Drugs to watch at ASCO 2013

Below is a recap of interesting data expected to be presented at the Annual meeting of The American Society of Clinical Oncology (ASCO).  This year’s meeting will have a strong focus on immunotherapy (PD-1 antibodies in particular) as well as novel oral agents for hematology. Companies for which important data are expected are reviewed as well.

PD-1 takes center stage

PD-1 antibodies are by far the hottest and most active field in cancer drug development. Although technically, all data to date were from phase I studies, there is wall to wall consensus that inhibitors of the PD-1 pathway will become a cornerstone in several cancer types and a $5-$10B franchise.

The undisputed leader is BMS’ (BMY) nivolumab, which is in 6 pivotal trials. The two closest competitors are Merck (MRK) and Roche, who are in phase II. The BMS and Merck antibodies bind PD-1 whereas Roche’s antibody binds PD-1 ligand (PD-L1). All three agents already generated exciting data and the main question is whether these agents are interchangeable or not (i.e do they have the same clinical profile?).

Data at ASCO could start to answer this question although it is unlikely to see unequivocal proof until late stage results are available. Given BMS’ clear early mover advantage, Roche and Merck are trying to differentiate their programs as early as possible. Focus is expected to be on cross-trial comparison per indication, activity spectrum in new indications, safety profile and biomarkers for patient selection.

PD-1 status update

PD-1 Data highlights

Combination of nivolumab and Yervoy (anti- CTLA-4, also from BMS) in melanoma – Response rate of 47%-50% at relevant doses appears numerically higher than that documented with either drugs alone but the sample size is small. BMS will start phase III trial for the combination later this year, which indicates updated results will be positive as well. BMS is the only one with an approved CTLA-4 antibody on the market, which could help it to corner the market, at least in melanoma.

Update from nivolumab’s phase I trial in melanoma, NSCLC and RCC – Although technically designated phase I, this was a huge study of over 250 patients. Response rates continue to look strong. As with Yervoy, there appear to be patients who achieve long term remissions that could eventually be seen as cure.

Median survival for melanoma, NSCLC and RCC respectively were 16.8, 9.6 and >22 (not reached) months respectively.  These values appear substantially better than historical data, although it is hard to interpret survival data from single arm trials. Initial results in PD-L1-positive melanoma patients also look promising with a dramatic difference in overall survival between PD-L1 positive (21.1 months) and PD-L1-negative (12.5 months).

Update from MPDL3280A’s phase I – The abstract discloses good activity (21% response rate) across various tumor types. Activity was seen in the 3 “usual suspects” (melanoma, NSCLC, RCC), generally in line with nivolumab’s early data but surprisingly, activity was seen in tumor types where nivolumab was not effective (colon cancer) or has not been evaluated (gastric, head and neck). This potentially unique activity spectrum could be an important differentiator for Roche, however, results are still very preliminary.

Another interesting point is that BMS also had a PD-L1 antibody (MDX-1105), which demonstrated lower response rates than nivolumab and MPDL3280A. The basis for this discrepancy is unclear although it appears that patients in the MPDL3280A trial were less heavily-pretreated compared to BMS’ trials. There is obviously the possibility that MPDL3280A is simply a better antibody.

One potential advantage of targeting PD-L1 is lower toxicity. MPDL3280A does not inhibit PD-L2 binding to PD-1, which may be associated with some of the pneumonitis (lung inflammation) seen with PD-1 antibodies (fatal is some cases). Indeed, to date no severe pneumonitis was reported with MPDL3280A, although follow up is still limited.

Abstracts also show clear correlation between PD-L1 expression and response across tumor types (39% of PD-L1–positive had a response vs. 13% for PD-L1–negative patients). This was more pronounced in the NSCLC cohort, where all 4 patients with PD-L1-positive tumors responded compared to 15% of PD-L1–negative patients. Needless to say, numbers are still small.

PD-L1-positive NSCLC will probably be Roche’s first indication based a single arm phase II trial expected to open this month. >25% response rate in this molecularly defined subpopulation should be enough for accelerated approval. BMS is also pursuing accelerated approval in NSCLC, but they focus on squamous histology rather than PD-L1 expression.

Update from Merck’s lambrolizumab (MK-3475) in melanoma– Data available only in melanoma patients, where response rate (>35%) is similar to slightly better than other PD-1 inhibitors. The abstract can be viewed as disappointing, as a previous update reported a response rate of 51%. This might be due to the fact that the prior update included also unconfirmed responses.

Merck is pursuing lambrolizumab in a large randomized phase II trial in pre-trated melanoma patients (Including Yervoy failures).

PD-1 table

Seattle Genetics – More ADC data from Roche

Seattle Genetics’ (SGEN) investors should track 2 data sets from Roche’s (Genentech) antibody-drug conjugate pipeline. As a reminder, Roche has 8 ADCs in clinical development, all of which utilize Seattle Genetics’ technology. As more data for these programs becomes available, the market is starting to realize the tremendous value these 8 programs represent. Today it is clear Seattle Genetics is much more than Adcetris but an ADC powerhouse with substantial economic interest in multiple partnered programs.

Until now, Roche unveiled positive phase I results for 3 ADCs targeting CD22, CD79b and MUC16 (Discussed here). In all 3 cases, a good safety profile and robust efficacy signals were noted. At ASCO, 2 additional ADCs will have initial results that appear positive based on the abstracts. This brings the number of partnered programs to which investors can ascribe value to 5.

SGEN- Genentech's pipeline

DNIB0600A (anti- NaPi2b) is in phase I for NSCLC and ovarian cancer. The abstract discloses 3 confirmed responses (still ongoing) and 1 unconfirmed response among 18 patients who received higher doses.

DSTP3086S (anti-STEAP1) is in phase I for prostate cancer. The abstract discloses a relatively modest activity profile but there were encouraging signs of CTC (circulating tumor cells) reductions that seem dose dependent.

Seattle Genetics’ agreements with Genentech entail a total of $1.4B in milestones and mid -single digit royalties per program. If only one or two of Genentech’s programs reach late stage testing, it should have a material impact on Seattle Genetics’ valuation (Best example is Immunogen’s mid single digit royalty stake in Kadcyla).

Additional partnered ADC programs to be presented at ASCO are Progenics’ (PGNX) anti-PSMA for prostate cancer and Genmab’s anti-TF (preclinical data).

Immunogen – First look at IMGN853

Immunogen (IMGN) will present phase I results for IMGN853 (anti-folate receptor), which is emerging as the company’s most important internal program. The company is going into ASCO with a positive sentiment towards IMGN853, which still needs to be corroborated by actual data.

