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Dr De's Opening Thoughts: Bad medicine
That's a quaint way to describe a medicine prescribed for children as safe against all evidence to the contrary, and which can lead to dangerous side-effects. This is GlaxoSmithKline's (GSK) paroxetine, a drug prescribed as an antidepressant since the 1990s. The famous, or infamous, study of 329 found that the drug had little benefit compared to placebo, and led to increased suicidal tendencies in children and adolescents. GSK paid a few million dollars in fines after facing extensive litigation and controversy.
I was recently reading an article about the Indian medical scenario vis-a-vis biosims, and the kind of things that happen is stunning, almost criminal. Apparently, the guidance document from the agency responsible for overlooking the approval process was written in conjunction with the medical industry, notably Biocon, Roche and Dr. Reddy's. If the article is right, the guidance is lax in quite a few respects, and standards have been made lower than they should be for large-molecule drugs that can contain upto 20,000 atoms per molecule.
Focus Ticker: Geron
Geron (NASDAQ:GERN) has achieved a major milestone, as the first patient enrolled in the Phase II trial to evaluate Imetelstat in patients with myelofibrosis (MF) was dosed. The clinical trial called IMbark[TM] is being conducted by Geron's partner Janssen Biotech (JNJ).
In little over a year, Geron has seen a remarkable turnaround. Imetelstat was placed on a clinical hold last year, a development that sparked a huge sell-off in Geron shares. GERN had been trading at around $5 when the clinical hold was placed. The development sent the stock below $2. While Geron shares have since recovered, they are still trading well below the pre-clinical hold levels. Considering that there is a higher probability of Imet reaching commercialization stage now than in March 2014, the current stock price is certainly not justified.
The Phase II trial has been designed to enroll approximately 200 patients with DIPSS Intermediate-2 or high-risk MF, who have relapsed after or are refractory to JAK inhibitor treatment. Unlike the current SOC for MF, Incyte's (INCY) Jakafi, Imet has shown potential to cure MF. This is why Geron's development and commercialization partner Janssen has huge expectations from the drug. Earlier this year, Janssen said that it saw Imet as one of the top drugs in its pipeline, with the potential to achieve blockbuster status.
From current levels, I see at least 40%-50% upside in GERN shares in the near term, as Imet trial continues to progress. But even after these gains, the stock would have only recovered to the levels seen in March 2014 when the FDA first placed a clinical hold.
Sector News
Amgen Acquires Dezima Pharma - Amgen (AMGN) announced the acquisition of privately held Dezima Pharma B.V., a Netherlands-based biotechnology company focused on the development of innovative treatments for dyslipidemia. Dezima's lead molecule, TA-8995, is an oral, once-daily cholesteryl ester transfer protein (CETP) inhibitor. As per the terms of the agreement, Amgen will pay $300 million in cash at completion of the transaction. In addition, Dezima is entitled to up to $1.25 billion in milestone payments.
Amgen Announces Strategic Collaboration with Xencor - Amgen and Xencor (XNCR) announced that they have entered into a research and license agreement to develop and commercialize novel therapeutics in the areas of cancer immunotherapy and inflammation. The research collaboration brings together Amgen's capabilities in target discovery and protein therapeutics with Xencor's XmAb[R] biospecific technology platform. The agreement includes molecular engineering by XNCR and the preclinical development of biospecific molecules for five programs proposed by Amgen, leveraging XmAb bispecific Fc domains to make half-life extended T cell engagers and dual targeting bispecific antibodies. It also includes a preclinical bispecific T cell engager program directed at CD38 and CD3 for multiple myeloma. As per the terms of the agreement, Amgen will make an upfront payment of $45 million. Xencor will be also entitled to up to $1.7 billion in clinical, regulatory and sales milestone payments in total for the six programs.
Opdivo Granted Breakthrough Therapy for Advanced Kidney Cancer - Bristol-Myers Squibb's (BMY) Opdivo has been granted a Breakthrough Therapy by the FDA for the treatment of advanced or metastatic renal cell carcinoma (RCC). The Breakthrough Therapy designation has been granted based on the results of Phase III study called CheckMate-025.
