Biotech portfolio update – 2012 summary and 2013 outlook

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In the last post of the year, I will try to provide a status update as well as key 2013 milestones for the stocks in our portfolio. I would like to use this opportunity to wish everybody happy holidays and a happy New Year.

Seattle Genetics

Seattle Genetics’ (SGEN) main task is expanding Adcetris’ use outside of approved niche indications (Hodgkin’s Lymphoma and ALCL). As an anti-CD30 antibody-drug conjugate (ADC), Adcetris has potential utility wherever CD30 is expressed by tumors.

Based on data at ASH earlier this month, the next major opportunity is CD30-positive diffuse large B cell lymphoma (DLBCL), where Adcetris demonstrated a 44% response rate with a response duration of 6 months. A 44% response rate is one of the highest response rates ever observed for a monotherapy in DLBCL (see figure below from a 2011 review by Anas Younes).


Source : Younes, A. Nat. Rev. Clin. Oncol. 8, 85–96 (2011)


This impressive single agent activity makes Adcetris one of the most active agents in DLBCL, which still remains an unmet need. Seattle Genetics did not announce its registration strategy, but an accelerated approval based on a single arm 100-patient trial in relapsed/refractory DLBCL looks reasonable given recent experience with hematology drugs (Folotyn in PTCL, Adcetris in HL and Kyprolis in multiple myeloma) 

Surprisingly, Adcetris appears effective even in patients with very low CD30 expression, which increases the amount of eligible DLBCL patients. ~30% of DLBCL cases are CD30-positive, which implies an annual incidence of 6,500 in the US alone. Around half (3250) of these patients could be candidates for Adcetris in the relapsed/refractory setting, translating to a $200M near term opportunity.

In 2013, investor attention could shift to Seattle Genetics’ partnered pipeline. The company’s main partner is Genentech, with 8 programs in clinical testing. Earlier this year at Roche’s analyst event, it presented 5 anecdotal objective responses, each with a different ADC.

At ASH, Genentech presented data for its CD22 and CD79b programs in non-Hodgkin’s Lymphoma (NHL). Both ADCs were active with a response rate of 27% and 31% in relapsed/refractory patients. At the higher doses, response rate was 41% and 47% for the CD22 and CD79b ADCs, respectively. These data appear slightly better than other antibody programs in NHL, primarily CD19 ADC and Fc engineered antibodies.


Exelixis (EXEL) continues to frustrate investors with its stock still hovering around $4.5. The company had 2 important milestones in November: Approval for cabozantinib (cabo) in a rare indication (medullary thyroid cancer, MTC) and initiation of phase III for its MEK inhibitor, GDC-0973, by its partner Roche.

Phase III readouts for cabo and GDC-0973 are expected only in mid-2014. Therefore, in 2013 focus will be directed towards cabo’s launch in MTC and potential off label use in prostate cancer. The company could also announce a licensing deal for EU or Japan rights for cabo, although it has enough cash to take the drug all the way to approval on its own.

Another important event in 2013 will be a decision regarding co-promotion of GDC-0973. Exelixis must decide whether to exercise its co-promotion option 12 months after phase III initiation and “must have the capability to co-promote, including an adequate internal sales and promotional infrastructure, and an experienced internal oncology sales force.” As the phase III trial started in November 2012, Exelixis will have to make a decision towards mid-2013 whether to establish its own oncology sales force. Alternatively, it might try to restructure the deal with Roche.    

In a sum of parts analysis, the MTC franchise and GDC-0973 probably represent half of the company’s valuation. The other half is attributed to cabo’s opportunity in prostate cancer, which can vary dramatically based on likelihood of success. Using Medivation’s (MDVN) $4B market cap as a benchmark (has 50% stake in an approved prostate cancer drug), it is clear the market is skeptical of cabo’s prospects.