There are high expectations around IMGN853 based on positive results from Endocyte’s (ECYT) Vintafolide (also targets folate receptor) in ovarian cancer. Vintafolide is a small-molecule drug conjugate and has inferior pharmacologic properties compared to ADCs, so IMGN853 may be more potent. Vintafolide is no active as a single agent, so if IMGN853 generates responses, it should be viewed as a superior competitor.

At ASCO, investors will look for preliminary signs of activity in the form of tumor shrinkage and CA125 reduction. The abstract has very limited data on efficacy but 1 case of an 82% reduction in CA125 in a patient receiving a high dose (3.3 mg/kg) is mentioned. The fact the drug could be escalated to 5 mg/kg is encouraging as these are levels where activity is typically observed with ADCs.

Exelixis – Promising activity in RET mutated tumors

Exelixis (EXEL) will have several clinical updates for cabozantinib (Cometriq) in different indications. Although none are expected to have a major impact on the stock, they could provide some read-across for the ongoing phase III in prostate cancer as well as insight on activity in RET-mutated tumors.

Survival data from prostate cancer phase II – Updated results from a single-arm phase II in prostate cancer will be reported. By definition, analyzing results from non-comparative trials is problematic but the trial was fairly large (144 patients).  As background, this was the trial that demonstrated the drug’s controversial bone scan normalization effect (reviewed here).

The abstract reports a median survival of 10.8 months, which is encouraging given the tough patient population (73% were ≥3rd line, many were taxane resistant/refractory). The most important read across is for the ongoing phase III trial, as investors will try to put cabozantinib’s survival in context with Zytiga’s and Xtandi’s post-chemo survival (with the caveats of cross trial comparisons).

Since patients in cabozantinib’s phase III are Zytiga and/or Xtandi failures, cabozantinib’s phase II data can be compared to the post-treatment survival in the phase III studies for both agents (8.5-10 months). At ASCO, survival for the Zytiga pre-treated subgroup will be particularly relevant as this subgroup more closely resembles the population being enrolled in the Phase 3 trial.

At first glance, cabo’s 10.8 months does not look impressive but it appears cabozantinib phase II had a much tougher patient population based on prior therapies and resistance to taxanes (Taxotere or Jevtana). In particular, all patients had progression within 6 months of last taxane dose and more than a third of patients progressed within 1 month after last taxane dose (highly aggressive tumors).

RET-mutated NSCLC as a new niche indication – Newly identified RET mutation (1-2% of NSCLC) represents a new niche indication for cabozantinib, which is also a RET inhibitor. At ASCO, a group from Memorial Sloan Kettering will report updated clinical experience with cabozantinib in this rare subset. Initial findings from this trial were recently published and showed promising activity in 3 patients with RET mutation, who appear to derive benefit from cabo (2 partial responses, 1 durable SD, see figure from the article below).

Cabo - RET

Source : Drilon A. Cancer Discov. 2013 May 7. [Epub ahead of print]

RET-mutated NSCLC represents a new opportunity for cabozantinib, currently not in anyone’s models. Assuming updated results are positive, Exelixis could pursue accelerated approval based on a small single-arm phase II trial. Since the drug is already approved for medullary thyroid cancer (MTC), physicians might use it off label in lung cancer. Challenges related to this program are the need for a validated diagnostic test for patient selection (Required by the FDA) and potential competition from other RET inhibitors, especially Ariad’s (ARIA) Iclusig which is also a highly potent RET inhibitor.

Subset analysis in RET mutated MTC – Subset analysis from cabo’s phase III trial in MTC shows dramatically better activity in RET mutated patients compared to non-mutated patients (PFS of 60 vs. 25 weeks). The drug is approved in MTC regardless of RET status, but this type of analysis could make physicians more inclined to use it in mutated patients. It could become even more important once overall survival data matures since the prior analysis found no benefit in the entire patient population. If the strong PFS signal in RET mutated patients is translated to a survival difference, it should bode well for Exelixis’ marketing efforts even if this does not go into the label.

Infinity –competition intensifies 

Infinity (INFI) lost half of its market cap in less than 2 months (it is still over $1B), which is largely attributed to concerns about competition in CLL and NHL. As background, Infinity’s  IPI-145 belongs to a new class of oral agents that are about to revolutionize the way many blood cancers are treated. The 3 most advanced are ibritunib (Btk inhibitor, Pharmacyclics/ J&J), idelalisib (PI3K-delta inhibitor, Gilead) and ABT-199 (Bcl-2 inhibitor, Roche/Abbvie). IPI-145, a PI3K gamma/delta inhibitor, is fourth in line, slightly behind ABT-199 and 1-2 years behind ibrutinib.

ASCO will have updates for IPI-145 (oral presentation for NHL) and competing drugs in CLL and lymphoma. While Infinity’s abstracts did not include new data, the competing molecules had positive updates which curbed investors’ enthusiasm regarding IPI-145.

Phacyclics’ (PCYC) ibrutinib – Continues to be the leader with “jaw dropping” results in CLL, multiple breakthrough designations and a very clean safety profile. Activity in CLL include objective responses in 67-81% of a very long progression free survival across different subsets and treatment lines. IPI-145 has a different, perhaps complimentary mode of action, but some claim it will be hard to gain market share or even recruit CLL patients for clinical trials once ibrutinib is on the market next year. A potential differentiator for IPI-145 could be activity in patients who progress on ibrutinib, which may be presented at ASCO.

Gilead’s (GILD) idelalisib – This is Infinity’s most direct competitor (target the same pathway) which is further along in development (multiple phase IIIs ongoing). Idelalisib is clearly an active drug but it is not as potent as ibrutinib and also appear to have some safety issues (potential liver toxicity). Until recently, IPI-145 appeared to be more effective, at least in CLL (55% response rate vs. 24% for idelalisib) and safer. However, updated results in idelalisib’s abstract show that response rate improved with time and it now stands at 56% (using a less strict criteria). This was the main trigger for the diminishing excitement around IPI-145, which derives most of it value as a better next-gen idelasilib.

Roche’s/Abbvie’s (ABBV) ABT-199 – This drug started to gain the market’s attention only recently, and initially was not perceived as a threat to IPI-145 due to unclear efficacy and a problematic safety profile (deaths due to tumor lysis syndrome  [TLS]). 2 ASCO abstracts show robust efficacy in CLL (85% response rate) and NHL (55%) and a surprisingly good safety profile. It also appears that investigators were able to find a regimen that minimizes TLS. Phase III trials are expected to begin shortly.