AbbVie Plans to Proceed to Phase III with Elagolix - AbbVie (ABBV) announced the completion of a Phase 2b clinical trial investigating the safety and efficacy of its elagolix as a monotherapy or in combination with add-back therapy compared to placebo in women with heavy menstrual bleeding (HMB) associated with uterine fibroids. Based on preliminary positive results, AbbVie is planning to proceed to Phase III evaluation of elagolix in patients with uterine fibroids. The Phase III program is expected to commence in the first quarter of 2016.
Baxalta Not Interested in Defensive Deal - Baxalta (BXLT) CFO Bob Hombach has said that the company does not plan to rush into a defensive deal in order to thwart Shire (SHPG). Shire has proposed to acquire Baxalta. Speaking during a presentation webcast from the Morgan Stanley Global Healthcare Conference, Hombach said that while the company was interested in doing strategic deals, it would not rush into one just to thwart an unsolicited takeover offer.
Aerie Pharmaceuticals Shares Soar on Positive Results - Aerie Pharmaceuticals (AERI) shares surged more than 77% in after-hours trading on Wednesday after the company reported positive results from its Phase III trial for Rhopressa. A novel once-daily, triple-action eye drop, Rhopressa, is being evaluated for its ability to lower intraocular pressure.
Intra-Cellular Surges on Positive Top-Line Results - Intra-Cellular Therapies (ITCI) surged more than 86% in regular trading on Wednesday after the company announced positive results from the Phase III clinical trial of ITI-007 for the treatment of patients with schizophrenia. The trial met its primary endpoint and demonstrated antipsychotic efficacy with statistically significant superiority over placebo at Week 4 as measured by the change from baseline on the Positive and Negative Syndrome Scale (PANNS) total score.
Mirna Therapeutics Appoints New CFO - Mirna Therapeutics announced the appointment of Alan Fuhrman as its CFO. Fuhrman has more than two decades of executive leadership experience in the life sciences and technology industries. Most recently, he was the CFO of Ambit Biosciences.
Drug Approvals
GlaxoSmithKline had a labeling revision for Breo Ellipta, a combination of fluticasone furoate, an inhaled corticosteroid ('ICS), and vilanterol, a long-acting beta2-adrenergic agonist ('LABA), indicated for long-term, once-daily, maintenance treatment of airflow obstruction and reducing exacerbations in patients with chronic obstructive pulmonary disease ('COPD). It is also indicated for once-daily treatment of asthma in patients aged 18 years and older. Jointly developed with Theravance (THRX), which will receive a 15% royalty for sales up to $3 billion, Breo started slow, with $110 million in sales last year, but analysts expect is to cross the $1 billion mark by 2020. Breo comes with warning of asthma-related deaths.
Jazz Pharma (JAZZ) had a labeling revision and a supplement for Versacloz, an atypical antipsychotic indicated for treatment-resistant schizophrenia. No sales data available. It comes with warnings of Severe Neutropenia, Orthostatic Hypotension, Bradycardia, and Syncope, seizure, Myocarditis and Cardiomyopathy.
Impax Labs (IPXL) had an ANDA approval for pyridostigmine bromide, used to treat muscle weakness in people with myasthenia gravis. The market for the drug was approximately $20 million.
Salix, now an unit of Valeant (VRX), had a manufacturing addition for Osmoprep, an osmotic laxative indicated for cleansing of the colon as a preparation for colonoscopy in adults 18 years of age or older. In 2008, a year after approval, it had sales of $30 million.
Trials and Results
No trials and results to report.
Deals
Illumina to Collaborate with MSK - Illumina (ILMN) and Memorial Sloan Kettering Cancer Center (MSK) have entered into a collaboration to conduct research studies that are critical to understanding the biology of circulating tumor DNA (ctDNA). MSK and ILMN will conduct research trials designed to answer the biological and clinical questions about cDNA in multiple cancer types.
