Albert Bourla, AP Images

Pfiz­er re­veals de­lays across the board for its clin­i­cal gene ther­a­py pro­grams

At JP Mor­gan 2020, a year af­ter tak­ing the reins at Pfiz­er, Al­bert Bourla claimed the com­pa­ny was poised to be­come a leader in gene ther­a­py. He’s since thrown hun­dreds of mil­lions of dol­lars at those plans, in­clud­ing a $630 mil­lion deal just last month for two of Voy­ager’s AAVs.

Now, it ap­pears Phase III read­outs are fur­ther away than Bourla had hoped.

Set­backs have re­sult­ed in de­lays across all three of Pfiz­er’s clin­i­cal gene ther­a­py pro­grams, the com­pa­ny re­vealed in its Q3 re­sults on Tues­day — in­clud­ing one tri­al which was paused over safe­ty con­cerns.

Pfiz­er’s slam­ming the brakes on its Sang­amo-part­nered Phase III he­mo­phil­ia A study af­ter some pa­tients re­port­ed high lev­els of Fac­tor VI­II, an es­sen­tial blood-clot­ting pro­tein. Nor­mal Fac­tor VI­II lev­els typ­i­cal­ly range from 50% to 100%, and any­thing low­er than 50% could in­di­cate he­mo­phil­ia A. How­ev­er, lev­els above 150% are as­so­ci­at­ed with an in­creased risk of throm­bo­sis, or the for­ma­tion of clots in the blood ves­sels.

Mul­ti­ple pa­tients in Pfiz­er’s he­mo­phil­ia A tri­al had Fac­tor VI­II lev­els over 150%, though the com­pa­ny has not said how many. To date, no pa­tients have ex­pe­ri­enced throm­bot­ic events, and some are be­ing treat­ed with oral an­ti­co­ag­u­lants to re­duce the risk, Pfiz­er said.

The com­pa­ny says it has vol­un­tar­i­ly halt­ed screen­ing and dos­ing in the study — which is more than half-full — un­til it can amend the pro­to­col to pro­vide guide­lines for the man­age­ment of el­e­vat­ed Fac­tor VI­II lev­els.

This isn’t Pfiz­er’s on­ly he­mo­phil­ia set­back. Sep­a­rate­ly, the com­pa­ny said, “re­cent in­ter­ac­tions with the FDA” have led it to de­cide against con­duct­ing in­ter­im analy­ses for both its he­mo­phil­ia A and B stud­ies. In­stead, it will wait for full analy­ses based on at least 50 par­tic­i­pants in the he­mo­phil­ia A study and 40 par­tic­i­pants in the he­mo­phil­ia B study, push­ing out the time­lines for both of the pro­grams.

While Pfiz­er had been an­tic­i­pat­ing read­outs in 2022 for its he­mo­phil­ia B and A pro­grams, it is now look­ing at a Q1 2023 read­out for he­mo­phil­ia B. The com­pa­ny hasn’t pro­vid­ed a new time­line for the he­mo­phil­ia A pro­gram.

That could put Pfiz­er sig­nif­i­cant­ly be­hind its ri­val uniQure, which read out some pos­i­tive Phase III re­sults from a 54-pa­tient tri­al back in June. Over the course of a 52-week pe­ri­od, uniQure’s drug re­duced pa­tients’ an­nu­al­ized rate of bleed­ing re­quir­ing treat­ment by 80% from base­line. That tri­al was briefly put on hold in De­cem­ber fol­low­ing the di­ag­no­sis of HCC in one pa­tient, but reg­u­la­tors gave uniQure the OK to re­sume in April.

uniQure says it plans to sub­mit a BLA in the first quar­ter of next year.

Pfiz­er’s al­so ex­pect­ing a de­lay in its piv­otal DMD study, which is now ex­pect­ed to read out in 2023. The news comes af­ter safe­ty con­cerns led Pfiz­er to scale back that tri­al, cut­ting out pa­tients with cer­tain ge­net­ic mu­ta­tions.

Three cas­es of mus­cle weak­ness, two of which in­clud­ed heart in­flam­ma­tion, forced the tri­al amend­ment. In its Q3 re­sults, Pfiz­er said it’s be­lieved that an im­mune re­sponse against the mi­ni-dy­s­trophin pro­tein caused these cas­es. All pa­tients were treat­ed with high­er dos­es of steroids, and all im­proved with­in a few weeks, though some are still re­cov­er­ing.

“This type of re­ac­tion is a risk po­ten­tial­ly in­her­ent to any gene re­place­ment ther­a­py, and sim­i­lar se­vere ad­verse events re­port­ed in oth­er pro­grams sup­port the no­tion that this is a class ef­fect,” Pfiz­er said.

