The Daily Biotech Pulse: Arrowhead Out-Licenses NASH Drug, Tonix Touts COVID-19 Treatment Data, Aadi Jumps On FDA Nod For Rare Tumor Drug

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

Stocks In Focus

Synlogic Presents Positive Phase 2 Data For Synthetic Biologic Treatment In Inherited Metabolic Disorder

Synlogic, Inc. SYBX presented at the International Congress of Inborn Errors of Metabolism Meeting interim data from its Phase 2 SynPheny-1 clinical trial showing that treatment with the investigational synthetic biotic medicine SYNB1618 resulted in significant reductions in plasma phenylalanine levels in patients with phenylketonuria.

Eight patients treated with SYNB1618 saw a 40% reduction in D5-Phe absorption after a meal challenge, a 20% reduction in mean fasting plasma Phe across all subjects, and over 250 µM mean reduction in fasting plasma Phe among responder subjects, the company said.

The stock was adding 7.29% to $2.65 in premarket trading.

Amryt Announces 3-Month Extension In Review Period For Genetic Skin Disease Treatment

Amryt Pharma plc AMYT said the Food and Drug Administration has extended the review period for the new drug application for Oleogel-S10 for the treatment of the cutaneous manifestations of junctional and dystrophic epidermolysis bullosa, a rare and distressing genetic skin disorder affecting young children and adults for which there is currently no approved treatment.

Following a three-month extension, the new PDUFA goal date is Feb. 28, 2022. The extension is to allow time to review additional analysis of data previously submitted by Amryt.

The stock was down 5.42% to $10.64 in premarket trading.

Arrowhead To Out-License Phase 2 NASH Asset To GlaxoSmithKline For Up To $1.03B

Arrowhead Pharmaceuticals, Inc. ARWR announced that it entered into an exclusive license agreement with GlaxoSmithKline GSK under which the latter will develop and commercialize ARO-HSD, Arrowhead's investigational RNA interference therapeutic in a Phase 1/2 trial that is currently being developed as a treatment for patients with nonalcoholic steatohepatitis.

Under the terms of the agreement, Arrowhead will receive an upfront payment of $120 million and is eligible for additional payments of $30 million at the start of Phase 2 and $100 million upon achieving a successful Phase 2 trial readout and the first patient dosed in a Phase 3 trial.
Furthermore, should the Phase 3 trial read out positively, and the potential new medicine receives regulatory approval in major markets, the deal provides for commercial milestone payments of up to $190 million at first commercial sale, and up to $590 million in sales-related milestone payments. Arrowhead is further eligible to receive tiered royalties on net product sales.

Arrowhead shares were gaining 4.62% to $72.48 in premarket trading.

Bristol-Myers Squibb's Zeposia Gets The Nod For Ulcerative Colitis In Europe

Bristol-Myers Squibb Company BMY announced the European Commission has granted a marketing authorization for Zeposia for the treatment of adults with moderately to severely active ulcerative colitis, who have had an inadequate response, lost response, or were intolerant to either a conventional therapy or a biologic agent.

Tonix Announces Positive Results From Lab Studies Of COVID-19 Antiviral Treatment

Tonix Pharmaceuticals Holding Corp. TNXP announced publication in JCI Insight positive results for its sangivamycin against SARS-CoV-2.

The company said in vitro studies show sangivamycin, the active pharmaceutical ingredient in the its TNX-3500 product candidate, is a potent antiviral against SARS-CoV-2, the cause of COVID-19, and suppresses viral replication in tissue culture with greater efficacy than remdesivir, the active pharmaceutical ingredient of Gilead Sciences, Inc.'s GILD Veklury.

When tested in combination with remdesivir, both drugs had additive rather than competitive effect against SARS-CoV-2. The new data show that TNX-3500 has similar low nanomolar antiviral activity in laboratory-based assays against multiple variants of SARS-CoV-2, including the delta variant.

Separately, the company said the FDA has cleared the investigational new drug application to support the initiation of a Phase 2 study of TNX-1900 for the prevention of migraine headache in chronic migraineurs.

In premarket trading, the stock was up 20.08% at 55 cents.

Related Link: The Week Ahead In Biotech (Nov. 21-Nov. 27): Takeda, Aadi, Fennec FDA Decisions, Earnings In The Spotlight In Holiday-Shortened Week

Aadi's PEComa Treatment Fyarro Gets FDA Approval

Aadi Bioscience, Inc. AADI announced the FDA has approved Fyarro for intravenous use for the treatment of adult patients with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor.

Fyarro is the first and only FDA-approved treatment for advanced malignant PEComa in adults.

The stock was soaring 56.7% to $39.99 in premarket trading.

Click here to access Benzinga's FDA Calendar.

On The Radar

PDUFA Dates

The FDA is scheduled to rule on Takeda Pharmaceutical Company Limited's TAK new drug application for maribavir (TAK-620), which is being evaluated for the treatment of refractory cytomegalovirus infection and disease with genotypic resistance to ganciclovir, valganciclovir, foscarnet or cidofovir in transplant recipients. The PDUFA date is set for Tuesday, Nov. 23.

Clinical Readouts

ICIEM Meeting Presentations

Abeona Therapeutics Inc. ABEO: interim results of Transpher A, a multicenter, single-dose, phase 1/2 clinical trial of ABO-102 gene therapy for Sanfilippo syndrome type A (mucopolysaccharidosis IIIA)

Applied Therapeutics, Inc. APLT: new data on burden of illness for AT-007 in adults with galactosemia

Earnings

Medtronic plc MDT (before the market open)

Related Link: Attention Biotech Investors: Mark Your Calendar For November PDUFA Dates