Alex­ion's suc­ces­sor to Soliris hits co-pri­ma­ry end­points in a piv­otal, but can't sur­pass the big block­buster

With a group of ri­vals press­ing in from every side, look­ing to cap­ture a piece of Alex­ion’s $ALXN block­buster mar­ket for the rare dis­ease trend­set­ter Soliris, re­searchers for the biotech say the first of two piv­otal stud­ies for PNH came through with non-in­fe­ri­or da­ta on their next-gen drug dubbed ALXN1210. That should leave the com­pa­ny on track for a bad­ly need­ed reg­u­la­to­ry fil­ing lat­er this year.

John Orloff

But, the parox­ys­mal noc­tur­nal he­mo­glo­bin­uria drug al­so failed a key mea­sure of su­pe­ri­or­i­ty, leav­ing Alex­ion tout­ing the sim­i­lar­i­ties of the two ther­a­pies and an eas­i­er dos­ing sched­ule that may leave quite a few sup­port­ers a wee bit dis­sat­is­fied.

The co-pri­ma­ry end­points in the study were trans­fu­sion avoid­ance and lac­tate de­hy­dro­ge­nase (LDH) nor­mal­iza­tion, stacked along­side four key sec­ondary end­points. The analy­sis on su­pe­ri­or­i­ty quick­ly broke down on the ex­am­i­na­tion of break­through he­mo­lo­y­sis: 4% of pa­tients giv­en and 10.7% for pa­tients treat­ed with Soliris. That de­liv­ered a failed p val­ue of 0.074, halt­ing the su­pe­ri­or­i­ty re­view and leav­ing some an­a­lysts a bit frus­trat­ed.

Un­der new man­age­ment af­ter CEO Lud­wig Hantson swept the old crew out, the biotech has con­cen­trat­ed heav­i­ly on ALXN1210 to save the com­pa­ny’s on­ly big fran­chise. At $542,640 a year, Soliris re­mains one of the 10 most ex­pen­sive ther­a­pies on the plan­et and Alex­ion’s cash cow.

The stock spiked on the news, pick­ing up a 9% gain mid-morn­ing as in­vestors ab­sorbed the da­ta.

Ge­of­frey Porges, Leerink

Leerink’s Ge­of­frey Porges led the cheer­ing sec­tion this morn­ing, but al­so not­ed that the tri­al fell well short of a com­plete suc­cess.

This con­sis­tent pic­ture of pos­i­tive trends in fa­vor of ALXN1210 should re­as­sure in­vestors that Alex­ion’s 2nd gen­er­a­tion prod­uct is in­deed bet­ter than Soliris; we ex­pect the stock to re­act pos­i­tive­ly to this dis­clo­sure, al­though con­tro­ver­sy will still ex­ist about whether 1210 could have shown su­pe­ri­or­i­ty in a larg­er tri­al, and whether the up­com­ing switch study could still meet the high­er hur­dle.

R&D chief John Orloff said:

Soliris has es­tab­lished a high bar for ef­fi­ca­cy. Achiev­ing non-in­fe­ri­or­i­ty on both co-pri­ma­ry and all key sec­ondary end­points, as well as see­ing nu­mer­ic re­sults in fa­vor of ALXN1210, in such a rig­or­ous study met a very high hur­dle. We look for­ward to reg­u­la­to­ry sub­mis­sions of ALXN1210 in PNH in the U.S., EU, and Japan in the sec­ond half of 2018.

Porges and al­lies be­lieve the da­ta sets up 1210 for a near cer­tain ap­proval, guard­ing the com­pa­ny’s fran­chise for many more years. But Alex­ion isn’t op­er­at­ing alone here any­more. Sev­er­al ri­vals are well along with new PNH drugs that they be­lieve can out­per­form Soliris, and now 1210.

One of those ri­vals is Apel­lis $APLS, which com­plet­ed a $150 mil­lion IPO last No­vem­ber. In 3 pa­tients nev­er treat­ed with Soliris, in­ves­ti­ga­tors re­port­ed that all of them ex­pe­ri­enced a quick cor­rec­tion on a key bio­mark­er for lac­tate de­hy­dro­ge­nase, or LDH, af­ter be­ing treat­ed with the biotech’s drug. In 6 pa­tients not re­spond­ing well to Soliris, the av­er­age he­mo­glo­bin lev­el was brought up an av­er­age of 36%, LDH was cor­rect­ed and trans­fu­sions dropped from 3.4/month on eculizum­ab monother­a­py to 0.3/month when APL-2 was added to eculizum­ab. And the biotech raised no un­usu­al red flags on the safe­ty side. Now it’s aim­ing at a piv­otal of its own.

The big ques­tion at Alex­ion re­mains open. What will Hantson do with the pipeline now? He has sought to stream­line de­vel­op­ment ef­forts and quite a few an­a­lysts are press­ing hard to make them di­ver­si­fy be­yond PNH.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Gossamer Bio CEO Faheem Hasnain at Endpoints' #BIO22 panel (J.T. MacMillan Photography for Endpoints News)

Gos­samer’s Fa­heem Has­nain de­fends a round of pos­i­tive PAH da­ta as a clear win. But can these PhII re­sults stand up to scruti­ny?

