Everything's Coming Up Roches
Stephen Simpson, CFA
Stephen Simpson, CFA
Stronger First Quarter Sales Help Roche, But ASCO Probably Matters More
Stephen Simpson, CFA
Stephen Simpson, CFA
Poor Pipeline Productivity Has Left Roche More Vulnerable
Stephen Simpson, CFA
Stephen Simpson, CFA
Tue, May 24, 9:10 AM
- Thinly traded nano cap OXiGENE (NASDAQ:OXGN) is up 65% premarket on a 9x surge in volume in response to its announcement of successful results in a Phase 2 clinical trial, called Study GOG-01861, assessing lead product candidate, Fast Track-tagged CA4P (fosbretabulin), in women with recurrent ovarian cancer. The results were just published online in the Journal of Clinical Oncology.
- The open-label U.S.-based study randomized 107 patients across 67 sites to receive either CA4P with bevacizumab [Roche's (OTCQX:RHHBY) Avastin] or bevacizumab alone (control). It achieved its primary endpoint of showing a statistically valid improvement in progression-free survival (PFS) for the combination compared to Avastin alone (7.3 months versus 4.8 months; p=0.05).
- Preliminary median overall survival also favored the CA4P combination (24.6 months vs. 22.0 months), with a 15% reduction in the risk of cancer progression or death (hazard ratio: 0.85).
- No treatment-emergent serious adverse events were observed. The most common CA4P combination-related adverse events (grade 3 or higher) were hypertension (35%; n=18) versus 20% (n=10) for control.
- CA4P is a vascular disrupting agent. It fights tumors by compromising the blood vessels that feed them, causing widespread ischemia (insufficient blood flow) and necrosis (tissue death) of the cancer cells within the core of the tumor.
- A 436-subject Phase 2/3 study assessing CA4P + bevacizumab in platinum-resistant ovarian cancer is about to start. According to clinicaltrials.gov, the estimated study completion date is July 2018.
Mon, May 9, 4:57 PM
- Thinly traded nano cap EPIRUS Biopharmaceuticals (NASDAQ:EPRS) reprioritizes its pipeline, suspending development of lead product candidate BOW015, a biosimilar to J&J's (NYSE:JNJ) Remicade (infliximab), to focus on developing biosimilars for the treatment of rare diseases. For example, the company will allocate additional resources to advance BOW080, a biosimilar candidate to Alexion Pharmaceuticals' (NASDAQ:ALXN) Soliris (eculizumab), currently approved in the U.S. for the treatment of orphan diseases paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome, and BOW070, a biosimilar candidate to Roche's (OTCQX:RHHBY) Actemra (tocilizumab) for the treatment of a rare lymphoproliferative disorder called Castleman's disease.
- The company intends to rationalize the BOW015 program including possible partnerships, divestitures or other alternatives.
- The reallocation of resources will include a workforce reduction of up to 40%. Concurrently, President & CEO Amit Munshi has stepped down, replaced by board member Scott Rocklage, Ph.D., former CEO of Cubist Pharmaceuticals.
Tue, Mar. 29, 3:37 PM
- Thinly traded micro cap Ohr Pharmaceutical (OHRP +16.8%) is up on a 6x surge in volume in response to its announcement that it has initiated a Phase 3 clinical trial assessing lead product candidate, Fast Track-tagged squalamine lactate ophthalmic solution, 0.2% (OHR-102), in combination with Roche's (OTCQX:RHHBY +2.2%) Lucentis (ranibizumab), for the treatment of neovascular age-related macular degeneration (wet AMD). The study will enroll ~650 treatment-naive patients. The primary endpoint will be the change in visual acuity at Month 9.
- The product candidate failed to beat placebo in a Phase 2 study, called IMPACT, which precipitated a plunge in valuation.
- Squalamine, a small molecule drug in an eye drop formulation, blocks the development of abnormal microvascular networks in the eye (a process called neovascularization) by inhibiting vascular endothelial growth factor (VEGF), platelet-derived growth factor (PDGF) and basic fibroblast growth factor (bFGF). All play key roles in angiogenesis (formation of new blood vessels from existing ones) and ocular neovascular disease.