Where does it leave Infinity?– Although the market is more competitive than expected, IPI-145 is still promising and Infinity could find multiple differentiation routes. Comparison with idelalisib should be done after ASCO, where results will include higher doses and longer follow up, both expected to improve activity over time. IPI-145 may also be relevant in indications that are irrelevant to its competitors such as T cell and Hodgkin’s lymphoma. Additional trials in inflammation are ongoing (results expected next year), to which the market doesn’t ascribe real value.  Lastly, assuming available preliminary results are corroborated in later trials, IPI-145 has a very mild safety profile, which could be an advantage over idelalisib and ABT-199 (but not ibrutinib which looks extremely safe).

Synta – Updated survival data in NSCLC

Synta (SNTA) will present updated results from the GALAXY1 trial, a large randomized phase II evaluating its Hsp90 inhibitor (ganetespib) in NSCLC. Results have important implications for an ongoing phase III trial that was designed based on initial phase II data.

Preliminary results from GALAXY1 (which I discussed here) demonstrated a survival trend, especially in patients with a 6-month follow up. While the previous analysis included only 77 patients, the upcoming analysis should include all or most of the 250 adeno patients enrolled in the trial.

The past month provided some mixed signals about the data. Earlier this month Synta announced the departure of Dr. Sumant Ramachandra, President of R&D for “personal reasons”. This raised concerns in the market as some viewed it as a red flag, several weeks before ASCO. On the other hand, the presentation was chosen by ASCO as a Clinical Review (CRA) presentation, which means results will be released only on the day of presentation. This is typically viewed as a positive indication although it is unclear how much data the organizing committee had when it made its decision (Synta disclosed that an OS analysis is expected sometime prior to ASCO). Another positive sign was the initiation of the phase III trial, implying the survival trend was not completely lost.

At ASCO, investors will evaluate the OS curves for the entire patient population as well as patients with indolent disease (6 month from diagnosis). The latter subgroup was chosen by Synta as the phase III population due to a robust efficacy signal. Given the nervousness around Synta, any attenuation of the survival signal could have animpact on the stock.

Additional presentations include results from 2 investigator-sponsored trials for ganetespib in esophagogastric and prostate cancer, respectively. Both studies had disappointing results but there was a single case of a dramatic complete response which is still ongoing after 27.5 months. Interestingly, the patient harbored a KRAS mutation, a known client protein of Hsp90. KRAS mutations are rare in gastric cancer (0-10%), and  this case demonstrates again that Hsp90 inhibition might be very effective in niche indications (such as ALK and RAF mutated NSCLC).

Roche – Protecting the CD20 franchise

Roche will report phase III results for obinutuzumab (GA101), an anti-CD20 antibody viewed as Rituxan’s successor. Rituxan (also an anti-CD20 antibody) is one of Roche’s top selling drugs (over $6.5B in sales) and several companies intend to launch biosimilar versions of the drug. Ideally, Roche should replace Rituxan with GA101 in as many settings as possible in order to remain the dominant CD20 player in hematology.

At ASCO, investigators will present phase III results from 2 separate trials evaluating GA101 or Rituxan, respectively, when added to chemotherapy in CLL. Both agents (GA101 and Rituxan) led to superior PFS over chemotherapy alone with GA101 demonstrating a numerically superior PFS (23 vs. 15.7 months). Although this result is not a direct proof for GA101’s superiority over Rituxan, it is a good indication for that.

Roche will also have results from 2 ADCs (discussed above for Seattle Genetics) and ABT-199 (co developed with Abbvie, discussed above as competition for Infinity))

Additional companies to watch

Array Astrazeneca’s (AZN) selumetinib, a MEK inhibitor licensed from Array (ARRY), will have phase II data in uveal melanoma. There are no details as results are embargoed and will be released only at the day of presentation. This implies results are positive to some extent and may result in another phase III for selumetinib.

Array’s second MEK inhibitor, MEK162, (developed by Novartis [NVS]) will have results from a combination trial with Novartis’ BRAF inhibitor. The two drugs appear combinable at their single agent doses and preliminary signs of activity appear encouraging.

Curis Phase I results for the IAP inhibitor, CUDC-427 (GDC-0917), will be presented. Curis (CRIS), which licensed this drug from Genentech earlier this year, is facing a lot of skepticism due to Genentech’s decision to externalize the program. As promised by Curis’ management, the drug showed initial activity signs including 2 complete responses (ovarian cancer and MALT lymphoma) of 42 patients. Safety profile remains an issue due to a fair amount of toxicity, which could be an issue especially for combination regimens.

Genmab (GEN.CO) – The abstract for daratumumab (anti-CD38) does not reveal a lot of new data from the phase I in multiple myeloma. The antibody, which was recently granted breakthrough designation, led to 5 PRs and 3 minor responses in 12 patients at the higher doses. Authors rightfully concluded: “This is unprecedented for single-agent mAb treatment of MM.”

IncyteInvestigators evaluated Jakafi’s effect on bone marrow fibrosis in patients who participated in the drug’s phase I/II trial. After 2 years, improvement or stabilization of fibrosis was more common in patients treated with Jakafi compared to patients who were treated with hydroxyurea. This is a small and retrospective trial, but bodes well for Incyte’s (INCY) effort to convince the market Jakafi is not just a symptomatic drug but a potential disease modifier.

ArquleArqule (ARQL) will present phase II results for its MET inhibitor ,tivantinib, in colon cancer. Although the trial did not lead to a meaningful PFS improvement in the overall population, an encouraging trend in survival emerged (HR = 0.67). More important is subset analysis based on MET status. If positive, it will be the 3rd time tivantinib demonstrates better results in MET high tumors. The company already disclosed that retrospective analysis from the failed lung cancer study also generated a survival difference in MET-high patients. This should be seen as a positive read-through for the ongoing phase III in liver cancer.

Portfolio updates

We are adding a new position in Infinity in anticipation of strong results at ASCO and a second position in ArQule based on enhanced confidence in the liver cancer trial and early data with ARQ092 (Discussed here).

We are selling our AVEO (AVEO) positions following the negative ODAC panel and Astellas decision not to pursue RCC. We are also selling one position in Celldex (CLDX) for a profit of 128.5%.

Portfolio holdings – May 26th 2013

biotech portfolio - May 26th 2013 - after changes Biotech ETFs - May 26th 2013

138 thoughts on “Drugs to watch at ASCO 2013

    • I think IPI145 will eventually be approved with multiple marketopportunities. Worst case scenario is 300 mil if it becomes a niche product so i like risk/reward here.
      Re CLDX i still like itbut the risk is there and our position got substantial.



      • That’s a good question. The low hanging fruits (relapsed/refractory CLL, iNHL, MCL) may be more challenging.
        Worst case they can do a randomized p3 and enroll as many pts before ibrutinib and idelalisib are available.
        Perhaps the best way would be head to head vs. idelalisib


  1. Note we won’t see OS for the entire SNTA trial population – they of course still pretend that the non-adenocarcinoma patients that were being harmed don’t count.


