Pfizer Data on Phase 2 Study of 20-Valent Pneumococcal Conjugate Vaccine

The U.S. FDA awarded Breakthrough Therapy Designation for this potential indication based on these Phase 2 data

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The Biologics License Application is expected to be submitted to the U.S. FDA by the end of 2020, subject to the successful completion of Phase 3 studies

Pfizer Inc. (NYSE: PFE) announced today the presentation of data from a Phase 2 study of its 20-valent pneumococcal conjugate vaccine (20vPnC) candidate, PF-06482077, being investigated for the prevention of invasive disease and pneumonia caused by Streptococcus pneumoniae serotypes contained in the vaccine in adults aged 18 years and older. The presentation was delivered at the 29th European Congress of Clinical Microbiology and Infectious Diseases (ECCMID) in Amsterdam, Netherlands. Pfizer’s 20vPnC candidate includes the 13 serotypes contained in Prevnar 13 plus seven additional serotypes (8, 10A, 11A, 12F, 15B, 22F, and 33F).

“The safety and immunogenicity results from this study suggest that our 20vPnC candidate, which initiated Phase 3 development in adults last year, could potentially offer comprehensive coverage of additional serotypes causing pneumococcal disease globally and in the U.S. as substantiated by the receipt of FDA’s Breakthrough Therapy Designation,” said Kathrin U. Jansen, Ph.D., Senior Vice President and Head of Vaccine Research & Development, Pfizer. “We believe the full extent of Prevenar 13 protection of adults has yet to be fulfilled. At the same time, there continues to be a global health need to protect against the potential effects of invasive pneumococcal disease and pneumonia caused by additional serotypes not yet covered by existing conjugate vaccines.”

https://www.marketscreener.com/PFIZER-23365019/news/Pfizer-Announces-Presentation-of-Data-from-a-Phase-2-Study-of-its-20-Valent-Pneumococcal-Conjugate-28420157/

EASL 2019 – Alnylam lays out givosiran’s benefit

Full data from Alnylam’s pivotal givosiran programme show a big impact for severe patients, though a case still needs to be made for a broader population.

The Envision study of Alnylam’s acute hepatic porphyria therapy givosiran was already known to have shown a significant reduction in the life-threatening attacks associated with this disease. Today at EASL the company revealed to experts the magnitude of that benefit.

The mean annualised rate of composite attacks fell 74% relative to placebo, a number that should impress physicians and make regulators comfortable with granting approval. However, three serious safety events mean that this is not a dead cert.

Still, all bar one of the 94 subjects recruited opted to move into a long-term extension study, suggesting that patients with severe forms of AHP consider givosiran a therapy worth trying.

Another important point to remember is that only 50% of patients were attack free, compared with 16% for placebo. Dr Manisha Balwani, Envision’s lead investigator who presented the data to journalists at the conference yesterday, declined to comment on the responses seen in the other 50% of patients, saying that the data had yet to be fully analysed on a patient-by-patient basis.

This is certainly a question that regulators will want answered, while safety will be scrutinised. The three serious safety events – high fever, kidney disease and raised liver enzymes – were previously announced last month, with the hit on the top line (Alnylam touts givosiran win despite toxicity fears, March 6, 2019).

Theresa Heggie, Alnylam’s senior vice-president of Europe and Canada, pointed out that kidney and liver problems could be a burden of this disease, but acknowledged that she could not rule out an effect of givosiran.

“Transient changes in [signals of kidney disease] resolved, and we were encouraged by that. And in those patients we continued to measure liver enzymes, and they normalised. We believe the risk-benefit ratio is very much in favour of givosiran,” she told Vantage at EASL.

Many harbour concerns about underlying toxicity issues with the company’s RNAi technology platform, and will be waiting with interest to see what regulators will make of these signals.

Source: Alnylam press presentation.

Other data provided for the first time included the median annualised rate of composite attacks, which was lowered by 90% relative to placebo, and details on a number of secondary endpoints regarding disease biomarkers, all of which were lowered dramatically, by 73-93%, pointing to givosiran’s big impact on the underlying disease.

