Your Daily Pharma Scoop: KPTI Data, Allergan Write-Off, Celgene Down On Delay

Summary

• KPTI is up on strong data from MM study.
• Allergan writes off its entire Vitae acquisition.
• Celgene is down on ozanimod resubmission delay.

Analysis focus : KPTI

Karyopharm (NASDAQ:KPTI) slumped 17% in the pre-market yesterday, ahead of data from a mid stage study of selinexor in treatment-resistant multiple myeloma. A couple of years ago, this same drug was pitted against Darzalex in the same indication and showed similar efficacy. An FDA filing was planned in 2018, which seems to be on track. The sell-off was therefore probably profit taking ahead of expected good news.

That is just what has happened after the data was released. The stock was up 22% after news came out that selinexor showed treatment effect in the mid-stage study. The patient group was heavily pre-treated, meaning this was a very sick patient population, so the observed response rate of 25.4% was very good. That included two complete responses as well as 29 partial responses. Response mean duration was 4.4 months. This is comparable with CR in ASCT in MM. As has been shown before, CR is a very good indicator of OS in MM, so the spike on this data is justified.

KPTI will now submit an US NDA in the second quarter, and an EU MAA in 2019. According to published data:

MM is the third most common blood cancer in the U.S. (after lymphoma and leukemia). Men have a higher incidence than women, and African-Americans have more than twice the incidence and mortality rates of whites. In 2014, it was estimated that more than 24,000 people would be diagnosed with MM in the U.S. and that more than 11,000 would die from it.

MM is currently treated by immunomodulatory drugs like Darzalex, Revlimid, and Thalomid, with treatment costs ranging between the high $60 thousands to $100 thousands. There are also Proteasome Inhibitors like Farydak and Kyprolis, which cost almost half the immunomodulatory drugs, and there's also Farydak, a Histone Deacetylase Inhibitor and, of course, the long standard Doxil chemo regimen.

NB - KPTI came out with a dilutive offering after this was written.

Stocks in the News: Analysis of AGN, CELG

Allergan writes off lead candidate from $640M Vitae buy

Discussion: Allergan (AGN) purchased Vitae Pharma in September 2016 for $640mn, a 160% preimium,, and by November, promptly lost its first of two product candidates VTP-38543, which failed to demonstrate a treatment benefit in a Phase 2 study in atopic dermatitis. VTP-43742, which was in mid-stage development in psoriasis at that time, has now also failed its trial due to safety concerns. With that, Allergan has now written off the entire $640mn deal and recorded a $535mn impairment charges. One wonders: who does their DD before these buyouts?

Celgene down on extended timeline for ozanimod resubmission in the U.S.

Discussion: According to Morgan Stanley, Celgene (CELG) might take over a year to resubmit its US marketing application for ozanimod, a drug for multiple sclerosis. The molecule is an oral, once-daily selective sphingosine 1-phosphate 1 and 5 receptor modulator whose MS sales forecast is as much as $6bn. The FDA sent a refusal to file letter to Celgene in February probably due to incomplete pharmacology data. The stock has been depressed since that time. However, Evercone analysts think that the selloff is an overreaction and that the company will be able to refile by this year, and the two-year animal study could be done post approval.

In Other News

Edge Therapeutics (EDGE) is looking at strategic alternative including an outright sale, and it is also planning to downsize operations to extend cash runaway after the company bailed on late stage study of lead candidate EG-1962.

Solid Bioscience (SLDB) received a letter from the FDA which may resolve the clinical hold of its early stage gene therapy study of SGT-001 in DMD patients. The study was suspended after the first participant experienced a severe reaction requiring hospitalization. A partial hold for a manufacturing issue has also been resolved.

Capricor Therapeutics (CAPR) has started a Phase 2 clinical trial, HOPE-2, assessing CAP-1002 in boys and young men with Duchenne muscular dystrophy (DMD). The drug candidate is an allogenic cardiac cell therapy derived from donor heart tissue.

Ultragenyx (RARE) will launch its recently approved drug Crysvita in the U.S. The drug was approved last month for the treatment of X-linked hypophosphatemia in patients at least one year old.

Conatus (CNAT) has completed enrollment of a midstage study of emricasan in NASH cirrhosis. Topline data will be available in late 2018. Two other studies in NASH fibrosis and decompensated NASH cirrhosis are under way, and data is expected in 2019.

Pluristem (PSTI) is up after the FDA cleared the IND for PLX-R18 for acute radiation exposure. The drug will take the animal rule pathway, and may not require human trials at all.

Keryx Pharma (KERX) CEO Greg Madison departure leads to a sell-off despite news that Q1 Auryxia sales will be up to $21mn. KERX has been seeing depressed stock movements on revenue misses and guidance withdrawals from the November 2017 quarter, despite approval of expanded use of Auryxia.

Ablynx (ABLYF) completed enrollment in a mid-stage study of ALX-0171 in respiratory syncytial virus (RSV) infection. Data from this 180 patient study will be available in the second half of 2018.

Aerie Pharma (AERI) has launched its recently approved drug Rhopressa in the U.S. The drug is used for lowering intraocular pressure in patients with glaucoma and ocular hypertension.

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Analyst’s Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.