Janssen: Switch to Med Maintains HIV-1 Suppression, No Resistance to 96 Weeks

Long-term safety and efficacy data from Phase 3 EMERALD study presented at IDWeek 2018¹

Janssen Pharmaceutical Companies of Johnson & Johnson unveils new 96-week data for SYMTUZA™ (darunavir 200 mg, cobicistat 150 mg, emtricitabine 200mg, and tenofovir alafenamide 10 mg; D/C/F/TAF), a single-tablet regimen for the treatment of human immunodeficiency virus type 1 (HIV-1) in treatment-naïve and certain virologically suppressed adults, in a presentation at IDWeek 2018 in San Francisco, CA.

Results from the pivotal Phase 3 EMERALD study demonstrate that in adults with HIV-1 who are virologically suppressed, switching to SYMTUZA™ resulted in maintained high virologic suppression (91%, 692/763) and low virologic failure (1%, 9/763) at week 96 (per FDA-Snapshot); low cumulative virologic rebound (3.1%, 24/763); and no resistance development, up to 96-weeks.¹

‘The 96-week results from the EMERALD study demonstrate that SYMTUZA™ can offer clinically appropriate people living with HIV a single-tablet option that may help them maintain high rates of virologic suppression over time,’ said Joseph Eron, M.D., Professor of Medicine and Director, Clinical Core, University of North Carolina Center for AIDS Research, Chapel Hill, NC.

This 96-week extension study, which follows on from the earlier 24 – and 48-week results,^2,3,4 reinforced the long-term efficacy, resistance and safety profile of SYMTUZA™ as a treatment for virologically suppressed adults with HIV-1. The patient population studied in EMERALD included patients who may have experienced prior virologic failure and/or who may have resistance to emtricitabine.¹ SYMTUZA™ was well-tolerated with 2% (14/763) of people experiencing a study drug related grade 3 or 4 adverse event (AE) and 2% (17/763) AE-related discontinuations over 96 weeks.

The most common AEs (all grades, ≥10% of adults) in the extension period were upper respiratory tract infection, viral upper respiratory tract infection, diarrhea, headache and back pain. After initial increases between baseline through to week 48, the lipid profile among D/C/F/TAF patients remained stable thereafter. Improvements in renal and bone parameters were maintained in the SYMTUZA™ group over 96 weeks and consistent with known tenofovir alafenamide and cobicistat profiles.¹

In a separate analysis, switching treatment to SYMTUZA™ from the multi-tablet control regimen after 52 weeks achieved comparable efficacy and safety to the 48-week results in the group that switched immediately.¹ In this late-switch group, after 44 weeks of SYMTUZA™ exposure, the virologic suppression and virologic failure rates were 94% (330/352) and 2% (6/352) respectively at week 96 (per FDA-Snapshot), and the cumulative rebound rate was 2.3% (8/352) from switch at week-52 through week 96. Over 44 weeks, in this late-switch group, serious adverse events and adverse event-related discontinuations occurred in 6% (21/352) and 2% (7/352) of adults respectively while on SYMTUZA™.

‘These new data are extremely important, as they further demonstrate that through 96-weeks SYMTUZA™ offers sustained efficacy, a long-term safety profile and the protective barrier to resistance of darunavir in a single-tablet option for clinically appropriate adults. These are all important factors for people who require long-term HIV-1 therapy,’ said Richard Nettles M.D., Vice President, US Medical Affairs, Janssen Scientific Affairs, LLC.

