Sec­ond dose of Sophiris' prostate can­cer drug proves fu­tile, shares crash

Fol­low­ing a pa­tient death that was ruled un­re­lat­ed to the study drug, Sophiris Bio con­tin­ued test­ing a sec­ond ad­min­is­tra­tion of its ex­per­i­men­tal drug in pa­tients with prostate can­cer who re­spond­ed well to the first round of dos­ing. That faith was in vain, as re-treat­ment with the drug, top­salysin, failed to con­fer ad­di­tion­al ben­e­fit in the Phase IIb study, oblit­er­at­ing the San-Diego-based com­pa­ny’s shares $SPHS af­ter the bell on Mon­day.

Pre­vi­ous­ly, the biotech had re­port­ed that 10 out of 37 pa­tients with lo­cal­ized prostate can­cer in the tri­al demon­strat­ed a clin­i­cal re­sponse six months fol­low­ing a sin­gle ad­min­is­tra­tion of top­salysin — and that 6 of those 10 re­spon­ders ex­pe­ri­enced com­plete ab­la­tion of their tu­mor. On Mon­day, the com­pa­ny up­dat­ed its re­sults from the mid-stage tri­al say­ing that the 10 re­spon­ders who re­ceived a sec­ondary ad­min­is­tra­tion of the drug did not de­rive ad­di­tion­al ben­e­fit, ac­cord­ing to a tar­get­ed biop­sy con­duct­ed on them six months af­ter re-treat­ment.

Shares of the com­pa­ny, which said it was for­mu­lat­ing a de­sign for a Phase III study for sub­mis­sion to US and EU reg­u­la­tors, cratered about 40% af­ter-mar­ket.

In June, the com­pa­ny tem­porar­i­ly halt­ed sec­ondary dos­ing in pa­tients who had re­spond­ed well to the first ad­min­is­tra­tion of the drug, af­ter a pa­tient sud­den­ly died. By Au­gust, Sophiris said it would re­sume dos­ing af­ter re­searchers con­clud­ed the drug had not caused the death.

Mark Em­ber­ton

On Mon­day, lead study in­ves­ti­ga­tor Mark Em­ber­ton said that “tak­ing in­to ac­count the ob­served ef­fi­ca­cy and safe­ty pro­file to date fol­low­ing a sin­gle ad­min­is­tra­tion, we be­lieve urol­o­gists would wel­come a treat­ment like top­salysin for men with clin­i­cal­ly-sig­nif­i­cant lo­cal­ized prostate can­cer.” Mean­while, the com­pa­ny did not pro­vide any de­tails on re­ac­tion to the sec­ond dose, oth­er than to say it had not in­tro­duced any ad­di­tion­al ben­e­fit.

Top­salysin is de­signed to be ac­ti­vat­ed on­ly by en­zy­mat­i­cal­ly-ac­tive prostate spe­cif­ic anti­gen (PSA), which is over­pro­duced in pa­tients with prostate can­cer. The drug is be­ing de­vel­oped as an op­tion for pa­tients to de­lay or even pre­vent ag­gres­sive surg­eries such as rad­i­cal prosta­te­c­to­my, which comes with sex­u­al side-ef­fects and uri­nary in­con­ti­nence. The drug’s safe­ty pro­file could po­ten­tial­ly al­low it to po­si­tion it­self be­tween non-in­va­sive ther­a­peu­tic op­tions and in­va­sive sur­gi­cal pro­ce­dure, while al­so re­duc­ing or pre­vent­ing the off-tar­get ef­fects, HC Wain­wright’s Joseph Pant­gi­nis wrote in a note last month.

Oth­er than skin can­cer, prostate can­cer is the most com­mon can­cer in Amer­i­can men, ac­cord­ing to the Amer­i­can Can­cer So­ci­ety.

A new era of treat­ment: How bio­mark­ers are chang­ing the way we think about can­cer

AJ Patel was recovering from a complicated brain surgery when his oncologist burst into the hospital room yelling, “I’ve got some really great news for you!”

For two years, Patel had been going from doctor to doctor trying to diagnose his wheezing, only to be dealt the devastating news that he had stage IV lung cancer and only six months to live. And then they found the brain tumors.

“What are you talking about?” Patel asked. He had never seen an oncologist so happy.

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Michael Corbo, Pfizer CDO of inflammation & immunology

UP­DAT­ED: Plan­ning ahead for crowd­ed ul­cer­a­tive col­i­tis mar­ket, Pfiz­er spells out PhI­II da­ta on $6.7B Are­na drug

Pfizer has laid out the detailed results behind its boast that etrasimod — the S1P receptor modulator at the center of its $6.7 billion buyout of Arena Pharma — is the winner of the class, potentially leapfrogging an earlier entrant from Bristol Myers Squibb.

Pivotal data from the ELEVATE program in ulcerative colitis — which consists of two Phase III trials, one lasting 52 weeks and the other just 12 weeks — illustrate an “encouraging balance of efficacy and safety,” according to Michael Corbo, chief development officer of inflammation & immunology at Pfizer. The company is presenting the results as a late breaker at Digestive Disease Week.