Gossamer Bio $GOSS posted a statistically significant improvement for its primary endpoint in the key Phase II TORREY trial for lead drug seralutinib on Tuesday morning. But CEO Faheem Hasnain has some explaining to do on the important secondary of the crucial six-minute walk distance test — which will be the primary endpoint in Phase III — as the data on both endpoints fell short of expectations, missing one analyst’s bar on even modest success.

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Kristen Hege, Bristol Myers Squibb SVP, early clinical development, oncology/hematology and cell therapy (Illustration: Assistant Editor Kathy Wong for Endpoints News)

Q&A: Bris­tol My­er­s' Kris­ten Hege on cell ther­a­py, can­cer pa­tients and men­tor­ing the next gen­er­a­tion

Kristen Hege leads Bristol Myers Squibb’s early oncology discovery program carrying on from the same work at Celgene, which was acquired by BMS in 2019. She’s known for her early work in CAR-T, having pioneered the first CAR-T cell trial for solid tumors more than 25 years ago.

However, the eminent physician-scientist is more than just a drug developer mastermind. She’s also a practicing physician, mother to two young women, an avid backpacker and intersecting all those interests — a champion of young women and people of color in STEM and life sciences.

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US sup­ports ex­ten­sion for Covid-19 IP waiv­er de­ci­sion

After much debate, the US government is now calling for a deadline extension to discuss a controversial potential IP waiver for Covid-19 diagnostics and therapeutics.

Over the last five months, the Office of the United States Trade Representative said it has consulted with members of Congress, public health advocates, organized labor groups, academics, think tanks, companies and trade associations on the WTO’s recent TRIPS agreement, which established a 5-year waiver of certain patent requirements on Covid-19 vaccines.

Eu­ro­pean Com­mis­sion lays ground­work to un­wind Il­lu­mi­na's $7B+ Grail merg­er

The European Commission has recommended steps that — though not yet final — would require Illumina to “swiftly” unwind its controversial $7.1 billion Grail buyout.

The Commission delivered a “statement of objections” on Monday, detailing the process Illumina would need to take in divesting Grail, its blood testing spinout launched in 2016. Illumina re-acquired Grail back in August, despite criticism from both the FTC and EU.

Rick Modi, Affinia Therapeutics CEO

Ver­tex-part­nered gene ther­a­py biotech Affinia scraps IPO plans

Affinia Therapeutics has ditched its plans to go public in a relatively closed-door market that has not favored Nasdaq debuts for the drug development industry most of this year. A pandemic surge in 2020 and 2021 opened the doors for many preclinical startups, which caught Affinia’s attention and gave the gene therapy biotech confidence in the beginning days of 2022 to send in its S-1.

But on Friday, Affinia threw in the S-1 towel and concluded now is not the time to step onto Wall Street. The biotech has put out few public announcements since the spring of this year. Endpoints News picked the startup as one of its 11 biotechs to watch last year.

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Mar­ket­ingRx roundup: Phar­mas lay off Twit­ter ads for an­oth­er week; WPP un­cov­ers LGBTQ+ mar­ket­ing find­ings

When Twitter’s new owner Elon Musk tweeted this weekend, “Just a note to thank advertisers for returning to Twitter,” he likely wasn’t talking about big pharma companies. The vast majority of the top spending pharma advertisers had not returned last week, according to updated tracking data Pathmatic for Endpoints News.

Only three pharma advertisers spent any money at all, which is about the same as the past several weeks. AstraZeneca rejoined the active advertiser list, although at $700 spent hardly worth a personal Musk expression of gratitude. GSK remained active with $3,500 spent ad much lower than its previous spending, according to the Pathmatics data. Only Bayer spent any significant amount in advertising, with $244,000 spent last week, but that’s a considerable drop from almost $500,000 spent on OTC, prescription and corporate Twitter ads in each of the previous two weeks.

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Bob Duggan, Summit Therapeutics co-CEO

Bounc­ing from ma­jor set­back, Sum­mit hands out $500M cash for can­cer drug — thanks to a loan from bil­lion­aire CEO

After hitting a dead end with Summit Therapeutics’ lead program, Bob Duggan has found the drug that he believes will usher into a compelling second act. So compelling, in fact, that it involves $500 million cash — and he’s taking money out of his own pocket to fund the deal.

Striking a partnership with Akeso Therapeutics out of China, Summit is bringing in a bispecific antibody that blocks both PD-1 and VEGF called ivonescimab. Akeso, which has a PD-1/CTLA-4 bispecific approved in China, has already taken ivonescimab into multiple clinical trials, including a Phase III in lung cancer.

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Gilmore O’Neill, Editas Medicine CEO

Ed­i­tas re­ports ear­ly-stage da­ta from two pa­tients in sick­le cell dis­ease

One company is moving forward in its bid to make a cell therapy for sickle cell disease viable — and after previous setbacks, including a hold earlier this year, execs are touting some really early data.

Editas Medicine announced Tuesday morning that it had positive safety and efficacy data from two patients in a Phase I/II trial investigating EDIT-301, a cell therapy candidate that Editas developed. Safety was measured in both patients, and efficacy data are from only the first patient dosed.

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