Assuming cabo’s sales in prostate cancer will be half of those of Medivation’s Xtandi, the current valuation reflects ~15% chances of success (using acceptable discount rates). In my opinion, chances of approval are higher (33%) based on the dramatic and reproducible resolution of bone mets and Alpharadin’s survival effect, which works exclusively by targeting bone metastases. This translates to a target price of $8.80 excluding other indications such as renal, lung and liver cancer. In addition, if cabo is approved in prostate cancer, it will face little competition as opposed to Medivation’s Xtandi, which will have to compete with generic Zytiga.


Arqule (ARQL) is down 50% year to date, following tivantinib’s phase III failure in lung cancer. A phase III in liver cancer is about to start, which in contrast to the lung cancer trial, will recruit only patients with high Met expression.

The major catalyst for 2013 is phase II data in colorectal cancer in Q1, where tivantinib is added to a standard chemotherapy regimen. Importantly, the trial is recruiting patients regardless of their Met status, but analysis will include activity in Met+ tumors as well. Positive results in this trial could have a major impact, as expectations are very low.

Results from the failed lung cancer study will probably be presented next year, in an attempt to find correlation between Met expression and clinical benefit. Such correlation cannot be used for approval, but it can be viewed as validation of this strategy in other cancer types. It is still unclear what portion of patients will have this information, as Met status was not required at study entrance.

Arqule has 2 additional programs in phase I. An Akt inhibitor developed by Daiichi Sankyo and a wholly owned FGFR inhibitor. Both targets are considered promising but there are many competing programs in development.


Array (ARRY) has made tremendous progress with its proprietary and partnered pipeline in 2012.

The company’s lead program is ARRY-520, a KSP inhibitor for the treatment of multiple myeloma. Updated results at ASH were encouraging and included a response rate of 22% in late stage patients in combination with steroids. This level of activity appears competitive with other novel agents such as Celgene’s (CELG) pomalidomide. In the same meeting, Celgene reported a response rate of 21% with pomalidomide in combination with steroids. Although Array’s results are from a small trial (18 patients) patients were more heavily pretreated (median of 10 prior treatment lines vs. 5 with pomalidomide).

In 2013, Array will have plenty of catalysts for its portfolio of 15 clinical stage drugs. 4 drugs are expected to start phase III: 2 of Array’s wholly-owned compounds (AARY-520, ARRY-614) and two partnered compounds (selumetinib and MEK162), developed by AstraZeneca (AZN) and Novartis (NVS), respectively.


Source: Array Biopharma

Data readouts are expected for 7-10 programs, including randomized phase II results in asthma for ARRY-502. A positive outcome could lead to a licensing deal in the 2nd half of 2013.  The company also expects to license its p38 inhibitor (ARRY-797) based on positive randomized data in osteoarthritis. The drug appears as effective as opioids but a QTc signal was observed in patients who received the drug, which raised doubts regarding Array’s ability to monetize this asset.

With a market cap of $350M, a broad and diverse pipeline including 4 programs that are about to enter phase III and multiple data readouts in 2013, Array is probably the cheapest stock in our portfolio.


Curis’ (CRIS) derives the majority of its valuation from its royalty stake in Roche’s Erivedge. Despite very high hopes for hedgehog pathway inhibitors, so far their activity is limited to niche indications, primarily basal cell carcinoma (BCC). The drug was approved in the US in 2012 for advanced BCC and is expected to win approval in ex-US territories during 2013. 

Royalties for Erivedge are still low (~$2M expected in 2012) but are expected to gradually increase. This should come from continued penetration in the US and product launches outside of the US. Roche is evaluating Erivedge in less advanced stages of BCC, with initial encouraging results but unclear market potential. The drug is in multiple phase II trials in additional indications, sponsored by the NCI.

The company has 4 additional clinical stage programs, 3 of which could generate data in 2013.

Curis recently licensed an IAP inhibitor from Genentech, which is currently in phase I. The deal was received with skepticism given Genentech’s decision to outlicense the drug and the disappointing results to date with this class of molecules. The company will present first clinical data at ASCO 2013.

The company’s Hsp90 program (Debio 0932), partnered with Debiopharm, is in a phase I combination trial in lung cancer. The drug led to 2 objective responses (KRAS+ lung cancer and breast cancer) in a phase I among 45 evaluable patients. As an oral drug, Debio 0932 could have an advantage over other Hsp90 inhibitors (primarily Synta’s (SNTA) ganetespib), but it is still unclear whether it is as potent as other Hsp90 inhibitors.