- Lucentis is a monoclonal antibody fragment that binds to VEGF.
- Previously: Ohr Pharma presents data from failed Phase 2 study; Phase 3 planned for H2 (May 8, 2015)
Tue, Feb. 2, 5:37 PM
- Pacific Biosciences (NASDAQ:PACB) jumped into the close in the last hour, up 24.3%, on news that Roche Holding (OTCQX:RHHBY -2.3%) approached the firm to talk buyout, Reuters reports.
- Price disagreement means the talks haven't advanced yet, sources said.
- Roche's interest lies in Pacific Biosciences' advanced gene sequencing technology. The two formed a development/distribution partnership in 2013.
- A deal in this area would show continued signs of life in life science M&A. Roche has been making small deals over the past year to fill out some therapeutic areas.
Dec. 8, 2015, 5:04 PM
- The first presentation (Abstract P4-14-20) highlights data from a Phase 1b study of ONT-380 in HER2+ breast cancer patients who had failed treatment with Roche's (OTCQX:RHHBY) Herceptin (trastuzumab) and a taxane. The data show an overall response rate of 41% and a clinical benefit rate (CBR) of 59% in an advanced stage patient population [clinical benefit rate is considered vague but should include the overall response rate plus patients with stable disease (no progression); the term "disease control rate" is used more frequently]. 60% of the advanced stage population had a history of central nervous system metastases. The CNS CBR for patients with response assessable (unclear on how this is defined) CNS metastases was 64%.
- The second presentation (Abstract P4-14-19) combines data from the above trial and another Phase 1b assessing ONT-380 in combination with Herceptin and Roche's Xeloda (capecitabine). The data includes patients with previously untreated CNS metastases as well as those with progressive or new CNS metastases after prior treatment with radiation or surgery. No specific numbers are disclosed, only the statement "Responses and clinical benefit in the CNS were seen for both groups and in all combinations tested."
- The misdirection has clearly spooked investors. Shares are down 33% after hours on robust volume.
- In June, the company announced positive data from the Herceptin/Xeloda study that showed an 85% disease control rate (DCR) (partial responders + complete responders + those with stable disease) in all cohorts. The company also reported data from the Herceptin/taxane study that showed a 64% DCR (this equals the 64% CNS CBR stated above). Shares rallied that day.
- Previously: Oncothyreon up 46% premarket on ONT-380 study results (June 1)
Dec. 8, 2015, 8:53 AM
- Thinly traded nano cap Pieris Pharmaceuticals (NASDAQ:PIRS) is up 17% premarket on increased volume in response to its announcement that it has entered into a research collaboration and license agreement with Roche (OTCQX:RHHBY) in cancer immunotherapy. Specifically, Pieris will discover, characterize and optimize Anticalin-based drug candidates against an undisclosed target. Both firms will evaluate different drug formats against the target and advance them through preclinical development. Roche will be responsible for IND-enabling activities, clinical development and worldwide commercialization.
- Under the terms of the agreement, Pieris will receive an upfront payment of CHF 6.5M (~$6.4M), research funding, regulatory- and sales-based milestones and mid-single to low-double-digit royalties on net sales. Total payments to Pieris could reach CHF 415M (~$409M), excluding royalties, if all milestones are met.
- Anticalins are engineered lipocalins, endogenous proteins found in blood plasma and other body fluids that naturally bind, store and transport a wide range of molecules. They potentially offer improved efficacy and more convenient routes of administration versus existing biologics.
Oct. 12, 2015, 10:44 AM
- Thinly traded nano cap Celsion (CLSN +9.2%) moves up on a healthy 10x surge in volume in response to its announcement of positive preclinical results for its GEN-1 IL-12 immunotherapy. Specifically, the combination of GEN-1 IL-12 with Roche's (OTCQX:RHHBY +0.7%) Avastin (bevacizumab) and J&J's (JNJ +0.9%) Doxil (doxorubicin HCl liposome injection) showed a greater than 98% reduction in tumor burden compared to the combination of Avastin and Doxil alone in an ovarian cancer cell line called SKOV3 implanted into immunocompromised mice.
