Three months after Reata Pharmaceuticals suggested its Friedreich’s ataxia program omaveloxolone could be delayed, the company revealed that is indeed going to be the case.
Reata $RETA shares took a nosedive Wednesday after the biotech revealed that the FDA said supplemental data for its pivotal trial did not strengthen the case for approval. As a result, the drug is likely to need another study before the FDA takes up the case.
In early Wednesday morning trading, Reata stock was down roughly 8% off the news. It’s a smaller dive than when the company first said the program could be set back, though, as shares lost about a third of their value at the time.
As such, this news was not entirely unexpected but still proved disappointing to analysts.
“While the path forward for omaveloxolone in the US remains to be determined, we believe it is largely derisked by the positive results seen” in the study, SVB Leerlink’s Joseph Schwartz wrote to investors.
Baird’s Brian Skorney took a more bullish view, describing the update as merely an “incremental negative.” Skorney said he has been operating with a delay being the “base-case assumption” and that the news doesn’t affect Reata’s other program, bardoxolone for rare kidney diseases, which is expected to be filed in the first quarter of 2021.
The FDA did not rule out reconsidering omaveloxolone’s application once the new study has been completed, Reata noted.
Four months after nabbing an expanded label in the US for active psoriatic arthritis, J&J’s Tremfya has gotten the OK for the indication in Europe as well.
Tremfya, also known as guselkumab, had previously been approved on the continent for moderate to severe plaque psoriasis. The drug is a monoclonal antibody targeting p19 in the IL-23 and inhibits its interaction with the IL-23 receptor.
Wednesday’s approval comes on the basis of two Phase III studies looking at adults who had active PsA and those who were biologic-naïve. Both studies looked at patients who had previously failed standard therapies.
Back when it first got US approval in 2017, analysts pegged peak annual sales for the drug at $1.6 billion, which would make it a top 20 drug. Through the first three quarters of 2020, Tremfya had netted J&J and Janssen $965 million worldwide.
Since January 2020, the clinical trials ecosystem has quickly evolved to manage roadblocks impeding clinical trial integrity, and patient care and safety amid a global pandemic. Closed borders, reduced air traffic and delayed or canceled flights disrupted global distribution, revealing how flexible logistics and supply chains can secure the timely delivery of clinical drug products and therapies to sites and patients.
In mid-August, as Kodak’s $765 million government-backed push into drug manufacturing slowly fell apart in national headlines, Ginkgo Bioworks CEO Jason Kelly got a message from his company’s government liaison: HHS wanted to know if they, too, might want a loan.
The government’s decision to lend Kodak three quarters of a billion dollars raised eyebrows because Kodak had never made drugs before. But Ginkgo, while not a manufacturing company, had spent the last decade refining new ways to produce materials inside cells and building automated facilities across Boston.
Two biotechs said they got turned away by the FDA on Wednesday, in part due to pandemic-related travel restrictions.
North Carolina-based Liquidia Technologies was handed a CRL for its lead pulmonary arterial hypertension drug, citing the need for more CMC data and on-site pre-approval inspections, which the FDA hasn’t been able to conduct due to travel restrictions. The agency also deferred its decision on Revance Therapeutics’ BLA for its frown line treatment, because it needs to inspect the company’s northern California manufacturing facility. The action, Revance emphasized, was not a CRL.
→ San Diego protein degradation startup Vividion Therapeutics has made a change at the top with Jeffrey Hatfield taking the helm as CEO, replacing Diego Miralles six months after Roche forked over $135 million to collaborate with Vividion on their small molecule degraders. Hatfield is chairman of the board at miRagen Therapeutics and previously held the CEO job at Zafgen and Vitae Pharmaceuticals. He also had a series of leadership roles at Bristol Myers Squibb from 1996-2004, including SVP, immunology and virology divisions.
When Shanghai’s STAR board began opening its doors to biotech, it was considered not just a rival to Nasdaq but also the stock exchange in Hong Kong. Those perceptions may take an amicable turn as China expands a mutual access program with the city.
The changes mean investors in mainland China will be able to own Hong Kong biotech chapter stocks, while those in Hong Kong — a much more internationally connected group — would have access to those listed on STAR. In effect, it turns the Shanghai market into a globally accessible exchange overnight while also broadening a key source of revenue for HKEX.
It’s been a banner year for the once humdrum business of manufacturing drugs, particularly vaccines. Billions have been spent ramping up facilities for Covid-19 jabs, while individual CDMOs have expanded their facilities, apparently anticipating demand or responding to a government-led push to onshore drug manufacturing.
Now Baxter Biopharma Solutions, the CDMO wing of the many-armed healthcare giant Baxter, is getting in on the game. On Tuesday, they announced plans to spend $50 million to expand their flagship, 600,000 square-foot facility in Bloomington, IN.
Novartis is always one of the industry’s biggest R&D spenders. As they often do toward the end of each year, company execs are highlighting the drugs they expect will most likely be winners in 2021.
And they’re also dreaming about some potential big-time lottery tickets.
As part of its annual investor presentation Tuesday, where the company allows investors and analysts to virtually schmooze with the bigwigs, Novartis CEO Vas Narasimhan will outline what he thinks are the pharma’s “Wild Cards.” The slate of five experimental drugs are those that Novartis hopes can be high-risk, high-reward entrants into the market over the next half-decade or so, and cover a wide range of indications.
On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.
With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.
The European Union is looking at ways to bypass patent protections and make it easier to make generic drugs in cases of emergency such as the Covid-19 pandemic, a new document says.
Normally, under WTO regulations, the practice known as “compulsory licensing” is allowed in exceptional circumstances and could be applied as a waiver to bypass patent holders. Wednesday’s document was published as part of the EU’s plan to shore up the intellectual property rights of its member states.
三个月后，ReaTa Pharmaceuticals建议其Friedreich的Ataxia Program OmaveloxoLone可以被删除，事实就是如此。
总部位于北卡罗来纳州的Liquidia Technologies获得了其主要肺动脉高压药物的CRL，理由是需要更多的CMC数据和现场审批前检查。由于旅行限制，FDA一直未能进行这些检查。该机构还推迟了对Revance Therapeutics的BLA用于其皱眉线治疗的决定，因为它需要检查该公司在北加利福尼亚的制造设施。Revance强调，这次行动不是一次CRL。
圣地亚哥蛋白质降解初创公司Vividion Therapeutics进行了高层调整，Jeffrey Hatfield接替Diego Miralles担任首席执行官，六个月前，罗氏花了1.35亿美元与Vividion合作研发小分子降解剂。哈特菲尔德是miRagen Therapeutics公司的董事长，之前曾在Zafgen和Vitae Pharmaceuticals公司担任首席执行官。1996年至2004年期间，他还在百时美施贵宝公司担任过一系列领导职务，包括免疫学和病毒学部门高级副总裁。