Daily Biotech Pulse: Liver Disease-Focused Gene Therapy Pact, Assembly Bio Stops HBV Infection Trial, X4 Pharma Looks For Partners

Daily Biotech Pulse: Liver Disease-Focused Gene Therapy Pact, Assembly Bio Stops HBV Infection Trial, X4 Pharma Looks For Partners

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

Stocks In Focus

Verve Therapeutics, Vertex Join Forces For Liver Disease-Focused Gene Program

Vertex Pharmaceuticals Incorporated VRTX and Verve Therapeutics Inc VERV announced an exclusive, four-year global research collaboration to discover and develop an in vivo gene editing program for undisclosed liver disease.

Verve will receive an upfront payment of $60 million, including a $35-million equity investment in Verve. 

Verve is also eligible to receive up to $66 million in success payments, up to $340 million in development and commercial milestones and sales-based royalties.

Fourth Canadian Approval For Abbvie's JAK Inhibitor

Health Canada approved AbbVie Inc's ABBV Rinvoq (upadacitinib) for active ankylosing spondylitis in patients who did not respond to biologic treatments.

The approval is supported by efficacy and safety data of two pivotal studies in which Rinvoq demonstrated improvements in disease activity and function.

This marks the fourth Health Canada-approved indication for Rinvoq.

Disappointing Data Forces Assembly Bio Stop Combo Trial For HBV Infection 

Assembly Biosciences Inc ASMB will stop working on its first-generation core inhibitor, vebicorvir (VBR), as it prioritizes its next-gen core inhibitors ABI-H3733 and ABI-4334, and advances its research pipeline. 

The decision follows the review of interim efficacy from the two triple combination studies that did not show a benefit in multiple key viral parameters compared to the combinations without VBR.

The company said that its partner Arbutus Biopharma Corporation ABUS, will continue dosing in the Phase 2a triple combination evaluating AB-729, VBR, and nucleos(t)ide analog therapy for chronic HBV infection. Preliminary data are expected in 2H of 2022.

The company workforce will be reduced by approximately 30%.

X4 Pharma To Stop Further Work On Oncology Programs To Sharpen Strategic Focus

X4 Pharmaceuticals Inc XFOR updated its strategic priorities that include streamlining resources to focus on advancing its lead candidate, mavorixafor, in WHIM syndrome and other chronic neutropenic disorders.

The company will be progressing its clinical and preclinical oncology programs only via a potential partnership(s).

Discontinuation of further work on oncology programs and a workforce reduction of approximately 20% would save around $5 million in 2022 and $20 million in 2023.

Related Link:  Attention Biotech Investors: Mark Your Calendar For These July PDUFA Dates.

HilleVax's Norovirus Vaccine Shows Long-Term Immunogenicity

HilleVax Inc HLVX announced results from the NOR-213 Phase 2 trial of HIL-214, a virus-like particle-based vaccine for preventing moderate-to-severe acute gastroenteritis caused by norovirus infection.

Among all dose regimens of HIL-214, GI.1-specific, and GII.4-specific HBGA-blocking and pan-Ig responses to vaccination persisted to year five and, at year five, results were similar to those previously reported at year three. 

Karyopharm's Lead Asset Gets Complete European Approval For Multiple Myeloma

The European Commission has approved Karyopharm Therapeutics Inc KPTI and its partner Menarini Group's lead asset Nexpovio (selinexor) for pretreated multiple myeloma.

The approval comes as a combination therapy with bortezomib (Velcade) and low-dose dexamethasone for multiple myeloma patients who have received at least one prior therapy. 

With this approval for the extension of Nexpovio's indication in Europe, the conditional marketing authorization is now converted to full approval.

FDA Fast Tracked Olema Pharma's Breast Cancer Candidate

The FDA has granted fast-track designation to Olema Pharmaceuticals Inc's OLMA OP-1250 for ER-positive, human epidermal growth factor receptor 2-negative metastatic breast cancer that has progressed following one or more lines of endocrine therapy. 

OP-1250 is currently being evaluated as a single agent in an ongoing Phase 1/2 clinical trial and Phase 1b combination with palbociclib.

Insider Trading

Tricida Inc TCDA: 10% owner Nimish Shah acquired 150,000 shares at an average price of $9.18 for around $1.38 million.

Offerings

Verve Therapeutics Inc VERV: Priced the upsized underwritten public offering of 8.3 million at $27 per share, for total gross proceeds of approximately $225 million.

Shares are slipping 14.6% at $30.67 during the premarket session.

Zynerba Pharmaceuticals Inc ZYNE has entered into an equity purchase agreement for up to $20 million with Lincoln Park Capital Fund.

On The Radar

Earnings

Roche Holdings AG RHHBY: Before the market open.

IPOs

Virax Biolabs Group Limited: Priced its IPO of 1.35 million shares at $5.00 per share, for gross proceeds of $6.75 million. Trading will start today on NASDAQ under the ticker VRAX.

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