  2. Hi Ohad,

    I feel AVEO is cheap now mkt cap wise wrt to its pipeline and tivo for other indications.
    I am actually adding instead of selling my AVEO position. Any thoughts on STML? Thanks


    • Tivo was in an ideal position in RCC due to its at least as good as efficacy and superior safety profile. Things are very different in breast (avastin failed to prolong OS) and colon ( avastin approved with good safety profile).
      The HER3 program could become valuable if MACK has positive data but it is still early.

      STML had an impressive fundraising , will take a look at them again.



      • I am thinking MACK lead drug (encapsulated irinotecan) fails its pivotal later this year. They said in recent CC they are conducting phase 1 to better define subgroup responses I believe. Makes me think all-comer P3 unlikely to succeed.


      • I think MACK is unreasonably expensive. Its p3 program in pancreatic cancer is unlikely to succeed imo and MM-111 and MM-121 haven’t generated any positive efficacy signals. In general HER3 is being pursued by many companies but so far without any success (i admit it is still early though for a final verdict)



    • Hey Mike
      I took a look at STML… looks very interesting with two pivotal trials about to start. It looks like they know what they are doing. MArket cap is also very low IMHO. They are presenting at ASCO. Wil see what that brings. Have you bought shares?
      Thanks for the tip.


  3. Ohad, do you have any concerns for ARQL given that one of the indications PD1s seem to be pursuing is HCC? I assume not because ARQL is so much further ahead and MET is entirely different MoA that, in theory, could presumably be paired with PD1 but just throwing out for discussion.

    I am wondering if, in general, I should have any concern for some of my other holdings that are competing in spaces that PD1s are pursuing. But, I assume I shouldn’t be too worried where there is the angle of agents either much further ahead and/or potential to be used in combo with PD1. Also, it sounds like PD1 is primarily limited (at least so far) to solid tumors; sounds like companies competing in hem onc space may not have to worry about future competition from PD1.


    • With respect ARQL, we still don’t know how effective pd1 agents are, so hard to say. Anyway, i agree about tivantinib having a different potentially complementary moa.

      Hem onc is already pretty crowded even without PD1.



      • Thanks Ohad. I think I mixed up HCC with RCC. Not clear if PD1s pursuing HCC.

        Yes hem onc in general getting much more crowded but still think there are niches where drugs with competitive advantages can compete.


  4. Ohad,

    How do you see ARRY positioning 520? In combo with Carfilzomib? With dexamethasone? ARRY doesn’t seem to have the resources to take both 520 and 614 forward even with ROW partnerships, so what you expect ARRY to do with 520? Is a partnership with ONXX likely or even plausible?

    Does IMGN’s folate receptor drug pose problems down the road for ECYT’s drugs? Do you expect a positive EMA decision for vintafolide?


    • If response rate with dex exceeds 20% ARRY may pursue accelerated approval. If not they can either bet on enriching the population with their biomarker. In case they’ll pursue approval in combination with kyprolis they will have to partner the drug imo.
      Anybody with a myeloma franchise is relevant, ONXX, Takeda or JNJ come to mind.

      Re IMGN vs. ECYT it is too early to tell but on paper IMGN853 should be more effective.



      • I have huge profits in ECYT. I notice you don’t have them in your portfolio. Is there something you don’t like about the platform? The idea of small molecule antibody conjugates certainly sounds intriguing. It seems like an idea that could take off. MRK is testing vintafolide in other cancer indications besides ovarian. And the tubulysin drug also looks interesting. That one is proprietary to ECYT. With a low float, there is certainly a lot of leverage. How would you handicap the EMA decision with vintafolide in ovarian?


      • My initial concern is the drug’s short exposure (half life < 30 mins) and the fact there was no activity as single agent. On top of that the contradicting OS signal is something to consider.
        Hard to predict EMA decision, my guess, which is as good as anyone else's, is that they won't approve it.



  5. Ohad,

    Here’s a reply from a knowledgeable board poster that I know. How would you respond?


    No mention of responses (or even lack of responses) at all – just something that sounds more like from a preclinical study:

    Quote:In this heavily pretreated population, ASLAN001 led to a downregulation of signalling pathways responsible
    for cell proliferation, and a reduction in cell survival and cell proliferation.

    How did they know there was a reduction in cell survival and proliferation?


    • Correct, they didn’t say anything about responses only about proof of target inhibition and some effect on cell viability. They did it by looking at pre and post treatment biopsies.
      What’s important for ARRY is that they are starting a randomized p2 which will provide a more definitive answer- a free call option.



    • Actually, I thought initial signs with the TROP2 ADC were very encouraging. Quite surprising to see responses already at the 2 lowest doses, let alone with an ADC based on SN38 (irinotecan). Definitely something to follow up on (didn’t see anything on that program at ASCO 2013)



      • IMMU’s prior Phase 2 lupus data was mixed, at best. Hard to have confidence in the Phase 3 based on prior Phase 2 data IMHO. Stranger things have happened I suppose.


      • My impression with the p2b lupus trial was more positive despite the bell shaped efficacy curve. Regardless, this is for epratuzumab, a different program than the one i was talking about.



  6. Ohad:
    Have you taken a look at Epizyme / EPZM, ipo’d last night, up 40% today. Has strong very VC backing, appears to have strong Pharma collaborations (CELG, ROCHE, GSK, Eisai, ABT,) I think GSK may have taken $11mm of the ipo according to the “red”. Post ipo, balance sheet appears strong. Any thoughts on the technology???
    Thanks and appreciate all your quality work.


  7. Hey
    Have you looked at Biod? It has been on a solid run in the past week, up from $3.10 to $4.60 today… It seems that investors believe management is finally executing. Market cap is still only $60M compared to $2B for MNKD.

    Also, CLDX up in after hours after Cramer plugs it. Maybe going higher from here?


  8. There was an interesting article posted on Seeking Alpha about a week ago. It detailed PD-1 as being the second choice and PPHM’s Bavituximab (PPHM is presenting at ASCO) as being superior in all ways. I would be very interested and extremely appreciative to hear your input on that specific drug’s development and if there is promise.




      • Ohad,

        I have to respectfully disagree. The clinical reality is that “Bavituximab” has proven safe and effective on over 600 patients based on a broad range of trials including: Lung, Breast, Pancreatic, Liver. Trust me, if you look deep enough into Fargo, ND and CSM and the “many” affiliations up and down the line …. Peregrine has been keeping “quiet” because they were told to “keep quiet” and not by shareholders. Federal Investigations have been on-going and do you find it strange that a Phase III was FDA approved before ASCO 2013 for Bavituximab’s Phase IIb NSCLC that was found to be “in jeopardy” due to Fargo, ND?

