Di­al­ing down the tox­ic ef­fects of ra­di­a­tion on can­cer pa­tients, Galera gets break­through sta­tus on Phase IIb da­ta

A biotech­nol­o­gy com­pa­ny near Philadel­phia — armed with da­ta from a Phase IIb tri­al — just got break­through sta­tus from the FDA for a drug that might re­duce the harm­ful ef­fects of ra­di­a­tion ther­a­py for can­cer pa­tients.

The com­pa­ny, Galera Ther­a­peu­tics, has an in­tra­venous drug (meant to be tak­en right be­fore ra­di­a­tion treat­ment) that might mit­i­gate the tox­ic ef­fects. Es­sen­tial­ly, when pa­tients un­der­go ra­di­a­tion, their bod­ies are over­whelmed by a com­pound called su­per­ox­ide, which is de­ployed by the im­mune sys­tem to kill in­vaders. Nor­mal­ly, the body can break down su­per­ox­ide, con­vert­ing it to hy­dro­gen per­ox­ide and mol­e­c­u­lar oxy­gen. But when un­der­go­ing in­tense ra­di­a­tion, the body can’t keep up.

Mel Sorensen

“Our drug mim­ics the ef­fect of that nat­ur­al en­zyme, and does it po­tent­ly,” Galera’s pres­i­dent and CEO Mel Sorensen tells me.

First, Galera is tack­ling a se­vere side ef­fect of ra­di­a­tion that af­flicts head and neck can­cer pa­tients. These pa­tients of­ten de­vel­op a con­di­tion called se­vere oral mu­cosi­tis, or SOM, which hap­pens when sen­si­tive tis­sue in the mouth is sub­ject­ed to ex­ces­sive su­per­ox­ide gen­er­at­ed dur­ing ra­di­a­tion treat­ment. This breaks down the ep­ithe­lial cells that line the mouth, caus­ing pa­tients to suf­fer from se­vere pain, ul­cer­a­tions, and bleed­ing of the mouth. It’s of­ten so bad that the pa­tient can’t eat or drink.

“The prob­lem isn’t just the im­me­di­ate ef­fect of the pain, sore­ness, and lack of nu­tri­tion, but al­so the resid­ual ef­fects,” Sorensen said. “It can cause dry mouth for years af­ter treat­ment.”

In pa­tients with head and neck can­cer, ra­di­a­tion is a main­stay, and about 70 per­cent of pa­tients re­ceiv­ing this treat­ment de­vel­op SOM.

The FDA has grant­ed break­through ther­a­py des­ig­na­tion for Galera’s drug, coined GC4419, based on the da­ta from a 223-pa­tient, dou­ble-blind, ran­dom­ized, place­bo-con­trolled Phase IIb clin­i­cal tri­al in pa­tients with head and neck can­cer. In the tri­al, GC4419 re­duced the du­ra­tion of SOM from 19 days to 1.5 days (92%), the in­ci­dence of SOM by 34%, and the sever­i­ty of pa­tients’ oral mu­cosi­tis by 47%.

Al­though start­ing with SOM, Sorensen said Galera’s drug might have ap­pli­ca­tions in many dif­fer­ent can­cers. Hy­dro­gen per­ox­ide, the byprod­uct Galera’s drug cre­ates when break­ing down su­per­ox­ide, hap­pens to be tox­ic to tu­mor tis­sue, Sorensen said. The com­pa­ny is hope­ful that ad­min­is­ter­ing the drug in com­bi­na­tion with high-dose ra­di­a­tion might pro­tect nor­mal tis­sue while fight­ing tu­mors.

“If we can do both of those, we’ve ef­fec­tive­ly trans­formed ra­di­a­tion ther­a­py,” Sorensen said.

The com­pa­ny is now re­cruit­ing for a Phase I/II tri­al test­ing GC4419 against pan­cre­at­ic can­cer.

Since the com­pa­ny’s found­ing in 2009, it’s raised $77 mil­lion ($20 mil­lion Se­ries A, $57 mil­lion Se­ries B). Sorensen wouldn’t say how much run­way the com­pa­ny has or whether it would need to raise more mon­ey be­fore tak­ing GC4419 in­to Phase III tri­als. He did note, how­ev­er, that the com­pa­ny has a strong in­vestor syn­di­cate in­clud­ing No­var­tis Ven­ture Fund, New En­ter­prise As­so­ci­ates, Sofinno­va, and oth­ers.

“We’re look­ing at all ways to op­ti­mize the fi­nan­cial side of this,” Sorensen said. “We’re still think­ing about what’s the best way to fi­nance the Phase III, and we’re talk­ing with lots of peo­ple.”

Im­age: Can­cer cells. Shut­ter­stock

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

The End­points 11: They've got mad mon­ey and huge am­bi­tions. It's time to go big or go home

These days, selecting a group of private biotechs for the Endpoints 11 spotlight begins with a sprint to get ahead of IPOs and the M&A teams at Big Pharma. I’ve had a couple of face plants earlier this year, watching some of the biotechs on my short list choose a quick leap onto Nasdaq or into the arms of a buyer.

Vividion, you would have been a great pick for the Endpoints 11. I’m sorry I missed you.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Dave Lennon, former president of Novartis Gene Therapies

So what hap­pened with No­var­tis Gene Ther­a­pies? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

FDA+ roundup: Bs­U­FA III ready for show­time, court tells FDA to re-work com­pound­ing plan, new guid­ance up­dates and more

The FDA has now spelled out what exactly will be included in the third iteration of Biosimilar User Fee Act (BsUFA) from 2023 through 2027, which similarly to the prescription drug deal, sets fees that industry has to pay for submitting applications, in exchange for firm timelines that the agency must meet.

This latest deal includes several sweeteners for the biosimilar industry, which has yet to make great strides in the US market, with shorter review timelines for safety labeling updates and updates to add or remove an indication that does not contain efficacy data.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

Joshua Liang, Clover Biopharmaceuticals CEO

With world still in sore need of dos­es, Clover says its Covid-19 vac­cine is 67% ef­fec­tive in Phase III

With concerns about the Delta variant rising and much of the world still in desperate need of vaccine doses, a Chinese biotech announced Wednesday that a new shot has shown positive results in a large trial against Covid-19, including new variants.

Clover Biopharmaceuticals announced Wednesday that its vaccine candidate showed 79% efficacy against the Delta variant in a Phase II/III trial dubbed Spectra, and 67% effective against Covid-19 overall.

Jean Bennett (Brent N. Clarke/Invision/AP Images)

Lux­tur­na in­ven­tor Jean Ben­nett starts a new gene ther­a­py com­pa­ny to tack­le rare dis­eases left be­hind by phar­ma, VCs

A few years ago Jean Bennett found herself in a surprising place for a woman who invented the first gene therapy ever approved in the United States: No one, it seemed, wanted her work.

Bennett, who designed and co-developed Luxturna, approved in 2018 for a rare form of blindness, had kept building new gene therapies for eye diseases at her University of Pennsylvania lab. But although the results in animals looked promising, pharma companies and investors kept turning down the pedigreed ophthalmology professor.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,900+ biopharma pros reading Endpoints daily — and it's free.

Maureen Hillenmeyer, Hexagon Bio CEO

Hexa­gon Bio rais­es $61M to con­tin­ue ef­forts to turn fun­gi in­to drugs

A year after raising a $47 million launch round, the fungi-loving drug hunters at Hexagon Bio have more than doubled their coffers.

Hexagon announced today that it raised another $61 million for its efforts to design cancer and infectious disease drugs based on insights mined from the DNA in millions of species of fungi. The new financing brings Hexagon’s committed funding to over $108 million.