The Daily Biotech Pulse: Aeglea Stock Jumps On Inherited Metabolic Disease Data, Partial Hold Lifted On Gilead's 2 Blood Trials, Early Data From Novartis' KRAS Inhibitor And More

Zinger Key Points
  • The FDA identified deficiencies that preclude discussion of labeling and/or post-marketing requirements related to Myovant Sciences and Pfizer Myfembree for endometriosis-associated pain.
  • Takeda Pharmaceutical's Phase 3 SHP643-301 Takhzyro (lanadelumab) study in kids ages 2 to 12 has met its objectives for hereditary angioedema. 

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

Stocks In Focus

Aeglea Shares Additional Phase 3 Data In Inherited Metabolic Disease

Aeglea BioTherapeutics Inc AGLE shared additional data from the PEACE Phase 3 study in Arginase 1 Deficiency at the Society for Inherited Metabolic Disorders (SIMD) Annual Meeting.

In an analysis of individual patients, clinically significant differences between the pegzilarginase treated patients (n=17), and placebo (n=9) were observed in motor function.

Aeglea submitted an FDA marketing application for pegzilarginase in Arginase 1 Deficiency.

The stock was trading 31.7% higher at $3.20 in premarket trading.

Novartis Highlights Early Data From KRAS Inhibitor At AACR Meeting

Novartis AG NVS shared clinical data for JDQ443, an investigational selective, covalent and orally bioavailable KRASG12C inhibitor.

Preliminary Phase 1b data showed that JDQ443 demonstrated anti-tumor activity, high systemic exposure at its recommended dose and a favorable safety profile in KRAS G12C-mutated solid tumors.

FDA Identifies Labeling Deficiency In Pfizer-Myovant's Myfembree Supplemental Application

The FDA identified deficiencies that preclude discussion of labeling and/or post-marketing requirements related to Myovant Sciences Inc MYOV and Pfizer Inc's PFE Myfembree for endometriosis-associated pain.

The agency did not provide additional detail. 

Myovant and Pfizer will continue to work with the FDA to determine the next steps with the application.

Related Link: The Week Ahead In Biotech (April 10-April 16): Regeneron FDA Decision, Cancer Conference Presentations, And More.

FDA Green Lights Gilead's Magrolimab Studies In MDS, AML

The agency has lifted the partial clinical hold placed on Gilead Sciences Inc's GILD studies of magrolimab combined with azacitidine.

Enrollment in the U.S. can resume for magrolimab plus azacitidine studies in myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). 

Gilead is also working with the FDA regarding the remaining partial clinical hold on magrolimab studies in diffuse large B-cell lymphoma and multiple myeloma.

Revive Therapeutics Issues Update On Phase 3 COVID-19 Trial

The Data Safety and Monitoring Board (DSMB) will meet this quarter to evaluate Revive Therapeutics Ltd's RVVTF Bucillamine Phase 3 trial in mild to moderate COVID-19.

The DSMB can either make recommendations or ask to potentially halt the study early due to positive efficacy based on other clinical outcomes evaluated. 

IsoPlexis Cuts Workforce To Streamline Operating Structure

IsoPlexis Corporation ISO initiated a reorganization of its workforce. It will retain approximately 80% of its prior workforce

The company expects first-quarter FY22 sales of $4.8 million-$4.9 million, up approximately 48%-52% year-over-year.

Takeda's Hereditary Angioedema Drug Aces Phase 3 Trial

Takeda Pharmaceutical Co Ltd's TAK Phase 3 SHP643-301 Takhzyro (lanadelumab) study in kids ages 2 to 12 has met its objectives for hereditary angioedema

The safety profile was consistent with that seen in the clinical program for patients 12 years of age and older.

Actinium Inks Iomab-B Pact For Europe, Middle East, North Africa

Actinium Pharmaceuticals Inc ATNM and Immedica Pharma AB announced a license and supply agreement for Iomab-B under development for targeted conditioning to facilitate bone marrow transplant (BMT) and other cell and gene therapies. 

Actinium will receive an upfront payment of $35 million and will be eligible to receive an additional $417 million in milestones.

Stock is trading 34% higher at $7.09 during the premarket session.

Insider Trading

Biodesix Inc BDSX shares skyrocketed after a series of filings disclosing stock purchases by its chairman and a couple of its directors.

John Patience bought 279,329 shares at $1.79 per share, while Matthew Strobeck and Jack Schuler purchased 558,659 and 2.79 million shares.

The stock was trading 76.1% higher at $2.80 in premarket trading.

Clinical Readouts/Presentations

American Association for Cancer Research Meeting Presentations 

C4 Therapeutics Inc CCCC: Data from the Phase 1/2 study of CFT7455 in multiple myeloma.

Checkmate Pharmaceuticals Inc CMPI: Biomarker signature data from the Phase 1b study of vidutolimod in combination with Keytruda in melanoma

Gracell Biotechnologies Inc GRCL: Early results of a safety and efficacy study of allogeneic TruUCAR GC502 in patients with relapsed/refractory B-cell acute lymphoblastic leukemia.

Alpine Immune Sciences Inc ALPN: Results from the dose-escalation portion of Phase 1 clinical trial of davoceticept (ALPN-202) as monotherapy in advanced malignancies.

HOOKIPA Pharma Inc HOOK: Phase 1/2 data for HB- 201 and HB-202 in treatment-refractory HPV16+ cancers.

Schrödinger Inc SDGR: New preclinical data from its Wee1 inhibitor program.

Repare Therapeutics Inc RPTX: Updated data from its ongoing Phase 1/2 TRESR trial of RP-3500 for solid tumors with specific synthetic-lethal genomic alterations.


Hoth Therapeutics Inc HOTH priced a public offering of 8.2 million shares at 85 cents per share, for gross proceeds of approximately $7 million.

The company will use the proceeds for general corporate and working capital purposes.

Shares are slipping 19.2% at 85 cents during premarket trading.

On The Radar

Zosano Pharma Corp's ZSAN 1-35 reverse split is effective.

Evotec SE EVO (before the market open).

Analyst Ratings




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