The third program, CUDC-907, recently started phase I. Although it has a unique inhibition profile (PI3K and HDAC), it is unclear whether such broad inhibition of 2 classes of enzymes can be achieved without substantial side effects.  


Morphosys remains a top pick, with its huge partnered pipeline, an evolving proprietary pipeline and profitable operations. The recent sale of its research and diagnostics business makes it even more attractive.

The company has three wholly owned programs in the clinic: MOR103 (anti-GMCSF) for inflammatory diseases and MOR208 (anti-CD19) and MOR202 (anti-CD38) for B cell malignancies.

The company published MOR103 data in rheumatoid arthritis, which demonstrated clear activity with one of the doses (1 mg/kg). Strangely, a slightly higher dose (1.5 mg/kg) was substantially less effective. The short duration and the low number of patients might account for this difference, but it is hard to ignore this discrepancy. Going forward, acknowledging PK variability among patients, there is a risk MOR103 will not be able to replicate these results. The company expects to partner the program in 2013.

MOR208 is an Fc engineered CD19 antibody, which Morphosys in-licensed from Xencor. Phase I data at ASH showed the drug is active in CLL, but it will experience fierce competition from other CD19 antibodies as well as new agents in development (Ibrutinib, GS-1101 etc).

Morphosys’ most interesting asset is MOR202, despite the lack of published results. This is due to impressive activity with Genmab’s CD38 antibody, daratumumab, in multiple myeloma. Although there is no guarantee MOR202 will exhibit the same clinical profile, there is a fair chance for MOR202 to have activity in this setting.

First results for MOR202 are expected in 2013 and the company has already been approached by companies who tried to get daratumumab but lost to J&J (JNJ). Sanofi just opened a combination trial for its CD38 antibody (SAR650984), which was licensed from Immunogen (IMGN).

Morphosys’ partnered pipeline comprises of 16 (!) antibodies in clinical testing. The most advanced program is Roche’s gantenerumab (anti-beta amyloid) for Alzheimer’s disease. To date, none of Morphosys’ partnered programs were able to generate exciting data, but as more programs move to phase II proof-of concept trials, likelihood of success increases.

Novartis is Morphosys’ most notable partner, with tens of programs at various stages of development. At its R&D day last month, Novartis emphasized BYM338 (anti-ActRIIb) for a variety of conditions characterized by muscle loss. With several ongoing studies and potential fast route to market in niche indications, BYM338, could emerge as an important catalyst for Morphosys in 2013.


Source – Novartis R&D day, November 2012


Incyte (INCY) represents a classic launch story with its Myelofibrosis (MF) drug, Jakafi. The drug was approved in November 2011 and is doing well but not great, with an annual run rate of $140M in the US. As the only approved drug for MF, Jakafi has the potential to double sales in the coming 2 years, before competing drugs from Sanofi and YM Biosciences (about to get acquired by Gilead) reach the market.

At ASH, Incyte reported findings that can increase Jakafi’s adoption.

An updated survival analysis from two phase III studies showed the drug may lead to a survival advantage, which becomes apparent with a relatively short follow up. Data set is still immature, but the separation looks real and already reached statistical significance. The alleged survival benefit is unlikely to appear in Jakafi’s label, but it will surely encourage more physicians to put their patients on the drug, which until now was criticized by some as being a “symptomatic treatment”.

Another presentation involved dosing patients with low platelet counts using a lower starting dose followed by a dose optimization period. Results were positive, demonstrating the drug can be highly effective in these patients without causing severe complications. This titration strategy could expand Jakafi’s use in patients with low platelet count (who until now considered ineligible) or less symptomatic patients as well as increase treatment duration.

In 2013, Incyte will start receiving royalties on ex-US sales from its partner Novartis.

