VRTX had the Kalydeco sales guidance for 2016 at $670-$690M worldwide which is only $50M more than 2015, representing only about 200 additional patients. This suggests that expansion to the residual function mutations (there are 3000 ww) was not expected to have a significant impact on revenue this year. There are 200 Kalydeco-responsive patients who are coming off from the 661/Kalydeco trial alone.
The trial on which the expansion was based was very small -- 24 people participated and 21 people in the open label. Efficacy was good. It is most likely that the FDA wants to see repeat of the same trial with a larger number of participants.
It was an 8 wk trial. Since Vertex can spend more money now than before, they can run a trial quickly and get the expansion approval in a year or two latest.
Gilead doesn't have to spend much to buy several biotechs right now. Gilead earns at an annual rate of more than 18 B a year with a 30+ B revenue.
24/7 Wall St. also has a similar idea, Q. The report is difficult to open. So quote it below.
[[ Vertex Pharmaceuticals Inc. (NASDAQ: VRTX) recently had its cystic fibrosis treatment Kalydeco (ivacaftor), accepted for review of a supplemental New Drug Application (sNDA). The FDA granted Vertex’s request for Priority Review of this sNDA and a target review date of February 6. This could be a turning point for Vertex, as the stock has gotten beaten up over the past couple of weeks. Shares ended last week at $90.75, with a consensus analyst price target of $140.25 and a 52-week trading range of $81.98 to $143.45. ]]
As you may be aware, Vertex is quietly developing cancer drugs: three of them VX 970, VX 803, and VX 984 in clinical trials. They are inhibitors of DNA damage repair, and most effective when they are given together with DNA damaging agents such as chemo and radiation. In 2015, they estimated, there were 1.6 million newly diagnosed cancer cases in the US and 600 thousand of patients would die. About 2/3 of all cancer patients would receive radiotherapy in their illness. I highlight the above drugs as effective sensitizers to radiotherapy because radiation, particularly high energy radiation, is an effective DNA damaging agent, and can be delivered to (and focused on) a localized tumors without seriously damaging surrounding normal tissues.
Vertex is recruiting and also 'not-yet recruiting' participants to multiple clinical trials for VX 970, and some results will be available sometime this year. Both VX 970 and VX 803 block damage repair by allowing the cell division cycle to proceed so that damaged DNA leads to broken chromosomes. VX 984 blocks non-homologous end-joining repair, being an inhibitor of DNA dependent protein kinase. Chemo or radiation alone cannot kill all tumor cells, but there is a chance that they could kill almost all tumor cells when used with above sensitizers. Any remaining tumor cells can be mopped up with immuno-oncology drugs such PD-1 or PD-L1 inhibitors. Chemo and radiation would remain the first line of battle against tumors for a long time. About 1 million people would have received radiotherapy in 2015. Both radiotherapy and chemo are expensive and they better be used with sensitizers to make them effective.
Verity, the literature is clear in supporting the relationship between increased infection and defective CFTR ion transport. This link is explained by difficulty of removal of mucus trapped bacteria and decreased immune response. But if you are seeking a quantitative relationship between a degree of CFTR function improvement and the improvement from staph infection, then I am afraid that you may have to wait until clinical data from Vertex' triple combo is released. I'll keep searching animal model studies. If I misunderstood your question, let me know.
A majority of analysts give a high target price. I found the quote below from Google Finance today.
[[...Piper Jaffray reissued an “overweight” rating and set a $180.00 target price on shares of Vertex Pharmaceuticals in a research report on Tuesday. Vetr raised shares of Vertex Pharmaceuticals from a “buy” rating to a “strong-buy” rating and set a $141.69 target price for the company in a research report on Monday, December 7th. Stifel Nicolaus assumed coverage on shares of Vertex Pharmaceuticals in a research report on Tuesday, December 1st. They set a “buy” rating and a $160.00 target price for the company. JPMorgan Chase & Co. raised shares of Vertex Pharmaceuticals from a “neutral” rating to an “outperform” rating in a research report on Thursday, November 26th. Finally, Guggenheim reissued a “buy” rating on shares of Vertex Pharmaceuticals in a research report on Thursday, November 5th. One analyst has rated the stock with a sell rating, six have assigned a hold rating, twenty-one have given a buy rating and one has assigned a strong buy rating to the company’s stock. The stock has a consensus rating of “Buy” and an average price target of $149.31.]]
As it was reported in SCIENCE, they could snip out a faulty segment (exon) of the gene in mice. They have not yet replace the gap with a normal exon. It is more difficult.
[[ ... The red-hot genome editing tool known as CRISPR has scored another achievement: Researchers have used it to treat a severe form of muscular dystrophy in mice. Three groups report today in Science that they wielded CRISPR to snip out part of a defective gene in mice with Duchenne muscular dystrophy (DMD), allowing the animals to make an essential muscle protein. The approach is the first time CRISPR has been successfully delivered throughout the body to treat grown animals with a genetic disease...]]
A paragraph from an article commenting on the award: [[... Their work grew out of a finding that bacteria could remember viruses. Researchers had found segments of viral genes, sandwiched between repeated bacterial DNA sequences. These viral scraps serve as an infection memory bank: From them, bacteria create guide RNAs that can seek out the DNA of returning viruses before chopping up the viral genes with a nuclease, Cas9. Once this mechanism was understood, Doudna and Charpentier, among others, raced to adapt it to editing DNA in higher organisms.]]
These people who applied the principle to higher organisms would certainly gain the Nobel prize in near future.
When RNAi was chosen for Nobel prize in physiology in 2006, $ALNY took off from low teens to 20s. Vertex may rise too.
[[ JPMorgan’s Cory Kasimov and team name Biogen (BIIB), Vertex Pharmaceuticals (VRTX), and BioMarin Pharmaceutical (BMRN) their top biotech picks for 2016.]]
[[ ... we see Vertex as a very attractive investment with commercial momentum and potential bottom line leverage combined with a substantial amount of meaningful data and significant strategic value.]]
Good suggestion. I'll own many common shares when option expiration dates come in future months.
Lentiglobin is working well if beta-thalassemia patients do NOT have beta 0 homozygous genotype. Even for the homozygotes benefit is there. For SCD, more data are need to determine the value of the procedure. It appears that for some patients it is working; it may be age-related, or partial bone marrow cell ablation is the cause of poor results for others.
Rojo, the DNA sequence of inserted gene is different from the sequence of removed piece. GUIDE RNA complexed with Cas9 makes sure that the nuclease produces cleavages at the desired places on the basis of sequence homology. For example, in CF a segment of CFTR containing del F508 can be removed and replaced with a wild type segment. A well-engineered GUIDE RNA would certainly distinguish the mutated from the wild type. CRISPR is known for specificity.
As I quoted from FT before, Gilead is actively seeking biotechs to buy. Gilead has a gross annual profit of 21B. It can buy 10 bluebird size biotechs with a single year profit. They are particularly interested in CAR-T drugs. Juno has Celgene, and Kite works with Amgen. bluebird is a strong candidate for CAR-T oncology products they want to acquire. Vertex is also interested in gene therapy because they are very committed to treating CF patients. Vertex is also interested in SCD as shown by their deal with Crispr. I see a bidding war.
I have been an investor of Blue for several months, and suffered a loss. I plan to sell more puts, not buying puts. Blue is a high risk, high reward stock.
Gilead Sciences is considering acquisitions, according to Financial Times this morning. The management has been saying for some time about their interest in buying immuno-oncology area, particularly, CAR-T technology. GILD can easily pay a 100% premium to 2B market cap of Blue. Gene therapy would be a fantastic bonus.