatic Enzyme therapy (CF digestion & absorption)
NVLS, Nivalis - In phase II, promises to compliment/improve Kalydeco & Orkambi
PTCT, PTC - Late stage trials for nonsense mutations…(10% of the CF population)
PULM, Pulmatrix - Preclinical, focus on CF related fungal infections, CF-ABPA
CRBP, Corbus - In phase II trial for CF related inflammation
PRQR, ProQR - In phase I trial for RNAi therapy
Obviously there’s the big guys; Abbvie, Novartis, Genzyme, Bayer, Shire and Pfizer – each with a CF program or more. Who am I missing?
The Porcine-sourced PEP therapy market, which if approved Sollpura would dominate, was 1.8 Billion in 2008...is likely closer to 2.2 Billion now as CF patients are living longer lives. There are about 225,000 individuals in the U.S. suffer from EPI (exocrine pancreatic insufficiency) due to not only CF but also due to chronic inflammation of the pancreas, pancreatic HIV infection, surgical removal of all or part of the pancreas, and other digestive diseases.
Sentiment: Strong Buy
While Anthera's lead candidate is promising...Sollpura is why to own this stock, here's why: There are 75,000 Cystic Fibrosis patients around the world all take pig intestine derived capsules (like oink, oink) EVERY time they eat anything containing fat, (CF patients cannot/absorb digest fat...it's not just a lung disease) All Anthera has to do is get Sollpura approved and they will rock it (or get bought for a boat load). I am not a pumper but I do know an awful lot load about CF. A typical 12 year old with CF take 5-6 enzyme pills per meal (about 7,500 per year!) Anthera's science makes that 1-2 pills per meal and a yearly total of about 1,500!
Sentiment: Strong Buy
Congratulations, MB! Crossing fingers for you. Our 3 month supply is being Fed Exed as I type this message. Insurance was a SNAP! Doctors did NOT wait for a 3 month appointment...our clinic initiated the whole process. Less than 2 weeks from order to shipping...like we hit the "EASY" button.
Sentiment: Strong Buy
Vertex should get Orkambi approval by Monday or sooner, says Piper Jaffray
Piper Jaffray analyst Edward Tenthoff said he is confident that the FDA will approve Vertex's Orkambi in homozygous F508del cystic fibrosis by its PDUFA date of July 5, meaning a decision should come by either late Thursday or by Monday morning, due to the holiday. Pricing will be key, Tenthoff noted, adding that he expects it to be in-line with Kalydeco's $311,000, except with steeper gross-to-net discounts. Tenthoff expects Vertex to retain its dominance of CF therapy and keeps his Overweight rating and $146 target on its shares, which closed down $4.84 near $122 yesterday.
Galapagos and AbbVie are working together to develop a triple combination therapy for cystic fibrosis patients with the Class II (F508del) mutation. Novel potentiator GLPG1837 is currently in Phase 1 and is expected to enter a Phase 2 study in class III mutation patients before end 2015. Novel corrector GLPG2222 is expected to enter Phase 1 before end 2015. Galapagos and AbbVie expect to nominate a second novel corrector by Q3 2015 and thereby complete the triple combination therapy discovery phase. This second novel corrector is expected to enter Phase 1 in Q2 2016
It will be interesting to see how Proteostasis and Nivalis (N30) start to factor into the third leg on the stool equation. Their therapies might improve Orkambi and Kalydeco.
Not that the FDA is sentimental but June 24th is the 1 year anniversary of the Kalydeco/Lumacaftor phase III trial results. Could be as good as any day for a thumbs up!
Verty's got it right (been lurking). - most won't see the positive in this release but that's not my problem & VRTX management shouldn't care either. Remember...to a CF patient or parent...all we want is to arrest the disease; To [theoretically] stop the lung related affects of CF in its tracks. Which, for a growing # of CF-ers, is what VRTX science delivers. Trust me...if I could halt my kids lung function at 87%...I'd take it and RUN! The only other option is to watch these patients is to get sicker and sicker by the day - right before your eyes. Last part of my rant...VRTX has the corrector/potentiator market until at least 2022 and IMO, once K + Lumacaftor revenues come in, they will be active buyers.
Utterly fantastic win for VRTX today & for CF patients - a huge win/win! Arguments over pricing are ridiculous. VRTX CF drugs REDUCE the of CF patient care costs, a fact Insurance companies admit. Vertex and their investors will enjoy many years of happy returns...until another company comes along with a better drug that's safer and cheaper. The soonest that will be is 2022. Just incredible news for biopharma innovation...
More and more players in CF nowadays! Never enough me! The following is from their web site..."Our lead product Resunab™ is a novel oral anti-inflammatory drug that is expected to commence Phase IIa clinical trials for the treatment of cystic fibrosis and scleroderma, pending U.S. Food and Drug Administration (FDA) approval in the second half of 2014."
Big meetings are happening this week Bethesda, MD (CFF HQ)...so, Monday may be correct because BIO 2014 is in San Diego starting Monday June 23rd. San Diego/La Jolla is where Kalydeco and Lumacaftor were invented. The largest biopharma conference in the world seems to be an ideal place and time to release positive news.
Sentiment: Strong Buy