NovaBay Signs Agreement with ALLDocs - NovaBay (NBY) has signed an agreement under which its prescription daily eyelid and lash hygiene product Avenova will be marketed through ALLDocs Optometry Group. ALLDocs in the second-largest independent optometry group in the U.S.
Cesca Therapeutics Announces Collaboration - Cesca Therapeutics (KOOL) has announced a collaboration with Center for Immune Cell Therapies (CICT) at Memorial Sloan Kettering Cancer Center (MSK) focused on the CellWerks[TM] portfolio of devices for intra-laboratory stem cell purification applications. As per the terms of the agreement, CICT will use Cesca's newest evolution of the AutoXpress[R] platform under the company's Early Technology Access Program.
Secondary Offerings
Juniper Pharmaceuticals Files Form S-3 - Juniper Pharmaceuticals (JNP) filed a Form S-3 with the U.S. Securities and Exchange Commission. As per the filing, the company may from time to time offer, issue and sell together or separately the securities noted in the prospectus. The aggregate of the offering prices of the securities covered by the prospectus will not exceed $100 million.
IPOs
REGENXBIO Raises IPO Price - REGENXBIO has increased its proposed IPO price from a range of $17-$19 to $22. The company now plans to raise $122 million. Rockville, Maryland-based REGENXBIO is offering 5.6 million shares in its proposed IPO.
Earnings
No earnings to report.
Insider Transactions
BioMarin CEO Sells - Jean Jacques Bienaime, CEO at BioMarin Pharmaceutical (BMRN), sold 1,000 shares at $131.43. The total value of the transaction was $131,432.
Immune Pharmaceuticals Insiders Buy - Rene Pierre Azria, director at Immune Pharmaceuticals (IMNP), bought 50,000 shares at $1.48. The total value of the transaction was $73,850. Daniel Kazado, also a director at the company, bought 50,000 shares at $1.47. The total value of the transaction was $73,480.
Otonomy CEO Sells - David Allen Weber, President & CEO at Otonomy (OTIC), sold 5,216 shares at $24.95. The total value of the transaction was $130,139.
RadNet Insider Sells - Norman R. Hames, Executive Vice President at RadNet, sold 40,000 shares at $6.04. The total value of the transaction was $241,550.
Acceleron Insiders Sell - Steven D. Ertel, EVP & COO at Acceleron (XLRN), sold 2,000 shares at $33.61. The total value of the transaction was $67,220. John D. Quisel, SVP Gen. Counsel & Secretary at the company sold 5,500 shares at $33.35. The total value of the transaction was $183,425.
Immune Design Insider Sells - Wayne Gombotz, Chief Development Officer at Immune Design (IMDZ), sold 5,000 shares at $16.19. The total value of the transaction was $80,955.
Pluristem CEO Buys - Zami Aberman, Chairman & CEO at Pluristem Therapeutics (PSTI), bought 25,000 shares at $2.11. The total value of the transaction was $52,750.
China Biologic Insider Sells - Ming Yang, CFO at China Biologic (CBPO), sold 20,304 shares in two separate transactions. Yang sold 9,000 shares at $94.56 and 11,304 shares at $97.08.
Big Movers
NYSE - InVitae (NVTA) shares were among the major movers on the NYSE. The stock closed 6.26% lower.
NASDAQ - Intra-Cellular Therapies was one of the biggest biotech gainers on the NASDAQ. The stock closed 86.86% higher. Collegium Pharmaceutical (COLL) shares closed 16.24% higher. Xencor shares ended the day 14.98% higher. KalaBios shares closed 8.38% lower. Acceleron Pharma closed 7.46% lower.
NYSEMKT - NovaBay shares were among the major movers on the NYSEMKT. The stock closed 6.02% lower. Organovo (ONVO) shares tumbled 9.86% in after-hours trading on Wednesday. The stock closed 0.85% higher in regular trading after rallying on Tuesday.
OTC - Anavex Life Sciences (AVXL) shares were among the major movers on the OTC market. The stock closed 6.82% lower.
Editor's Note: This article covers one or more microcap stocks. Please be aware of the risks associated with these stocks.