Baricitinib is Incyte’s most advanced development-stage program, partnered with Lilly (LLY). The drug is in a broad phase III campaign, focusing on RA, where phase II results demonstrated similar efficacy to that of Pfizer’s recently approved Jak inhibitor (Xeljanz). Originally, Baricitinib was expected to be launched in a market with 2 established oral drugs (Pfizer’s Xeljanz and AstraZeneca/Rigel’s (RIGL) fostamatinib. Recent disappointing phase II data for fostamatinib imply it is not as effective as hoped and should be viewed as an incrementally positive development for Incyte.


Celldex (CLDX) saw its shares more than double in 2012. The company’s lead program, rindopepimut, is a cancer vaccine currently in phase III for brain cancer. Results are expected only in 2015, which creates a value creation gap for 2013-14. 2 programs that may fill that gap are CDX-011 (anti-GPNMB ADC for breast cancer) and CDX-1127 (anti-CD27 antibody for cancer).

Earlier this month the company presented updated results from a randomized trial evaluating CDX-011 vs. chemotherapy. Celldex should be commended for running such a rigorous study, which sets a higher bar compared to placebo controlled trials. In the entire population, CDX-011 had similar activity to chemotherapy (a result some might say is a positive by itself). In patients with high GPNMB expression, CDX-011 was superior in progression-free survival (2.7 vs. 1.5 months) and overall survival (10 vs. 5.7 months). Although numbers were small (n=33), the robust survival difference over an active regimen clearly indicates CDX-011 has promise for that subset of patients.

In 2013, investors expect Celldex to provide a registration strategy for CDX-011. The company stated it would take the phase II data to the FDA and negotiate a development plan in the near future. One direction the company is considering is narrowing target population even further to triple-negative tumors with high GPNMB expression. In the phase II study, the effect this subset was more pronounced, although the sample size was very small (n=16). I find this approach risky, as it relies on s very small subset analysis and a problematic patient stratification based on markers that are absent as opposed to ones that are present in the tumor.

Based on the high response rate among the GPNMB-high patients, Celldex might be able to pursue accelerated approval with a single arm phase II. In parallel, it could start a large randomized phase III for earlier stage disease. One can only hope that Celldex will run the studies under SPA, to avoid Genentech’s experience with T-DM1.

CDX-1127 should be viewed as a “wild card”, as there are limited expectations from this agent. CDX-1127 belongs to a class of immune-modulating antibodies, a field that is garnering a lot of attention thanks Yervoy and anti-PD1 antibodies. Therefore, any signs of efficacy in phase I next year could have a meaningful impact on Celldex.      


Synta (SNTA) is in final preparations for launching a pivotal trial for its Hsp90 inhibitor, ganetespib in lung cancer. Updated results from a large phase II trial showed an impressive overall survival signal across certain subsets, primarily, tumors of adenocarcinoma histology. The company is finalizing the phase III design based on FDA’s feedback and expects to activate the trial in Q1 of 2013.

An interesting twist in the phase III trial design is the exclusion of “rapid progressors” by requiring patients to have at least 6 months from diagnosis. In the phase II trial, the latter group derived a dramatic benefit in overall survival (HR = 0.367). Despite the strong results, applying such criterion bears some risk as it has never been used for approving a cancer drug to the best of my knowledge. Updates from the lung cancer phase II study are expected throughout 2013.

Ganetespib is in several phase II trials across several indications, which may read out during 2013. Studies that are of particular interest are two trials in ALK+ lung cancer alone or in combination with Xalkori, respectively. The trials, which started in August 2012, should read out in 2013 and provide a good sense of ganetespib’s activity in this subset. Another trial in breast cancer is expected to report data in the first half of 2013.

Synta’s competitors are advancing their own Hsp90 inhibitors as well. In contrast to what I originally thought, both Novartis and Astex (ASTX) are forging ahead with their respective programs. Novartis is pursuing AUY922 in multiple indications, including lung cancer but its emphasis is on a different subset of patients with EGFR mutated tumors. Astex is conducting several combination trials, including a large phase II trial in combination with Xalkori in ALK+ or ROS+ lung cancer. Debiopharm’s Debio 0932 is an earlier stage compound, which had anecdotal activity in 2 patients (KRAS+ lung cancer and breast cancer) in phase I.      

