Ian Mortimer, Xenon Pharmaceuticals CEO

Xenon jumps on pos­i­tive da­ta-to-im­me­di­ate pub­lic of­fer­ing train to bankroll PhI­II epilep­sy study

Af­ter woo­ing in­vestors last Oc­to­ber with a suc­cess­ful Phase IIb, Xenon Phar­ma­ceu­ti­cals is back with more da­ta on an ex­pan­sion study for its dai­ly epilep­sy pill, and the biotech im­me­di­ate­ly went to the pub­lic mar­kets to drum up the cash need­ed for a Phase III that it hopes will gar­ner an FDA nod.

The Phase III tri­al of XEN1101 is slat­ed to be­gin in the sec­ond half of this year and will set the grounds, along with the Phase IIb da­ta, for a reg­u­la­to­ry fil­ing, the com­pa­ny said. To bankroll that work and oth­er stud­ies of the drug, the British Co­lum­bia-based biotech laid out plans Wednes­day night for a $250 mil­lion pub­lic of­fer­ing that is ex­pect­ed to close Mon­day.

Xenon’s stock $XENE dropped about 11.5% short­ly af­ter the open­ing bell Thurs­day to ap­proach the of­fer­ing price of $30.50 per share.

The biotech’s drug re­duced fo­cal on­set seizure (FOS) with­in one week for all three dos­es com­pared to place­bo, Xenon said. A whop­ping 96% of pa­tients, or 275 peo­ple, in the ran­dom­ized por­tion of the Phase IIb X-TOLE study moved in­to the open-la­bel ex­pan­sion phase, with 54 of them so far go­ing at least two years on treat­ment.

That fast on­set pro­vides a boon for the drug’s po­ten­tial go­ing for­ward, with Jef­feries an­a­lysts not­ing XEN1101 might beat out al­ready ap­proved com­peti­tors and the new da­ta “in­cre­men­tal­ly sup­ports our view the drug will be used in ear­li­er set­tings.” With ef­fi­ca­cy hap­pen­ing as ear­ly as week 1, Jef­feries an­a­lysts not­ed oth­er drugs can take mul­ti­ple weeks, in­clud­ing Xco­pri, GSK’s Lam­ic­tal and UCB’s Vim­pat.

Pa­tients in the open-la­bel ex­pan­sion had at least a 70% re­duc­tion in me­di­an month­ly seizure fre­quen­cy af­ter three months and greater than 80% re­duc­tion af­ter 12 months, the com­pa­ny said. Fur­ther yet, Xenon said the drug led to at least six con­sec­u­tive months of seizure free­dom in 19.6% of pa­tients in the open-la­bel ex­ten­sion and more than 12 months in 9.5% of pa­tients.

“A ‘time course to ef­fi­ca­cy’ analy­sis shows that all dos­es of XEN1101 rapid­ly re­duced the fre­quen­cy of fo­cal on­set seizures with­in one week com­pared to place­bo, sug­gest­ing that XEN1101 may of­fer a com­pelling and dif­fer­en­ti­at­ed op­tion for pa­tients seek­ing to quick­ly re­duce seizure fre­quen­cy,” CEO Ian Mor­timer said in a state­ment.

With the da­ta and at the re­quest of study in­ves­ti­ga­tors, Xenon will ex­tend the open-la­bel ex­pan­sion from three to five years, CMO Christo­pher Ken­ney said in a state­ment.

Com­ment­ing on Wednes­day’s new da­ta, William Blair an­a­lysts said they be­lieve the drug’s case for a high­ly dif­fer­en­ti­at­ed pro­file is bol­stered go­ing in­to the Phase III tri­al.

The com­pa­ny is al­so test­ing the drug in mid-stage stud­ies in pri­ma­ry gen­er­al­ized ton­ic-clonic seizures and ma­jor de­pres­sive dis­or­der. Jef­feries an­a­lysts pro­ject­ed XEN1101’s sales, if ap­proved, could reach $1 bil­lion in epilep­sy, more than $1 bil­lion for MDD and eclipse $400 mil­lion in PGTCS.

Xenon had $537.9 mil­lion in cash as of March 31, which was enough in the bank to last “at least” in­to 2024, Xenon said last month.