On September 25, 2017, SYMTUZA™ was approved for the treatment of HIV-1 infection by the European Commission based on results from a bioequivalence study that compared SYMTUZA™ with the combined administration of the separate agents darunavir [D] 800mg, cobicistat [C] 150mg, and emtricitabine/tenofovir alafenamide [FTC/TAF] 200mg/10mg fixed-dose combination.^5,6FDA approval was granted on July 17, 2018 based on the results from the two pivotal Phase 3 studies, EMERALD and AMBER.^5,7

https://www.marketscreener.com/JOHNSON-JOHNSON-4832/news/Johnson-Johnson-Janssen-Reports-Switching-to-SYMTUZA-trade-Results-in-Maintained-High-Virologic-27365077/

BTG upgrades sales guidance after strong first half

Pharmaceutical firm BTG upgraded its sales forecast on Thursday after it included its varicose vein treatment Varithena in its oncology and vascular portfolio and saw strong demand for its tumour-targeting medicines.

Shares in the group rose 4 percent in early deals.

The company said it expected product sales for the combined portfolio to grow by 15-17 percent this year, up from its previous 13-15 percent forecast, while sales of its pharmaceutical products would be at the upper end of guidance.

It had guided to flat to single digit percentage declines in pharmaceutical sales this year when it published its full-year results in May.

The company also said its chairman Garry Watts would retire at the end of the year.

https://www.marketscreener.com/BTG-9583552/news/BTG-Pharma-upgrades-sales-guidance-after-strong-first-half-27366793/

Why Does Immunotherapy Cost So Much?

Immunotherapy – reprogramming your immune system to attack cancer cells – is a rapidly growing and promising field in cancer research and treatment. Traditional cancer chemotherapy and radiation are non-specific, killing both the cancer and the patient’s own tissue. The power of immunotherapy lies in its specificity.

For example, chimeric antigen receptor T-cell therapy (CAR-T) utilizes the patient’s own immune cells. Their T-cells are removed, genetically engineered to recognize a certain protein expressed on cancer cells and infused back into the patient. These modified T-cells selectively seek out and destroy cancer cells.

Immune checkpoint inhibitors are another type of immunotherapy, which target the molecular ‘brakes’ of the immune system, such as CTLA-4 and PD-1, allowing the immune system to more successfully recognize and destroy cancer cells. In fact, this work was just awarded the 2018 Nobel Prize in Physiology or Medicine, highlighting its importance in cancer treatment today.

These personalized medicines and novel immunotherapies are providing substantial clinical benefit, especially in patients who had little to no hope even a decade ago. They have ‘raised the hope of significantly improving cancer survival’ across many kinds of cancer.

“A potential revolution in cancer care may be stymied by the high price of drugs, which suggests that we need to reconsider how we price them,” Ezekiel Emanuel, vice provost and chair of the department of medical ethics and health policy at the University of Pennsylvania, wrote in the Wall Street Journal (WSJ).

Why Such High Costs?

The oncology market remains very active and is moving towards targeted treatments and combination therapies. In 2016, there were over 600 molecules in late-stage development – 90 percent of which were targeted therapies.

As cancers develop treatment resistance, combinations of old and new therapies will become increasingly important to effective treatment. The use of multiple, potentially costly, drugs during combination therapy, increased duration of novel therapies and the possibility of patients needing multiple rounds of therapy all contribute to the high costs in oncology.

Between 2012 and 2016, the global cost of oncology therapeutics and supportive care drugsincreased from $91 billion to $113 billion – 46 percent of which was for the U.S. alone. Cancer care costs are expected to continue rising rapidly, estimated to be $173 billion by 2020.

The average annual cost of cancer drugs has jumped from less than $10,000 in 2000 to more than $120,000 in 2015. During that time, the average income for a family of four dropped 8 percent to $52,000, furthering the potential financial burden of these cancer drugs. Increasing drug costs and the number of approved therapies, especially targeted therapies, are driving this cost increase.

For example, two recently approved CAR-T treatments, Novartis’ Kymriah and Gilead Sciences’ Yescarta, sport price tags between $373,000 and $475,000 per patient, depending on the type of cancer being treated. Each CAR-T treatment is made specifically for each patient, taking more than three weeks of lab work to make. Therefore, the manufacturing and processing cost is high – about $60,000 per patient.

