Q4 2014 NDA/BLA Round-up Part II: Can Sarepta Succeed with Only n=12?

For project 6 at PSIC, we shift our time horizon longer and decide to look at candidates which will have NDA/BLA filing due this quarter. This means the FDA decision will come 6-9 months later after our research.

For this round, we have 1 PDUFA around May and 14 NDA/BLA filing due Q42014. A round-up of these company and their drug candidates is summarized below:

Q4 2014 NDA/BLA Round-up Part II

Ticker $ Cap Drug Disease
HRTX 7.18 215.0M APF530 (Sustol) Acute and Delayed-onset chemotherapy-induced nausea and vomiting (CINV)
LGND 50.07 1.02B Melphalan IV (Captisol enabled) Pretreament of SC transplant for Multiple Myeloma
NKTR 12.4 1.69B BAX 855 Hemophilia A
OPK 8.25 3.57B CTAP101 (Rayaldee) Secondary hyperparathyroidism (SHPT) in chronic kidney disease (CKD)
RPRX 6.31 242.88M Androxal Secondary hypogonadism
SPPI 7.4 496.28M Melphalan IV (Captisol enabled) Pretreament of SC transplant for Multiple Myeloma
SRPT 15.57 638.4M Eteplirsen Duchenne muscular dystrophy

SRPT: eteplirsen

First-in-class: RNA-based therapeutics for Duchenne muscular dystrophy (DMD)

Mechanism: exon 51 skipping to produce functional dystrophin.

FDA concern: FDA Questions Dystrophin as a Biomarker Due to Failed Studies of Other Investigational Drugs for DMD; FDA Questions 6-Minute Walk Test Results for Eteplirsen, Suggesting Study Population Should Be Stable Over Two-Year Timeframe Due to Recent Natural History Data; FDA Requests Further Discussion on Endpoints, Design of Confirmatory Clinical Study

Art Krieg as CSO

Trials: PIIb (three years, n=12) -> NDA -> PIII (confirm).

Main competitor: Drisapersen of Prosensa (NASDAQ: RNA); PIII done “-/+”. [link]

Company history (was AntiVirals Inc.; then AVI BioPharma); current name in 2012 (with 1:6 reverse split)

Recent PO price: $25 in 2012, $38 in 2014
Update 10.27.2014 link: FDA demands more for NDA; NDA delayed to 1H2015
-34% drop as of writing

Melphalan IV (Captisol)

Disease: Multiple myeloma, (may expand to ovarian cancer)

Unmet: current formula use propylene glycol (PG), renal and cardiac side eff.

Drug: Melphalan, developed by GSK before 1982 (Alkeran), generic available

Company: SPPI (Spectrum) partner with LGND (Ligand; provides Captisol), Orphan drug

LGND: 12 drugs in market already, little revenue … (50M/y)
SPPI: 5 drugs in market, sales 170M/y

Probably will approve since both drug and formula are very mature

HRTX: APF530 (Sustol)

Disease: Acute and delayed-onset chemotherapy-induced nausea and vomiting (CINV)

CRLed: 03/2010; Resub 09/2012; CRLed: 03/2013

Stock: NASDAQ: APPA -> OTCBB in 03/2011 -> Rename -> NASDAQ: 01/2014

Drug: novelty: Biochronomer (polymer-based platform)
active ing.: granisetron (generic; serotonin 5-HT3 receptor antagonist)

Data: more side effects than Aloxi. Similar efficacy as Aloxi

NKTR: “longer-acting” rFVIII

Partner with Baxter NYSE:BAX (36B)

BAX 855: full-length longer-acting rFVIII for Hemophilia A (just like insulin for Diabetes)

  • utilize NKTR’s PEGylation and polymer conjugate technology platforms

NKTR: 1 product; 7 in P-III

OPK: Rayaldee Capsules

Secondary hyperparathyroidism (SHPT) in chronic kidney disease (CKD)

Rayaldee Capsules is a modified-release formulation of 25-hydroxyvitamin D3 (calcifediol) being developed as a new monotherapy to treat secondary hyperparathyroidism (SHPT) associated with vitamin D insufficiency in patients with Stage 3 through 4 chronic kidney disease (CKD).

to gradually restore serum total 25-hydroxyvitamin D concentrations to (or above) the currently accepted minimum adequate level of 30 ng/mL.

application as an adjunctive therapy to vitamin D hormone replacement therapy for SHPT in patients with Stage 5 CKD.

also as adjunctive therapy to bisphosphonate, selective estrogen receptor modulator (SERM) and anti-RANK ligand (RANKL) treatment for metabolic bone diseases, including osteoporosis and metastatic bone cancer.

RPRX: clomiphene (Androxal)

hypogonadism due to an impaired secretion of gonadotropins, including follicle-stimulating hormone (FSH) and luteinizing hormone (LH), by the pituitary gland in the brain, and in turn decreased gonadotropin levels and a resultant lack of sex steroid production.

Clomiphene is a selective estrogen receptor modulator (SERM) that has become the most widely prescribed drug for ovulation induction to reverse anovulation or oligoovulation.

We are developing Androxal® for men of reproductive age with low testosterone due to secondary hypogonadism, associated with obesity and we believe it is among the most common causes of low testosterone in men. It is estimated that 13 million men in the U.S. experience low levels of testosterone, and the condition is becoming recognized with more frequency.

Generic. Leading candidate. No product.