4-Year-Old May Lose Vision After Battling Flu

Jade DeLucia, 4, of Iowa is home after a bout of the flu landed her in the intensive care unit fighting for her life and her eyesight.

Jade was flown to the University of Iowa Stead Family Children’s Hospital and diagnosed with the flu on Dec. 24, according to Jennifer Brown, PhD, a spokesperson for the Iowa City hospital.

The girl’s grandmother, Courtney Frey, told a local television station that the infection made the girl’s brain swell. On the family Facebook page to help support Jade’s fight, there was mention of eyesight problems.

She returned home on Jan. 9.

“Jade continued to improve gradually and her strength and ability to respond to commands are improving consistently. However, her vision is affected, probably at the level of brain, and she needs more diagnostic tests. She will get regular follow up visits with the neurology and ophthalmology teams as an outpatient,” said Aditya Badheka, MD, medical director of the Pediatric Intensive Care Unit at the hospital.

Jade’s mother, Amanda Phillips, talked about what happened ahead of her discharge on the Fundraiser for Jade Facebook page .

“We’re doing one final test here soon to see if her brain will react to her eyes,” Phillips wrote. “If her brain does respond then there is hope for her eyesight returning. If it doesn’t then we’ll be taking home the bravest, strongest, kindest, blind girl I will every meet.”

Aaron Glatt, MD, chair of medicine at Mount Sinai South Nassau and a professor of medicine at the Icahn School of Medicine at Mount Sinai, says the flu can cause problems in the nervous system. He did not treat Jade.

“Fortunately it isn’t very common, but it is not unheard of,” Glatt says. “Breathing problems can lead to problems in the brain,” with the lack of oxygen affecting how well the brain works.

The flu virus can also affect the eyes, experts say.

This year’s flu season is shaping up as a bad one, according to the CDC. For the week ending Dec. 28, seasonal flu activity in the U.S. is high, the CDC says, and continues to increase.

Influenza B/Victoria viruses are most common now nationally, and that is unusual for this time of year, the CDC says. Next most common are influenza A (H1N1) strains.

Five new child deaths blamed on the flu were reported during the 2019-2020 season the week of Dec. 28. The total for the season is 27 child deaths, as of Dec. 28.

Children younger than 5 years old, and in particular those younger than 2, are at high risk of getting serious flu-related problems, the CDC says. The flu vaccine is recommended each year for children 6 months and older.

Complications include pneumonia, dehydration, brain problems, sinus problems, and ear infections. Since 2010, hospitalizations for children under 5 have ranged each year from 7,000 to 26,000, the CDC says.

Deaths from the flu are relatively rare in children. Since 2004-2005, the number of deaths reported annually has ranged from 37 to 187; but the CDC says the deaths may be underreported. For instance, during the 2017-2018 season, the number may have been closer to 600.

https://www.webmd.com/cold-and-flu/news/20200109/4-year-old-may-lose-vision-after-battling-flu

NanoString: Prelim Operating, Financial Results for Fourth Quarter and FY 2019

— FY19 Product and Service Revenue of $103.7 million, including GeoMx DSP Revenue of $10.8 million, Exceeds High End of Guidance Ranges —

— Over 90 Cumulative GeoMx DSP Orders as of Year End —

NanoString Technologies, Inc. (NASDAQ:NSTG), a leading provider of life science tools for translational research, today provided preliminary operational and financial results for the fourth quarter and fiscal year ended December 31, 2019.

“Last year was transformative for NanoString. The launch of the GeoMx™ Digital Spatial Profiler (DSP) accelerated our growth, with product and service revenue increasing by 24% for the full year, and by 42% during the fourth quarter. Our strategic transaction to license our nCounter® diagnostic business to Veracyte has sharpened our focus on GeoMx and other growth opportunities in our research markets,” said NanoString president and CEO, Brad Gray. “We expect this transformation to continue in 2020, as we enjoy our first full year of GeoMx revenue, and expand our addressable markets into discovery research through the launch of GeoMx compatibility with next-generation sequencers.”