        I have nothing against PD-1 or any drug that can help cancer… but when there is a clear, constructive, well thought out plan that almost worked until Peregrine salvaged the data and found some interesting details.., some have lost control of the situation and many that have continued the story have no idea how far up the Federal Investigations go.

        You just may have stumbled upon the PD-1 story but you’d have the blog of a lifetime if you traced the story back to its roots.

        Good Luck!


      • Thank you. Given Fink’s comments below that PPHM may have been under wraps,…and now have a p3 approval,….is more to the story to come this weekend or does the clinical side already tell the story?


    • Rex,

      I happened to have read the article and that is probably one of the most “scientific” explanations in how Bavituximab works. Peregrine Pharmaceuticals seemingly has something under their sleeve because they are presenting at ASCO 2013 on a previously presented Phase IIb NSCLC from ASCO 2012 that was found to have “errors” made by a 3rd party. If you look into the 3rd party, Clinical Services Management out in Fargo, ND.. its CEO is affiliated with Bristol Myers Squibb and quite interesting to say the least.

      Since that time… Peregrine could not comment, communicate to shareholders or relay any information at all due to FDA investigations.. though all has ended with Phase III approval. More interesting is the Phase III approval comes before the data is presented at ASCO 2013? It all sounds like only a few inside Peregrine and the FDA knows the direction of the Bavi pipeline. Next week… their shareholders finally get a glimpse of whats being kept under wraps this entire 2012 of the Bavi pipeline.

      Good luck!


  9. Hey Ohad
    what do you make of the ARRAY results in uveal melanoma…. what is the take away, in terms of other applications of the drug? Thanks!


      • If I had to guess we’re talking about ~1000 cases in the US with metastatic disease. Despite teh confounding effect of the crossover in the selumetinib trial, it looks likely one of ARRY’s MEK will get to pivotal testing in uveal melanoma.



  10. Ohad
    interesting article about the top 10 experimental cancer drugs in 2013
    very positive about: PCYC ibrutinib (3 breakthrough designations); BMY nivolumab (clear leader in PD-1 space); Genmab – daratumumab; GILD idelalisib,and more. The last in the list is Bind Therapeutics – BIND-014. Unfortunately they are private company.Do you expect for them to have an IPO?
    Do you consider adding PCYC to the portfolio and what would be a good entry?


  11. For SGEN investors, Ohad suggested looking also at the results from PGNX Phase 1 for prostate using SGEN’s ADC. I am an SGEN investor, so I did the work, and the results follow. The outcome appears generally positive, and certainly not negative. Also, PGNX is moving forward with a phase 2 study. I’m buying more SGEN and maybe some PGNX. See specifics below.

    For the phase 1 study, N=52. Abstract is at http://meetinglibrary.asco.org/content/112557-132.

    PGNX also put in an abstract (not presented) for measuring changes in PAS during dosing of the 52 , and found a correlation showing PSA expression decreasing during dosing. See http://meetinglibrary.asco.org/content/112918-132. That helps to explain the results explained in the phase 1 abstract, which is below.

    PGNX also put in an abstract (not presented) for its in vitro study of combining its ADC with other PMSA drugs such as XTANDI. Results are positive, but again that’s in a dish. Abstract is: http://meetinglibrary.asco.org/content/114803-132 (Key quote is ” Results: Enzalutamide (1 mM) increased PSMA expression by approximately 2.5-fold in LNCaP (androgen-dependent) and C4-2 (androgen-independent) cells, with maximal expression observed after approximately 4 weeks’ culture. PSMA expression returned to basal levels within days following removal of enzalutamide from the culture. Enzalutamide and PSMA ADC exhibited synergistic antitumor activity in LNCaP and C4-2 cells (P < 0.05). Similar results were observed for abiraterone in C4-2 cells. Less induction of PSMA expression by antiandrogens and modest effects on cytotoxicity were observed using 22Rv1 cells, an androgen-independent cell line with low basal expression of PSMA. Conclusions: Enzalutamide and abiraterone significantly and reversibly augmented PSMA expression and potentiated the activity of PSMA ADC in PCa cell lines in vitro. The findings support clinical exploration of regimens that combine potent antiandrogens and PSMA-targeted therapies.')

    Key Quote from abstract on Phase 1 – "Antitumor activity was manifested as reductions either in PSA or in CTCs in approximately 50% of patients at ≥ 1.8 mg/kg PSMA ADC. Exposure to PSMA ADC increased with dose and was ~1,000-fold greater than MMAE exposure.

    Full abstract from phase 1:
    Background: The abundant expression of prostate specific membrane antigen (PSMA) on prostate cancer cells provides a rationale for antibody therapy. PSMA ADC is a fully human antibody to PSMA linked to the microtubule disrupting agent monomethyl auristatin E (MMAE). It binds PSMA and is internalized within the cancer cell where cleavage by lysosomal enzymes release free MMAE, causing cell cycle arrest and apoptosis. A phase 1 dose escalation study of PSMA ADC in taxane-refractory mCRPC has been completed. Methods: Patients with progressive mCRPC following taxane-containing chemotherapy and ECOG status of 0 or 1 were eligible. PSMA ADC was administered by IV infusion Q3W for up to 4 cycles. Safety, pharmacokinetics, PSA, circulating tumor cells (CTC), immunogenicity and clinical progression were assessed. Serum PSMA ADC and total anti-PSMA ADC antibodies were measured by ELISA, and free MMAE was measured by LC/MS/MS.The dosing cohorts ranged from 0.4 mg/kg to 2.8 mg/kg. Subjects who benefitted from PSMA ADC were eligible for treatment in an extension study. Results: 52 subjects were dosed in 9 dose levels. All subjects received prior docetaxel, 6 also received cabazitaxel and 3 subjects also received paclitaxel. PSMA ADC was generally well tolerated with the most commonly seen adverse events being anorexia and fatigue. 16 patients reported peripheral neuropathies, including 3 with grade 3. Dose limiting toxicities (DLT) seen at 2.8 mg/kg were neutropenia (one death) and reversible elevations in liver function tests (LFTs). Antitumor activity was manifested as reductions either in PSA or in CTCs in approximately 50% of patients at ≥ 1.8 mg/kg PSMA ADC. Exposure to PSMA ADC increased with dose and was ~1,000-fold greater than MMAE exposure. There was no accumulation. Conclusion: PSMA ADC in this study was generally well tolerated in subjects with progressive mCRPC, previously treated with taxane. Antitumor activity was seen at doses ≥ 1.8 mg/kg. DLTs were neutropenia and reversible LFT abnormalities. The maximum tolerated dose was determined to be 2.5 mg/kg. A phase 2 trial of PSMA ADC in taxane refractory mCRPC has been initiated at 2.5 mg/kg. Clinical trial information: NCT01414283.


