The Daily Biotech Pulse: Aquestive's Positive FDA Meeting, Prevail Awarded Patent, Neuro-Oncology Conference Gets Underway

Here's a roundup of top developments in the biotech space over the last 24 hours.

Scaling The Peaks

(Biotech Stocks Hitting 52-week Highs Nov. 18)

• Amicus Therapeutics, Inc. FOLD
• Atara Biotherapeutics Inc ATRA
• Biodesix Inc BDSX
• C4 Therapeutics Inc CCCC
• CareDx Inc CDNA
• Generation Bio Co GBIO
• Halozyme Therapeutics, Inc. HALO
• Immunovant Inc IMVT
• Intellia Therapeutics Inc NTLA
• Insmed Incorporated INSM
• Kazia Therapeutics Ltd KZIA ( reacted to positive brain tumor study)
• Keros Therapeutics Inc KROS
• Kodiak Sciences Inc KOD
• Kymera Therapeutics Inc KYMR
• Morphic Holding Inc MORF
• NeoGenomics, Inc. NEO
• Oncorus Inc ONCR
• Pacira Biosciences Inc PCRX (announced European regulatory nod for drug to treat post-operative pain)
• Prelude Therapeutics Inc PRL
• PTC Therapeutics, Inc. PTCT(announced rare pediatric disease designation and orphan drug designation for PTC596 in the treatment of rare cancer of smooth muscle tissue)
• Replimune Group Inc REPL
• Rocket Pharmaceuticals Inc RCKT
• Seres Therapeutics Inc MCRB
• Shockwave Medical Inc SWAV
• SpringWorks Therapeutics Inc SWTX
• Spero Therapeutics Inc SPRO
• Sutro Biopharma Inc STRO
• Trillium Therapeutics Inc TRIL
• Twist Bioscience Corp TWST
• Veracyte Inc VCYT

Down In The Dumps

(Biotech Stocks Hitting 52-week Lows Nov. 18)

• Codiak BioSciences Inc CDAK
• Polypid Ltd PYPD

Stocks In Focus

Aquestive Plans Resubmission of Seizure Cluster Drug NDA Following Positive Type A Meeting With FDA

Aquestive Therapeutics Inc AQST announced the completion of a Type A meeting with FDA, confirming a pathway for resubmission of the NDA for its drug candidate Libervant buccal Film for management of seizure clusters.

The original application was handed with a complete response letter in late September.

The FDA confirmed that the issues identified in the CRL may be addressed by utilizing modeling and simulations based upon the information provided by Aquestive in its FDA meeting package submitted in October 2020.

Based on the FDA's preliminary comments and the discussion with the FDA during the Type A meeting, Aquestive said it continues to believe that no additional clinical studies will be required for the resubmission of the NDA for Libervant. Aquestive will work to prepare the analysis requested and schedule the follow-up meeting with the FDA as soon as is practical. The company expects to resubmit the NDA for Libervant as soon as possible after the follow-up meeting, which has yet to be scheduled.

The stock gained 7.50% to $6.74 in after-hours trading.

Prevail Therapeutics Announces Patent Award Related to Gene Therapy Candidate For Neurological Disorders

Prevail Therapeutics Inc PRVL said the U.S. Patent and Trademark Office issued a composition of matter patent, with claims directed to the AAV vector used in PR001, its experimental gene therapy program for the treatment of Parkinson's disease with GBA1 mutations and neuronopathic Gaucher disease. The base patent term extends until Oct. 3, 2038, excluding patent term extensions or coverage in additional related patent filings.

Qiagen to Collaborate With BioNTech For Companion Diagnostic Test

Qiagen NV QGEN announced a strategic collaboration with BioNTech SE – ADR BNTX to develop and commercialize a tissue-based companion diagnostic to be used with investigational cancer treatment BNT113, which identifies patients with squamous cell carcinoma of the head and neck that are caused by specific infections by human papilloma virus.

Pfizer Announces Publication of Positive Phase 3 Results For Lung Cancer Drug

Pfizer Inc. PFE announced publication in the New England Journal of Medicine positive results from the Phase 3 CROWN trial of Lorbrena in people with previously untreated ALK-positive advanced non-small cell lung cancer. At a planned interim analysis, Lorbrena treatment resulted in statistically significant and clinically meaningful improvement in progression-free survival, according to blinded independent central review, the primary endpoint, compared to Xalkori, corresponding to a 72% reduction in the risk of progression or death.

The trial is continuing for the secondary endpoint of overall survival, which was not mature at the time of analysis.

Separately, Pfizer announced a collaboration with LianBio, aimed at developing and commercializing transformative pharmaceutical products in Greater China.

Orchard Announces FDA Nod For its IND Application For Gene Therapy to Treat Rare Inherited Disease

Orchard Therapeutics PLC – ADR ORTX said the FDA has cleared its investigational new drug application for OTL-200, an autologous, hematopoietic stem cell, lentiviral vector-based gene therapy in development for the treatment of metachromatic leukodystrophy.

Lantheus Announces FDA Approval For Room Temperature Formulation For Contrast Agent Used In Echocardiograms

Lantheus Holdings Inc LNTH said the FDA has approved the supplemental new drug application for Definity Room Temperature injectable suspension.

"With the approval of DEFINITY RT, we expand our microbubble franchise offering to include a room temperature formulation, in addition to our market leading, refrigerated DEFINITY that our customers and patients have trusted to enhance suboptimal echocardiograms for 19 years," the company said.

The product will be commercially available in early 2021.

The stock ended up 8.58% to $13.41 in after-hours trading.

Offerings

MeiraGTx Holdings PLC MGTX said it has commenced an underwritten public offering of 5 million shares of its ordinary shares. All of the ordinary shares are being offered by the company.

The stock fell 8.43% to $13.15 in after-hours trading.

Stoke Therapeutics Inc STOK announced a proposed underwritten public offering in which it intends to offer up to 2.5 million shares of its common stock. All of the shares of common stock are being offered by Stoke.

Immutep ADS Representing 10 Ord Shs IMMP said it has successfully completed a A$29.6 million a private placement of ordinary shares to professional and institutional investors.

The stock was down 5.94% to $1.90 in after-hours trading.

On The Radar

Clinical Readout/Presentations

2020 Society for Neuro-Oncology Meeting Presentations:

VBI Vaccines Inc VBIV: data from Part B of the ongoing Phase 1/2a study of VBI-1901, its cancer vaccine immunotherapeutic candidate, in recurrent glioblastoma

Kintara Therapeutics Inc KTRA: update on Phase 2 studies of VAL-083 in unmethylated recurrent glioblastoma multiforme

Plus Therapeutics Inc PSTV: new interim data from the ongoing NIH-sponsored ReSPECT Phase 1 clinical trial evaluating its lead investigational asset, Rhenium NanoLiposome in patients with recurrent glioblastoma

Kazia Therapeutics: initial Phase 1 data for Paxalisib in diffuse intrinsic pontine glioma

Earnings

Entera Bio Ltd ENTX (before the market open)
Precipio Inc PRPO (after the close)