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An NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

'Xeno­trans­plan­ta­tion is com­ing': New NE­JM pa­per gives de­tailed look in­to 2 pig-to-hu­man kid­ney trans­plant cas­es

The thymokidney is a curious organ, if you could call it that. It’s a sort of Frankensteinian creation — a system of pig thymus embedded underneath the outer layer of a pig’s kidney, made for human transplantation.

In the first case of pig-to-human xenotransplantation of a kidney into a brain-dead patient, the thymokidney quietly featured front and center.

In that experiment, which took place in September of last year, NYU researchers led by Robert Montgomery sutured a pig thymokidney onto the leg of a brain-dead 66-year-old woman. That case was widely reported on by a horde of major media outlets, including the New York Times, the BBC, and an in-depth feature by USA Today.

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Vlad Coric, Biohaven CEO

UP­DAT­ED: Fresh off $11.6B sale to Pfiz­er, New Bio­haven hits Phase III set­back just weeks af­ter Vlad Coric chalked up promise

When Pfizer bought up Biohaven’s migraine portfolio in the largest M&A deal of the year earlier this month, Biohaven CEO Vlad Coric promised the rest of the pipeline, which will live on under the umbrella of New Biohaven, still has a lot to offer. But that vision took a dent Monday as the drugmaker revealed it’s once again flopped on troriluzole.

The glutamate regulator failed to meet the primary endpoint on a Phase III study in patients with spinocerebellar ataxia, an inherited disorder that impairs a person’s ability to walk, speak and swallow. SCA can also lead to premature death.

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Mihael Polymeropoulos, Vanda Pharmaceuticals CEO

Phar­ma com­pa­ny con­tin­ues its FDA law­suit spree, this time af­ter agency de­nies fast-track des­ig­na­tion

Vanda Pharmaceuticals is making a name for itself, at least in terms of suing the FDA.

The DC-headquartered firm on Monday filed its latest suit against the agency, with the company raising concerns over the FDA’s failure to grant a fast track designation for Vanda’s potential chronic digestive disorder drug tradipitant, which is a neurokinin 1 receptor antagonist.

Specifically, Vanda said FDA’s “essential point” in its one-page denial letter on the designation pointed to “the lack of necessary safety data,” which was “inconsistent with the criteria for … Fast Track designation.”

Mod­er­na seeks to dis­miss Al­ny­lam suit over Covid-19 vac­cine com­po­nent, claim­ing wrong venue

RNAi therapeutics juggernaut Alnylam Pharmaceuticals made a splash in March when it sued and sought money from both Pfizer and Moderna regarding their use of Alnylam’s biodegradable lipids, which Alnylam claims have been integral to the way both companies’ mRNA-based Covid-19 vaccines work.

But now, Moderna lawyers are firing back, telling the same Delaware district court that Alnylam’s claims can only proceed against the US government in the Court of Federal Claims because of the way the company’s contract is set up with the US government. The US has spent almost $10 billion on Moderna’s Covid-19 vaccine so far.

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(Credit: Shutterstock)

Cracks in the fa­cade: Is phar­ma's pan­dem­ic ‘feel good fac­tor’ wan­ing?

The discordant effects of the Covid-19 pandemic on pharma reputation continues. While the overall industry still retains a respectable halo from its Covid-19 quick response and leadership, a new patient group study reveals a different story emerging in the details.

On one hand, US patient advocacy groups rated the industry higher-than-ever overall. More than two-thirds (67%) of groups gave the industry a thumbs up for 2021, a whopping 10 percentage point increase over the year before, according to the PatientView annual study, now in its 9th year.

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Saqib Islam, SpringWorks CEO

Pfiz­er spin­out Spring­Works will ship its first drug to the FDA be­fore year’s end with PhI­II win

SpringWorks Therapeutics thinks it has cemented the backbone for its first “pipeline-in-a-product” oncology treatment and will send it to the FDA before the clock strikes 2023 with a Phase III win on Tuesday.

The oral gamma secretase inhibitor, dubbed nirogacestat, beat placebo on the primary goal of progression-free survival in adults with progressing desmoid tumors.

The soft-tissue tumors can lead to long-lasting pain, disfigurement and amputation, and there are currently no approved meds for the rare oncology indication. The tumors typically impact patients aged 20 to 44 years old and disproportionately affect women at rates 2 to 3 times higher, with up to a total of 1,650 new cases diagnosed in the US annually, according to SpringWorks.

Robert Califf (Michael Brochstein/Sipa USA via AP Images)

House Re­pub­li­cans at­tack Chi­na-on­ly da­ta in FDA sub­mis­sions, seek new in­ves­ti­ga­tion in­to re­search in­spec­tions

Three Republican representatives are calling on the FDA to take a closer look at the applications including only clinical data from China.

The letter to FDA commissioner Rob Califf late last week comes as the agency recently rejected Eli Lilly’s anti-PD-1 antibody, which attempted to bring China-only data but ran into a bruising adcomm that may crush the hopes of any other companies looking to bring cheaper follow-ons based only on Chinese data.

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