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Monday, October 8, 2018

Lilly Gains on Upbeat Drug Data and an Upgrade


Eli Lilly stock is trading higher Friday, after touching all-time highs Thursday on positive data about its diabetes drug Empagliflozin, on the heels of positive analyst commentary.
Where we were: Eli Lilly is up nearly 36% since the start of the year, and got a boost from a trial that showed Empagliflozin met its primary end points.
Where we’re headed: Analysts are bullish on the stock’s ability to keep outperforming.
On Thursday, Eli Lilly (LLY) rose to an all-time intraday high (even though it did slip from them by the close) after the diabetes data. The stock saw more gains Friday, following an upgrade from BMO Capital Markets.
Analyst Alex Arfaei upgraded Eli Lilly to Outperform from Market Perform, with a $130 price target, writing that he’s more confident in company’s portfolio of drugs. He expects that Eli Lilly’s GLP-1 class of diabetes drugs will notch a compounded annual growth rate of 15% between this year and 2027, and that its Trulicity diabetes franchise can reach $11 billion in sales, which should help expand margins.
Arfaei is also bullish on pain drug Tanezumab, developed with Pfizer (PFE), and argues that investors aren’t assigning much value to Eli Lilly’s Alzheimer’s franchise. That makes the stock attractive, even with its year-to-date rally, he argues.
Nor is he the only analyst who likes Eli Lilly’s prospects. JPMorgan’s Chris Schott reiterated an Overweight rating and raised his price target on Eli Lilly to $123 from $117 Friday. He cites the upbeat diabetes data for his estimate increase, and writes that the stock “remains one of our favorite names in the group.”
He believes that the data suggest potentially a “best-in-class” profile for the drug, and it could become a multi-billion dollar product. Schott too believes that the stock can keep climbing despite its year-to-date run, given that Eli Lilly can generate mid-single digit sales growth and a low-teen earnings per share compound annual growth rate for “much of the next decade.”
Eli Lilly is up 1.7% to $114.89, while the SPDR S&P Pharmaceuticals ETF(XPH) is down 0.2% to $45.92, and the Health Care Select Sector SPDR ETF(XLV) is down 0.2% to $94.24.

Novartis in FDA, EMA filing acceptance of MS med application


Novartis International AG / Novartis announces FDA and EMA filing acceptance of siponimod, the first and only drug shown to meaningfully delay disability progression in typical SPMS patients . Processed and transmitted by West Corporation. The issuer is solely responsible for the content of this announcement.
  • There is a critical need for safe and effective treatments for secondary progressive multiple sclerosis (SPMS) – a highly debilitating form of MS characterized by gradual, irreversible worsening of disability, largely independent of relapses 
  • If approved, siponimod (BAF312) would be the first oral disease-modifying therapy with the potential to delay progression and expand possibilities for SPMS patients 
  • Filings are supported by Phase III EXPAND data, which showed siponimod had beneficial effects on disability, relapses and magnetic resonance imaging (MRI) disease activities in typical SPMS patients[1]
  • Novartis used a priority review voucher to expedite review of siponimod in the US to ensure patients could benefit from the drug as soon as possible, pending approval.

Sunday, October 7, 2018

Alnylam’s Onpattro highly efficacious, safer than competitor, says Piper Jaffray


Piper Jaffray analyst Edward Tenthoff notes that Ionis Pharmaceuticals (IONS) announced FDA approval of TEGSEDI for treatment of hATTR polyneuropathy. The analyst also points out that the label includes a Black Box warning for thrombocytopenia and glomerulonephritis requiring every 2-week monitoring, and will be marketed by Ionis subsidiary Akcea (AKCA) with a Risk Evaluation and Mitigation Strategy. TEGSEDI is administered subcutaneously every week versus Alnylam’s (ANLY) ONPATTRO intravenously every 3 weeks with steroids, he adds. Tenthoff believes that ONPATTRO appears safer and highly efficacious, and should win out. The analyst reiterates an Overweight rating and $160 price target on Alnylam shares.