    • How can you make heads or tails of the PGNX data? There’s nothing related to traditional endpoints like partial responses, ORR, stable disease, or OS. Just very hard to know if they have anything at all IMHO.


      • It is interesting that to date, ADCs did not lead to robust responses (e.g ORR in soft tissue lesions) but both PGNX and Genentech’s ADC demonstrated biomarker changes in PSA and CTC count (a marker with growing popularity).

        Jury is still out on these programs imo.



  12. Ohad, ARQL P2 CRC data is out at ASCO for tivantinib and it’s, well, quite confusing. I think there are more questions than answers. I don’t think data is as bad as some have made it out to be (complete failure) as I think there are certainly trends for tivantinib across the board and in select sub-groups. But, the key is whether or not the sub-group data is just a bunch of noise. E.g., check out the presentation slides at: http://files.shareholder.com/downloads/ARQL/2508840080x0x668052/14606ce0-7a88-43ac-8398-8ab794b2d453/CRC%20Ph%202%20ASCO%202013.pdf .

    Even though not stat sig, there is three-month OS advantage for tivantinib in all-comers group with HR=0.70. I think that’s pretty good for such a small trial. Slide 16 is strange as it shows OS HR=1.09 for all men in the trial and OS HR=0.45 for all women, suggesting the drug works substantially better for women (and actually harms men). Just a total fluke? Also, patients that had prior treatment with oxaliplatin did much better with tivantinib with OS benefit almost stat sig (and HR=0.58). But, again, is this just a fluke?


    • It does not look like they are confused about SNTA…. disappointing results. INFI, I am not sure myself. Bought some this morning. But I do think that the valuation is high, when the marke is competitive, and their drug does not have a clear approval path…. I think this is what the market is realizing, the previous valuation of INFI was exhorbitant…. ($50). It may still be high now goven the uncertainty on approval path, and then commercial opportunity of IPI 145. This is just my opinion…


      • Market is definitely disappointed with SNTA and INFI, in both cases people expected improvement in the extent of benefit and got good but not stellar results.
        I still fidn’t have a chance to hear their analyst events but for both cases the bull story is definitely there. SNTA has a clear OS signal (avastin like) in a large data set of all adeno pts even if one doesn’t buy the subset they chose for p3. Note also there were relatively few events to date. Re INFI, people wanted to see better CLL response rate but given the short follow up and the fact nodal responses appear to convert to objective responses with time, the drug looks better than idelalisib.



      • Yes unless I learn anything dramatic from the analyst events.
        If the story is still there, I prefer to stick with fundamentals no matter how cruel market reaction is.



      • Ohad
        does the CLVS results EGFR, targeted, have implications for AVEO, as they are pursuing selective markers… or at least paint a more rosy pictures for a strategy )marker specific targeting of therapeutics)? Thanks


      • Ohad, did you look at the results for CLVS? and is this a competitor to ARRY and SNTA?


      • Looks very impressive, people have been trying to cope with Tarceva resistant tumors for years. Funny how this compound turned out to be the star in Avila’s pipeline and not their Btk inhibitor (CELG).
        Don’t think EGFR mutated NSCLC is a relevant market for ARRY or SNTA.



      • Dude, very bad calls on INFI and SNTA. With AVEO in your track record I think you should update your portfolio to show all your dogs. Three strikes in a row. Is your analysis any better than flipping a coin?
        Uggh. Sorry for negativity but very disappointed. Think you need to qualify downside risk more clearly bro.


  13. Ohad,

    Why do you think EXEL is down so much today? Were the results that bad? And why is ARRY down? The uveal melanoma data looked very good. Is this all an effect of the PD-1 inhibitors on some small-cap bios in the oncology space?

    EXEL at ASCO

    The stock is down today. I guess the Market didn’t like the results. Comments?

    Exelixis Announces Presentation of Updated Phase 2 Data for Cabozantinib in Men with Heavily-Pretreated Metastatic Castration-Resistant Prostate Cancer

    — Median Overall Survival of 10.8 months in a population where 73% of patients had 2+ prior therapies —

    — Responder analyses show that bone scan, circulating tumor cell, and pain responses are associated with longer overall survival —
    Business WirePress Release: Exelixis, Inc. – Sat, Jun 1, 2013 9:00 AM EDT

    Symbol Price Change
    EXEL 4.67 -0.17


    Exelixis, Inc. (EXEL) today announced the presentation of updated interim data from 144 docetaxel-pretreated patients with metastatic castration-resistant prostate cancer (mCRPC) and bone metastases treated with cabozantinib in an ongoing non-randomized expansion (NRE) cohort of its phase 2 randomized discontinuation trial. Median overall survival was 10.8 months in a patient population in which 73% of patients had received two or more prior therapies including docetaxel and abiraterone, enzalutamide, and/or cabazitaxel. Furthermore, a retrospective analysis of the interim data shows that early responses in bone scan, circulating tumor cell (CTC) levels, and pain are associated with longer median overall survival (OS) as compared to non-responders. These post hoc findings, particularly the bone scan response results, support the rationale for potential future prospective validation of the association of bone scan response with OS in Exelixis’ ongoing phase 3 COMET trials in mCRPC. Howard I. Scher, M.D., Chief of the Genitourinary Oncology Service at Memorial Sloan-Kettering Cancer Center and a principal investigator on the study, presented the data today in a poster-discussion session at the 2013 Annual Meeting of the American Society of Clinical Oncology (ASCO) (Abstract #5026), in Chicago, Illinois.

    “This clinical trial enrolled a heavily pretreated mCRPC population that has not been studied previously. These patients experienced disease progression despite treatment with docetaxel and additional therapies that included abiraterone, enzalutamide, or cabazitaxel. In this context the results are encouraging,” said Dr. Scher. “Patients with such advanced disease have limited options, and the data from this cohort suggest that cabozantinib has the potential to be an important treatment option for patients with mCRPC. The preliminary data suggest that these early response indicators are associated with longer median overall survival and warrant further prospective evaluation in phase 3. The ongoing phase 3 trials will help to define the potential utility of cabozantinib in mCRPC.”