This isn’t the first time the DMD pro­gram has hit a road­block on its late-stage path, with the com­pa­ny re­port­ing in May that it was meet­ing head­winds from the FDA on the qual­i­ty of its po­ten­cy as­says.

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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ProFound Therapeutics founding team

Flag­ship's lat­est biotech could turn some of the thou­sands of new pro­teins it dis­cov­ered in­to ther­a­pies — and it has $75M to start

Flagship Pioneering, the incubator of Moderna and dozens of other biotechs, says it has landed upon tens of thousands of previously undiscovered human proteins. The VC shop wants to potentially turn them into therapeutics.

Like other drug developers that have turned proteins into therapeutics (think insulin for diabetes), Flagship’s latest creation, ProFound Therapeutics, wants to tap into this new trove of proteins as part of its mission to treat indications ranging from rare diseases to cancer to immunological diseases.

Richard Silverman, Akava Therapeutics founder and Northwestern professor

This time around, Lyri­ca's in­ven­tor is de­vel­op­ing his North­west­ern dis­cov­er­ies at his own biotech

Richard Silverman was left in the dark for the last five years of clinical development of the drug he discovered. The Northwestern University professor found out about the first approval of Lyrica, in the last few days of 2004, like most other people: in the newspaper.

What became one of Pfizer’s top-selling meds, at $5 billion in 2017 global sales before losing patent protection in 2019, started slipping out of his hands when Northwestern licensed it out to Parke-Davis, one of two biotechs that showed interest in developing the drug in the pre-email days, when the university’s two-person tech transfer team had to ship out letters to garner industry appetite.

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David Ricks, Eli Lilly CEO (David Paul Morris/Bloomberg via Getty Images)

Eli Lil­ly set to in­vest $2.1B in home state man­u­fac­tur­ing boost

Eli Lilly is looking to expand its footprint in its home Hoosier State by making a major investment in manufacturing.

The pharma is investing $2.1 billion in two new manufacturing sites at Indiana’s LEAP Lebanon Innovation and Research District in Boone County, northwest of Lilly’s headquarters in Indianapolis.

The two new facilities will expand Lilly’s manufacturing network for active ingredients and new therapeutic modalities, including genetic medicines, according to a press release.

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Pfiz­er and CD­MOs ramp up Paxlovid man­u­fac­tur­ing with Kala­ma­zoo plant ex­pan­sion lead­ing the way

As the Covid-19 pandemic continues to evolve, pharma companies and manufacturers are exploring how to step up production on antivirals.

Pfizer is planning to expand its Kalamazoo-area facility to increase manufacturing capabilities for the oral Covid-19 antiviral Paxlovid, according to a report from Michigan-based news site MLive. The expansion of the facility, which serves as Pfizer’s largest manufacturing location, is expected to create hundreds of “high-skilled” STEM jobs, MLive reported. No details about the project’s cost and timeline have been released, but according to MLive, Pfizer will announce the details of the expansion at some point in early June.

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FDA spells out the rules and re­stric­tions for states seek­ing to im­port drugs from Cana­da

The FDA is offering more of an explanation of the guardrails around its program that may soon allow states to import prescription drugs in some select circumstances from Canada, but only if such imports will result in significant cost reductions for consumers.

While the agency has yet to sign off on any of the 5 state plans in the works so far, and PhRMA’s suit to block the Trump-era rule allowing such imports is stalled, the new Q&A guidance spells out the various restrictions that states will have to abide by, potentially signaling that a state approval is coming.

Pfizer CEO Albert Bourla at the World Economic Forum (Gian Ehrenzeller/Keystone via AP Images)

All about ac­cess: Pfiz­er moves to a non-prof­it mod­el for drug sales in 45 low­er-in­come coun­tries

Leading the way to increase access to cheaper drugs worldwide, Pfizer said Wednesday it will provide all current and future patent-protected medicines and vaccines available in the US or EU on a not-for-profit basis to about 1.2 billion people in 45 lower-income countries.

Rwanda, Ghana, Malawi, Senegal and Uganda are the first five countries to sign on to this accord, which will also seek to blaze new paths for quick and efficient regulatory and procurement processes to reduce the usual delays in making new medicines and vaccines available in these countries.

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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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Up­dat­ed: US sees spike in Paxlovid us­age as Mer­ck­'s mol­nupi­ravir and As­traZeneca's Evusheld are slow­er off the shelf

New data from HHS show that more than 162,000 courses of Pfizer’s Covid-19 antiviral Paxlovid were administered across the US over the past week, continuing a streak of increased usage of the pill, and signaling not only rising case numbers but more awareness of how to access it.

In comparison to this week, about 670,000 courses of the Pfizer pill have been administered across the first five months since Paxlovid has been on the US market, averaging about 33,000 courses administered per week in that time.