Secondary endpoints regarding pain and fatigue, which can blight the lives of sufferers of AHP in between the extreme attacks, were not hit. Dr Balwani said that responses on these measures had been seen in earlier trials, and that she hopes to see a benefit as treatment progresses. The primary and secondary endpoints were measured at six months, a relatively short time for a therapy that will presumably be taken chronically.

Evaluate’s sellside consensus has givosiran sales reaching $397m by 2024, and analysts currently assume that the therapy will be restricted to more severe patients, those who suffer several attacks a year. For Alnylam to persuade patients and payers that less severe patients could benefit, those more subjective secondary endpoints need to start looking more encouraging over time.

Ms Heggie confirmed that the company intends to pursue value-based pricing agreements with payers in the US, a strategy that no doubt speaks to the company’s intention to build a case for the therapy in a wider set of patients.

Alnylam plans to complete regulatory filings for givosiran in the coming months in both the US and Europe; Ms Heggie said the group will be ready to launch in the US by the end of the year.

If all goes smoothly that would be two approvals in as many years for the rare disease company, which should go some way to removing lingering  scepticism about its technology.

https://www.evaluate.com/vantage/articles/events/conferences/easl-2019-alnylam-lays-out-givosirans-benefit

Biotech week ahead, April 15

Biotech stocks witnessed a slowdown in momentum last week, which was a relatively slow one in terms of news flow. Amgen, Inc. AMGN 0.33%‘s unique drug to treat osteoporosis in postmenopausal women at high risk of a fracture cleared the FDA hurdle after an earlier unsuccessful attempt.

The highlights of the week were presentations at the European Association for the Study of the Liver, or EASL, International Liver Congress held in Vienna, Austria.

Here are some key catalysts on tap for the unfolding week.

Conferences

• 2019 Muscular Dystrophy Association, or MDA, Clinical & Scientific Conference – April 13-17, at the Hyatt Regency in Orlando
• 29th European Congress of Clinical Microbiology & Infectious Diseases, or ECCMID – April 13-16 in Amsterdam, Netherlands
• World Vaccine Congress 2019 – April 14 – 17, in Washington
• 5th World Heart Congress – April 15-16, in Amsterdam
• 12th International Conference on Genomics and Molecular Biology – April 15-17, in Berlin, Germany
• 2019 ESM-EVBO (European Society for Microcirculation – European Vascular Biology Organization) Conference – April 15-18, at the MECC in Maastricht

Clinical Trial Readouts

Wave Life Sciences Ltd WVE 0.09% is due to present new Phase 1 safety and tolerability data for WVE-210201 for Duchenne muscular dystrophy, or DMD, Exon 51 at the MDA Clinical & Scientific Conference.

Sarepta Therapeutics Inc SRPT 2.5% will present data on MYO-1 for DMD – LGMD2E at the 2019 MDA Clinical & Scientific Conference on April 16.

Novavax, Inc. NVAX 4.78% will present already-released Phase 3 top-line data for RSV vaccine in healthy pregnant women to protect infants via maternal immunization. The datareleased in late February showed that the primary endpoint was not met.

ContraFect Corp CFRX 5.48% will present new Phase 2 data for CF-301 in treating serious infections caused by Staphylococcus aureus, including MRSA. The data is due to be presented April 16. The stock was one of the worst performers among biotechs in the first quarter.

SCYNEXIS Inc SCYX 1.08% is scheduled to present interim Phase 3 open-label data for SCY-078, oral, in invasive candidiasis at the ECCMID on April 16.

Earnings

Wednesday

• Abbott Laboratories ABT 0.64% (before the market open)

Thursday

• Intuitive Surgical, Inc. ISRG 0.34% (after the market close)

IPOs

Brainsway, a maker of medical devices that use magnetic stimulation to treat depression and obsessive-compulsive disorder, is set to offer 2.5 million shares in an IPO, with the price estimated between $9 and $11. Shares are to be listed on the Nasdaq under the ticker symbol “BWAY.”

