The Daily Biotech Pulse: Protagonist's Ulcerative Colitis Study Disappoints, Gilead's Remdesivir Approved For Pediatric COVID-19 Patients, FDA Gives Nod To Gamida's Cell Therapy Trial, Fast Track Tag For Vascular Biogenics Ovarian Cancer Gene Therapy

Zinger Key Points
  • The FDA has approved Gilead Sciences' supplemental marketing application for Veklury (remdesivir) for pediatric COVID-19 patients older than 28 days.
  • The approval covers patients weighing at least 3 kg and are either hospitalized with COVID-19 or have mild-to-moderate COVID-19 and are considered high risk for progression to severe infection.

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

Stocks In Focus

Protagonist's Ulcerative Colitis Study Disappoints With Higher Dose Of PN-943

Protagonist Therapeutics Inc PTGX announced topline results from the Phase 2 IDEAL study of PN-943 in moderate-to-severe ulcerative colitis patients.

While the 450 mg BID dose did not meet the prespecified primary endpoint, the 150 mg dose achieved 27.5% clinical remission with a delta of 13% versus the placebo and a delta of 16% (p=0.04) in the bio-naïve group. 

In addition, the 150 mg BID data showed strong concordance across multiple parameters.

Shares are falling 32.7% at $12.66 during the premarket trading session.

Gilead's Remdesivir Becomes First Approved Treatment For Pediatric COVID-19 Patients

The FDA has approved Gilead Sciences Inc's GILD supplemental marketing application for Veklury (remdesivir) for pediatric COVID-19 patients older than 28 days.

The approval covers patients weighing at least 3 kg and are either hospitalized with COVID-19 or have mild-to-moderate COVID-19 and are considered high risk for progression to severe infection.

Gamida Cell Shares Surge On FDA Nod For Cell Therapy Study In Blood Cancer Settings

The FDA has signed off and removed the clinical hold for a cryopreserved formulation of Gamida Cell Ltd's GMDA GDA-201, an off-the-shelf cell therapy candidate for follicular and diffuse large B cell lymphomas. 

Gamida Cell expects to initiate a Phase 1/2 clinical study in patients with follicular and diffuse large B-cell lymphomas in 2022.

GMDA shares are up 30.9% at $3.69 during the premarket session

Valneva, Pfizer Post Encouraging Data From Lyme Disease Vaccine Candidate

Valneva SE VALN and Pfizer Inc PFE have reported Phase 2 pediatric data for their Lyme disease vaccine candidate, VLA15. 

VLA15 was found to be more immunogenic than in adults with both two-dose and three-dose vaccination schedules tested.

No vaccine-related serious adverse events were observed.

Marker Therapeutics Inks Services Agreement With Wilson Wolf For $8M

Marker Therapeutics Inc MRKR has entered into a services agreement with Wilson Wolf Manufacturing Corporation. 

The agreement includes an $8-million upfront cash payment by Wilson Wolf to Marker in exchange for services relating to Marker's expertise in manufacturing cell therapies. 

Wilson Wolf has agreed to pay Marker an additional $1 million if the certain work, as defined in the services agreement, is completed within one year from the onset of the services agreement.

Shares are up 18% at $0.39 during premarket trading.

After Bristol Myers Cuts Ties, Nektar Enacts Cost Restructuring

Nektar Therapeutics NKTR announced a new strategic plan to prioritize key research and development efforts and cut 70% of its workforce, including several changes to its executive team. 

After accounting for BEMPEG wind-down and restructuring costs, Nektar expects to end the year with approximately $440 million-$450 million in cash and investments. 

The company said it expects to take a charge of $150 million-$160 million, a substantial portion of which will be recorded in the second quarter. 

Shares are down 4.03% at $4.53 during the premarket session.

Related Link: The Week Ahead In Biotech (April 24-April 30): Bristol-Myers Squibb, Supernus, Hutchmed, Axsome, Coherus FDA Decisions, Big Pharma Earnings, IPOs.

Ambrx Biopharma's ARX788 Selected For Phase 2 Adaptive Breast Cancer Trial

Ambrx Biopharma Inc's AMAM antibody-drug conjugate (ADC) ARX788 has been selected for a new investigational treatment arm in the Quantum Leap Healthcare Collaborative-sponsored I-SPY 2.2 TRIAL.

ARX788 will be evaluated as a monotherapy, combined with the PD-1 targeting checkpoint inhibitor cemiplimab, in HER2-positive early-stage breast cancer in the neoadjuvant setting. 

Enrollment will start in May 2022.

Biocytogen Signs RenMab/RenLite Licensing Agreement With BeiGene

BeiGene Limited BGNE has in-licensed Biocytogen Pharmaceuticals (Beijing) Co Ltd's fully human antibody RenMab/RenLite mice platforms to develop fully human monoclonal antibodies, bispecific antibodies, and other types of antibody drugs.

BGNE stock is up 0.1% at $162.96 in premarket trading.

VBL Biogenics Receives Fast Track Tag For Ovarian Cancer Gene Therapy

The FDA has granted Fast Track designation to VBL Biogenics Ltd's VBLT ofra-vec (ofranergene obadenovec or VB-111) combined with paclitaxel for platinum-resistant ovarian cancer.

VBL recently completed patient enrollment of 409 patients in this global trial. In addition, the Independent Data Safety Monitoring Committee unanimously recommended continuing the trial as planned, following a review of unblinded data from 370 randomized patients.

Shares are up 11.7% at $1.62 during the premarket session Tuesday. 

Clinical Readouts/Presentations

Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR Tandem Meetings (TCT) 

Magenta Therapeutics Inc MGTA: Phase 2 Study of MGTA-145 + Plerixafor in hematopoietic stem cell mobilization for autologous stem cell transplant in multiple myeloma patients.


Nkarta Inc NKTX has priced an underwritten public offering of 13.3 million shares at $15 per share, for gross proceeds of $200 million

Underwriters have an option to purchase up to 2 million additional shares.

Shares are down 4.59% at $17.86 during premarket trading.

On The Radar

Novartis AG NVS Q1 Earnings.

Agile Therapeutics Inc's AGRX 1-for-40 reverse stock split effective tomorrow.

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