Roche, Ab­b­Vie forced to slam brakes on myelo­ma stud­ies of star drug Ven­clex­ta af­ter re­searchers flag high­er risk of death

Two heavy­weight phar­ma play­ers to­day said they’ve been forced to stop re­cruit­ing pa­tients for all on­go­ing stud­ies in­volv­ing Ven­clex­ta (vene­to­clax) for mul­ti­ple myelo­ma. Roche and Ab­b­Vie say the drug arm has been linked to a high­er rate of death than the con­trol group, prompt­ing the FDA to is­sue a par­tial hold or­der­ing them to sus­pend any new pa­tient re­cruit­ment for the blood can­cer tri­als un­til they can sort out what is hap­pen­ing.

In the study, 21.1% — 41 of 194 pa­tients — in the vene­to­clax arm com­pared with 11.3% — 11 of 97 pa­tients — in the place­bo arm died, cre­at­ing a haz­ard ra­tio of 2.03.

Michael Sev­eri­no

Ini­tial­ly stamped with an ac­cel­er­at­ed ap­proval for CLL back in 2016, this drug has been hur­ried along by the FDA with a to­tal of 5 break­through drug des­ig­na­tions. Both com­pa­nies are cred­it­ed with top in­ves­tiga­tive teams and a keen in­ter­est in the on­col­o­gy mar­ket.

The sud­den halt on re­cruit­ment will like­ly put a crimp in their plans to hit $3 bil­lion in peak sales for the drug, which was ini­tial­ly lim­it­ed to pa­tients with a 17p dele­tion, ac­count­ing for about 10% of all cas­es. Last sum­mer they got an ap­proval to use the drug with Rit­ux­an for re­lapsed CLL, an add-on that Leerink’s Ge­of­frey Porges es­ti­mat­ed could be worth $690 mil­lion this year alone.

This was the Phase III BELLI­NI tri­al (M14-031), one of dozens of on­go­ing clin­i­cal tri­als that the phar­ma gi­ants are pur­su­ing for this ther­a­py, ac­cord­ing to a quick check of clin­i­cal­tri­als.gov.

“We will con­tin­ue work­ing with the FDA and world­wide reg­u­la­to­ry agen­cies to de­ter­mine ap­pro­pri­ate next steps for the mul­ti­ple myelo­ma pro­gram,” said Ab­b­Vie re­search chief Michael Sev­eri­no in a pre­pared state­ment.

MedTech clinical trials require a unique regulatory and study design approach and so engaging a highly experienced CRO to ensure compliance and accurate data across all stages is critical to development milestones.

In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

Avance Clinical is the Australian CRO for international biotechs providing world-class clinical research services with FDA-accepted data across all phases. With Avance Clinical, biotech companies can leverage Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend. The CRO has been delivering clinical drug development services for international biotechs for FDA and EMA regulatory approval for the past 24 years. The company has been recognized for the past two consecutive years with the prestigious Frost & Sullivan CRO Best Practices Award and a finalist in Informa Pharma’s Best CRO award for 2022.

Illustration: Kim Ryu for Endpoints News

Why non-opi­oid pain drugs keep fail­ing — and what's next for the field

In 1938, Rita Levi-Montalcini was forced to move her lab into her bedroom in Turin, as Mussolini’s facist government expelled Jewish people from studying or working in schools in Italy. Levi-Montalcini, then just a few years out of medical school and using sewing needles as scalpels in her makeshift lab, would soon discover nerve growth factor, or NGF, in chicken embryos.

Her discoveries formed the basis of our understanding of the peripheral nervous system and how cells talk to each other, and Levi-Montalcini went on to win the Nobel Prize in 1986. Much later, NGF was hailed as a promising target for new pain therapies, with some analysts quoting an $11 billion market. However, the latest anti-NGF candidate, Pfizer and Eli Lilly’s tanezumab, was rejected by the FDA last year because of a side effect that dissolved bone in some of its patients.

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Who are the women blaz­ing trails in bio­phar­ma R&D? Nom­i­nate them for End­points' 2022 spe­cial re­port

Over the past three years, Endpoints News has spotlighted 60 women who have blazed trails and supercharged R&D across the biopharma world. And judging from the response we’ve received, to both our special reports and live events, telling their stories — including any obstacles they may have had to overcome — has inspired our readers in many different ways.