PTC Therapeutics announces initial data from patient registry


PTC Therapeutics announced preliminary data from the first international drug registry for Duchenne patients receiving Translarna, underscoring the long-term clinical benefit of Translarna when used in routine clinical practice in delaying irreversible muscle loss in children with Duchenne caused by a nonsense mutation, when compared with published natural history. The data show that children and adolescents receiving Translarna in the real-world setting are continuing to walk years longer than untreated children, and are staying more physically able. A time-to-event analysis for loss of ambulation has shown that patients on Translarna had a median age of loss of ambulation of 16.5 years of age – up to 5 years later than seen with natural disease progression in untreated children. The data were presented as a late breaker at the 23rd International Annual Congress of the World Muscle Society in Argentina. Patients who received Translarna in routine clinical practice also experienced a slower decline in their physical function compared with the placebo arm of Phase 3 Study 020, as measured by a series of timed function tests. Safety outcomes for patients in the STRIDE Registry were consistent with the known safety profile of Translarna. The analysis was based on data captured from 216 patients, the majority of whom had not been previously enrolled in an ataluren clinical trial, across 11 European countries and Israel. Patients had a mean age of 9.8 years at first assessment and had been diagnosed with Duchenne caused by a nonsense mutation at approximately five years of age. Almost 90% had previously or were still receiving corticosteroids.

Loxo Oncology announces LOXO-292 durability update in RET-Mutant MTC


Loxo Oncology announced updated interim clinical data for LOXO-292 from the global Phase 1/2 LIBRETTO-001 trial in patients with RET-mutant medullary thyroid cancer and RET fusion-positive thyroid cancer who were initially included in the LOXO-292 presentation at the 2018 ASCO Annual Meeting. In these 38 patients, approximately 3.5 months of additional patient follow-up were available, as were first follow-up scans for the nine patients most recently enrolled. Sixteen of 17 responding RET-mutant MTC patients remained on therapy, with median follow-up of 8.4 months. Seven of seven responding RET fusion-positive thyroid cancer patients remained on therapy, with median follow-up of 8.5 months. Inclusion of new restaging data for the most recently enrolled patients resulted in a 59% overall response rate in the presented subset of RET-mutant MTC patients, and a 78% confirmed overall response rate in the presented subset of RET fusion-positive thyroid cancer patients. These data were presented at the 88th Annual Meeting of the American Thyroid Association. The data presented were based on a July 19, 2018 data cut-off date and included the 29 patients with RET-mutant MTC and the nine patients with RET fusion-positive thyroid cancer who were initially included in the LOXO-292 presentation at the 2018 ASCO Annual Meeting. Patients were heavily pretreated, having received a median of three prior systemic treatment regimens. Of the patients with RET-mutant MTC, 79% had previously received cabozantinib or vandetanib and 45% had received prior treatment with both agents. Of the patients with RET fusion-positive thyroid cancer, 78% had previously received radioactive iodine and 78% had previously received sorafenib or lenvatinib. Anti-tumor activity was observed regardless of RET mutation, RET fusion partner, and prior multikinase inhibitor treatment. One patient, with RET fusion-positive thyroid cancer, had RECIST target lesions in the central nervous system and exhibited an intracranial partial response by RECIST 1.1, pending confirmation. Of the 82 patients in the safety analysis, most treatment-emergent adverse events were Grade 1 in severity and judged by the investigator as not related to LOXO-292.

Loxo Oncology’s 292 remains on top after RET data updates, says Piper Jaffray


Piper Jaffray analyst Tyler Van Buren reiterates an Overweight rating and $200 price target for Loxo Oncology (LOXO) after the company provided an update of the thyroid patients in the Phase I/II study of LOXO-292. The analyst highlights that in the largest patient population the response rate increased from 45% to 59% with three additional patients responding between 9 and 12 months. Blueprint Medicines’ (BPMC) competing BLU-667 reported a 49% ORR in a similar patient population and recall that adverse events of hypertension are still a concern, he notes. Van Buren believes that the 667’s safety profile could continue to look worse as it continues to administer the drug to patients at higher doses and that treatment discontinuations could increase. Overall, the analyst believes Loxo’s 292 appears to be the superior RET inhibitor in development, both in terms of efficacy and safety and could easily become a $1B+ drug.

