As the third-quarter earnings season ends this week, the focus in the biotech sector is mostly on pipeline and key regulatory updates. Novavax NVAX was in focus this week as investors were disappointed with the lowered guidance.

Recap of the Week’s Most Important Stories:

Novavax’s Q3 Results

Novavax incurred a third-quarter 2024 loss of 76 cents per share, narrower than the Zacks Consensus Estimate of a loss of 87 cents. In the year-ago quarter, the company reported a loss of $1.26 per share.

Revenues in the quarter amounted to $85 million, which beat the Zacks Consensus Estimate of $57 million. However, the top line declined 55% on a year-over-year basis.

Novavax expects 2024 total revenues to be in the range of $650-$700 million, down from its previously issued guidance of $700-$800 million due to lower COVID-19 vaccine uptake in ex-U.S. markets.

Novavax also announced that the FDA has lifted the clinical hold on its two pipeline programs, COVID-19-Influenza Combination (CIC) and stand-alone influenza vaccine candidates. The agency had initially placed this hold after a study participant who took the CIC shot as part of a phase II study reported nerve damage. With this hold removed, management is working with study investigators and other partners to start the phase III immunogenicity study on both vaccines as quickly as possible.

Novavax currently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Syndax Announces Data From AML Study

Syndax Pharmaceuticals SNDX announced positive top-line results for revumenib, an oral small-molecule menin inhibitor, from the AUGMENT-101 study. The results were announced from the relapsed or refractory (R/R) mutant NPM1 (mNPM1) acute myeloid leukemia (AML) cohort in the phase II portion of the AUGMENT-101 study of revumenib.

The primary endpoint was met with a complete remission (CR) plus CR with partial hematological recovery (CRh) rate of 23%. The overall response rate was 47% in a heavily pre-treated population, including 75% with prior venetoclax exposure in the efficacy population. 17% of patients who achieved an overall response underwent hematopoietic stem cell transplant following treatment with revumenib, with three resuming revumenib therapy post-transplant. The candidate showed a favorable safety and tolerability profile, as only 5% of patients discontinued due to treatment-related adverse events.

The new drug application (NDA) for revumenib, an oral menin inhibitor, for the treatment of adult and pediatric relapsed or refractory (R/R) KMT2A-rearranged (KMT2Ar) acute leukemia was granted Priority Review and is being reviewed under the FDA’s Real-Time Oncology Review Program with a target action date of Dec. 26, 2024.

Syndax plans to submit a supplemental NDA (sNDA) for treatment of R/R mNPM1 AML in the first half of 2025.

However, shares declined despite the positive data readout as the efficacy data did not meet investors’ expectations.

RNA Surges on Study Data

Avidity Biosciences RNA announced that it is expanding its current RNA-based pipeline of rare muscle disorders to explore a new therapeutic area — precision cardiology. The company is advancing two new wholly-owned pipeline drugs, namely AOC 1086 and AOC 1072, targeting two rare genetic cardiomyopathies, PLN cardiomyopathy and PRKAG2 syndrome, respectively. Shares of the company surged on the news.

RNA stated that both AOC 1086 and AOC 1072 have been designed to address the root causes of the genetic diseases. Based on preclinical studies conducted by Avidity, both drugs have demonstrated robust delivery of siRNA (a type of RNA molecule) against targets in the heart and reduced the number of disease-causing genes by nearly 80%.

Avidity also shared first look at the next generation, including siRNA modifications and evolved antibody engineering. In preclinical studies, these advancements have shown a 30-fold increase in siRNA delivery to skeletal muscle and extended durability, achieving sustained target inhibition for three months.

Performance

The Nasdaq Biotechnology Index has lost 1.94% in the past five trading sessions and Moderna’s shares have lost 18.66%. In the past six months, shares of MRNA have lost 66.47%. (See the last biotech stock roundup here: Biotech Stock Roundup: FOLD Settles Patent Dispute, Updates From EDIT & More)

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What's Next in Biotech?

Stay tuned for more pipeline updates.

 

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