But change takes time, and the fact remains that women are still underrepresented at the upper ranks of the drug-making world.

Up­dat­ed: Amid mas­sive re­struc­tur­ing, Bio­gen looks to re­duce phys­i­cal pres­ence in Boston

Biogen is putting a sizable chunk of office and research space in Kendall Square and Weston, MA up for sublease, marking another big change as the biotech grapples with the aftershock of a disastrous and controversial rollout for its Alzheimer’s drug.

The subbleases are “part of Biogen’s overall implementation of the ‘Future of Work,’ which is allowing us to optimize our footprint and reduce the amount of space we occupy, taking into consideration new elements such as the hybrid work model,” Biogen spokesperson Ashleigh Koss wrote in a statement to Endpoints News, adding that the company has had subleases across several buildings for years.

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Saurabh Saha, Centessa CEO (BIO19)

One of 2021's star biotech play­ers flags an­oth­er big set­back for the pipeline

Two months after scuttling their lead drug, Centessa’s executive team is back with the latest in a series of setbacks that have tanked its stock and blown holes in its strategic lineup of biotech subs.

The company reported in its Q2 post today that it has decided to scrap ZF874 after a patient demonstrated elevated liver enzymes — a classic red safety flag — in a Phase I study for alpha-1-antitrypsin (A1AT).

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Pfiz­er launch­es re­bate pro­gram for rare dis­ease pa­tients who have to stop tak­ing Panzy­ga

Pfizer is launching its second-ever rebate program, this time for Panzyga, its treatment for a rare neurological disease of the peripheral nerves.

The program began last month, according to STAT which first reported the news, and offers a refund of out-of-pocket costs for patients who must discontinue their course before the fifth treatment for “clinical reasons.”

Panzyga was approved back in 2018 to treat primary immunodeficiency (PI) in patients two years and older and chronic immune thrombocytopenia (cITP) in adults. It has since picked up an indication in chronic inflammatory demyelinating polyneuropathy (CIDP), a condition that’s characterized by weakness of the arms or legs, tingling or numbness, and a loss of deep tendon reflexes, according to the NIH.

Horizon's back-to-school campaign for children with cystinosis includes an all about me poster as part of a care package box.

Hori­zon read­ies kids and fam­i­lies for back to school with week­long ac­tiv­i­ties around rare dis­ease cysti­nosis

Going back to school is usually a bumpy readjustment from summer freedom for all kids, but especially for kids with chronic health conditions. Horizon Therapeutics is hoping to help smooth the way for some who have the rare disease cystinosis. Cystinosis is a genetic disease that causes the amino acid cystine to build up in different tissues and organs.

The “Gear Up” for school campaign is running all week with different online and at-home events and activities for families and children with cystinosis. Each family who signed up receives a care package mailed to their home including an activity coloring book “Michael’s Show-and-Tell.” The book tells Michael’s story about living with cystinosis while offering kids matching, coloring and finding object games along with information.

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Alessandro Maselli, Catalent CEO

Catal­ent ac­quires North Car­oli­na CD­MO for $475M, boost­ing oral solids work

As Catalent has been expanding its reach in the US this year, as well as recently completing a C-suite shuffle, the company announced last night that it has acquired the CDMO Metrics Contract Services for $475 million from Mayne Pharma Group.

The acquisition will increase Catalent’s capabilities in oral solid formulation development, manufacturing and packaging as well as expand its capacity to handle more highly potent compounds.

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Phar­mas spend mil­lions on di­a­betes ad­ver­tis­ing, but few pa­tients can re­call brand names — sur­vey

While many Big Pharma diabetes brands spend millions of dollars on TV ads every year, most people with type 2 diabetes don’t recognize specific drug brand names, according to a new study.

No brand garnered more than 30% recognition in Phreesia Life Science’s latest in-office patient survey of more than 4,000 adults with type 2 diabetes. Eli Lilly’s Trulicity topped the list as the most recognized brand with 29% of those surveyed recalling it, followed by Boehringer Ingelheim and Lilly’s Jardiance at 27% and Merck’s Januvia and Novo Nordisk tying for the third spot with 24%. Meanwhile, 76% of the patients surveyed were familiar with the generic treatment metformin.

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