Hikma reaffirms full-year revenue forecast, cites better generics business

Hikma Pharmaceuticals Plc on Friday reaffirmed its revenue guidance for the full year, noting its generics business was performing better due to a favourable product mix, despite continued pricing pressure in the United States.

Shares of the company were up as much as 4 percent at the market open before settling up 0.7 percent at 0725 GMT.

The drugmaker, which was forced to cut revenue guidance for its generics business three times in 2017, said it continued to expect revenue from the business to be between $550 million (407 million pounds) and $600 million in 2018.

Sigurdur Olafsson, former generics chief at drug giant Teva Pharmaceuticals, was named Hikma’s chief executive officer in February.

The company suffered a setback in March for its generic version of GlaxoSmithKlineblockbuster lung drug Advair, after the U.S. Food and Drug Administration asked Hikma to conduct a further clinical study evaluating the drug, dashing hopes to get it to the market this year.

“The study is proceeding as planned and we expect to submit a response to the FDA with the new clinical data as early as possible in 2019”, the company said on Friday.

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Genentech’s Alecensa Beats Out Pfizer’s Xalkori in Late-Stage Lung Cancer Trial

Genentech, a Roche company, released follow-up data from its Phase III ALEX trial comparing its Alecensa (alectinib) to Pfizer‘s Xalkori (crizotinib) in late-stage non-small cell lung cancer (NSCLC).

The results showed that Alecensa significantly cut the risk of disease progression or death by 57 percent compared to Xalkori after two years of follow-up in individuals with anaplastic lymphoma kinase (ALK)-positive metastatic (advanced) NSCLC. The median progression-free survival (PFS) for patients on Alecensa more than tripled compared to those on Xalkori. The PFS for the Alecensa group was 34.8 months compared to 10.9 months for the Xalkori group.

“Follow-up results from the ALEX study demonstrate the significant sustained benefit of Alecensa, showing that people with metastatic ALK-positive non-small cell lung cancer lived for almost three years without their disease progressing,” said Sandra Horning, Roche/Genentech’s chief medical officer and head of Global Product Development, in a statement. “These results further support the use of Alecensa as a standard of care for people who are newly diagnosed with this form of lung cancer.”

Alecensa is currently approved by the U.S. Food and Drug Administration (FDA) to treat ALK-positive metastatic NSCLC as detected by an FDA-approved clinical diagnostic test.

The ALEX trial is a randomized, multicenter, open-label Phase III trial comparing Alecensa to Xalkori in treatment-naïve patients with ALK-positive NSCLC which was diagnosed by the Ventana ALK (D5F3) CDx Assay, a companion immunohistochemistry test developed by Roche Tissue Diagnostics. Patients were randomized one-to-one to receive either of the drugs. The primary endpoint was PFS as assessed by the investigator. Secondary endpoints included Independent Review Committee (IRC)-assessed PFS, time to CNS progression, objective response rate (ORR), duration of response (DOR) and overall survival (OS).

The Roche trial involved 303 patients at 161 sites in 31 countries. The data will be presented at the 2018 American Society of Clinical Oncology (ASCO) Annual Meeting on Sunday, June 3 at 8:00 to 11:30 a.m. CDT.

Genentech also released positive results from its Phase III IMpower 150 trial of Tecentriq (atezolizumab) and Avastin (bevacizumab) plus carboplatin and paclitaxel (chemotherapy) for first-line treatment of patients who had not received chemotherapy with metastatic NSCLC. Interim data analysis indicated that the combination helped people live significantly longer compared to Avastin plus carboplatin and chemo.

“The IMpower 150 study results showed a significant survival benefit, adding to the clinical evidence supporting the combination of Tecentriq and Avastin as an initial treatment for metastatic non-squamous non-small cell lung cancer,” Horning said in a statement. “An overall survival benefit was also observed in key populations such as people with EGFR- and ALK-positive mutations and those with liver metastases. We are working with health authorities around the world to bring this potential Tecentriq combination regimen to people living with this disease.”

The combination of Tecentriq and Avastin plus carboplatin and paclitaxel was recently given Priority Review from the U.S. Food and Drug Administration (FDA) for first-line treatment of chemotherapy-naïve patients with metastatic NSCLC. The agency is expected to make a decision by September 5, 2018.

This data will be presented at ASCO on Monday, June 4 at 3:45 to 3:57 p.m. CDT.

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At ASCO, One Study Pits Novartis and Gilead CAR-T Therapies Head-to-Head

Wednesday was the big day for pharma and biotech companies to unveil abstracts ahead of the annual American Society of Oncology meeting in Chicago next month. Some companies have unveiled positive results that are making investors happy, while others… not so much. BioSpace looks at a handful of the abstract reports.