“We enter 2020 with a substantially strengthened financial profile,” said Tom Bailey, chief financial officer of NanoString. “With $157 million in cash and short-term investments, we have the resources to sustain our growth in 2020 and beyond.”

Financial Highlights:

For the fourth quarter of 2019, product and service revenue is expected to be $33.6 million, an increase of 42% compared to the prior year period and exceeding the upper end of the company’s guidance range of $30 to $32 million. This includes nCounter revenue of $25.2 million and GeoMx DSP revenue of $8.4 million, as compared to prior guidance for fourth quarter nCounter revenue of $24 to $26 million and prior guidance for fourth quarter GeoMx DSP revenue of $6 million.

Product and service revenue for full year 2019 is expected to be $103.7 million, an increase of 24% compared to the prior year and exceeding the upper end of the company’s previous guidance for revenue of $100 to $102 million.

Total revenue for the fourth quarter of 2019 is expected to be approximately $36.6 million, including approximately $3 million of collaboration revenue, as compared to prior guidance for total revenue of $33 to $35 million. Total revenue for full year 2019 is expected to be approximately $125.7 million, including approximately $22 million of collaboration revenue, as compared to prior guidance of $121 to $123 million.

Cash and short-term investments totaled $157.3 million as of December 31, 2019.

Operational Highlights:

• Generated orders for approximately 20 GeoMx DSP systems during the fourth quarter.
• Shipped 34 GeoMx DSP systems during the fourth quarter, and completed the installation of 25 of those 34 shipped units.
• Shipped $0.7 million in GeoMx consumables during the fourth quarter.
• Completed 35 Technology Access Program projects during the fourth quarter, bringing the total number of completed projects to more than 200 since inception of the program.
• Sold approximately 125 nCounter Analysis Systems in 2019, increasing our installed base to approximately 855 nCounter Analysis Systems as of December 31, 2019, an increase of approximately 17% over fiscal year 2018.
• Licensed our nCounter-based diagnostic business to Veracyte for initial consideration of $50 million. Certain operating costs were eliminated as a result of the transaction and combining the impact of these cost savings with the impact of the supply agreement on Prosigna® revenue and gross profit results in an approximate $14 million improvement in NanoString’s 2018 operating loss on a pro forma basis and an approximate $12 million improvement in NanoString’s anticipated 2019 operating loss on an annualized pro forma basis.

Brad Gray will give a corporate update at the JP Morgan Healthcare conference at 3:00 p.m. PST on Wednesday, January 15th, 2020. A live webcast of the presentation will be available online from the investor relations page of the company’s corporate website at www.nanostring.com. After the live webcast, the presentation will remain available on the website for approximately 30 days.

https://www.marketscreener.com/NANOSTRING-TECHNOLOGIES-13479937/news/NanoString-Technologies-Provides-Preliminary-Operational-and-Financial-Results-for-Fourth-Quarter-29826999/

Adverum: Momentum with Gene Therapy Trial in Wet AMD; 2020 Outlook

— OPTIC phase 1 trial progressing with plans to complete patient dosing in cohort three, begin enrollment in cohort four in 1Q20 —

— IND application planned for ADVM-022 in diabetic retinopathy in 1H20; Patient enrollment targeted for 2H20 —

Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, today reviewed recent business and development progress and provided an outlook for 2020.

“In 2019, we made significant progress advancing our lead gene therapy candidate ADVM-022 in the ongoing OPTIC phase 1 dose-ranging clinical trial in patients with wet age-related macular degeneration (AMD),” said Leone Patterson, president and chief executive officer of Adverum Biotechnologies. “Patients with wet AMD typically require frequent ocular anti-VEGF injections to maintain their vision. Yesterday, additional data were presented from the first cohort of patients in OPTIC, which demonstrated zero anti-VEGF rescue injections required following a single intravitreal injection of ADVM-022 and with median follow up of 44 weeks, ADVM-022 treatment was safe and well-tolerated. This year, we look forward to presenting additional data from all four cohorts of patients in OPTIC. Additionally, we plan to submit an IND for ADVM-022 in a second indication, diabetic retinopathy, and begin enrolling patients in a new clinical trial. We believe ADVM-022 has the potential to be a paradigm-changing treatment for patients with wet AMD and for patients with diabetic retinopathy.”