- GEN-1 is an immunotherapy consisting of an interleukin-12 (IL-12) DNA plasmid vector encased in a nanoparticle delivery system. It is administered via an intraperitoneal (body cavity) injection.
- The company is currently enrolling patients in a Phase 1b trial called OVATION. A Phase 1/2 combination study should commence in mid-2016.
Sep. 18, 2015, 8:43 AM
- Thinly traded micro cap Tracon Pharmaceuticals (NASDAQ:TCON) is up 70% premarket on average volume in response to its announcement that a patient with an aggressive form of uterine cancer is experiencing an ongoing complete response after being treated with the company's investigational TRC105 in combination with Roche's (OTCQX:RHHBY) Avastin (bevacizumab). The results were presented at the 18th World Congress on Gestational Trophoblastic Diseases in Bali, Indonesia.
- The single patient was suffering from persistent and unresectable metastatic choriocarcinoma which is an aggressive form of gestational trophoblastic neoplasia (GTN), a rare form of uterine cancer. Under a compassionate use protocol, she was dosed with TRC105 10 mg/kg weekly, in combination with Avastin 10 mg/kg every other week. After 28 weeks of treatment (seven cycles), her beta human chorionic gonadotropin (bHCG), a reliable biomarker in choriocarcinoma, decreased from more than 3,000 IU/L to less than 1 IU/L from cycles four through seven, indicating a complete response.
- Choriocarcinoma is the second tumor type known to express high levels of a protein called endoglin, which plays a key role in angiogenesis (development of new blood vessels that "feed a tumor"). The other, angiosarcoma, also responds well to treatment with TRC105 and a VEGF inhibitor like Avastin.
- TRC105 is a novel antibody to endoglin.
- The company plans to initiate a Phase 2 study in GTN in Q4 and apply to the FDA for Orphan Drug status in GTN and soft tissue sarcoma by the end of the year.
Aug. 14, 2015, 7:16 AM
- Based on additional analyses of the data from a Phase 2a clinical trial in wet age-related macular degeneration, Avalanche Biotechnologies (NASDAQ:AAVL) decides not to proceed to Phase 2b with its lead product candidate AVA-101. Instead, it will conduct further preclinical studies to clarify the optimal dose and delivery of AVA-101 and AVA-201 compared to standard-of-care anti-VEGF protein therapy before advancing the best candidate back into the clinic. The process should be completed by the end of the year.
- The company's decision shouldn't be a surprise. The stock plummeted 40% in June after the company reported less-than-expected results from the Phase 2a study.
- The value proposition of AVA-101, a gene therapy that contains a gene that encodes a naturally occurring anti-VEGF protein, is a single subretinal injection compared intravitreal (inside the eye) injections as frequent as once per month for Regeneron's (NASDAQ:REGN) Eylea and Roche's (OTCQX:RHHBY) Lucentis.
- Shares are down 14% premarket on light volume.
- Previously: Avalanche Bio plummets 40% after hours on less-than-expected AVA-101 data; chances dim for pick up by Regeneron (June 15)
Jul. 13, 2015, 8:42 AM
- Results from a 20-subject Phase 2 trial assessing Ohr Pharmaceutical's (NASDAQ:OHRP) OHR-102 (squalamine lactate ophthalmic solution 0.2%) in patients with macular edema secondary to branch and central retinal vein occlusion show that treatment with a combination of OHR-102 and Roche's (OTCQX:RHHBY) Lucentis (ranibizumab injection) produced greater gains in visual acuity compared to Lucentis alone. At week 38, the mean gain in visual acuity from baseline in the combination arm was +27.8 letters compared to +23.3 letters for Lucentis. The difference was clinically meaningful but unclear as to statistical significance. The data were presented at the 2015 Annual Meeting of the American Society of Retina Specialists in Vienna, Austria.
- Shares are up 23% premarket on robust volume.
Jul. 1, 2015, 11:08 AM
- Exelixis (EXEL -7.7%) slumps on average volume in response to the FDA's decision to extend its action date for its review of Genentech's (OTCQX:RHHBY) New Drug Application (NDA) for cobimetinib to November 11 from August 11. The agency feels that the extension is necessary in order for it to review additional data from coBRIM, the Phase 3 trial of cobimetinib and vemurafenib (Zelboraf) in patients with BRAF V600 mutation-positive advanced melanoma. The additional data were requested by the regulator.