    The interim results presented today comprise data from 144 men with mCRPC in the NRE cohort of an ongoing phase 2 randomized discontinuation trial. All patients had disease progression in either bone or soft tissue disease within 6 months of completion of docetaxel treatment, and the protocol differed from typical CRPC studies in that it excluded patients who progressed by PSA criteria alone, who generally have a better overall prognosis. All patients also had bone metastases on bone scan and 31% had measurable soft tissue disease. Seventy three percent of patients had received at least two prior lines of therapy for mCRPC, including docetaxel in all patients. In 36% of patients, disease progression was very rapid, occurring less than one month following the last dose of taxane therapy. Clinically significant pain, defined as baseline pain score by Brief Pain Inventory (BPI) = 4, was present in 47% of patients. Eighty percent of patients had a CTC count = 5/7.5 mL of blood. Patients received a 100 mg or 40 mg daily dose of cabozantinib.

    The median OS in the 144 patients was 10.8 months (95% confidence interval 9.1-13.0). Bone scan response (= 30% decrease from baseline in the Technetium-99m methylene diphosphonate bone scan lesion area determined by computer assisted detection) was observed in 65% of evaluable patients, CTC response (conversion from = 5 CTC/7.5 mL of blood to

    Safety data for the NRE were presented at two medical meetings in 2012. At the ASCO Annual Meeting in June 2012, Dr. Matthew Smith of Massachusetts General Hospital presented data from 93 patients receiving a 100 mg daily dose of cabozantinib (Abstract #4513). At the European Society for Medical Oncology Congress in September 2012, Dr. Johann de Bono, of The Institute of Cancer Research, London, presented data from 51 patients receiving a 40 mg daily dose of cabozantinib (Abstract #897O). Across dose levels, the most common adverse events (AEs) of grade 3 or higher were fatigue, hypertension, and venous thrombosis. 25% of patients in the 40 mg dose cohort experienced one dose reduction due to an AE, and 84% of patients in the 100 mg dose cohort experienced at least one dose reduction due to an AE. Overall, the lower starting dose of 40 mg was better tolerated with lower rates of dose reduction and interruption.

    “These data provide additional evidence that cabozantinib has clinical activity against multiple aspects of metastatic disease in this patient population,” said Michael M. Morrissey, Ph.D., president and chief executive officer of Exelixis. “The overall survival results are encouraging given that the patients enrolled in the NRE cohort comprise a highly refractory and advanced mCRPC population, and are similar to those being enrolled in our ongoing phase 3 COMET trials. We believe that these data provide continued support for our clinical strategy in this indication.”

    About Cabozantinib

    Cabozantinib inhibits the activity of tyrosine kinases including RET, MET and VEGFR2. These receptor tyrosine kinases are involved in both normal cellular function and in pathologic processes such as oncogenesis, metastasis, tumor angiogenesis, and maintenance of the tumor microenvironment. COMETRIQ™ (cabozantinib) is currently approved by the U.S. Food and Drug Administration for the treatment of progressive, metastatic medullary thyroid cancer.


  14. “Didn’t see anything dramatic in EXEL. Overall results were good but nothing revolutionary beyond the abstracts.”

    On the conf. Call they mentioned cabo for bladder cancer…one doctor stating they’ve been waiting ten years for something like this….very interesting…not many options for these patients


  15. ARRY offering convertible debt…


    one better news is this one
    www morphosys.com/pressrelease/morphosys-signs-global-license-agreement-glaxosmithkline-anti-inflammatory-program-mor103

    first outlicensed mab of morphosys


    • Im not sure what the conversion price is. I like that its 2020, and hope the conversion price is north of $10 as that usually puts a ceiling on the SP in the short to intermediate term. Ive read the 10K and 10Q and the Deerfield terms were horrendous in my opinion. I think this is a much better option and personally I am planning to buy some of the convertible debt as I see more and more that one if not both MEKs will make it to the market in a few years.


  16. Dude, very bad calls on INFI and SNTA. With AVEO in your track record I think you should update your portfolio to show all your dogs. Three strikes in a row. Is your analysis any better than flipping a coin?
    Uggh. Sorry for negativity but very disappointed. Think you need to qualify downside risk more clearly bro.


    • Indeed bad calls… I’ve had my share of bad calls in the past and will probably have more in the future but this comes with the territory when u deal with smid cap biotechs, where most drugs fail. The idea is having a diversified portfolio of high risk stocks and with the hope that eventually the winners will more than compensate for the losers.
      I post portfolio updates on over post which always include the entire portfolio, including the losers…

      btw, in contrast to AVEO, the jury is still out on both SNTA and INFI.



      • That’s it! The a v e r a g e return of your portfolio is very good!

        Will you hold Morphosys long-term after the deal for MOR103?

        Thanks, Toby


    • Ohad: I started reading your blog right before AVEO, since you built some reputation on the internet… and then 3 bad calls in a row. Ouch. In this field reputation means a lot, I figure that’s your purpose when you created this blog too. At this rate, you may lose what you built. Maybe take a break and reassess the way you evaluate things?

      I’ve never followed your advice or any blogger’s advice, nor do I think any investor should. So I think it’s unreasonable for readers to vent their frustration/blame their loss on you. But I’m trying to help man. If it were me, I’d rather make solid calls once in awhile than post often with good and bad calls at the same ratio as coin flipping.


      • I have a suggestion…. research “Peregrine Pharmaceuticals” “Bavituximab” “Dr. Philip Thorpe” “Dr. Rolf Brekken” “Dr. Robert Garnick” “Avid Bioservices” and when all is said and done…. my only call for the last couple years that has been slowly and surely waiting for the right time.. is right now. The key word is “Safety” and “Effective” as it extends survival and mixes well with all chemo’s and most importantly: it mixes well with many other SOC drugs from Big Pharma. Keep and eye because the ball is just about to start rolling on this one as partnering talks are back after ASCO 2013.

        Pipeline 100% owned… IP all locked up… Global Rights… and very shortly will be one Big Pharmas guarantee to staying on the top.
        140M shares OS
        $1.60 pps ..

        Ohad.. you can reem yourself for those last 3 picks…

        PPHM – Strong Buy


    • From their note:

      “So what would change sentiment or could turn it around? The docs defended the data and said they were surprised by the reaction today. Stock now trades at $785M market cap or $485M EV ($300M cash) or roughly $685M EV by year end or early 14. This is below even DNDN ($900M EV) and around ITMN ($650M EV). Sentiment may take some time to change but two tangible things to watch are 1) GILD could file GS-1101 on Phase II iNHL data at Lugano this month (Phase I is 58% ORR and 19mo duration and INFI has higher responses) so investors may realize that iNHL is a $1B potential fast-to-market that people forgot after CLL, and 2) ASH where INFI will report 150+ pts and durability and will be prophylaxing so if no major new infections/deaths this could reverse today’s perception.”