Recent Data Presented for First Cohort of OPTIC Phase 1 Clinical Trial (n=6)
On January 11, 2020, Adverum presented median 44-week (range of 40-52 weeks) data at the Atlantic Coast Retina Club and Macula 20/20. These data demonstrated that patients in the first cohort of OPTIC achieved vision maintenance and improvements in retinal anatomy with zero anti-VEGF rescue injections required for any patient. Additionally, the first patient treated in OPTIC has reached 52 weeks post ADVM-022 administration. ADVM-022 has been safe and well-tolerated, with no dose-limiting toxicities through the latest time point at December 1, 2019 and inflammation has been manageable with topical eye drops.

OPTIC Phase 1 Trial Execution
After completing patient enrollment in the first and second cohorts, Adverum dosed the first patient in the third cohort (n=9, dose of 2×10^11 vg/eye) in October 2019. In the third and fourth cohorts, Adverum plans to use prophylactic steroid eye drops instead of prophylactic oral steroids to manage inflammation.

2020 Outlook

First quarter of 2020:

• Move to new corporate headquarters in Redwood City, CA, allowing for the expansion of the company’s in-house process development capabilities to the 1,000-liter production scale
• Complete patient dosing in the third cohort, begin enrollment in the fourth cohort, and determine if additional cohorts are needed in the OPTIC trial

First half of 2020:

• Present longer-term data from the first cohort of patients in OPTIC
• Present 24-week data from the second cohort of patients in OPTIC
• Submit an investigational new drug application for ADVM-022 in diabetic retinopathy, a key VEGF-driven cause of vision loss among working-age adults

Second half of 2020:

• Present longer-term data from the first cohort and second cohorts of patients in OPTIC trial
• Present clinical data from the third and fourth cohorts of patients in the OPTIC trial
• Begin enrolling patients in a planned phase 1/2 clinical trial for ADVM-022 in diabetic retinopathy to expand Adverum’s clinical development pipeline

Upcoming Events
Adverum plans to participate in the following upcoming conferences:

• J.P. Morgan’s 38^th Annual Healthcare Conference in San Francisco on January 15, 2020 at 2:30 pm PST
• Angiogenesis, Exudation, and Degeneration 2020 in Miami on February 8, 2020 at 2:44 pm EST. Dr. David Boyer will present the 24-week data from the second cohort for the first time as well as an update from the first cohort of patients in the OPTIC trial. A KOL event and simultaneous webcast to discuss the data presented with management and an expert panel of retina specialists will take place on February 9, 2020 at 10:00 am EST
• SVB Leerink’s 9^th Annual Global Healthcare Conference in New York on February 25, 2020 at 9 am EST
• Cowen’s 40^th Annual Health Care Conference in Boston from March 2-4, 2020

https://www.marketscreener.com/ADVERUM-BIOTECHNOLOGIES-27535811/news/Adverum-Biotechnologies-Reports-Continued-Momentum-with-OPTIC-Trial-for-ADVM-022-Intravitreal-Gene-T-29827000/

Alnylam: Prelim Q4, Full Year ’19 Global Net Product Revenue, Additional Update

− Achieved Fourth Quarter and Full Year 2019 ONPATTRO^® (patisiran) Preliminary Global Net Product Revenues of Approximately $56 Million and $166 Million, Respectively –

– As of Year-End 2019, Over 750 Patients Worldwide Receiving Commercial ONPATTRO and Over 1,000 Total Patients Worldwide Being Treated with Patisiran –

– Achieved Strong Initial Demand for GIVLAARI™ (givosiran) in the U.S., with 13 Start Forms Received in First Six Weeks after FDA Approval –

– Maintained Strong Balance Sheet with Year-End Cash and Investments Balance of Approximately $1.5 Billion –

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced its preliminary* fourth quarter and full year 2019 global net product revenues for ONPATTRO and GIVLAARI and provided additional updates on the products’ commercial launches.