Pharma companies justify pricing by highlighting the value of these immunotherapy drugs and the continual investment of their profits into R&D to discover new cancer drugs. The exorbitant $1 billion (or higher) price tag to bring a drug to market is also a key factor in high prices. However, the companies say that ‘significant discounts’ are offered to many patients.

Although the annual cost of these immunotherapy drugs far exceeds almost all other cancer drugs, it may be considered ‘cheap’ given that leukemia patients may receive over $2 million hospital bills over the course of their treatment.

“If the therapy is curative, the total cost of therapy might be less than would be the case for repeated courses of less expensive, but less effective treatments such as chemotherapy,” Mark Faries, director of therapeutic immunology at the John Wayne Cancer Institute at Providence Saint John’s Health Center in California, told Healthline.

Potential Changes in Pricing

One way to affect drug prices may be to change laws involving drug pricing. Proposed legislature, such as the Medicare Negotiation and Competitive Licensing Act of 2018, aims to negotiate the prices of drugs prescribed under part D of Medicare. However, many patients receiving immunotherapy aren’t on Medicare. ‘More than 175 million Americans have private insurance that wouldn’t be covered by Medicare negotiations,’ Emanuel wrote in the WSJ.

But legislature isn’t the only way to lower drug prices. In September 2017, the National Institute for Health and Care Excellence (NICE), Britain’s ‘healthcare cost watchdog,’ recommendedBristol-Myers Squibb’s cancer immunotherapy drug Opdivo to some lung cancer patients only after a discounted price was agreed upon.

“This new deal means that we can give patients access to what we know is a promising treatment whilst more evidence is gathered on its value,” Carole Longson, director of the NICE center for health technology evaluation, told Reuters.

While the answer to how to manage the costs of novel cancer immunotherapies may be complex, the need to make this effective treatment accessible to patients is clear.

https://www.biospace.com/article/why-does-immunotherapy-cost-so-much-/

Guardant Health 12.5M share IPO priced at $19.00

The deal priced above the $15.00-$17.00 range, as expected. JPMorgan and BofA/Merrill acted as joint book running managers for the offering.
https://thefly.com/landingPageNews.php?id=2799471

Kodiak Sciences 9M share IPO priced at $10.00, below the range

The deal priced below the $13.00-$15.00 range. Morgan Stanley and BofA/Merrill acted as joint book running managers for the offering.

https://thefly.com/landingPageNews.php?id=2799473

Approval of Noxivent has mitigating factors for Mallinckrodt, says BMO

BMO Capital analyst Gary Nachman kept his Outperform rating and $37 price target on Mallinckrodt (MNK), saying the FDA’s approval of Praxair (PX) ANDA for Noxivent, which is a generic form of gas used in the Inomax device for “treating neonates with respiratory failure”, has some mitigating factors. The analyst notes that Praxair’s device will still have to be approved by the FDA and litigation appeal between the two companies regarding Inomax patents is still ongoing until the first half of next year, with the possibility of a favorable settlement between the two still possible. Moreover, Nachman points to Mallinckrodt’s “very strong relationships” with partner hospitals’ NICUs, as 25% of its U.S. book of business has been committed for the next couple of years.

https://thefly.com/landingPageNews.php?id=2799474

Twist Bioscience on deck for IPO

San Francisco, CA-based Twist Bioscience (TWST) has filed a preliminary prospectus for an $86M IPO.

The synthetic biology company has developed a DNA synthesis platform that it says enables the industrialization of biology engineering. The core of the platform provides a method of making synthetic DNA by “writing” it on a silicon chip. It plans to develop products like synthetic genes, tools for next-gen sample preparation and antibody libraries for drug discovery and development.

2018 Financials (9 mo.)($M): Revenues: 17.0 (+133.1%); Operating Expenses: 67.9 (+38.0%); Net Loss: (51.4) (-21.5%); Cash Consumption: (49.5) (-24.4%).

https://seekingalpha.com/news/3394796-twist-bioscience-deck-ipo