Poor Hospital Compliance With Patient Record Requests


US hospitals continue to place cost and processing obstacles in the way of patients requesting their personal medical records, according to a study published online October 5 in JAMA Network Open.
The study highlights the need for tougher enforcement of state and federal policies on the accessibility of protected health information to ensure a smooth, timely, and affordable process for patients, write Carolyn T. Lye, BA, a medical student at Yale School of Medicine, New Haven, Connecticut, and colleagues.
In its survey of 83 top hospitals across 29 states, the study reports that many facilities do not comply with the Privacy Rule of the Health Insurance Portability and Accountability Act (HIPAA) of 1996. This federal law guarantees that patients have access to their protected health information within 30 days in their preferred format and at a reasonable charge.
The investigators evaluated adherence to HIPPA guidelines through prescripted telephone interviews conducted with one person at each of the hospitals’ medical records departments from August 1 to December 7, 2017. The elite hospitals were ranked as the top 20 for each of the 16 adult specialties in the 2016-2017 US News & World Report’s Best Hospitals National Ranking.
The survey found contradictory information about records entitlement, varying release times, and higher-than-recommended fees.
“There were overwhelming inconsistencies in information relayed to patients regarding the personal health information they are allowed to request, as well as the formats and costs of release, both within institutions and across institutions,” Lye said in a JAMA news release. “We also found considerable noncompliance with state and federal regulations and recommendations with respect to the costs and processing times associated with providing access to medical records.”
In 2017, a report from the US Department Health and Human Services’ Office of the National Coordinator for Health Information Technology revealed barriers to patients’ obtaining their medical records.
Although all 83 facilities in the study communicated by telephone that they could release a patient’s entire medical record, only 53% explicitly stated this on their paper authorization forms, and only 11% stated on forms that they could release physicians’ orders.
Discrepancies about available formats emerged in telephone statements vs statements on forms, pointing to noncompliance with federal regulations that require release in a patient’s preferred format. There was a phone-vs-form discordance of 83% vs 48% on the option of picking up the records in person and of 24% vs 17% for transmission by fax. For other release options, the phone-form discrepancies were 47% vs 33% for email, 66% vs 42% for CDs, and 25% vs 40% for online patient portals. There was no discrepancy for release by regular mail (100% both).
As for fees, 43% of hospitals provided no fee schedule on the authorization form or on the webpage for downloading the form. Whereas some hospitals charged no processing fees, 48 (59%) charged costs higher than the Office for Civil Rights recommendation of a flat fee of $6.50 for electronically maintained records — in one case charging $541.50 for a 200-page record.
In the telephone interviews, 82 hospitals disclosed costs for a paper release, and one was unable to comment because costs were calculated by an external party. Of the 29 hospitals that disclosed fees on authorization forms, nine (31%) charged the same fees as those disclosed by telephone, 10 (34%) charged less, three (10%) charged more, and seven (24%) charged nothing.
On the issue of timing, 71 hospitals provided mean times of release for paper records in telephone interviews, with the time ranging from same day to 60 days. Of the hospitals that responded with processing times, seven centers were not compliant with government requirements.
Ten institutions gave a maximum time of release, whereas two could not specify either a mean or maximum. Of those supplying processing time information, 17 (21%) reported mean times of fewer than 7 days, 21 (25%) of 7 to 10 days, 26 (31%) of 11 to 20 days, four (5%) in 21 to 30 days, and three (4%) of more than 30 days. Almost 15% were “unable to say.” Release times were generally shorter for electronic records than for paper records.
“As legislation, including the recent 21st Century Cures Act and government-wide initiatives such as MyHealthEData continue to stipulate improvements in patient access to medical records, attention to the most obvious barriers should be paramount,” the authors write.
“We are also in an era in which patients are participants in their own health care. Inhibiting access for patients to their own medical records with complicated, lengthy, and costly request processes prevents patients from obtaining information that they may need to better understand their medical conditions and communicate with their physicians,” Lye added in the news release.
The authors’ relevant financial relationships are listed in the original article.
JAMA Network Open. Published online October 5, 2018. Full text