YM Biosciences

Uncertainty around YM Biosciences’ (YMI) Jak inhibitor came to an end with the recent Gilead (GILD) acquisition. The acquisition price represented an ~80% premium over the market price. Gilead’s move ended almost a year of speculations but it is unlikely to change controversy around CYT387’s alleged anemia effect. The deal negatively affected Incyte, as Gilead is expected to start an aggressive phase III program for CYT387, a direct competitor for Jakafi.  Nevertheless, Incyte still has 2-3 years before Gilead reaches the market.


In 2012 Genmab underwent a transformation and emerged as an antibody powerhouse, with a promising pipeline as well as several high profile technology collaborations.

Historically, the market ascribed value only to Genmab’s royalty stake in Arzerra (marketed by GSK) while the rest of Genmab’s pipeline was ignored. In June, the company released spectacular phase I data for its CD38 antibody, daratumumab. This was followed by a $1.1B licensing deal with J&J in August.

On the technology front, Genmab announced some big name partners for its bispecific antibody technology (DuoBody). The deals with Novartis, J&J and Kyowa Hakko Kirin were modest in their initial size but served as a testament to Genmab’s capabilities in the field. A fourth Duobody collaboration was announced but the partner’s identity remains undisclosed.

At its analyst event last week, Genmab provided expected milestones for 2013. Most importantly, J&J will advance daratumumab into a global clinical development program. Genmab will start phase I with its Tissue-Factor ADC, in collaboration with Seattle Genetics. The company also expects to announce additional licensing deals around its bispecific antibody technology and the Hexabody platform that was unveiled at the event.

Inevstors should also track two additional CD38 programs: MOR202 from Morphosys and SAR650984 from Sanofi. Both programs could report clinical data for the first time in 2013. Genmab claims daratumumab’s performance will be hard to match due to a unique set of distinguishing properties (primarily CDC and phagocytosis). If this is the case, daratumumab could easily become a $1B drug very quickly, assuming widespread adoption in relapsed refractory patients.   


AVEO (AVEO) is the only company on the list with a potential FDA approval in 2013. After reporting positive data for lead program Tivozanib for renal cancer in June 2012, the company filed an NDA with an expected decision by July 28th 2013. Tivozanib was compared head to head vs. Nexavar and led to a higher progression-free survival with substantially fewer side effects. Nevertheless, the market is very cautious on tivozanib’s regulatory and commercial outlook.

The regulatory concerns focus on inferior survival rate with Tivozanib compared to the control arm. Although this can be easily explained by the cross-over study design, many suggest the FDA will require another trial for approval to assure tivozanib does not adversely affect survival.

From a commercial standpoint, some claim that even if the drug gets approved, it will face a lot of competition from at least 3 approved agents, including a market leader (Sutent) that is expected to go off patent in the coming years. Going forward, additional competition could come from PD-1 antibodies, given impressive activity demonstrated by BMS’ PD-1 antibody.

AVEO’s current valuation represents a low likelihood of approval and/or commercial success. This creates a buying opportunity with an attractive risk/reward ratio, as I believe the drug will eventually get approved and that once in the market, it will gain a reasonable market share thanks to its differentiated safety profile. Tivozanib is not a revolutionary drug, but it is likely to become the drug of choice for many patients who cannot tolerate or will want to avoid common side effects with current drugs. It benign safety profile should make it very combinable with other drugs down the road.

Portfolio holdings – December 23rd, 2012

Biotech portfolio - Dec 23rd 2012 Biotech etfs - Dec 23rd 2012

95 thoughts on “Biotech portfolio update – 2012 summary and 2013 outlook

  1. I have a question for you: If the market is facing a big correction, do you close some positions without imminent catalysts or take some steps to hedge your position?



  2. Thanks Biochem.
    It is very difficult to predict which stock will be affected more than its counterpart (which is why it’s important to have 10-20 stocks at a given time). I would feel more comfortable in selling portions of each position.
    In our portfolio, we have 20% in cash.