  17. Ohad, your blog gives a pretty good insight into the biotech world. I am a big fan of it for a year already and follow closely all your posts. Just pay no attention to those shameless faultfinders that might bother you and others in this blog. They just don`t deserve our time! Thank you, Ohad


    • Ohad, let me also add my thanks and appreciation to what Andriy just expressed, as well. The two commenters who have been critical do not appear to understand the extraordinary risk and unpredictability associated with all biotech investing. I have been a biotech investor for a number of years and have had many losses, including several total write-offs when companies I had invested in ceased operations entirely. I’ve done much, much better since I discovered your blog! The level of insight and detailed analysis that you provide far surpasses anything I have seen elsewhere, including for-fee subscription newsletters. Please don’t be dissuaded from continuing your outstanding analysis.
      Thanks, as always …


  18. SNTA did very well for a very long time, i always take some profit when i am up 50% then over 100%, and again if it cont to go up as in SNTA case. I have a little snta left…… still over my buy price. Anyone who accumulated snta when ohad recommended it and never took some profit has only him/her self to blame


  19. Ohad, regarding ARQL any additional thoughts post ASCO, do you see any near future catalyst or do you think current price is a fair one.


  20. Ohad, what do you think about INCY’s balance sheet. I knew you have address their debt issue quite a while ago. But until today, they still own around 400M debt. Do you have any worries about that?


  21. Hey Ohad
    What do you make of the Aveo announcement? Is there any value in their EGFR MAB? Clovis is in the same field. I am still holding to the Aveo shares… thus my question! Sorry. I know you have liquidated that position….


    • Yes, I feel comfortable with the current positions, which are relatively modest and represent the high risk nature of the stock. Valuation looks good as a p3 company with optionality on Akt and FGFR programs that might attract partnerships.



  22. Ohad,
    After reviewing the ASCO information on SYNTA, I still feel the drug has an excellent chance for approval. What do you see as the strengths and weakness
    of ganetespib?


      • Ohad, when is ALK+ data coming out? Always thought that was lowest-hanging fruit for ganetespib given prior data. Also, I see TNBC data is due out later this year. How optimistic are you on a signal there?


      • Both trials in ALK ( monotherapy and combination with criz) are enrolling for over a year. I expected an update by now at least from the monotherapy trial but I am not sure how successful they are at recruiting naive patients. Perhaps this explains why nothing has been presented.
        Agree about ALK being the low hanging fruit. Hsp90 inhibitors (3 different molecules) clearly have single agent activity there. I expect them to be added to ALK inhibitors as I am not sure they are potent enough as single agents though.



  23. Hello Ohad
    you wrote regarding tivos ongoing trails –
    Things are very different in breast (avastin failed to prolong OS) and colon ( avastin approved with good safety profile).
    my questions:
    in breast the trail tests tivozanib plus paclitaxel vs. placebo paclitaxel ,what this got to do with avastin?
    and in colon , tivozanib plus FOLFOX can extend os / pfs even if avastin is approved and safe. am I wrong?


      • Avastin also wasn’t tested in the specific TNBC/CRC biomarker subgroups that tivo is being tested in I don’t believe. Hopefully that makes a difference.


  24. hi, ohad –

    i’ve enjoyed ur blogs and the scientifically oriented insight they’ve provided. as a former practicing member of NYSSA, i admire and appreciate the “box score” you regularly publish. very few of my former colleagues practiced that kind of full-disclosure.

    i’m interested in HALO; and i understand they submitted 1 or 2 abstracts at ASCO; but i don’t know if there were presentations, as well. if so, i would appreciate your comments. tuvm.



    • Thanks, JJJ. No doubt that having an ongoing “box score” is not always pleasant, especially in this business 🙂

      I don’t follow HALO closely. Their p1b in pancreatic cancer had good data in terms of response rate but I am still a little bit skeptic with respect to their approach. Dissolving the matrix around pancreatic cancer is a major challenge and it is unclear to how that can be used with their enzyme when given systemically especially with the amounts they used. But again, can’t say I really know this program.



  25. Interesting, Bruce Kovner buys 2M shares in SNTA. ($9.3M) in addition to 20.5M shares he already owns. He must have strong faith in the company (and can afford it).


      • Hey Ohad
        so you think SNTA is a buy at these levels? I am concerned by their phase 3 trial? have they rushed into it too soon? And what do you make of the decreasing efficacy of their compound (to the control group) in the longer terms, as Adama Feuerstein reported in a good write up?
        Thanks for the help


  26. Ohad,
    Do you think CLDX run up this week is related to the focus on its CDX-1127 immunotherapy- in light of ASCO PD-1 data? And what are your thoughts on chances of success?


  27. Thanks for your wisdom. You were early in seeing the significance of anti PD1, Ipilumumab. Any thought on Innate Pharma. They are in phase 1 with Bristol Myers in combining Nivolumab and there anti Kir mab. Novo Owns 15 percent. The total market cap is 150 million. Also any opinion on early stage Compugen. Thank you again for your work.


    • I love this board because of the new companies I discover. Had never heard of Innate before. Thanks for the heads-up Scott! At first blush, looks interesting given big name partners (BMY, NVO), small marketcap, and involvement in immunotherapy. Do you know when PoC results will be out on their drugs partnered with BMY? Also, any idea on royalty rate and if Innate has option to co-promote?

      My issue with Compugen is, while they seem to discover apparently novel targets, they license them out before any PoC and thus settle for paltry single-digit royalties. I don’t like that type of business model myself.


    • Thanks. So far the data I have seen weren’t so impressive but if BMS thought the program was attractive there’s gotta be something there.
      Re CGEN, sorry but I prefer not to discuss Israeli companies as there might be overlap with my work at Pontifax.



      • Ohad, do you have any links to the Innate data you didn’t find so impressive or otherwise some more info? Thanks for any help.


  28. Ohad,

    How about PARP inhbitor as many are starting Phase 3 study this year. Clovis, Biomarin, Tesaro, AZ and I believe Abbvie also has PARP inhbitor. Can you compare them based on results so far or best one to win.


    • Definitely an exciting class of drugs where interest has been rekindled. Astrazeneca is leading the race but others are not far behind. All appear active but they are being pursued in different settings (e.g. BRCA mutants, ovarian/breast, monotherapy/combination).
      I don’t have a clear winner.



  29. Ohad,

    I noticed you have not mentioned a company by the name of MSTX which has a drug in phase III trials. Do you consider this company worth mentioning or looking to establish position? Please give me a feedback on whether this company is worth an investment or not. Thanks


  30. Looks like we have now race for PARP inhibitor clearing with Olaparaib filing for EMA in Ovarian cancer, BMN-673 from biomarin described in ECCO/ESMO as ” flat out success ” and inititated Phase 3 in BRC MUT BREAST CANCER and Tesaro with Phase 3 in Ovarian cancer. Only one out of race it seems like Abbvie’s veliparib


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