La Jol­la whips up some love for iron over­load drug with promis­ing da­ta snap­shot

With rev­enue trick­ling in from its first prod­uct and an NDA in place for a sec­ond, La Jol­la Phar­ma­ceu­ti­cal is ready to shine a bright light on the third can­di­date com­ing through its pipeline.

Jeff Vacir­ca NY Can­cer & Blood

An in­ter­im analy­sis of a Phase II study sug­gests that LJPC-401 could sig­nif­i­cant­ly low­er the ex­cess iron lev­els in pa­tients with hered­i­tary he­mochro­mato­sis. The drug — a sub­cu­ta­neous in­jec­tion of syn­thet­ic hu­man hep­cidin — in­duced a mean re­duc­tion in TSAT from base­line of 42%, ver­sus 6% on place­bo (p<0.0001), mark­ing a clear score on the pri­ma­ry end­point.

It’s a small study with 12 and 14 pa­tients on the drug and place­bo co­horts re­spec­tive­ly but ap­par­ent­ly looked promis­ing enough to send the stock $LJPC surg­ing 84%.

Jeff Vacir­ca, an in­ves­ti­ga­tor and chief of clin­i­cal re­search at New York Can­cer & Blood Spe­cial­ists, ex­plained his en­thu­si­asm this way:

There have been no new treat­ment modal­i­ties in­tro­duced for pa­tients with hered­i­tary he­mochro­mato­sis in more than a decade. In light of the neg­a­tive im­pact that re­peat­ed phle­boto­my pro­ce­dures have on pa­tient qual­i­ty-of-life, pa­tients would wel­come a phar­ma­co­log­ic treat­ment that they can self-ad­min­is­ter and that ad­dress­es the un­der­ly­ing patho­phys­i­ol­o­gy of the dis­ease.

LJPC-401 al­so met the key sec­ondary end­point of low­er­ing the fre­quen­cy of phle­boto­my pro­ce­dures with pa­tients av­er­ag­ing 0.06 phle­botomies per month (3 pro­ce­dures be­tween 2 pa­tients) com­pared to place­bo-treat­ed pa­tients, who had 0.41 (9 pa­tients col­lec­tive­ly re­ceived 24). The p-val­ue came in at 0.003.

In­jec­tion site re­ac­tions, which on­ly hap­pened to 6% of the place­bo group, oc­curred in 79% of pa­tients treat­ed with the drug — though La Jol­la would em­pha­size they were mild.

LJPC-401 was a key part of the San Diego biotech’s pitch for a $100 mil­lion pub­lic of­fer­ing in 2018 along­side LJPC-0118, the se­vere malar­ia treat­ment that re­cent­ly re­ceived a break­through ther­a­py des­ig­na­tion. Topline re­sults are ex­pect­ed lat­er in the year.


Im­age: La Jol­la via Linkedin

As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Ab­b­Vie wins an ap­proval in uter­ine fi­broid-as­so­ci­at­ed heavy bleed­ing. Are ri­vals My­ovant and Ob­sE­va far be­hind?

Women expel on average about 2 to 3 tablespoons of blood during their time of the month. But with uterine fibroids, heavy bleeding is typical — a third of a cup or more. Drugmakers have been working on oral therapies to try and stem the flow, and as expected, AbbVie and their partners at Neurocrine Biosciences are the first to make it across the finish line.

Known chemically as elagolix, the drug is already approved as a treatment for endometriosis under the brand name Orilissa. It targets the GnRH receptor to decrease the production of estrogen and progesterone.

Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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Sanofi brings in 4 new ex­ec­u­tives in con­tin­ued shake-up, as vac­cines and con­sumer health chief head out the door

In the middle of Sanofi’s multi-pronged race to develop a Covid-19 vaccine, David Loew, the head of their sprawling vaccines unit, is leaving – part of the final flurry of moves in the French giant’ months-long corporate shuffle that will give them new-look leadership under new CEO Paul Hudson.

The company also said today that Alan Main, the head of their consumer healthcare unit, is out, and they named 4 executives to fill new or newly vacated positions, 3 of whom come from both outside both Sanofi and from Pharma.

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Roger Perlmutter, Merck R&D chief (YouTube)

Backed by BAR­DA, Mer­ck jumps in­to Covid-19: buy­ing out a vac­cine, part­ner­ing on an­oth­er and adding an­tivi­ral to the mix

Merck execs are making a triple play in a sudden leap into the R&D campaign against Covid-19. And they have more BARDA cash backing them up on the move.

Tuesday morning the pharma giant simultaneously announced plans to buy an Austrian biotech that has been working on a preclinical vaccine candidate, added a collaboration on another vaccine with the nonprofit IAVI and inked a deal with Ridgeback Biotherapeutics on an early-stage antiviral.

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Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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As­traZeneca’s $7B ADC suc­ceeds where Roche failed, im­prov­ing sur­vival in gas­tric can­cer

Another day, another win for Enhertu.

The antibody-drug conjugate AstraZeneca promised up-to $7 billion to partner on has had a quite a few months, beginning with splashy results in a Phase II breast cancer trial, a rapid approval and, earlier this month, breakthrough designations in both non-small cell lung cancer and gastric cancer.

Now, at ASCO, the British pharma and their Japanese partner, Daiichi Sankyo, have shown off the data that led to the gastric cancer designation, which they’ll take back to the FDA. In a pivotal, 187-person Phase II trial, Enhertu shrunk tumors in 42.9% of third-line patients with HER2-positive stomach cancer, compared with 12.5% in a control arm where doctors prescribed their choice of therapy. Progression-free survival was 5.4 months for Enhertu compared to 3.5 months for the control.

Once a gem, now just a rock, Take­da punts PhI­II IBD drug as ri­vals mus­cle ahead

Back in 2016, when then-Shire CEO Flemming Ørnskov picked up a promising clinical-stage IBD drug from Pfizer, the Boston-based biotech dubbed it SHP647 and moved it into the gem section of the pipeline, with rosy expectations of registration-worthy Phase III data ahead.

This was a drug that the EC wanted Takeda to commit to selling off before it gave their blessing to its acquisition of Shire, to settle some deep-seated concerns revolving around the potential market overlap with their blockbuster rival Entyvio. And Takeda, which took on a heavy debt load to buy Shire, clearly wanted the cash to pay down debt.

Ear­ly sur­vival da­ta boost Zio­phar­m's 'con­trolled IL-12' im­munother­a­py for glioblas­toma

An unconventional pairing of a gene therapy and an oral drug that promises to attack recurrent or progressive glioblastoma with controlled release of IL-12 has turned up more promising — if early — overall survival data. On top of boosting its case as a monotherapy, the data can also bode well for a combination with Regeneron’s PD-1 inhibitor, Libtayo.

Both the treatment and its developer, Ziopharm Oncology, have come a long way. The stock price peaked in 2015 but cratered in 2016 following a patient death in a Phase I.