  3. Wow, excellent article and enjoy your insight and knowledge.

    I’m surprised you didn’t sell YMI in your portfolio. Do you intend to hold on to GILD assuming the sale goes through?

    You mention frustrated EXEL investors but then put only a 33% chance of approval of cabo for prostrate. Is this a typical buying opportunity you would observe as a way to beat the market?

    Lastly, you mention ARRY as the “cheapest” stock in the portfolio. It seems they ave a vast number of trials going on but keep needing cash. Were you surprised at all by this and from this do you assume they intend to go it alone?

    Have a great Christmas, and thanks again for all your hard work.

    Dan S.


  4. Thanks, Dan.
    I don’t see any urgency selling YMI.

    Re EXEL – Unfortunately most drugs fail and no matter how promising I find cabo’s data, a 33% chance looks reasonable. Indeed, I think it is a good buy at these levels.

    ARRY – Agree that cash burn is the main concern with ARRY, although the recent transaction alleviated near term concerns. A lot depends on Array’s ability to partner at least one program in 2013 (ARRY-502 or ARRY-797) and milestone payments from Novartis, Amgen and Genentech.



  5. i hold some isis, why you think alny would be better pick……they are only in early stage vs isis
    HAPPY NEW YEAR and thanks again for all the informative imput


  6. Ohad, if u like rnai, how about ARWR? I think ALNY is partnered with them on one program, they have a whole rnai pipeline/platform and real tiny market cap.


  7. Bj – I follow ZIOP but don’t have a concrete opinion one way or the other. Re teh RheoSwitch, it’s a very cool concept but we need more clinical data to assess potential.

    Mcbio – What I like in ALNY is the specific TTR program. It has positive read-throghs to other programs targeting liver related conditions. With respect to RNAi, my working hypothesis that after the meltdown of the field, it will have several therapeutic indications and several programs will reach the market. ARWR have a lot going on but most of it is early stage, could be a nice speculative bet, who knows but in the meantime I prefer ALNY (and perhaps Tekmira)



  8. I just think the ARWR pipeline looks much more interesting than TKMR and like the fact that ARWR is starting to sign up big pharma and other biotech partners. Obviously TKMR is in much better financial shape.


  9. ARWR claims on slide 18 of their corporate presentation that it “is the first company to have successfully systemically administered sIRNAs and the only biotech that showed clinical success in anything other than liver.”

    Any comments on those claims?


  10. Ohad
    what is your opinion on the recent GERN Phase 2 trial of imetelstat in ET? It looked promising to me:
    “The observed hematologic response rate of 100%, accompanied by a molecular response rate of 86% among the patients who had a JAK2 V617F mutation.” This drug, if successful amy be a competitor to Jak1-2 inhibitors for some indications.
    Thanks for your opinion on this


  11. Dan, that sounds interesting on GERN; I missed those results. I had assumed that GERN really didn’t have anything left. Maybe it’s time to take a look again. Sounds like imetelstat may have a role in ET, MM, and possibly myelofibrosis. Anxious to hear Ohad’s thoughts here.


  12. Dan, their drug is clearly active (all 14 pts respondede). The unmet need and potential market size are unclear to me. INCY also had good data in ET but they are not pursuing it, which implies they don’t find it attractive enough.



  13. Ohad: Listened to recent CC and it sounds like GERN pursued ET initially because it was a quick PoC for the drug in this kind of setting. They think becaue it worked against ET it will work against myelofibrosis as well. Also, the early data against MM looks encouraging. Finally, per the recent CC their rationale for working in these indications is that there are short telomeres in these patients. GERN claims that’s where imetelstat works best. They even claim there were stat sig PFS results in the sub-pop of the NSCLC trial that had short telomeres so they are still continuing on in this specific NSCLC pop as well.


  14. Mcbio, yes, I agree. They seen convinced the drug will work with most hematological idseases where short telomeres are common. I think Chip Scarlett knows what he’s doing.


  15. Take a look at Titan Pharmaceuticals, TTNP. Recently signed partnership and received priority review for drug delivery system PDUFA April 30, 2013 for FDA action on the NDA. Market cap less than 80 million. Under partnership deal, Titan will get 50 million if approved.

