Overweight SGMO with core position and small trading position.
BIVV entry level on valuation and SGMO collaberation
ONCE took entry level position after they raised $380m secondary in Aug.
Target AVXS currently too expensive. No collaboration. One clinical trial = concentrated risk. Still researching
Target ABEO looks like solid candidate. Limited cash suggests they will do a raise in NTM which might be a good entry point. With 4 ODD and 7 preclinic candidates they look more compelling than most.
Target RGNX still evaluating
Spark Therapeutics, Inc. (“Spark”) (NASDAQ:ONCE) announced today the closing of the previously announced underwritten public offering of its common stock pursuant to an automatically effective shelf registration statement that was previously filed with the Securities and Exchange Commission, including the exercise in full by the underwriters of their option to purchase an additional 690,789 shares from Spark at the public offering price of $76.00 per share, less the underwriting discount. The exercise of the option brought the total number of shares sold in the offering to 5,296,053, and increased the aggregate net proceeds to Spark to approximately $380.4 million, after deducting underwriting discounts and before offering expenses.
Uploaded a new spreadsheet today in first post of this thread to include ONCE secondary etc
The ability to truly compare the various companies is particularly limited. The array of approaches and therapeutic approaches varies as does their pipeline disclosure policies. To somewhat limit the attempt to compare disimilar companies I'll group KITE, JUNO and BLUE in group one and put the others in group two.
Group One represents the large cap more advanced companies
Mkt Cap $8.1b
JUNO rumored in play > Celgene
Mkt cap $7.7b
KITE acquired > Gilead
Juno and Kite are primarily cell therapy engineered CAR-T and TCRs for oncology. Juno has a 10 yr collaboration with Celgene which is the likely driver for the M&A discussions. The valuation of cell therapy companies has aggressively moved up after Novartis FDA approval for Kymriah which then led to the Gilead acquisition of KITE and now Celgene acquisition of Juno.
Novartis move signals further gene therapy resurgence Date January 25, 2018 Less than two years ago Novartis’s decision to disband its cell and gene therapy division caused a crisis among investors. Events since then have shown how misplaced the fears were: Novartis and other large groups have continued showing an interest in gene therapies, likely driven by an increasingly positive regulatory stance.
Yesterday the Swiss giant, the first to launch a CAR-T therapy, doubled down on emerging technologies when it paid Spark Therapeutics $105m for Europe rights to Luxturna, the first bona fide gene therapy approved in the US. The deal might disappoint Spark bulls, but it does feature as one of the largest up-front fees for a gene therapy in recent years (see table below).
As far as big pharma buy-in is concerned, however, Novartis is up against Pfizer, which has probably committed more than any other large group to gene therapy. Novartis’s Spark deal came a few months after Pfizer licensed Sangamo’s haemophilia A gene therapy, and is outstripped by Pfizer’s $193m takeout of Bamboo Therapeutics last year.
Indeed, Spark itself already counts Pfizer as a partner, having licensed its haemophilia B gene therapy to the big pharma group back in 2014. It was at roughly that time that Uniqure launched Glybera, the west’s first ever approved gene therapy, in the EU, though the product was a commercial flop and ended up being withdrawn.
Despite that failure gene therapies have continued to impress clinically – witness Biomarin’s spectacular results for valoctocogene roxaparvovec in haemophilia A, presented at Ash last year – and have won further regulatory backing: Spark’s Luxturna bagged US approval for vision loss caused by biallelic RPE65 mutation-associated retinal dystrophy last month.
As such, Spark could probably not have hoped for a better Luxturna partner than Novartis. The Swiss group boasts the muscle of its Alcon eyecare division, as well as having ex-US rights to Lucentis, one of the bigger eyecare drugs. And it already has a gene therapy deal under its belt – a 2010 hearing loss tie-up with Genvec.
All the signs point to growing confidence in gene therapy as a viable technology. However the $105m that Novartis has paid Spark is still a significant bet to make on a product that could have difficulties gaining reimbursement among austere European payers. Regulatory successes notwithstanding, whether gene therapies are economically viable is a major missing piece of the puzzle.
That said, Spark’s bullish investors have assigned a market cap to the group of over $2bn, and the Novartis deal could disappoint those who had banked on a takeout. Stifel analysis today wrote that Spark trading ex-US sales “for what essentially amounts to $3/share and a mid-20% royalty rate” would raise valuation concerns.
It could be that big pharma has not bought into this area as much as exuberant investors have.
Sanofi, for one, seems to have significant doubts, last year canning a 2015 deal with Voyager despite what looked like promising data in Parkinson’s disease, and this week buying Bioverativ in a clear snub to haemophilia gene therapies (Sanofi bets against haemophilia gene therapy, January 22, 2018).
The markets do not look like being put off just yet, but today’s planned float of Solid Biosicences will provide a major test. The group upsized the IPO to $130m yesterday, but only this morning revealed that its lead asset, the Duchenne muscular disease project SGT-001, had been placed on partial US clinical hold in November – before it first filed to float.
With some venture capitalists hailing 2018 as the year in which gene therapy finally comes of age this advanced therapy niche still has it all to do.
Under: Been on the sidelines for a bit holding (building) cash. Now that "BIGLEY" has rolled out the tax plan its time to jump in.
Dec 21, 2017 19:06:02 GMT -6
martyc: Looks like you are buying Msft again!
Dec 15, 2017 11:23:29 GMT -6
martyc: The news that Trump called Rupert to congratulate him sure seems to indicate that this is heading to approval
Dec 15, 2017 11:22:23 GMT -6
Under: DIS finally getting some traction.?
Dec 14, 2017 17:08:45 GMT -6
martyc: I took an entry level position in DIS. Will add eventually to overweight when it becomes clearer that the deal will go thru. Can't believe how well positioned they will be. 60% Hulu. 20% of content watched on NFLX they can pull. More in thread
Dec 14, 2017 11:05:16 GMT -6
Under: Great posts on $DIS
Dec 13, 2017 17:50:49 GMT -6
Under: $ROKU Citron on a war path.
Nov 28, 2017 15:11:20 GMT -6
Under: $HAS takeover bid for $MAT?
Nov 10, 2017 16:16:07 GMT -6
martyc: Not looking like the market will provide any discounted opp for SGMO. Call was just too professional and all signs indicate they are on a great path for commercialization. Happy with core but wish I had some trading shs
Nov 10, 2017 9:04:05 GMT -6
martyc: For anyone looking to find an entry point into SGMO, I'm almost hoping is sells off in next few days so I can add more. They are really clicking but the fact they haven't signed new deals might cause some to exit. Watching as I have room for trading shs
Nov 9, 2017 18:28:09 GMT -6
martyc: Been an interesting ride so far. I figured the Bears would be about this good but hoped the O wouldn't look so lame. Another building yr but still possible to get to 8-8 IMO
Nov 9, 2017 18:26:08 GMT -6
Under: whats up with your Bears this year Marty?
Nov 9, 2017 17:35:25 GMT -6
martyc: Hope you were long ROKU. I wanted to see Q first so missed out
Nov 9, 2017 7:08:53 GMT -6