Most HALO Shorts are Blinded, but the Crucial PegpH20 study ISN'T!
Yes that's what they're telling us- but there is some delay between the end of the meeting, the minutes review, and the announcement of news, So I'm not expecting it by Easter.
Open-label study A clinical study in which the patients/subjects and investigators know which product each patient/subject is receiving, which is the opposite of a blinded study.
Segen's Medical Dictionary.
"To compare the treatment effect of PEGPH20 combined with nab-paclitaxel and gemcitabine (PAG) to nab-paclitaxel and gemcitabine (AG) in subjects with Stage IV pancreatic cancer.
The Phase 2 will study safety and treatment effect in 237 subjects (2:1 randomization, PAG:AG), preceded by two run-in phases (the first to assess safety and tolerability and a second to assess a new formulation of PEGHP20), 16 subjects total (randomized 3:1).
Metastatic Pancreatic Cancer
Drug: nab-paclitaxel + gemcitabine
Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label" ***
Yes the CEO, CFO, and new CMO have each alerted us to a scheduled FDA Mtg "by the end of Q1" for the purpose of discussing the benefit/risk of PegpH20, the use of PFS as an accelerated endpoint (instead of MOS) and the start of a Phase 3 Registration study to begin by end of '15 or start of '16.
You mean the Former CCO of ONYX- who was given responsibility for launching their most important Rx- Kyprolis- the drug which was the target of the takeover of ONYX by her former home Amgen? That CEO? Did you see Kramer sharing his personal memories of the takeover? She is a Biotech executive with a storied past- multiple launches at AMGN- doesn't throw around the term "Blockbuster" in Reuters without putting her name on the line. Oh, on that note- you know and I know, but I'm not sure everyone knows that 202 is an open label study- they know who is getting what and how long they go without progression or worse on a day to day basis. Considering the reputation and fortune of Torley, Ramsey, etc rides on this study- they know. Nice of HALO's CEO to share her experienced, considered opinion with the rest of us.
Great points- yes the Fast track means the FDA agrees that PegpH2- meets an unmet need- now HALO wants to take it to the next step by proving that Pegis better than what's avaiable- 9.2 mo is the # they're bringing, which CLEARLY is- Then they want to try for accelerated approval, which is why they state that they are attempting to get PFS established for the first time in this diagnosis as the primary endpoint. Proving PegpH20-Gem-Abraxane is better than the standard- Abrax-Gem (5.5mo) for an established clinical endpoint- PFS and you have the requirements for an accelerated approval. Clearly, there is more here than meets OUR eyes- 202 is an Open Label Study- Torley will be able to give the FDA another interim update. Since the meeting has been confirmed multiple times- as late as 3/2 Q4 2014 Earning call, this post halt data musty at least support the 9.2 mos. the meeting will probably take a day or two, but there's a delay until we hear- minutes review, etc- a good point made here in the past by another. This is getting closeer to what longs have been waiting for- the heavy tree shaking is a result.
Torley considering doing a deal for HALO owned PD-1 inhibitor - I hope that's an indication that she and Kirk are keeping it solo here for a while.
PFIZER using it to study Small cell ca and ovarian- not NSCLC or Gastric- HALO can deal for the rights by giving up royalties. Most other PD-1 inhibitiors are signed up- Medivation just signed Curetech's antibody. Helen said she might sign up her own- let's hope she can.
Think about it- HALO didn't bring in outside PR help- let alone hire them at VP level- for CRL or HALT- so this is an indication that Helen Torley expects that her dealings with the FDA so far will lead to PegpH20 approval on an expedited basis. Then she'll need to deal with all the CAR-T and anti PD-1 makers who want to address solid tumor and admit in the press (JUNO) that they need help in doing so.
"SAN DIEGO, Aug. 1, 2012 /PRNewswire/ -- Halozyme Therapeutics, Inc. (NASDAQ: HALO) today announced that the Center for Biologics Evaluation and Research (CBER) of the U.S. Food and Drug Administration (FDA) has issued a complete response letter (CRL) for Baxter's HyQ Biologics License Application (BLA). HyQ is an investigational product that includes plasma-derived Immune Globulin (IG) 10% and Halozyme's recombinant human hyaluronidase (rHuPH20) for subcutaneous administration in patients with primary immunodeficiency disease.
The letter requested additional preclinical data to support the BLA. The primary issues raised in the letter focused on non-neutralizing antibodies generated against recombinant human hyaluronidase and the possible effects of these antibodies on reproduction, development and fertility. Elevated anti-rHuPH20 antibody titers were detected in the registration trial, but have not been associated with any adverse events."
In clinical medicine, an imperfect test means any indication that a tumor has a near-high Hyaluronan count will be reason enough to try PegpH20 with the prescribed combination therapy- for pancreatic ca- with folfirinox (pending S1313) or with Gem-Abrax. If I had pancreatic cancer, I would find a pegpH20 trial and enter it. Yes, I'd like to know to the value of 0.000001 my Hyaluronan content, but if the assay off a bit but "acceptable" well that's what it is. The more HALO errs on the high side in the trials- saying only 40-50% are "High Hyaluronan", the better the separation of effect they will show. After which 63% or so can be treated.
Same reason? Curious thought- HyQ received a CRL on antipH20 antibodies- PegpH20 trial held on TE's. Cure for 1st- trial design and careful clinical observation= FDA Approved with a less onerous label than universally expected. Cure for 2nd= LMW Heparin- Trial resumed and ongoing for 9mos without another hitch. Wow, fear mongering is REALLY an INEXACT Science.
Once the world acknowledges what HALO's data at AACR and presented earlier in Analyst Meeting in Jan - shows-that PegpH20 enables Tcells, NK cells, and PD-1 inhibitors into solid tumors- we'll need more.
"Results: Analytical sensitivity studies identified an optimal probe dilution of 0.417 µg/mL on an immunostainer based on dynamic range in 4 human tumor xenografts containing differential levels of HA. The frequency of high HA observed was 62.7% of archival PDA (N=75). During analytical specificity, 5.1% of 75 PDA and 3 normal adjacent tissue samples had faint or focal staining after pre-digestion with recombinant PH20 hyaluronidase. No cross-reactivity was identified in 99 normal human tissues across a panel of histotypes; only 1 (1.0%) colon sample had staining of macrophages. Within-laboratory precision among three observers’ annotations had average %CVs as follows: between day 4.8%, between run 12.2%, repeatability 16.6% and total imprecision across serial sections 24.9%."
This puts the old "87%" value based on the old assay and "40-60%" estimate-based on the Study 202 interim findings- in perspective. Once PegpH20 is proven to add 67 or so percent to the APPROVED MPACT Study findings- Abrax-Gem- Analysts will estimate that around 2/3 of Pancreatic Ca patients will be eligible for Rx with PegpH20.
Did someone announce today that the 9.2 mo interim PFS was in question? nope.
Was there a 202 halt on some AE not covered by LMW Heparin? uh uh
Did HALO say their upcoming data to be presented at AACR15 in weeks Doesn't show that NK and T cells penetrate solid tumors better with PegpH20? Not a chance-
So Buy Away.
No- ATNM and BIIB watching today to keep my mind off the shenanigans of the option writers