The Daily Biotech Pulse: Kezar Life's Lead Asset Fails, Brickell Biotech Offloads Late-Stage Asset, Connect Biopharma Ulcerative Colitis Trial Disappoints

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

Stocks In Focus

Kezar Life's Lead Asset Fails To Top Placebo In Muscle Inflammation Disease

Kezar Life Sciences Inc KZR announced topline results from the PRESIDIO Phase 2 trial of zetomipzomib (KZR-616) in patients with dermatomyositis (DM) and polymyositis (PM), chronic inflammatory diseases of muscle.

Topline results showed that most DM and PM patients saw clinically meaningful improvements in Total Improvement Score, but zetomipzomib demonstrated no significant differentiation from placebo. 

Zetomipzomib was well tolerated. 

There were three Grade 3 serious adverse events in the zetomipzomib arms. All deemed unrelated to zetomipzomib.

AstraZeneca's Imfinzi/Chemo Combo Under Priority FDA Review For Bile Duct Cancer

The FDA has accepted AstraZeneca plc's (NASDAQ) supplemental marketing application seeking approval for Imfinzi (durvalumab), in combination with standard-of-care chemotherapy, for locally advanced or metastatic biliary tract cancer.

Under the Priority Review status, the FDA action date is in the third quarter of 2022.

Click here to access Benzinga's FDA Calendar

Brickell Biotech Shares Surge After Offloading Late-Stage Asset To Australia-based Firm

Brickell Biotech Inc BBI agreed to sell sofpironium bromide rights to Australia-based Botanix Pharmaceuticals Limited.

Brickell will receive upfront and potential near-term regulatory milestone payments over the next 18 months of up to $9 million.

Brickell also is eligible to receive additional milestone payments of up to $168 million and tiered earn-out payments.

Sofpironium bromide gel, 15% recently completed a U.S. Phase 3 program for primary axillary hyperhidrosis or excessive underarm sweating. 

Shares are trading 30.3% higher at 0.30 cents during the premarket session.

Connect Biopharma Ulcerative Colitis Candidate Flunks In Mid-Stage Study

Connect Biopharma Holdings Limited CNTB announced topline results at 12 weeks from its Phase 2 trial for CBP-307 (CBP-307CN002) for ulcerative colitis.

CBP-307 0.2 mg demonstrated a numerical reduction for the primary endpoint of least squares mean change from baseline in adapted Mayo Score at Week 12 that did not meet statistical significance. 

The Mayo Score (stool frequency, rectal bleeding, and endoscopy scores) at Week 12 for CBP-307 0.2 mg and placebo were -2.65 and -2.01, respectively (p=0.103). 

The company intends to engage in partnership discussions for the future development of CBP-307 to focus on the lead program CBP-201.

Shares closed 33.5% lower at $1.29 during after-hours trading.

Phathom's Vonoprazan Regimens Scores FDA Approval For H. pylori Infection

The FDA has approved Phathom Pharmaceuticals Inc's PHAT Voquezna (vonoprazan) regimens for Helicobacter pylori infection in adults.

The approval covers Voquezna Triple Pak (vonoprazan tablets, amoxicillin capsules, clarithromycin tablets) and Voquezna Dual Pak (vonoprazan tablets, amoxicillin capsules).

Voquezna triple and dual paks are expected to be available in the U.S. in Q3 of 2022 and marketed exclusively by Phathom.

Novartis's Kymriah CAR-T Cell Therapy Scores European Approval For Follicular Lymphoma

The European Commission has approved Novartis AG's NVS Kymriah (tisagenlecleucel), CAR-T cell therapy for follicular lymphoma (FL).

The approval covers adult patients with relapsed or refractory (r/r) FL after two or more lines of systemic therapy.

This approval marks the third indication for Kymriah and makes it the first CAR-T cell therapy approved in the EU for these patients.

Also Read: Attention Biotech Investors: Mark Your Calendar For May PDUFA Dates.

New Data Shows Novartis' Kisqali Extends Survival In Postmenopausal Breast Cancer Setting

Novartis AG NVS has announced updated median overall survival (OS) results for Kisqali (ribociclib) combined with fulvestrant for postmenopausal women with hormone receptor-positive, human epidermal growth factor receptor-2 negative advanced or metastatic breast cancer. 

The analysis of patients treated in first-line with Kisqali plus fulvestrant demonstrated a significant OS benefit of nearly 16 months compared to those treated with fulvestrant alone. 

The final analysis demonstrated a statistically significant OS benefit for Kisqali combined with fulvestrant and a relative reduction in the risk of death by 28% compared to fulvestrant alone.

Aptose's HM43239 Fast Track'd In US For Relapsed/Refractory AML Setting

The FDA has granted Fast Track designation to Aptose Biosciences Inc's APTO HM43239 for relapsed or refractory acute myeloid leukemia (AML) with FLT3 mutation. 

HM43239 received orphan drug designation from the FDA for acute myeloid leukemia in 2018.

Data Presentations

Association for Research in Vision and Ophthalmology (ARVO) 

Applied Genetic Technologies Corporation AGTC, Multiple presentations related to ongoing X-linked retinitis pigmentosa and achromatopsia trials.

UNITY Biotechnology Inc UBX: 24-week results of a Phase 1 study of UBX1325 in advanced DME or wet AMD patients.

ProQR Therapeutics N.V. PRQR: Efficacy and safety of sepofarsen for CEP290-associated Leber congenital amaurosis.

Insider Trading

Tempest Therapeutics Inc TPST: Director Thomas Woiwode reported the purchase of 2.12 million shares at $2.36/share.

Offering

Allena Pharmaceuticals Inc ALNA has announced a registered direct offering of 1,436.0688 shares of its Series D Convertible Preferred Stock and 1,436.0688 shares of its Series E Convertible Preferred Stock for gross proceeds of approximately $2.8 million. 

On The Radar

Earnings

Deciphera Pharmaceuticals Inc DCPH: (Before Market Open).

Amneal Pharmaceuticals Inc AMRX: (Before Market Open).

Amarin Corporation plc AMRN: (Before Market Open).

Regeneron Pharmaceuticals Inc REGN: (Before Market Open).

G1 Therapeutics Inc GTHX: (Before Market Open).

Moderna Inc MRNA: (Before Market Open).

Seres Therapeutics Inc MCRB: (Before Market Open).

United Therapeutics Corporation UTHR: (Before Market Open).

Ionis Pharmaceuticals Inc IONS: (Before Market Open).

Apellis Pharmaceuticals Inc APLS: (Before Market Open).

Regenxbio Inc RGNX: (After Market Close)

Kura Oncology Inc KURA: (After Market Close)

Rain Therapeutics Inc RAIN: (After Market Close)

Fate Therapeutics Inc FATE: (After Market Close)

Zymeworks Inc ZYME: (After Market Close)

Allogene Therapeutics Inc ALLO: (After Market Close)

AxoGen Inc AXGN: (After Market Close)

Mirati Therapeutics Inc MRTX: (After Market Close)

Sarepta Therapeutics Inc SRPT: (After Market Close)

Jazz Pharmaceuticals PLC JAZZ: (After Market Close)

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