La Jol­la CEO de­camps just as biotech faces a reck­on­ing for iron over­load drug

Months af­ter tout­ing how LJPC-401 ap­pears to sig­nif­i­cant­ly low­er ex­cess iron lev­els in a small study in­volv­ing pa­tients with hered­i­tary he­mochro­mato­sis, La Jol­la Phar­ma­ceu­ti­cal is re­con­sid­er­ing its plans as the drug flopped for be­ta tha­lassemia pa­tients. And they’ll be re­view­ing their fu­ture un­der a new CEO — with a se­ri­ous­ly de­flat­ed share price.

George Tid­marsh

The San Diego-based biotech is gut­ting that study, LJ401-BT01, im­me­di­ate­ly. A re­cent in­ter­im analy­sis, which in­clud­ed about half of the en­rolled pop­u­la­tion, sug­gest­ed a lack of ef­fi­ca­cy. As far as they could tell, pa­tients on the treat­ment and con­trol arms saw the same changes in car­diac iron lev­els, the pri­ma­ry end­point, and in oth­er iron, hema­tol­ogy and en­docrine mea­sure­ments.

“The Com­pa­ny ex­pects to re-eval­u­ate its cur­rent op­er­at­ing plan in light of the mixed re­sults of these stud­ies and to make ad­just­ments as ap­pro­pri­ate to man­age the Com­pa­ny’s avail­able cash re­sources,” it said in a state­ment, pri­or­i­tiz­ing the sales of its sole mar­ket­ed prod­uct and reg­u­la­to­ry ac­tiv­i­ties for a sec­ond ex­per­i­men­tal drug.

CEO George Tid­marsh won’t be around for the soul search­ing. In a sep­a­rate an­nounce­ment, La Jol­la dis­closed that its chief of sev­en years has left to pur­sue oth­er in­ter­ests.

As board di­rec­tors Kevin Tang and Craig John­son step in to over­see the man­age­ment team, they are tasked with max­i­miz­ing sales of Gi­apreza — a vaso­con­stric­tor ap­proved to in­crease blood pres­sure in adults with sep­tic or oth­er dis­trib­u­tive shock in the US, and to treat re­frac­to­ry hy­poten­sion for the same pop­u­la­tion in Eu­rope — and seek an FDA green light for the malar­ia ther­a­py LJPC-0118. La Jol­la has re­cent­ly filed an NDA for the drug.

La Jol­la still counts $104.8 mil­lion in cash as of Sep­tem­ber 30, com­pared to $172.6 mil­lion at the end of last year. Gi­apreza has brought in $15.8 mil­lion in the first three quar­ters of 2019, a far cry from its peak sales es­ti­mate of $600 mil­lion.

LJPC-401 was a key part of the biotech’s pitch for a $100 mil­lion pub­lic of­fer­ing in 2018, just af­ter it rolled out Gizpreza. And in­ves­ti­ga­tors in the hered­i­tary he­mochro­mato­sis tri­al gushed over it as it promised to be the first new treat­ment for this dis­ease in more than a decade, sav­ing pa­tients from fre­quent phle­boto­my pro­ce­dures.

The com­pa­ny said topline re­sults in that tri­al were con­sis­tent with the in­ter­im num­bers re­port­ed in June, in which the drug con­ferred a sta­tis­ti­cal­ly sig­nif­i­cant re­duc­tion in trans­fer­rin sat­u­ra­tion and re­quire­ment for phle­boto­my pro­ce­dures com­pared to place­bo.

LJPC-401-treat­ed pa­tients had a mean re­duc­tion in TSAT of 33% com­pared to place­bo-treat­ed pa­tients who had a mean re­duc­tion of 3% (p<0.0001). […] LJPC-401-treat­ed pa­tients had 0.10 phle­botomies per month com­pared to place­bo-treat­ed pa­tients who had 0.50 phle­botomies per month (p<0.0001).

In­vestors’ en­thu­si­asm is dis­ap­pear­ing al­most as swift­ly as they shot up. Shares $LJPC were down 53.58% to $2.59 fol­low­ing the news on Mon­day.

Tal Zaks, Moderna CMO (Moderna via YouTube)

UP­DAT­ED: NI­AID and Mod­er­na spell out a 'ro­bust' im­mune re­sponse in PhI coro­n­avirus vac­cine test — but big ques­tions re­main to be an­swered

The NIAID and Moderna have spelled out positive Phase I safety and efficacy data for their Covid-19 vaccine mRNA-1273 — highlighting the first full, clear sketch of evidence that back-to-back jabs at the dose selected for Phase III routinely produced a swarm of antibodies to the virus that exceeded levels seen in convalescent patients — typically in multiples indicating a protective response.

Moderna execs say plainly that this first stage of research produced exactly the kind of efficacy they hoped to see in humans, with a manageable safety profile.

