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.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

All about Omi­cron; We need more Covid an­tivi­rals; GSK snags Pfiz­er’s vac­cine ex­ec; Janet Wood­cock’s fu­ture at FDA; and more

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Usama Malik

Ex-Im­munomedics CFO charged with in­sid­er trad­ing, faces up to 20 years in prison af­ter al­leged­ly tip­ping off girl­friend and rel­a­tives of a PhI­II suc­cess

The former CFO of Immunomedics, who helped steer the company to its $21 billion buyout by Gilead last year, has been charged with insider trading, the Department of Justice announced Thursday.

Usama Malik tipped off his then-girlfriend and four others that a Phase III study for Trodelvy would be stopped early four days before Immunomedics publicly announced the result in April 2020, DoJ alleged in its complaint. The individuals then purchased Immunomedics shares, selling them after the news broke and Immunomedics’ stock price doubled.

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Merck's new antiviral molnupiravir (Quality Stock Arts / Shutterstock)

As Omi­cron spread looms, oral an­tivi­rals ap­pear to be one of the best de­fens­es — now we just need more

After South African scientists reported a new Covid-19 variant — dubbed Omicron by the WHO — scientists became concerned about how effective vaccines and monoclonal antibodies might be against it, which has more than 30 mutations in the spike protein.

“I think it is super worrisome,” Dartmouth professor and Adagio co-founder and CEO Tillman Gerngross told Endpoints News this weekend. Moderna CEO Stéphane Bancel echoed similar concerns, telling the Financial Times that experts warned him, “This is not going to be good.”

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Pfiz­er, Am­gen and Janssen seek fur­ther clar­i­ty on FDA's new ben­e­fit-risk guid­ance

Three top biopharma companies are seeking more details from the FDA on how the agency conducts its benefit-risk assessments for new drugs and biologics.

While Pfizer, Amgen and Janssen praised the agency for further spelling out its thinking on the subject in a new draft guidance, including a discussion of patient experience data as part of the assessment, the companies said the FDA could’ve included more specifics in the 20-page draft document.

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Vas Narasimhan, Novartis CEO (Thibault Camus/Pool via AP Images)

With gener­ic com­pe­ti­tion heat­ing up, Vas Narasimhan out­lines No­var­tis' growth plans at R&D day

Thursday marks Novartis’ annual R&D day, and with it comes CEO Vas Narasimhan’s attempt to spotlight the company’s pipeline strategy and emerging stars.

The biggest question entering Thursday’s presentation dealt with how the big biopharma will make up revenues from upcoming generic competition — Novartis says within the next five years, generics will eat away roughly $9 billion in sales. To offset this, Narasimhan outlined a strategy for 4% growth or higher until 2026, focusing on six key medicines he believes will see multibillion dollar profits during this time.

In­cor­po­rat­ing Ex­ter­nal Da­ta in­to Clin­i­cal Tri­als: Com­par­ing Dig­i­tal Twins to Ex­ter­nal Con­trol Arms

Most drug development professionals are familiar with the nerve-racking wait for the read-out of a large trial. If it’s negative, is the investigational therapy ineffective? Or could the failure result from an unforeseen flaw in the design or execution of the protocol, rather than a lack of efficacy? The team could spend weeks analyzing data, but a definitive answer may be elusive due to insufficient power for such analyses in the already completed trial. These problems are only made worse if the trial had lower enrollment, or higher dropout than expected due to an unanticipated event like COVID-19. And if a trial is negative, the next one is likely to be larger and more costly — if it happens at all.

Reshma Kewalramani, Vertex CEO (Vertex via YouTube)

Bat­tling a line­up of skep­tics, Ver­tex claims an­oth­er ear­ly clin­i­cal win — this time in kid­ney dis­ease

Vertex claimed its second early-stage win of the fall Wednesday, announcing positive results in a small study on a genetically defined form of kidney disease.

The 16-patient, Phase II trial focused on patients with focal segmental glomerulosclerosis, a rare disease where kidneys are unable to filter blood properly. Over 13 weeks on an experimental pill, the level of protein in the patients’ urine fell by an average of 47.6%.

Ab­b­Vie tacks on a new warn­ing to Rin­voq la­bel as safe­ty frets crimp JAK class

The safety problems that continue to plague the JAK class as new data highlight some severe side effects are casting a large shadow over AbbVie’s Rinvoq.

As a result of a recent readout highlighting major adverse cardiac events (MACE), malignancy, mortality and thrombosis with Xeljanz a couple of months ago, AbbVie put out a notice late Friday afternoon that it is adding the new class risks to its label for their rival drug.

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