For ONPATTRO, the Company reported:

• Preliminary global net product revenues for the fourth quarter and full year 2019 were approximately $56 million and $166 million, respectively.
• As of year-end 2019, over 750 patients worldwide were receiving commercial ONPATTRO, and over 1,000 total patients worldwide were being treated with patisiran, when also including those patients in clinical studies and in the Company’s global Expanded Access Program (EAP).
• In the U.S., 44% of Start Forms submitted in 2019 came from cardiologists, 38% from neurologists, and 18% from other physician specialties.
• Significant ONPATTRO new patient growth in the fourth quarter was seen in the U.S. and EU, along with a strong launch in Japan.
• In the U.S., Alnylam has now completed definitive value-based agreements (VBAs) with 14 commercial payers, including each of the top 5 commercial payers and 8 of the top 10, with signed VBAs now covering over 130 million U.S. lives in the aggregate.

For the period following approval of GIVLAARI in the U.S. on November 20 through year end, the Company reported:

• A total of 13 Start Forms were submitted.
• Preliminary global net product revenues were approximately $0.2 million representing initial channel stocking.
• The Company has made significant progress toward establishing VBAs, including a Prevalence-Based Adjustment feature, with multiple ongoing discussions with payers.

“In 2019 we achieved continued and steady growth of patients on ONPATTRO and we expect sustained growth in 2020 and beyond. We believe our preliminary results reflect strong patient and physician demand and excellent execution by our commercial teams around the world. With the early U.S. approval of GIVLAARI, we are now a multi-product commercial company, and are pleased with the strong initial interest from patients and physicians in the short period since the drug’s approval by the FDA. We’re also encouraged by the receptivity of U.S. payers to the value that GIVLAARI has the potential to deliver,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “In 2020, we look forward to the continued launches of ONPATTRO and GIVLAARI, bringing the benefits of these innovative therapies to patients around the world. We also look forward to advancing lumasiran – as our third wholly owned RNAi therapeutic – as well as inclisiran – with our partners at Novartis – toward the market in 2020 and achieving additional milestones across our broad late-stage pipeline of investigational RNAi therapeutics, notably in our ATTR amyloidosis clinical programs.”

In addition, the Company today reported that at December 31, 2019, it had preliminary cash, cash equivalents, marketable debt and equity securities, and restricted investments of approximately $1.5 billion, as compared to $1.1 billion at December 31, 2018.

Alnylam management will discuss these preliminary selected financial results and commercial updates during a webcast presentation at the 38^th Annual J.P. Morgan Healthcare Conference in San Francisco, California tomorrow, Monday, January 13, 2020, at 10:30 a.m. PT (1:30 p.m. ET).

https://www.marketscreener.com/ALNYLAM-PHARMACEUTICALS-8322/news/Alnylam-Pharmaceuticals-Announces-Preliminary-Fourth-Quarter-and-Full-Year-2019-Global-Net-Produc-29827002/

Aquestive Therapeutics Receives FDA Negative Response to Citizen’s Petition

Aquestive Therapeutics, Inc. (NASDAQ: AQST), a specialty pharmaceutical company focused on developing and commercializing differentiated products that meet patients’ unmet needs and solve therapeutic problems, today reported that the U.S. Food and Drug Administration (FDA) issued a response letter (Response) dated January 10, 2020 denying Aquestive’s Citizen’s Petition received by the FDA on November 1, 2019, including the supplement to the Citizen’s Petition received by the FDA on December 4, 2019 (Docket No. FDA-2019-P-5121) (Petition).  The Petition requested, among other things, that the FDA stay approval of a New Drug Application for Valtoco^® (diazepam nasal spray) submitted by Neurelis, Inc. until additional clinical studies were conducted.  In the Response, the FDA indicated that it had approved Neurelis’s NDA for Valtoco on January 10, 2020.  Valtoco has received orphan drug exclusivity from the FDA’s Center for Drug Evaluation and Research commencing as of January 10, 2020 for the labeled indication of acute treatment of intermittent stereotypic episodes of frequent seizure activity (i.e., seizure clusters, acute repetitive seizures) that are distinct from a patient’s usual seizure pattern in patients with epilepsy six years of age and older.

