This week, focus was on data presented at the annual conference of the American Society of Hematology ("ASH") from December 9-12 with some companies emerging winners while others lost ground on disappointing data.
Recap of the Week’s Most Important Stories
ASH Roundup: Several companies were at ASH with data on approved and pipeline drugs. While some companies soared on data, others were hit by disappointing results.
One of the companies that scored a win was bluebird BLUE, which along with partner Celgene CELG, announced updated results from an ongoing phase I study of bb2121 anti-BCMA CAR T cell therapy in patients with late stage relapsed/refractory multiple myeloma. The companies said that the overall response rate (“ORR”) was 94% in patients in active dose cohorts while a complete response (“CR”) rate of 56% observed in patients in active dose cohorts. Bluebird’s shares shot up 15.2% while Celgene gained 1.8% (Read more: Bluebird's Shares Jump on Strong Data for CAR-T Therapy). bluebird is a Zacks Rank #3 (Hold) stock. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Kite, a Gilead GILD company, was also there at ASH with updated results from the ongoing phase I/II ZUMA-3 study of KTE-C19, a CAR T cell therapy, for the treatment of adult patients with relapsed or refractory acute lymphoblastic leukemia (“ALL”). High rates of response were observed – 71% of ALL patients on KTE-C19 achieved complete tumor remission. Long-term data was also presented on Yescarta from the pivotal ZUMA-1 study in patients with refractory large B-cell lymphoma. 42% of patients continued to respond to therapy including 40% in complete remission, at a median follow-up of 15.4 months. Yescarta was approved in the United States recently and is currently under review in the EU with potential approval expected in the first half of 2018 (Read more: Gilead Posts Updated Data on CAR-T Therapy Yescarta).
Juno Therapeutics JUNO, another major name in the CAR T category, provided additional data from the TRANSCEND study on JCAR017 in patients with relapsed or refractory aggressive B-cell non-Hodgkin lymphoma. Although data looked good, shares declined reflecting investor expectation for better results. The company expects to finish submitting a regulatory application in the United States in the second half of 2018, with approval expected as early as the end of 2018 (Read more: Juno Reports Additional Data on CAR-T Therapy, Shares Drop).
Another company that was hit by ASH data is Spark Therapeutics ONCE which saw it shares plunging 34.9% on preliminary data on its investigational hemophilia A treatment, SPK-8011 (Read more: Spark Therapeutics Stock Plunges on Hemophilia Therapy Data).
Gilead to Buy Cell Design Labs, Strengthen Cellular Therapy Efforts: Gilead announced its next acquisition deal which comes a few months after the company acquired Kite Pharma for approximately $11.9 billion. This time round, Gilead is acquiring Cell Design Labs, Inc., with the deal expected to add new technology platforms to Gilead’s portfolio. Gilead will shell out up to $567 million including an upfront payment of approximately $175 million. Additional payments of up to $322 million will be made on the occurrence of certain events, including the achievement of development and approval milestones. With this acquisition, Gilead is looking to enhance its R&D efforts in cellular therapy (Read more: Gilead Sciences to Acquire Cell Design Labs for $567 Million)).
Vertex Selects First Gene Edited Treatment under CRISPR Collaboration: Vertex Pharmaceuticals VRTX announced the selection of CTX001 as the first gene edited treatment that will be co-developed and co-commercialized under its agreement with CRISPR Therapeutics. The companies will split all R&D costs and profits worldwide. The plan is to commence a phase I/II study in Europe for β-thalassemia in 2018 while an Investigational New Drug (“IND”) application will be submitted in the United States to support the commencement of a phase I/II study in sickle cell disease.
Meanwhile, on the cystic fibrosis (“CF”) front, Vertex said that Kalydeco met the primary endpoint in an open-label phase III study in children (1 to 2 years of age) with CF. Vertex expects to file for approval in the United States and the EU for this patient population in the first quarter of 2018 (Read more: Vertex Kalydeco Study Successful in Children Aged 1-2 Years). Vertex’s shares are up 91.3% year to date, compared to the 3% gain recorded by the industry it belongs to.
Repros to be acquired by Allergan: Repros Therapeutics saw its shares gaining 41.5% on news that it will be acquired by Allergan for a cash payment of 67 cents per share. The acquisition is expected to close in the first quarter of 2018. With this acquisition, Allergan is looking to boost its women’s health pipeline. However, Repros’s Proellex ran into trouble with the program being placed on partial clinical hold, and the FDA asking for a large pre-approval safety data base to support future development.
Medical - Biomedical and Genetics Industry 5YR % Return
Medical - Biomedical and Genetics Industry 5YR % Return
The NASDAQ Biotechnology Index gained 2.4% over the last five trading sessions. Among major biotech stocks, Celgene gained 6.3% while Amgen declined 1.4%. Over the last six months, Biogen was up 19.2% while Regeneron REGN lost 19.9% (See the last biotech stock roundup here: FDA Nod for Amgen PCSK9 Inhibitor, Revance Up on RT002 Data).
What's Next in the Biotech World?
Watch out for the usual pipeline and regulatory updates.
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Vertex Pharmaceuticals Incorporated (VRTX) : Free Stock Analysis Report
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