- Cobimetinib was discovered by Exelixis. Its collaboration with Genentech began in late 2006.The companies will co-promote the product in the U.S. while Genentech will promote it ex-U.S.
- Previously: Roche's melanoma combo NDA gets Priority Review (Feb. 19)
- Previously: Roche submits NDA for skin cancer med (Dec. 15, 2014)
Jun. 15, 2015, 6:52 PM
- Relatively recent IPO Avalanche Biotechnologies (NASDAQ:AAVL) craters 40% after hours on robust volume in response to its announcement of top-line results from a Phase 2a study assessing its lead product candidate, AVA-101, in patients with wet age-related macular degeneration (wet AMD). The primary endpoint of the trial was safety, but secondary efficacy measures have apparently disappointed investors despite the study not being powered to show statistical significance.
- The trial enrolled 32 wet AMD patients at least 55 years old. They were randomized to receive AVA-101 (n=21) or control (n=11). Both arms received two ranibizumab injections [Roche's (OTCQX:RHHBY) Lucentis] at day 0 and week 4. Almost all (n=29/32) had received prior anti-VEGF therapy with a median of 10 prior injections.
- The mean change in best corrected visual acuity (BCVA) from baseline in the AVA-101 cohort was +2.2 letters compared to -9.3 letters for control. In addition, 42.9% of patients (n=9/21) receiving AVA-101 improved or maintained stable vision with two or fewer rescue injections compared to 9.1% (n=1/11) for control. BCVA improvement of at least 10 letters with no more than two rescue injections occurred in 23.8% of the AVA-101 arm versus 0% in control.
- The modest average letter gain for the AVA-101 arm is substantially less than published studies of Eylea and Lucentis, which have demonstrated mean improvements in BCVA scores of 18.9 letters and 14.2 letters, respectively. If the difference persists in subsequent studies, then Regeneron will be less likely to take advantage of its right of first negotiation for the rights to AVA-101.
- The value proposition of AVA-101, a gene therapy that contains a gene that encodes a naturally occurring anti-VEGF protein, is a single subretinal injection compared intravitreal (inside the eye) injections as frequent as once per month for Regeneron's (NASDAQ:REGN) Eylea and Roche's Lucentis.
Jun. 2, 2015, 1:09 PM
- Thinly traded micro cap Cerulean Pharma (CERU -12.3%) moves south on 50% higher volume, albeit on turnover of only 430K shares, in response to the presentation of data (abstract #4543) at ASCO from a Phase 1b/2 trial assessing its lead product candidate, CRLX101, in combination with Roche's (OTCQX:RHHBY -0.4%) Avastin (bevacizumab), for the treatment of patients with metastatic renal cell carcinoma (mRCC).
- The study met its primary endpoint of at least half of the patients achieving progression-free survival (PFS) of four months or greater (n=12/22). Median PFS was 9.9 months. Overall response rate (ORR) was 23%. Average durability of response was 3.5 months from partial response to time off the study.
- The source of investors' lack of enthusiasm over the data may its comparison with Roche's AVOREN study that assessed bevacizumab in combination with interferon alfa (IFN) in mRCC. Median PFS was 10.2 months for Avastin + IFN compared to 5.4 months for placebo + AFN while ORR was 30% for Avastin + IFN versus 12% for placebo + IFN.
- CRLX101 is nanoparticle-drug conjugate (NDC) that delivers highly toxic camptothecin directly to cancer cells.
Jun. 2, 2015, 11:56 AM
- Puma Biotechnology (PBYI -13.3%) drops again on 3x normal volume as investors continue to head for exits in response to less-than-expected results from a Phase 3 study, called ExteNET, that assessed its lead product candidate, PB272 (neratinib) versus placebo after adjuvant treatment with Roche's (OTCQX:RHHBY -0.6%) Herceptin (trastuzumab) in women with early stage HER2-positive breast cancer. The data were presented yesterday (abstract #508) at ASCO. Shares have slumped 26% since then.