“This patient population has been underserved for some time with little choice beyond the rectally administered gel and choice is important.  We believe that our candidate drug Libervant™ (diazepam) Buccal Film will, if approved by the FDA, further expand patient choice as the first orally administered dosage form for this patient population,” said Keith J. Kendall, Chief Executive Officer of Aquestive.

“We appreciate that the FDA has confirmed in the Response its guidance that 505(b)(2) drugs can be approved without proving bioequivalence if they demonstrate “relative bioavailability” to the reference drug. The FDA stated in the Response, when granting exclusivity based upon “major contribution to patient care” over and above already approved products for the indication, it may consider such factors as convenience of treatment location, duration of treatment, patient comfort, reduced treatment burden, advances in ease and comfort of drug administration, longer periods between doses, and potential for self-administration.  In making this determination for Valtoco, the FDA indicated in the Response that the intranasal route of administration provides a major contribution to patient care over the rectal route of administration by providing a significantly improved ease of use.”

“We look forward to working with the FDA in the coming months to demonstrate why we believe that our product candidate Libervant, as an orally delivered product for this indication, has one or more of the attributes required by the FDA to be considered a major contribution to patient care relative to the currently approved products,” concluded Mr. Kendall.

Although we cannot be assured of FDA approval of Libervant, Aquestive remains committed to helping people affected by seizure clusters by looking to bring important innovative products to the market that will improve the lives of patients.

We provided preliminary 2020 revenue guidance in our press release dated January 10, 2020.  As a reminder, we did not include any Libervant revenues in that guidance.

https://www.marketscreener.com/AQUESTIVE-THERAPEUTICS-I-45049850/news/Aquestive-Therapeutics-Receives-FDA-Response-to-Citizen-s-Petition-29827005/

Exelixis sees $245M in Q4 revenue

Ahead of its JPM20 presentation, Exelixis (NASDAQ:EXEL) announces preliminary 2019 results and its outlook for this year.

2019: Q4 and full-year revenues expected to be ~$245M and ~$972M, respectively. Product sales should be ~$200M and ~$765M, respectively.

Final results will be released on Wednesday, February 26, after the close.

2020 guidance: Revenues: $850M – 900M; product sales (cabozantinib): $725M – 775M.

Selected 2020 milestones:

Topline data from Phase 3 Checkmate 9ER study of cabozantinib + Opdivo (nivolumab) in treatment-naïve advanced/metastatic renal cell carcinoma expected in H1.

Preliminary data from Phase 3 COSMIC-311 study of cabozantinib in patients with radioactive iodine-refractory differentiated thyroid cancer who have progressed after up to two VEGF receptor-targeted therapies (and the completion of enrollment) expected in H2.

Complete enrollment in Phase 3 COSMIC-312 study of cabozantinib + Roche’s Tecentriq (atezolizumab) versus Bayer’s Nexavar (sorafenib) in H1. Analysis of progression-free survival and interim overall survival could happen in H2.

#JPM20

https://seekingalpha.com/news/3531138-exelixis-sees-245m-in-q4-revenues

Iovance in-licenses gene-editing technology from Cellectis

Iovance (NASDAQ:IOVA) inks an agreement with Cellectis (NASDAQ:CLLS) for exclusive global rights to its TALEN gene-editing technology aimed at developing genetically edited tumor infiltrating lymphocytes (TIL) that are more effective at treating cancer.

Under the terms of the deal, Iovance will pay Cellectis undisclosed milestones and royalties on net sales of TALEN-modified TIL products. Specific financial terms remain confidential.

https://seekingalpha.com/news/3531137-iovance-in-licenses-gene-editing-technology-from-cellectis