The biotech’s quick da­ta drop to im­me­di­ate pub­lic of­fer­ing fol­lows the suit of mul­ti­ple drug de­vel­op­ers this year, all try­ing to raise cap­i­tal on the back of pos­i­tive da­ta amid a tu­mul­tuous bear mar­ket.

Day One Bio­phar­ma­ceu­ti­cals did so last week af­ter pos­i­tive da­ta on its pe­di­atric brain can­cer drug. That fol­lowed the tra­jec­to­ry of oth­ers, like Phath­om Phar­ma­ceu­ti­cals, Re­zo­lute, Tar­sus, As­cendis Phar­ma, ar­genx and Apel­lis Phar­ma­ceu­ti­cals.

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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Spe­cial re­port 2022: Meet 20 women blaz­ing trails in bio­phar­ma R&D

When you run a special report for a fourth year, it can start feeling a little bit like a ritual. You go through the motions — in our case opening up nominations for top women in biopharma R&D and reviewing more than 500 entries — you make your choices of inclusion and exclusion. You host a ceremony.

But then things happen that remind you why you do it in the first place. Perhaps a Supreme Court rules to overturn the constitutional right to abortion and a group of women biotech leaders makes it clear they strongly dissent; perhaps new data on gender diversity in the industry come out that look all too similar to the old ones, suggesting women are still dramatically underrepresented at the top; perhaps protests and conflicts around the world put in stark terms the struggles that many women still face in earning the most basic recognition.

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Prometheus moves to raise cash hours af­ter PhII da­ta leads to stock surge

After releasing better-than-anticipated data on two mid-stage studies Wednesday morning, Prometheus Biosciences’ CEO said the company would “take some time to assess” its next financing options.

It only needed about seven hours. Wednesday afternoon after the market closed, the biotech announced it would seek $250 million through an equity offering as the company looks to edge out anti-TL1A competitor Pfizer and its new partner Roivant.

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Mark McKenna, Prometheus Biosciences chairman & CEO

With clear PhII win in IBD, Prometheus thwarts Pfiz­er com­par­isons as it fol­lows Hu­mi­ra 'play­book'

Prometheus Biosciences reported a clear Phase II win in two inflammatory bowel disease conditions in a clinical development race with Pfizer, planting the biotech’s flag in a field of antibodies attempting to go against black box-cornered JAK inhibitors and AbbVie’s Humira.

Shares $RXDX have soared since the summer — a small dip last week notwithstanding when rival Pfizer teamed up with Roivant on a new company for their competing anti-TL1A monoclonal antibody. And they skyrocketed once again Wednesday morning, climbing from $36 apiece to more than $100 on the back of two Phase II studies: one placebo-controlled in ulcerative colitis and the other an open-label trial in patients with Crohn’s disease.

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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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Alex Martinez, Intrinsic Medicine CEO

They with­drew their IPO. Then, they broke off their SPAC merg­er. Now what?

If at first an IPO doesn’t succeed, try, try a SPAC. But what happens when that fails too?

Intrinsic Medicine and its blank-check partner Phoenix Biotech Acquisition Corp. called off their reverse merger Tuesday night, citing “current market conditions” as the reason it went kaput. The pair decoupled just weeks after agreeing to combine in late October as investors’ appetite for new IPOs and SPACs has been limited, at best.

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Just days be­fore pre­sent­ing at ASH con­fer­ence, Syn­dax out­lines plan to raise $150M

Syndax is banking that upcoming clinical data will be enough to woo investors — to the tune of $150 million.

The biotech kept it short in its announcement Tuesday afternoon, saying it is planning to sell $150 million worth of shares of its stock via a public offering. While no date or exact number of shares was given for the offering, the biotech noted it will be giving underwriters 30 days to purchase up to an additional 15% of shares sold in the public offering.

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Pen­ny stock pulls back on of­fer­ing up more of its shares amid ‘cur­rent mar­ket con­di­tions’

As some biotechs continue to rethink IPOs or SPAC deals in the current market, other companies are pausing on selling off more shares.

The Boston-based RNA biotech TransCode Therapeutics on Tuesday announced that it has withdrawn its S-1 filing with the SEC that it made in late November. TransCode said in a brief press release that the withdrawal is reflective of TransCode’s belief that “current market conditions are not conducive for an offering on terms that would be in the best interests of the company’s stockholders.”