- The ExteNET trial randomized 2,840 patients in 41 countries with HER2+ breast cancer who had undergone surgery and adjuvant (following) treatment with Herceptin. The two treatment arms were neratinib and placebo.
- The primary endpoint was invasive disease-free survival (DFS). Patients treated with neratinib showed a 33% lower risk of invasive disease recurrence or death compared to placebo (p=0.009), but there was only a modest separation in the two-year DFS rates: 93.9% for neratinib versus 91.6% for placebo.
- The secondary endpoint was disease-free survival including ductal carcinoma in situ (DFS-DCIS). Patients receiving neratinib showed a 37% reduction in risk of disease recurrence including DCIS or death versus placebo (p=0.002) but the difference in 2-year DFS-DCIS rates was, again, slight: 93.9% for neratinib versus 91.0% for placebo.
- Subgroups of patients whose tumors were assessed in a single reference location and those positive for hormone receptor (HR) showed higher reductions in risk of recurrence and greater differences in DFS compared to placebo.
Jun. 1, 2015, 8:56 AM
- Micro cap Vascular Biogenics (NASDAQ:VBLT) is up 11% premarket on robust volume in response to its announcement of positive interim results from a Phase 2 study assessing its lead product candidate, VB-111, in patients with recurrent glioblastoma (rGBM), one of the most aggressive primary brain tumors. The data showed a statistically significant benefit in overall survival (OS) in patients treated with VB-111 followed by VB-111 in combination with Roche's (OTCQX:RHHBY) Avastin (bevacizumab) upon disease progression compared to patients treated with VB-111 followed by bevacizumab alone (p=0.05). The full results were presented earlier this morning at a meeting for investors in conjunction with ASCO.
- 46 patients with rGBM were treated with VB-111. Upon disease progression, 23 were treated with VB-111 + bevacizumab and 22 received bevacizumab alone (one patient was stable on VB-111 alone after 18 months). Median OS in the combination arm was 16 months compared to eight months in the bevacizumab alone arm (p=0.05).
- VB-111 is an anti-angiogenic agent which is designed to restrict tumor growth by inhibiting the proliferation of new blood vessels that form to support rapidly growing cells.
- The company intends to initiate a Phase 3 trial later this year.
May 29, 2015, 5:24 PM
- Bristol-Myers Squibb (NYSE:BMY) stumbled 7% today on a 4.5x surge in volume in response to the presentation of an abstract (#109) at ASCO that underwhelmed many investors. The data were from Phase 3 study, called CheckMate-057, assessing the company's PD-1 inhibitor, Opdivo (nivolumab) compared to the standard-of-care chemotherapy agent docetaxel in treatment-experienced patients with advanced, non-squamous non-small cell lung cancer, the most common form of lung cancer. A drug used after another (treatment experienced) is called a second line setting.
- The study met its primary endpoint of a statistically significant 27% reduction in the risk of cancer progression or death compared to docetaxel (p=0.0015). The trial also evaluated the efficacy of Opdivo compared to docetaxel by the level of PD-L1 expression, 1%, 5% and 10%. As expected, the median overall survival (OS) in the Opdivo group was significantly longer than the chemo cohort for all three levels where PD-L1 was highly expressed (greater than or equal to 1, 5, 10%).
- What raised eyebrows was the absence of a treatment benefit compared to docetaxel in the three groups with low PD-L1 expression (<1%, <5%, <10%). Median OS was barely higher in the Opdivo group with <1% expression and lower than docetaxel in the other two. This potentially opens the door for eventual competitors such as Roche (OTCQX:RHHBY) and Merck (NYSE:MRK) whose offerings show strong efficacy in tumors that highly express PD-L1. Had Opdivo shown a significant OS advantage in low PD-L1 expression tumors, its dominance in the second line setting would have been virtually assured.
- CheckMate-057 was stopped early based on the successful achievement of its primary endpoint, per the recommendation of the independent Data Monitoring Committee.
- Previously: Phase 3 study stopped early after Bristol-Myers' Opdivo hits efficacy endpoint (April 17)
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