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Trans­port Sim­u­la­tion Test­ing for Your Ther­a­py is the Best Way to As­sure FDA Ex­pe­dit­ed Pro­gram Ap­proval

Modality Solutions is an ISO:9001-registered biopharmaceutical cold chain engineering firm with unique transport simulation capabilities that support accelerated regulatory approval for biologics and advanced therapeutic medicinal products (ATMP). Our expertise combines traditional validation engineering approaches with regulatory knowledge into a methodology tailored for the life sciences industry. We provide insight and execution for the challenges faced in your cold chain logistics network.

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Who are the women blaz­ing trails in bio­phar­ma R&D and lead­ing the fight against Covid-19? Nom­i­nate them for End­points' spe­cial re­port

One of the many inequalities the pandemic has laid bare is the gender imbalance in biomedical research. A paper examining Covid-19 research authorship wondered out loud: Where are the women?

It’s a question that echoes beyond our current times. In the biopharma world, not only are women under-represented in R&D roles (particularly at higher levels), their achievements and talents could also be undermined by stereotypes and norms of leadership styles. The problem is even more dire for women of color.

Sanjiv Patel, Relay CEO

Re­lay reaps $400M IPO wind­fall af­ter draw­ing the cur­tain on mo­tion-based drug de­sign pipeline

A red-hot venture market back in 2018 propelled Relay Therapeutics to a whopping $400 million crossover round. And now, the biotech is riding on a red-hot IPO market to a public debut that brought in the same exact amount.

The $400 million raise breaks down to 20 million shares sold at $20 each — above even the amended proposed range of $16 to $18.

It’s also double what Relay had penciled in with the initial S-1 filing, in yet another illustration that those figures are often nothing more than a placeholder these days.

BioAt­la rakes in $72.5M Se­ries D, ad­vanc­ing re­search for pH-de­tect­ing can­cer treat­ments

A little over a year after agreeing to a worldwide collaboration with hefty Chinese biotech BeiGene worth up to $270 million, BioAtla is ready for another haul.

The San Diego-based biotech pulled in a $72.5 million haul for their Series D financing, which the company announced Wednesday. Funds will go toward their four main clinical programs developing cancer treatments, two of which are currently in Phase II, BioAtla president and former Celgene COO Scott Smith said.

Mene Pangalos, AstraZeneca R&D chief (AstraZeneca via YouTube)

A day af­ter Mod­er­na vac­cine re­sults, ru­mors swirl of pend­ing As­traZeneca da­ta

A day after Moderna and the NIH published much-anticipated data from their Phase I Covid-19 vaccine trial, attention is turning to AstraZeneca which, according to a UK report, is expected to publish its own early data tomorrow.

ITV’s Robert Peston reported that AstraZeneca will publish the Phase I data in The Lancet. 

AstraZeneca and Moderna represent the two most ambitious Covid-19 vaccine efforts, having set the quickest timelines for approval (though they were recently joined in that regard by the Pfizer-BioNTech partnership) and some of the loftiest goals in total doses. Yet there is even less known about AstraZeneca’s vaccine’s effect on humans than there was about Moderna’s before yesterday. Although, in a controversial move, Moderna released some statistics from its Phase I in May, AstraZeneca has yet to say anything about what it saw in its Phase I trial — a move consistent with the scientific convention to withhold data until it can be published in a peer-reviewed journal.

Ludwig Hantson, Alexion CEO

Why pay $4B for a steady di­et of dis­ap­point­ment? Porges turns thumbs down on Alex­ion’s M&A strat­e­gy, of­fers some point­ers

When Alexion announced recently that it was paying $1.4 billion to bag Portola and its underperforming Factor Xa inhibitor reversal agent, you could hear the head-scratching going on around virtual Wall Street.

Why was Alexion going down the discount lane for new products? And why something like this? Analysts have been urging Alexion to get serious about M&A for years if it was serious about diversifying the company beyond Soliris and its successor drug. But this wasn’t the kind of heavy-impact deal they were looking for.

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Mil­li­pore­Sig­ma to build new $20 mil­lion, 12,000-square-foot lab in Switzer­land

On the heels of opening a new laboratory in Shanghai last week, MilliporeSigma is continuing its construction push.

The Merck KGgA life science subsidiary announced Wednesday its intentions to build a new $20 million lab in Buchs, Switzerland to support its reference materials business. It’s estimated that the new facility will be completed in December 2021 and open in early 2022 and is expected to be 12,000 square feet.

Stéphane Bancel, Moderna CEO (Steven Ferdman/Getty Images)

‘Plan­ning to vac­ci­nate every­one in the US,’ Mod­er­na out­lines ef­forts to sup­ply their Covid-19 vac­cine as man­u­fac­tur­ing ramps up ahead of PhI­II

Twelve days from the planned start of their Phase III pivotal trial, the executive crew at Moderna has set up the manufacturing base needed to begin production of the first 500,000 doses of their Covid-19 vaccine with plans to feed it into a global supply chain. But the initial batches will likely be ready in the US first, where company CEO Stéphane Bancel plans to be able to vaccinate everyone.

“We have started making commercial product at-risk, and will continue to do so every day and every week of the month,” Bancel told analysts during their morning call on the Phase I data just published in the New England Journal of Medicine.

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