Novartis vs. Gilead – One abstract that may get a lot of attention is a Chinese study that has pitted the only two U.S. Food and Drug Administration CAR-T drugs against each other in a head-to-head trial. The Chinese group ran a parallel trial to compare the CD28 (Gilead’s Yescarta) and 4-1BB (Novartis’ Kymriah) CD19-positive relapsed/ remitting B-cell acute lymphoblastic leukemia. The study included 19 patients in CD28 group and 28 patients in 4-1BB group. The abstract results seem to show that Novartis Kymriah has an advantage. The objective response rate of the 4-1BB group was 100 percent. The ORR of CD28 group was 89 percent. Kymriah also appears to be safer, according to the abstract data.  Nearly all the patients in the groups, 45 of 47, experienced some degree of cytokine release syndrome, a common occurrence with CAR-T treatment. Of those 45 patients, 5 patients who had grade III-IV of CRS were all in the group who received Gilead’s Yescarta. All adverse events were effectively controlled within one month, the Chinese group said.

Syndax Pharmaceuticals – Waltham, Mass.-based Syndax released updated results from multiple cohorts of the ongoing Phase II ENCORE 601 trial of entinostat in combination with Merck’s Keytruda across multiple cohorts of PD-(L)1 treatment-naïve and pre-treated cancers, including non-small cell lung cancer (NSCLC), melanoma and microsatellite stable colorectal cancer (MSS-CRC). The company said confirmed objective responses with the combination regimen have been observed across all cohorts. Additionally, the company said the therapeutic has a manageable toxicity profile. The news hasn’t moved the needle much on early trading. Shares are up just over 1 percent in premarket trading to $11.25.

Jounce Therapeutics – Cambridge, Mass.-based Jounce Therapeutics released data on its lead product candidate, JTX-2011, a monoclonal antibody that binds to and activates the Inducible T-cell CO-Stimulator (ICOS) and the news doesn’t appear to be good – investors have bailed on the company in early trading. Shares of Jounce are down more than 34 percent as Jounce showed off data from its therapeutic combined with Bristol-Myers Squibb’s OPDIVO. The study is examining the efficacy of the drug as a monotherapy and in combination across four different cancer types. The company said the treatment is well tolerated but it appears that in gastric cancer the efficacy is not what was hoped for. Results show low response rates to the therapy and that has sent investors running.

Tesaro, Inc. – Waltham, Mass.-based Tesaro Inc. presentation of five Zejula (niraparib) abstracts across multiple indications at ASCO. The company has paired Zejula, its PARP inhibitor, with checkpoint inhibitors in patients with triple-negative breast cancer or recurrent ovarian cancer. Tesaro is also testing Zejula in combination with Genentech’s Avastin (bevacizumab) in ovarian, breast and lung cancer. Nothing seems to be amiss in the abstracts as investors have responded favorably. Shares of Tesaro are up nearly 2 percent in early trading, which follows its nearly 3 percent gain on Wednesday.

Nektar Therapeutics – Shares of Bay Area’s Nektar are down about 5 percent after the company’s abstract showed its CD122-biased agonist NKTR-214 in combination with BMS’ OPDIVO did not generate as strong of an efficacious response as hoped. The combination revealed an objective response rate of 52 percent in the Phase I/II trial. For renal cell carcinoma, the ORR was reported to be 54 percent. Those middle-of-the-road rates may not be satisfactory following BMS’ heavy investment of $3.6 billion into Nektar earlier this year. Nektar’s experimental drug is designed to expand cancer-fighting T cells and natural killer (NK) cells in the tumor environment.

https://bit.ly/2Ld3Lz0

FDA Warns Pharma Firms Blocking Sample Requests of Generics Makers

In order to drive the manufacture of affordable generic drugs, the U.S.Food and Drug Administration is naming names of companies that have attempted to block competition.

Building on the momentum of President Donald Trump’s proposals to bring down the cost of prescription drugs, FDA Commissioner Scott Gottlieb announced some steps his agency will take to spur affordable drugs and improve access – including the prevention of branded companies gaming the system. To help improve generic competition and reduce the price of medicines, Gottlieb said the FDA said it will have to address some of the tactics banded drug companies use to protect their medications from generic competition, particularly the way branded companies prevent generic drugmakers from gaining access to samples of brand medicines.

Those samples are what generic drugmakers used to develop their products or use in head-to-head clinical trials. Gottlieb said the FDA has received more than 150 inquiries from generic drug developers who have requested assistance in obtaining samples from brand companies. Without them, alternative medicines cannot be developed.