Turning Point Therapeutics, an early-stage biotech developing therapies to treat cancer, plans to offer 7.352 million shares in an IPO, with an estimated price range of $16-$18. The company seeks to list shares on the Nasdaq under the ticker symbol “TPTX.”

Hookipa Pharma is gearing up for a 6.67-million share IPO, with an estimated price range of $14-$16. The developer of T cell immunotherapies for treating cancer and infectious diseases is due to list shares on the Nasdaq under the ticker symbol “HOOK.”

https://www.benzinga.com/general/biotech/19/04/13516145/the-week-ahead-in-biotech-conferences-clinical-trial-readouts-earnings-and-ipos

Dr Reddy Laboratories Announces Acquisition of ANDA Portfolio

Dr. Reddy’s Laboratories Ltd. (BSE: 500124, NSE: DRREDDY, NYSE: RDY, along with its subsidiaries together referred to as “Dr. Reddy’s”) announced today that it has entered into a definitive agreement to acquire a portfolio of 42 approved, non-marketed Abbreviated New Drug Applications (ANDAs) in the U.S.

The portfolio includes more than 30 generic injectable products. These products will require to be technology transferred and could be launched within the next one to two years. The value of total addressable market for these products in the U.S. is approximately $645 million for the calendar year ending in December 2018 according to IQVIA.

Erez Israeli, Chief Operating Officer of Dr. Reddy’s Laboratories, commented, “The acquisition is in line with our stated strategy to significantly enhance our portfolio in our chosen growth markets. This transaction will help augment our injectables product portfolio in the U.S. market and globally.”

https://www.marketscreener.com/DR-REDDY-S-LABORATORIES-9058869/news/Dr-Reddy-Laboratories-Announces-Acquisition-of-ANDA-Portfolio-28420513/

Knight Therapeutics Subdermal Implant for Opioid Disorder Gains Across Canada

Knight Therapeutics Inc. (TSX: GUD) (“Knight”), a Canadian specialty pharmaceutical company, is pleased to announce that PROBUPHINE™, a new treatment in the fight against opioid addiction, is now being implanted in patients across the country.

Approved by Health Canada in April 2018, PROBUPHINE™ is the only subdermal implant designed to deliver buprenorphine continuously for 6 months following a single treatment. The product must be inserted and removed by a healthcare professional who has successfully completed a live training program, the PROBUPHINE Education Program.

Knight has the exclusive right to distribute PROBUPHINE™ in Canada under a license agreement with Titan Pharmaceuticals, Inc. (NASDAQ:TTNP).

“Probuphine provides a unique new option in the treatment of opioid abuse, one that overcomes the barriers of daily adherence and multiple pharmacy and clinic visits,” said Dr. Ken Lee, London site lead and head physician at the Rapid Access Addiction Medicine (RAAM) Clinic.

https://www.marketscreener.com/KNIGHT-THERAPEUTICS-INC-15925497/news/Knight-Therapeutics-Use-of-PROBUPHINE-trade-for-Opioid-Disorder-Gains-Momentum-Across-Canada-Subd-28399191/

Time to buy CVS Health stock, Barron’s says

CVS should discuss its expansion plans for HealthHub at a June investor date, and while the stock has tumbled on stalled earnings growth and concerns that margins as a drug-price negotiator will fall, CVS’ assets leave it “uniquely well positioned” for a future when consumers gain more control over healthcare and prices fall, Jack Hough writes in this week’s edition of Barron’s. It might be time to buy the shares, Hough contends.

https://thefly.com/landingPageNews.php?id=2892193

Alcon stock might deserve a long look, Barron’s says

Alcon (ALC) is back on the public market after more than eight years under the wing of Swiss drug giant Novartis (NVS) and investors should raise their sights on the stock, Andrew Bary writes in this week’s edition of Barron’s. While Alcon’s business suffered from “some neglect” under Novartis’ control and had lackluster financial results, but that should change now that it is independent, he contends.

https://thefly.com/landingPageNews.php?id=2892195