Researchers from MD Anderson Cancer Center have published new findings that argue for a role of the long noncoding RNA (lncRNA) metastasis-associated lung adenocarcinoma transcript 1 (MALAT1) as a metastasis suppressor, rather than promoter, as previous work had suggested. Read More
In the company's second program with UX-007 (triheptanoin, or trihep), Ultragenyx Pharmaceutical Inc.'s data are "much more robust" – enough so that the FDA recently said the firm could file an NDA based on phase II findings, spokesperson Danielle Keatley told BioWorld. Read More
Shares of Syndax Pharmaceuticals Inc. (NASDAQ:SNDX) fell 17.1 percent to $5.01 Friday on news that adding its HDAC inhibitor, entinostat, to an aromatase inhibitor failed to deliver a statistically significant improvement in the length of time women with hormone receptor-positive, HER2-negative breast cancer lived without worsening of their cancers. The outcome, from the ongoing phase III study E2112, dashed hopes of an early regulatory filing, putting the focus on the trial's other co-primary endpoint, overall survival (OS). Read More
A day after Biogen Inc. and Eisai Inc. thoroughly confused and mainly disappointed the Alzheimer's disease (AD) field with updated data on BAN-2401, Anavex Life Sciences Corp. took the podium at the 2018 Clinical Trials on Alzheimer's Disease (CTAD) meeting in Barcelona for an oral presentation on lead candidate ANAVEX-2-73. In a starkly different turn from the amyloid beta approach, the oral sigma-1 receptor (S1R) agonist, a tetrahydrofuran derivative, binds to muscarinic acetylcholine 1, seeking to improve measures such as Alzheimer's Disease Cooperative Study-Activities of Daily Living (ADCS-ADL), psychomotor function, attention and working memory. Read More
HAMBURG, Germany – Regenerative medicine startup company Precise Bio Inc. is launching a dedicated ophthalmology business unit to help raise funds and attract partners as it continues to develop bio-printed tissues and organs technology that one day could result in a 3D-printed cornea graft in the human eye. Read More
HONG KONG – Leading Chinese vaccine maker Sinovac Biotech Ltd. resorted to the Hong Kong courts to fight off an attempted "coup" by a minority shareholder – an attempt that involved presenting allegedly fake documents filed in Hong Kong that suggested top management at Sinovac Biotech's holding company had resigned. Read More
Biomarin Pharmaceutical Inc., of San Rafael, Calif., reported that it generated total revenues of $391.7 million for the third quarter, compared to $334.1 million for the quarter ending Sept. 30, 2017, an increase of 17 percent. Net product revenues were $386.3 million, compared to $298.8 million in 2017. Read More
Gamida Cell Ltd., of Boston, said it priced its IPO of 6.25 million shares at $8 each, for aggregate gross proceeds of $50 million. The underwriters have been granted a 30-day option to purchase up to 937,500 additional shares at the initial offering price. The company's shares began trading on Nasdaq under the ticker symbol GMDA, closing Friday at $8.44. Read More
Quadrant Biosciences Inc., of Syracuse, N.Y., is collaborating with the Autism Speaks Autism Treatment Network. Quadrant will provide financial support for the Signature Study, testing the antibiotic minocycline on patients with autism spectrum disorder, and will collect salivary RNA data to further elucidate epigenetic factors associated with the disease. Read More
