CymaBay shares get a bounce on pos­i­tive PBC PhII da­ta

Gideon Hirschfeld

CymaBay Ther­a­peu­tics got a bad­ly need­ed boost this morn­ing af­ter its lead drug scored some pos­i­tive da­ta in a Phase II tri­al.

The Newark, CA-based biotech — the old Metabolex, which has had its share of ups and downs over the years — says two dos­es of saledel­par demon­strat­ed an abil­i­ty to slash lev­els of al­ka­line phos­phatase among treat­ment-re­sis­tant pa­tients suf­fer­ing from pri­ma­ry bil­iary cholan­gi­tis. The 5 mg and 10 mg dos­es cut AP lev­els by 39% and 45% from base­line af­ter 12 weeks of ther­a­py.

That’s what in­vestors want­ed to hear. The stock $CBAY shot up 23% on the news of this drug, which still has a con­sid­er­able clin­i­cal path­way ahead.

Not on­ly were there no se­ri­ous ad­verse events in the study — a key con­sid­er­a­tion giv­en the tar­get — in­ves­ti­ga­tors re­port­ed that transam­i­nase lev­els dropped. Liv­er mark­ers of cholesta­sis — in­clud­ing gam­ma glu­tamyl trans­ferase and to­tal biliru­bin — were al­so im­proved, ac­cord­ing to the com­pa­ny. And there was no ev­i­dence of drug-in­duced itch­ing, or pru­ri­tis.

CymaBay float­ed its IPO back in 2014, af­ter the com­pa­ny — as Metabolex — had burned through $349 mil­lion and watched Sanofi punt a big part­ner­ship in 2011. Since then it’s been do­ing a con­sid­er­able amount of re­or­ga­niz­ing.

“The da­ta emerg­ing from this study are im­pres­sive and sup­port our hy­poth­e­sis that low­er dos­es of se­ladel­par than pre­vi­ous­ly stud­ied re­tain strong ef­fi­ca­cy with­out rais­ing a con­cern with transam­i­nase el­e­va­tions. We al­so see that se­ladel­par ac­tiv­i­ty is not as­so­ci­at­ed with drug-in­duced itch, an im­por­tant ben­e­fit for pa­tients with PBC. If these re­sults are main­tained over longer pe­ri­ods, we think that se­ladel­par could of­fer pa­tients sig­nif­i­cant ad­van­tages over ex­ist­ing treat­ments,” said Gideon Hirschfeld of the Cen­tre for Liv­er Re­search, Uni­ver­si­ty of Birm­ing­ham, UK.

Im­ple­ment­ing re­silience in the clin­i­cal tri­al sup­ply chain

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.

Gen­mab ax­es an ADC de­vel­op­ment pro­gram af­ter the da­ta fail to im­press

Genmab $GMAB has opted to ax one of its antibody-drug conjugates after watching it flop in the clinic.

The Danish biotech reported Tuesday that it decided to kill their program for enapotamab vedotin after the data gathered from expansion cohorts failed to measure up. According to the company:

While enapotamab vedotin has shown some evidence of clinical activity, this was not optimized by different dose schedules and/or predictive biomarkers. Accordingly, the data from the expansion cohorts did not meet Genmab’s stringent criteria for proof-of-concept.

Vas Narasimhan, Novartis CEO (Jason Alden/Bloomberg via Getty Images)

Vas Narasimhan's 'Wild Card' drugs: No­var­tis CEO high­lights po­ten­tial jack­pots, as well as late-stage stars, in R&D pre­sen­ta­tion

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.

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In fi­nal days at Mer­ck, Roger Perl­mut­ter bets big on a lit­tle-known Covid-19 treat­ment

Roger Perlmutter is spending his last days at Merck, well, spending.

Two weeks after snapping up the antibody-drug conjugate biotech VelosBio for $2.75 billion, Merck announced today that it had purchased OncoImmune and its experimental Covid-19 drug for $425 million. The drug, known as CD24Fc, appeared to reduce the risk of respiratory failure or death in severe Covid-19 patients by 50% in a 203-person Phase III trial, OncoImmune said in September.

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Pascal Soriot (AP Images)

UP­DAT­ED: As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

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.

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News brief­ing: Gilead part­ner Gala­pa­gos sells off CRO for $37M; Polyphor bags $3.3M from CF Foun­da­tion

Close Gilead ally Galapagos is selling off one of its contract research organizations to a Polish pharma company.

Galapagos has agreed to sell 100% of the outstanding shares in the CRO Fidelta to Selvita, in a deal worth roughly $37 million expected to close in the first week of January. The acquisition is expected to nearly double Selvita’s revenues, the company says, as well as expand its drug discovery efforts.

Michelle Longmire, Medable CEO (Jeff Rumans)

Med­able gets $91M for vir­tu­al clin­i­cal tri­als, bring­ing to­tal raise to $136M

As biotechs look to get clinical studies back on track amid the pandemic, Medable returned to the venture well for the second time this year, bagging a $91 million Series C to build out its virtual trial platform.

The software provider recently launched three new apps for decentralizing clinical trials, and saw a 500% revenue spike this year. And it isn’t alone. Back in August, Science 37 secured a $40 million round for its virtual trial tech, with support from Novartis, Sanofi Ventures and Amgen. Patients and researchers are taking a liking to the online approach, suggesting regulators could allow it to become a new normal even after the pandemic is over.

PhRMA sues Trump gov­ern­ment over drug im­por­ta­tion rule — days be­fore it's set to be ef­fec­tive

Ever since President Donald Trump floated the idea of using state-sponsored importation to lower drug prices, PhRMA has made its opposition abundant. Not only is the proposal dangerous and futile,  but the trade group has also argued that it may even be illegal.

Now that the FDA has issued its final rule permitting states to bring certain drugs from Canada, PhRMA is taking the government to court — just a few days before the rule is slated to take effect.

Feng Tian, Ambrx CEO (Ambrx)

Af­ter 5 qui­et years, a for­mer Scripps spin­out rais­es $200M and an­nounces plans to try again at an IPO

The first time San Diego biotech Ambrx tried to go public in 2014, they failed and the company’s board switched to a radically different strategy: They sold themselves for an undisclosed amount to a syndicate of Chinese investors and pharma companies.

Now, after 5 quiet years, that syndicate has raised a mountain of cash and indicated they’ll soon make another bid to go public.

Earlier this month, Ambrx raised $200 million in what they billed as a crossover round financed by Fidelity, BlackRock, Cormorant Asset Management, HBM Healthcare Investments, Invus, Adage Capital Partners and Suvretta Capital Management. It’s the largest amount they’ve ever raised and, according to Crunchbase figures, more than doubles the total amount of VC capital collected since their launch 17 years ago.