“Yet, the FDA has heard that some brand companies will adopt tactics to make it hard for the generic companies to purchase these brand drugs at a fair value and in the open marketplace. The FDA is taking new steps to address this issue,” Gottlieb said.

One step is to publicly shame companies that block access. The FDA has created a public list of companies that have been blocking access. Some of the companies on the list include Roche, Novartis, Cubist Pharmaceuticals, Jazz Pharmaceuticals, BioMarin Pharmaceutical, Gilead Sciences, Inc. and more. In all, the FDA’s list includes more than 50 instances where companies have attempted to block generic manufacturers from getting samples. Several of the companies, such as Novartis, appear more than once on the list. Gottlieb noted that generic drugmakers need between 1,000 and 5,000 samples to complete their clinical processes.

Additionally, the FDA will post a list of the inquiries the regulatory agency has received from generic drug developers who report having trouble accessing testing samples. Through the increased transparency or shame, the FDA hopes that will reduce some of the hurdles generic drugmakers create. For example, the FDA has received 13 requests regarding samples of Celgene’s multiple myeloma drug Revlimid, as well as 10 requests for cancer drug Thalomid. Celgene isn’t the only company to be called out multiples times. Gilead Sciences was called out 10 times for its pulmonary arterial hypertension (PAH) treatment Letairis. The FDA has had 14 requests regarding Actelion Pharmaceuticals PAH drug Tracleer.

“… I want to be very clear: a path to securing samples of brand drugs for the purpose of generic drug development should always be available,” Gottlieb said.

There have been other tactics taken to limit challenges to branded drugs. Challenges to the Inter Partes Review process were popular, although in April the U.S. Supreme Court ruled the IPR is constitutional.

Before that ruling companies went to great lengths to avoid IPR challenges, perhaps most notably was Allergan’s attempt to circumvent patent laws by patents for Restasis to the Saint Regis Mohawk Tribe in New York. The company made the move last year as part of an attempt to take advantage of the Mohawk tribe’s sovereign immunity would shield the drug from IPR challenges to its patents on Restasis.

Gottlieb said the FDA will continue to look at ways it can address the access concerns. Gottlieb also noted that the FDA will continue to work with the Federal Trade Commission where there may be anticompetitive business practices. He added that the FDA will continue to strengthen its internal processes for handling inquiries related to problems generic drug developers report having in obtaining samples of brand products.

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Aon to empower self-insured employers with clear prices and quality data

Professional services firm Aon announced Thursday it has partnered with two companies to provide cost and quality information to self-insured employers so they can better negotiate surgical prices with providers. This is the latest move by employers to seek out innovative solutions to lower healthcare spending as costs rises.

Aon has collaborated with Amino, a company that provides consumers with healthcare cost information, and MPIRICA, a start-up that uses CMS data to illustrate surgical outcomes for roughly 123,400 U.S. surgeons and nearly 5,000 hospitals.

The partnership allows Aon’s self-insured employer clients to leverage cost and quality data when it contracts with providers. Its become more common for large and even small companies to shift to self-funded health plans with the implementation of the Affordable Care Act. The insurance pool now includes sicker, costlier individuals so employers with healthy, low-cost employees are incentivized to switch to their own plan.

The hope is that with the additional data, employers will be able to select hospitals and physicians for their employees at the lowest cost for the best quality, said Todor Penev, leader of actuarial innovation at Aon which has about 3,000 clients.

“We want to use the data to provide an independent benchmark of what is happening in their market so employers can be better informed about who they should partner with that can deliver the best combination of cost and quality,” he said.

About 28% of employers currently have benefit design packages that incentivize employees to use high quality hospitals or physicians, according to research from Aon, which surveys its employer clients. And 58% of employers are interested in such a strategy in the next two to three years.

Aon will present the data from Amino and MPIRICA to its employer clients that consult with Aon on benefit design packages. Employers will likely use the data for expensive, common procedures with a lot of variation in cost and quality depending on the hospital or physician, Penev said. The data is currently limited to metropolitan markets because that is where information is most robust. Aon hopes to eventually expand the data offerings to maternity care.

Very few employers are large enough to analyze the data themselves, usually because they don’t have enough employees in a given zip code, so they have to rely on outside data companies. “We are trying to bring that (data) to them,” Penev said.

Doug Emery, director of benefits innovations solutions at Altarum, said he doesn’t expect the partnership to have a big impact. Third-party administrators and health plans can already access this kind of data, and it hasn’t made much difference on healthcare costs.

“Until there is a new operating system for transacting benefits and contracts between self-funded companies and providers of healthcare, this can only make a marginal difference,” he said.