    To a new year as good or better than the last.


  16. Ohad, I am glad you are interested. As always, it will be very helpful to get your opinion and insight into any drug candidate and their chances.
    Thanks a lot


  17. Ohad: On imetelstat safety, 6 of 7 eligible patients chose to remain on treatment beyond a year. Obviously some patients dropped out, whether for lack of efficacy or safety, given study started with 14, but presumably this speaks to some degree to safety of the drug. Also, I assume the JAK2s have their own safety questions. Would be interested to know how durable JAK2 responses are as well. Finally, I like the fact that imetelstat is an entirely different MoA from the JAK2s and could presumably be used in combo with them or at least treat JAK2 failures.


  18. Ohad: did you catch ARRY today at JPM? Pretty important news on ARRY-614 front IMHO. Disclosed that FDA has agreed to hematologic improvement (HI) as primary endpoint for pivotal trial, as opposed to having to have OS as primary endpoint (much higher bar). Also, NVS started P3 NRAS melanoma trial for MEK162. Expected to complete in October 2014 and ARRY thinks they can begin receiving revenues in 2015 on MEK162.


  19. Ohad: ARQL CRC data sounds bad today but would just say way too early to call the trial a fail I think. They don’t have the results stratified by MET status yet. Sounds like these will be available middle of this year (ASCO?). Need to see specific results in high MET patients I think before we judge this trial. OS results in high MET HCC were very positive.


  20. why is the big gap betwin Estimated Study Completion Date: March 2016
    and Estimated Primary Completion Date: February 2013
    for Sanofi SAR302503 ?


  21. SAR302503 – They’ll have efficacy data this year, the 2016 date probably relates to long term safety and extension cohort.

    I still think ARQL represents a good risk/reward ratio, waiting to see subset results from both NSCLC and CRC trials.



  22. Re: ARQL, in addition to the critical subset data in CRC around middle of this year, we also may get P2 data from signal generating studies of single-agent tivantinib and tivantinib+sorafenib in breast, head and neck, kidney, multiple myeloma, and prostate cancers. I’m long and will continue to hold, for better or worse.


  23. Best data – INFI, most advanced – GS101.

    One clear advantage INFI has activity on gamma isoform which makes it relevant for T cell malignancies and Hodgkin’s lymphoma. (still don’t know if potent enough as they need to reach higher doses to inhibit the isoform efficiently)

    In B cell diseases (CLL, iNHL) will be interesting to see if INFI can demonstrate superiority over GS-101 as the bar is very high especially in combination with Rituxan/ Treanda.
    Inflammation is another area of potential differentiation.

    Gilead also disclosed it started p1 with a dual gamma/delta inhibitor.

    TGTX – still no clinical data .
    MRK will probably start p1 with XL499 soon.



  24. Hi Ohad,

    You wrote that Morphosys remains a top pick. Do you find its current level attractive? (about $40) Would you initiate a position now?
    Thanks as always,


  25. Ohad:
    Thanks for all your continued thoughtful analysis. Have you ever taken a look at Astx. Seems to have a reasonable number of drugs in their pipeline, aprx. $130mm cash on bal.sheet and currently has a fair number of deals with various pharmas.ongoing. Appreciate any color.


  26. Chris – Yes I think it is still attractive after the AbD serotec sale. A lot depends on their CD38 program, if it is as good as Genmab’s then the stock is very cheap.

    Eagle75- Yes I am following them, even listened to their R&D webcast last month. I agree they meet a lot of criteria and have the right ingredients on paper. I still couldn’t find a real value creator in their pipeline , perhaps the Hsp90 program or next-gen Dacogen?




  27. Ohad- am long ASTX and i think 2nd gen dacogen is def their key drug. Do also like that their hsp90 drug is pursuing a diff strategy than snta drug by going after prostate cancer since they are behind snta. Finally they have a partnered cdk 4/6 drug in same class as pfe drug that recently had strong breast cancer data along with partnered bace for AD.


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