The Daily Biotech Pulse: Pfizer-BioNTech's COVID-19 Booster For Kids 5-11 Coming Soon, Ultragenyx In-Licenses Gene Therapy For Genetic Disorder, Ampio's AP-013 Trial Under Independent Investigation

Zinger Key Points
  • MediWound has appointed Ofer Gonen as CEO of MediWound, effective June 30, succeeding Sharon Malka, who will join the company's board. 
  • Gonen has been a MediWound board member and the CEO of Clal Biotechnology Industries Ltd, MediWound's largest shareholder, for the last five years.

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

Stocks In Focus

FDA May Authorize Pfizer-BioNTech's COVID-19 Booster For 5-11 Year Kids Soon: NYT

The FDA is expected to authorize a booster shot of Pfizer Inc PFE/BioNTech SE's BNTX COVID-19 vaccine for children ages 5 to 11, likely Tuesday, the New York Times reported, citing people familiar with the matter.

It is unclear how much demand there is for the third dose in the age group. According to the U.S. Centers for Disease Control and Prevention, just 28.8% of children ages 5 to 11 are fully vaccinated.

Applied Genetic's Vision Loss Gene Therapy Shows Encouraging Safety, Efficacy At Three Months

Applied Genetic Technologies Corporation AGTC reported three-month interim data from its ongoing Phase 2 Skyline trial of AGTC-501 for X-linked retinitis pigmentosa, an inherited condition causing progressive vision loss in boys and young men. 

AGTC-501 is a recombinant AAV viral vector-based gene therapy targeting mutations in the RPGR gene.

There were robust improvements in visual sensitivity, the trial's primary efficacy endpoint, in multiple patients three months after dosing, with a 62.5% response rate in dose group B and a 25% response rate in dose group A.

Gilead Sciences Can Resume Injectable Lenacapavir Trial For HIV Infection

The FDA has lifted the clinical hold placed on Gilead Sciences Inc's GILD Investigational New Drug Application to evaluate injectable lenacapavir for HIV treatment and HIV pre-exposure prophylaxis. 

The agency has removed the clinical hold after reviewing Gilead's comprehensive plan and corresponding data on the storage and compatibility of lenacapavir injection with an alternative vial made from aluminosilicate glass.

Ampio's Independent Committee Launches Investigation Related To AP-013 Trial

Ampio Pharmaceuticals Inc's AMPE independent special Committee of the Ampio Board of Directors has started an internal investigation regarding Ampio's AP-013 trial with the assistance of independent legal counsel.

FDA has communicated to Ampio that it does not consider data from AP-013 to be sufficient to demonstrate efficacy as a second pivotal trial for Ampion. 

Click here to access Benzinga's FDA Calendar.

Sernova, Evotec Join Forces To Develop 'Functional Cure' For Diabetes

Sernova Corp and Evotec SE EVO have announced an exclusive global strategic partnership to develop cell therapy treatment for insulin-dependent diabetes. 

The companies will leverage their respective technologies and scientific expertise to develop an implantable iPSC-based (induced pluripotent stem cells) beta cell replacement therapy.

The partnership provides Sernova with an exclusive global option to license Evotec's iPSC-based beta cells to treat type 1 and type 2 diabetes. 

MannKind Acquires Zealand Pharma's Wearable Insulin Delivery Device

MannKind Corporation MNKD has agreed to acquire Zealand Pharma A/S ZEAL V-Go for $10 million, with additional sales-based milestones plus the cost of certain inventory. 

The acquisition of V-Go allows MannKind to expand its portfolio and strengthen its commitment to providing innovative mealtime diabetes solutions.

V-Go is a once-daily, wearable insulin delivery device that helps control blood sugar for everyday lifestyles. It is worn like a patch and eliminates the need for taking multiple daily shots.

MediWound's CEO Transition

MediWound Ltd MDWD has appointed Ofer Gonen as CEO of MediWound, effective June 30, succeeding Sharon Malka, who will join the company's board. 

Gonen has been a MediWound board member and the CEO of Clal Biotechnology Industries Ltd, MediWound's largest shareholder, for the last five years.

Ultragenyx In-Licenses Abeona's Gene Therapy For Rare Genetic Metabolism Disorder

Ultragenyx Pharmaceutical Inc RARE has announced an exclusive license agreement for Abeona Therapeutics Inc's ABEO AAV gene therapy ABO-102 (now UX111) for Sanfilippo syndrome type A (MPS IIIA). 

Ultragenyx will assume responsibility for the ABO-102 program, and Abeona is eligible to receive tiered royalties of up to 10% on net sales and commercial milestone payments following regulatory approval.

FDA Plans AdComm To Discuss Cytokinetics' Heart Drug Potential

The FDA plans to convene an Advisory Committee meeting for Cytokinetics Incorporated's CYTK omecamtiv mecarbil marketing application. 

Omecamtiv mecarbil is an investigational, selective, small molecule cardiac myosin activator for heart failure with reduced ejection fraction. 

The FDA has assigned the application a standard review with a PDUFA target action date of November 30.

Clinical Readouts/Presentations

American Society of Gene & Cell Therapy (ASGCT) 25th Annual Meeting 

Ultragenyx Pharmaceutical Inc RARE: Safety and efficacy data from Phase 1/2 trial of DTX301 in adults with late-onset ornithine transcarbamylase Deficiency A.

RegenXbio Inc RGNX Interim Analysis of Data from the First in Human Study of RGX-111 Gene Therapy for Severe Mucopolysaccharidosis Type I.

Cabaletta Bio Inc CABA: Early cohort data from Phase 1 Trial of targeted DSG3-CAART cell therapy in mucosal-dominant pemphigus vulgaris.

American Thoracic Society (ATS) 2022 International Conference

Bellerophon Therapeutics Inc BLPH: Phase 2 Trial of INOpulse in Patients with Sarcoidosis Associated Pulmonary Hypertension Requiring Supplemental Oxygen.

Akari Therapeutics Plc AKTX: Two posters reporting results from the CORONET and CASCADE studies of nomacopan in COVID-19 pneumonia patients.

BELLUS Health Inc BLU: Safety and efficacy of BLU-5937 for a refractory chronic cough from the Phase 2b Soothe Trial.

On The Radar

IPO

OKYO Pharma Limited OKYO priced its IPO of 625,000 ADSs at $4/ADS. Trading will start today.

Stock Split 

PolarityTE Inc PTE: 1-for-25 reverse stock split.

Earnings

ObsEva SA OBSV: Before the market open.

Vascular Biogenics Ltd VBLT: Before the market open.

Bio-Path Holdings Inc BPTH: Before the market open.

Posted In: COVID-19 CoronavirusCOVID-19 Vaccinegene therapyBiotechEarningsLarge CapNewsPenny StocksHealth CareSmall CapFDALegalManagementGeneral