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CMS considers paying all Medicare providers for cancer gene therapies

CMS is considering reimbursing all Medicare providers for one-time cancer treatments that genetically alter patients’ cells to fight the disease. The move could increase revenue by millions at each hospital offering the therapy.

The FDA cleared two forms of chimeric antigen receptor-T or CAR-T therapies last year for patients battling leukemia and lymphoma. Novartis and Gilead manufacture the two cell therapies marketed as Kymriah and Yescarta. The treatments, which are meant to be a one-time event, work by manipulating immune cells to attack cancer cells.

While local Medicare contractors can cover CAR-T treatments already, there is no national payment policy to reimburse hospitals which typically administer the treatments. The CMS pays hospitals $500,839 for administering Kymriah and $395,380 for Yescarta. Hospitals also can secure more revenue from inpatient stays stemming from the treatment’s side effects. The patient then also undergoes chemotherapy and if complications arise, might need to be hospitalized.

The total bill could add up to $1.5 million per patient, between actual treatment costs and inpatient stays.

UnitedHealthcare urged the CMS to cover the treatments nationally via a letter to the agency earlier this year.

“We believe there is an industry-wide need for a national coverage determination to ensure a level playing field across Medicare Advantage plans,” UnitedHealthcare said. “Absent a national coverage determination providers and beneficiaries could get inconsistent treatment decisions and inconsistent Medicare Administrative Contractors decisions.”

The CMS will accept comments through June 15 on the request. The agency also will host an advisory committee meeting to discuss the proposal on Aug. 22.

The CMS said it is concerned about the lack of research about whether Medicare enrollees use CAR-T therapy. That could pose a barrier to national coverage. It also raised concerns about adverse event rates.

The CMS anticipates that it will release a proposed coverage decision by Feb. 16, 2019, and a final decision by May 17, 2019.

https://bit.ly/2wQNNYl

Mount Sinai’s Icahn medical school launches $10M investment fund

The Icahn School of Medicine at Mount Sinai has created a $10 million accelerator fund to speed up the commercialization of research at the school. It’s starting with two investments in drug-development ventures.

The i3 Asset Accelerator will provide capital and business development support to its researchers. Icahn joins a growing group of local medical schools—including NYU Langone, Weill Cornell, Columbia and SUNY Downstate—that have dedicated funds to commercialize their research.

“This is a really great way to seed the companies,” said Nicole McKnight, managing director at BioLabs New York, which runs a co-working space with NYU Langone for early stage biotech companies in Manhattan. “As funding for basic science kind of shrinks, it’s also a key way for the institutions to raise money through royalties.”

The state and the city have committed nearly $1.2 billion combined to develop New York’s biotech industry, a source of middle-class jobs, through tax credits, real estate development and some seed funding. Several local drug-development companies have raised investment rounds of $50 million or more this year.

Expediting investments

The goal of the accelerator—i3 refers to innovation, inflection and impact—is to help Mount Sinai’s researchers traverse the “valley of death” in biotech, where startups may perish if they don’t get the funding needed to pay for clinical trials. Those results are vital to attracting investment capital from pharmaceutical companies or venture capital firms.

“It was important for the institution to have a fund that could provide funding in a rapid format for a high-potential program,” said Erik Lium, senior vice president of Mount Sinai Innovation Partners. “The kind of research we will be funding may not be the type of research that falls within the National Institutes of Health.”

Lium said the medical school expects to evaluate potential investments within two to three months. By contrast, it can take years to get funding through other methods.

The money initially will support two research projects. Geneticist Ross Cagan, senior associate dean at Icahn’s Graduate School of Biomedical Sciences, and chemist Arvin Dar, an assistant professor of oncological sciences and pharmacological sciences at Icahn, are collaborating on research on colorectal and liver cancer. They have developed a platform to identify potential drugs that has so far yielded several promising candidates, Cagan said.

But pharma companies and venture capital firms aren’t quite ready to invest in the venture. “Their message is, ‘Come back when you’re much further along,’ ” Cagan said.

Mount Sinai is funding the research before a company has been formed. If the work yields intellectual property and a company is later created, the medical school will take an equity stake in the venture. “The next step forward is getting data so people want to invest,” Cagan said.

The second project the accelerator will fund is research on new therapies for influenza B viral infections, which is being conducted by Florian Krammer, an associate professor of microbiology at Icahn.

Mount Sinai Innovation Partners, which has 36 employees, filed 226 patents last year. The medical school has received $29.2 million from outside sources to fund research.

Lium said he hopes to expand his team to ensure Mount Sinai can capitalize on opportunities that could ultimately translate into treatments.

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